Your recommendations are fine but the title is just anti-biotech clickbait. For once please write an article that helps bring together the inventors and funders who take extraordinary risks with the positive result for patients when it occasionally works.
Do you want to preserve capitalism? Stop worrying about Bernie and Elizabeth and AOC. This is what will kill capitalism.
I have to quibble about the semantics.
It does not cost $2M, Novartis is charging $2M.
There is a significant and important to solving the problem the price indicates, difference in meaning there.
Data shows SMA occurs in 1 in 10,000 live births meaning that about 378 babies will have some variance of this condition. there are multiple types/severities, and I could find nothing about which are treatable with Zolgensma. The trial was for type 1, the most severe, with 12 babies.
It is unclear how effective the drug is over the long term as it must be evaluated over 15 years. All that can be said is that a majority of treated infants don't require to be on a ventilator.
Now $2M for a treatment does seem high, but I'd assume insurance pays for it in almost every case. Drug development is risky, and successful drugs need to pay for their development along with those that fail.
1
I am sorry to say this but I question whether extremely high cost treatments is something our insurance system, private or public, should be paying for. One million dollars could buy a lot of rubella vaccines, or insulin, or pay for MS treatments, or whatever. Why is our government encouraging R&D for treatments that they know will be extremely expensive? There are many areas with a better ROI, such as looking for the next generation of antibiotics since the current generation is losing efficacy due to immune bugs.
I would add a fourth recommendation:
Refunds, with interest, for ineffective drugs. If a drug makes it to market, is taken by patients, and does not provide a demonstrable benefit to those patients* within a reasonable period of time** then the drug company should be required to refund any money paid for them, by individuals or insurance companies, with interest. If drug companies aren't held financially liable in any way for ineffective drugs, then the incentives are all on the side of pushing these through any way possible, and charging as much as possible for them before their failure becomes apparent.
* "Benefit" could perhaps be something like an average increase in life expectancy of at least two months--this could mean a little bit of an increase for most people who take the drug or a significant increase for a lucky few.
** The evaluation period would depend on the condition being treated; in the case of spinal muscular atrophy, it ought to be clear within a few years whether babies are living longer or not, while a slower-moving disease like some cancers might require a longer evaluation period.
This is atrocious to say the least. Is this medicine made of gold, even then its price will never be that high.
1
The Editorial Board deftly danced around the central problem with government polices regarding the cost of medicine and the medical business in general, their laws manufactured by rich politicians who benefit the uber-rich.
The inequality of power and income is harming and killing poorer people. There's no point in getting around this nor talking with people who want to skirt the issue especially people who should know better, such as current NYTEB writers.
How can rich politicians tell investors, or executives, that they aren't entitled to above average income and wealth, if they insist on an excessive standard of living for themselves?
Hence, we see the Editorial Board talk about vague limited-time price controls until the effectiveness of medicines is "fully proven."
The Editorial Board won't forthrightly state how their proposals will maintain income and wealth inequality.
We will never put a stop to the medical business harming and killing poorer people until we get rid of rich politicians.
4
I've almost become complacent about the problems with the FDA approval process. They've only been documented regularly for the past 20-30 years. Several documentaries, case studies, lawsuits against drug companies, etc. And let's not forget the FDA's role in the opioid epidemic.
The cost of skepticism and disillusionment is distrust of doctors and pharmaceuticals. Voila, you have anti-vaxxers and more moderate people like me who simply presume against any medication until a limb is about to fall off. In one case, it was me gritting my teeth to deal with pain rather than accepting an opioid prescription, which I'm proud to say was the correct decision in light of how irresponsible these dangerous drugs are handled.
It seems like something someone running for president might want to take a look at. Too boring?
Novartis sure is proud of this drug. What's it made of ... a Martian's tears?
I was just prescribed a drug that is more than my house payment -- for one monthly dose. I had to jump through burning hoops of fire to get my insurance company to pay for it. They hadn't counted on my actually already having gone through all the numerous try-and-fail treatments.
1
Capitalism is the ultimate "death panel" as the Republicans like to say. If you want a purely market driven medical-industrial complex, then this is the result.
4
Obviously no middle class person can foot the bill for this treatment. You need to have a good healthcare insurance or government healthcare program, and you need to have access to the drug in the first place. Sadly, a lot of Americans enjoy the technological advances, largely thanks to immigrant scientists. Many of them come from countries where patients can’t get access to these treatment.
I work in an institution that is famous for developing one of these “miracle” treatment, and on my Facebook feeds I see children in Vietnam dying because the drug is not available. It is bittersweet to read the news where another American kid get saved while the kid from your country is doomed.
1
There's a basic disagreement over whether EVERYONE ought to have access to a drug with the potential to cure or ameliorate a disease, no matter what the cost to individuals or taxpayers- or proof of its efficacy.
David Whitrap's comment (wrt the FDA's speed approvals of specially targeted drugs) cannot be ignored: “In some instances, we are getting incredible innovations. . . But in others, we are getting completely ripped off, and we don’t have a good mechanism for telling the two apart.”
Complicating things further is the influence that "big Pharma" has over legislation, and the acceptance of their "right" to make obscene amounts of money for a few, while gouging whomever pays the bill. ( No argument about the right to make a profit; but definite questions as to obligations to society).
When it comes to health value for the investment - as some have pointed out - when EPA rules which protect air and water( two essentials for survival) are being trashed -do we have to weigh assuring safe water to all before we guarantee access to any treatment for all? One has a massive impact on the health on millions; the other affects a few hundred. Can we do it all? How?
Reassigning the enormous sums wasted on our war machine will not happen. Killing corporate campaign financing would be a step in getting to an objective appraisal process.
Inflated drug costs do not occur as an independent variable, but in relation to how businesses coexist with government and the public.
@cheryl: No public health care plan can survive without triage.
1
NYT should understand that the cost to develop a drug does not change whether the drug treats 2 million patients or 20. If a Drug maker develops a drug that treats 2 million patients, you can charge $ 1000 per patient per year, giving you a gross revenue of $ 2 Billion, which is s decent sized block buster after costs. If you have a drug that treats 20 patients and you charge $ 2 million per patient, that is only $ 40 million a year, which will never recover the cost that went into that drug, let alone pay for the 99.9 % of drugs that fail in clinical trials. This means that such a drug will probably never make it to the market. Drug companies routinely shelve promising new drugs because there is no path to profitability based on what payers (Insurance, Government Agencies) are willing to pay. $ 2 million per patient is the amount that the Insurance or medicaid pays and the patient pays a very small part. NYT has this wrongheaded notion that the health care crisis will be solved by taking a hatchet and cutting off R&D. The real crisis is the waste, duplication and inefficiency of the system where the providers and insurers employ armies of people to fight with each others and unnecessary tests and procedures are performed for profit and to protect against liability. By taking the side of insurers against drug companies you help no one as the insurers are as much beasts out to make profits as the pharma companies.
The failure of some drug studies are the cost of doing business in the pharmaceutical industry and should not be reflected in the cost of every single drug. Put aside this new medication, how do you justify the massive price increases on decades old medications like insulin and epinephrine? Like this drug they mean the difference between life and death, but for millions of people. I agree that insurance companies are no angels and deserve their fair share of the blame for the way they refuse to cover certain drugs and require high copays when they do. But it all starts with the pharmaceutical companies and their pricing. It is greed pure and simple and neither the pharmaceutical companies nor the insurance companies care about the lives they are harming and/or destroying.
Read the article below this one about the carve out for dialysis to treat end stage renal disease. Compare how differently we treat these two difficult conditions.
We really need to think logically about what medical interventions we are willing to pay for and which ones we are not, knowing that medical needs are infinite, and our resources are finite.
As a physician I get so discouraged because Americans remains totally unwilling to have this conversation about what health care should look like.
We are terrified about trying to think about what we can afford because that is the "death panels" and it would not be perfect, and we all think we should live forever as long as we do not have to pay for it, but we are not so sure all the effort is worth it for the next guy down the block, and no one is honest about the fact that we waste so much money on futile end of life treatments that if even if temporarily successful, do little or nothing to improve the quality of life.
4
@Jim P: What doctors call "triage", patients call "death panels".
Government is already paying the vast majority of basic science and early clinical development cost through its basic science and small business grants. It should also take ownership of the patents and follow through until Phase II clinical trials are finished. At that point it should let pharmaceutical companies bid to take over the drugs with a clause dictating how much can be charged for it.
1
@Ivan..."Government is already paying the vast majority of basic science"....Basic science and drug discovery and development are not the same thing. Basic science suggests an idea. Applied science takes the idea and makes it a reality. Both are critical, both are difficult, neither is superior, and you cannot substitute one for the other.
1
Globally, there are millions of people that die every year from vaccine preventable illnesses, unsafe drinking water, and malnutrition. Such outrage about the list price of an orphan drug seems fundamentally misplaced when access to food, clean water, and inexpensive vaccines would save so many more lives.
1
@JP: Saving lives while otherwise stunting people fails to make the transition to a long-life low birth rate stable population.
1
This drug will let its company executives & large investors buy even bigger, faster, & more luxurious private jets.
1
@Beartooth
There is a myth here. The high cost of drugs while saving millions of lives, prevents the loss of far too many millions more lives due to unaffordability of drugs for those who need them.
A clear example is an estimated 10 million preventable deaths occurred in Sub-Saharan Africa, which also created nearly that many more innocent AIDS orphans, since 1996 when the life-saving 3-drug-cocktail for AIDS was discovered. The drug still costs annually >$10K/year/patient. This drug is now procured for around $100! It was available for about $400 by 2000. In 2003 GW Bush allocated $15 billion saving lives by the millions in Africa.
There is an interesting story about how the Clinton administration unleashed generic manufacturing of this three-drug-cocktail all over the world in 2000.
The myth is few investors & drug-company executives make that much money from high cost of drugs: Very few investors, not traders, made any money on buying drug-company stocks: After an initial fast run between 1995 & 2000, the share prices of major drug companies styed stagnant, actually dropped by about half and stayed low for about 15 years. Most investors lost money during that period. Drug-company CEOs don't make that much more than the average top 200 CEOs. But sure they managed to figuratively commit genocide meanwhile!
1
There's a far too simplistic solution to the high cost of lifesaving drugs, which may appear too ridiculous or too good to be true. Bernie Sanders had hinted it.
The total annual R&D budgets of all U.S. based companies will be under $90 billion - Pfizer's is about $10 billion. About half of that budget is spent on promotion.
The total amount Americans spend on drugs is about $370 billion annually. Eliminate patents on (lifesaving) drugs, on moral grounds. Let taxpayers pay the entire R&D cost/budget of all drug-companies based in the U.S.
If patents are eliminated, the cost of drugs will come down at least by $100 billion.
On top of that whenever a new drug is approved by the FDA the company will be awarded, say a minimum of $100 million, which will increase to up to say $1 billion based on the importance & commercial use of that drug. Once a drug is approved, its generic version can be manufactured by any company, the quality of which will be certified by FDA or a similar entity. This way price of the drug is determined by market forces, not by the company that invented or discovered.
Ten% of the reward money ought to go to the scientists & technicians who conducted the research, which will be a great incentive for them to work on the drugs, including orphan drugs with very little commercial value.
1
@A.G....Many people do not understand that a patent is a two way street. In return for the exclusive use of their invention for 20 years, the inventor gives the public detailed information about the invention and how it is used. A patent provides a strong incentive to scientists for making their work public.
@A.G.: Collectively, the drug industry probably spends $30 billion per year on prescription drug advertising.
1
@W.A. Spitzer: Scientific discoveries are not eligible to patent.
Fast-tracking a drug that failed Phase II and is made by the acting FDA commissioner’s old company!
How is that draining of the swamp going?
2
Big Pharma making millions for stockholders while Americans die due to the cost of drugs. It not about safety or affordability for Big Pharma, only the bottom line. Soon they will find only a few will buy their drugs not because they aren't innovative, but because they cannot afford them.
This is all morally wrong at a fundamental level.
In capitalism, you have the right to walk away from any cost you don't like. That option doesn't exist with health care.
It's not capitalism. It's extortion.
As with the woman who received a check for $5k from her insurance company for having her knee replacement surgery in a hospital in Mexico, clinical trials and other costly related steps, could also be undertaken outside the U.S. A win-win for colaborative research and affordable drugs.
Thanks for focusing your editorial board power on this tragedy.
2
Having worked as a chemist in pharma for 16 years and now for a chemical manufacturing company, I see firsthand the high costs of producing these drugs. I can’t even imagine what it costs to produce a custom nucleic acid such as Novartis’ drug, but surely it’s a lot. $2.1m? Probably not, but even so this one would not be cheap. I’ve also seen that even in this age of loosened regulations plenty of drugs fail clinical trials. A balance needs to be struck between keeping the profit incentives to produce these transformative products and ensuring accessibility to as many patients as possible (all, ideally). Government incentives to promote competition in the same disease spaces, prevention of mergers and acquisitions, and price negotiation will go a long way to keeping costs in check.
1
Actually, production of nucleic acids is quite cheap. You can order 1kb DNA fragment for less than 100$, meaning that cost of the synthesis is much low than that. Once you have template you can use several means of production depending on delivery system and mechanism of action.
August 14, 2019
User rights to claim ownership of the results of the medicine taken for the life of the patient. As such user publishing can and should offset the medicine costs and countered by the patient costs; as drug Rx application remedy is published to interested parties and all in medical journals. Win Win when we create the best solutions that benefit life's goals.
1
That 2M could go to save the lives of HUNDREDS of children, if used wisely. We need to prioritize as with all aspects of life. Drug companies are not the problem, they are the solution. Fully 85% of all drug prescriptions are "generic" (ever buy a "generic" Porsche?). Ever take an antibiotic, an aspirin? If you want to lower healthcare costs, focus on physicians salaries...
I never take antibiotic for 2 mln dollars. Pharma could be solution but they are also a problem, because their pricing practices. Insulin is an example. It is not a new drug, but the price keeps rising and makes insulin unaffordable for many, how that is not a problem?
And I am not so sure if how much solution is the big pharma . This drug was developed by AveXis company that was founded in 2015 based on research of French researcher. The company was acquired by Novartis in 2018. Clearly Novartis didn't have much time to do any research on the drug at all, but even AveXis didn't have much time for full research, what makes me think that majority of the work was done in French Academia, and companies just did regulation effort and marketing.
1
Hint:. The physicians do good stuff for us. If you want to eliminate waste, it's in the overhead of pharma companies and hospital corporations.
If drug or physician prices were cost of production based and payment was proportional to treatment success, providers would lose their incentive to promote medical services with low success rates and society would be paying what is deserved without excessive/ unwarranted premiums.
It doesn't cost $2 million. It costs $2 million A YEAR!!!!
@Curiousone No, this treatment actually only costs $2 million once via a single treatment. If it is successful, the patient is cured.
1
We can stop Big Pharma with free medication for all. I tend to be a conservative but socialized drugs is as natural as a socialized army. There are a few things that should not be left to the private sector and this small part of health care is one of them.
Taxpayers should have whatever they need and reap the immediate benefits of advances. Nurse Practictioners can prescribe and renew at little cost resulting in basic health care for all.
Patents (monopolies) are not necessary to encourage medical progress. There are better ways to manage research.
2
Ethical issues continue to thrive re drugs, their cost, distribution, and the ability to meet, ALL, the sick patient needs. Is anyone in charge?
1
If I understand trump followers correctly, here's the argument:
1) Drug companies make drugs that are hard to make. It's like magic, called R&D, very specific work that every other high tech company also uses.
2) Drug companies find a useful drug to treat little babies who would die without it. Pro-lifers are thrilled because now careless women won't abort this fetus!
3) The price per patient is.....gulp...$2M per patient? How do the insurance companies feel about this?
4) Capitalistic America does a quick monetary calculation while the Pharma lobbyists stand quietly sobbing, showing how much expensive R&D magic went into this feat.
5) The price is allowed to stand, NOT because the numbers won't work out fairly, but because Capitalistic America puts capitalism first, so sorry. Unlike Salk's polio vaccine, it's corporate profit that drives our healthcare decisions now. Proof? The comments in here excusing Novartus because they had to do research.
Like all other technologies, drug innovation stands on the steps that were built before them. It's not like these companies have to re-map the human genome. Are they 'accounting' for the infrastructure already in place for them to build the next step?
So instead of getting a reasonable profit, they'll be allowed to wring millions by only providing this to the babies of rich people. Pro-lifers shrug, although this means there is no hope for those other parents.
2
I thought Gilead Sciences was bad (they still are) for pricing hepatitis C cures around $1000/pill, taken 1 a day for 90 days. This, however, is criminal. The day pharmaceutical companies were given permission (by a Republican President led Congress). I wish nothing but ebola and the black plague upon the FDA, Novartis, and Gilead executives. They deserve it. I believe in karma. The execs who price these drugs, along with the FDA allowing it.
1
My husband was a cancer patient. I left my job to care for him so I could spend my time calling my insurance company to determine where they were in the process of determining the medical necessity of the procedures and drugs the doctor ordered weeks before. When I wasn’t doing that I spent free time worrying about how much longer it would take for us to be bankrupted by costs not covered by insurance. I wouldn’t go through that again for anything. Life at any price? I don’t think so. Everybody dies even in the land of the free and the home of the brave.
1
How is anyone surprised at all? This is par for the course. The taxpayers foot the bill for the R&D by paying for those grants and tax breaks that the pharmaceutical company gets- which then uses our tax dollars to bribe more congressional reps and senators to make laws that benefit them and by either bribing FDA officials or using regulations they wrote to fast track their product. They then bleed the ones least able to afford this taxpayer funded product and hold it for ransom.
The greed and corruption of the pharmaceutical industry is staggering- but they aren't alone in this clown car circus that's become our reality. Every corporation is slurping at the trough that's only gotten wider since the orange fruitcake took charge.
1
@SR.... "The taxpayers foot the bill for the R&D by paying for those grants and tax breaks that the pharmaceutical company gets-"...To make this statement credible you need to provide details and examples.
Where do you thing the Government takes money to offer the grants or to cover for uncollected taxes from Pharma?
1
This is a national disgrace, committed by the drug companies and supported by our paid-off lawmakers.
And it not's the only one. As Bernie Sanders has pointed out, across the border in Canada are countless medicines easily affordable that can't be touched in the US because of price.
1
Your article says "families who desperately need Zolgensma are struggling to afford it."
Struggling? What families are you referring to mr author?
Who has the capacity albeit a struggle to afford to buy a2 million dollar drug? You are obviously moving in elite circles.
3
It would be helpful if the article mentioned that the only alternative therapeutic to treat SMA currently costs $750,000/year for the first year and then $375,000/year thereafter for life. Zolgensma potentially offers a lifetime cure in a single treatment.
Many pharmaceuticals cost well over $2 million over the lifetime of a patient, allowing manufacturers to recoup their costs and profit over an extended period. This isn't so easy with single treatment gene therapies.
Additionally, for rare diseases such as SMA the options with our current capitalist drug development system are to either charge a high cost relative to treatments for more common diseases (there are ~25,000 patients in the US with SMA, and more than 100 million with high blood pressure, for example) or to not develop treatments at all.
2
With such a small patient base, the mathematics shouldn't be so hard to figure out. 400 patients at $2 million each is $800 million. Did Novartis spend that much on research? How much Federal aid did they get (Novartis didn't pay that)? Where could the $800 million have come from? I don't know any of this, but this looks like an easy example to use to figure out what the pharmaceutical companies are doing.
@Scottilla....For every drug that starts clinical trials, only 1 in 7 is ever approved. And just getting a compound to clinical trials in the first place is a very significant accomplishment. You need to include the cost of those failures.
The question that I never seen answered is how they justify a TWO MILLION dollar price tag on a single drug.
All we ever hear from the pharmaceutical companies is "it is expensive" to research and develop.
Prove it.
1
Before people get their knickers in a twist, I would remind them that the human genome project completed just 16 years ago required 13 years at a cost of $2.7 billion dollars. Today you can do the same thing in 2 weeks for about $20 thousand dollars. Science moves forward. We learn things. What was impossible yesterday, is possible today, and will become conventional tomorrow. Be thankful that someone spent the time and effort to find a cure.
2
Yeah, but human genome is now available for free to everybody, speeding up the progress to whole society. Here we are talking about 2 mln drug that benefits few, but should be paid by everybody (including the public research that was the basis for the drug) except big pharma.
@yulia. Now that Novartis has proved that there is an effective treatment, it is possible for other scientists to take that information, improve on it, and make a new drug that is better and less expensive. My point is that you should not dismiss what has been accomplished. It is not the end, but rather the beginning.
My husband has SMA type II. Insurance won't pay for this treatment. The Muscular Dystrophy Association won't pay for it. SMA patients have been left to tend to their own health needs their entire lives. You can argue all you want about who will pay and when, but the fact remains that the treatment was created for use, but only for a profit.
6
I was recently diagnosed with amyloidosis, a rare heart condition. My monthly copay for a new drug is $1,800. We must fix our health care system, including drug prices.
5
This is obscene.
I am a physician, nearly 60, and I never thought that my profession would become such a black hole of fraud, greed, and political power. All across the country, down to the community level, the people making the decisions in healthcare place profit and power first with virtually no concern for patient and local community welfare and wellness. The people who actually treat patients - doctors, nurses, and other healthcare workers - have virtually no voice in how hospitals and medical communities work. This is a big change from when I started medical practice in 1992.
We need to remove the ability of bad people and greedy companies to wildly profit from sick patients. We need to have a healthcare system which requires direct and open local community oversight and involvement. We need to pay healthcare workers better wages with better benefits and job security. We need to stop paying hospital administrators and medical company administrators seven and eight figure salaries and bonuses.
We need a massive overhaul of the US healthcare system. The primary focus of every individual in US healthcare should be patient and community welfare. Obscene profit, bloated administrative paychecks and retirement packages, price gouging, fraud, and the like should be addressed with prosecution and jail time.
The quickest way to stop this type of behavior is to elect Elizabeth Warren for president in 2020 and to gain Democratic control of both houses of Congress.
8
Let's do the math for fun.
Let's admit that there are 3,850,000 newborns a year in the USA.
Statistics say that spinal muscular atrophy in the U.S. affects from 1 in 20,000 (minimum) to 1 in 6,000 (maximum) infants per year.
This would result in 192 (minimum) to 642 (maximum) new cases per year.
At the cost of 2,100,000 dollars per patient for a resolutive treatment of the disease (I say "resolutive" - correct me if I was wrong to understand), the total annual expense to be incurred to treat new patients would vary from 403,200,000 dollars (minimum) to 1,348,200,000 dollars (maximum).
Novartis made a net profit of $7,700,000,000 in 2017.
If Novartis decided to "give away" the drug, its net profit would decrease from 5.24% (minimum) to 17.51% (maximum).
I note that the budget of the U.S. Ministry of Defense amounts to over 640,000,000,000 per year.
End of the game.
Best regards from Padua (EU).
3
Now factor in the percentage of people able to afford 2 million who have an infant afflicted and requiring treatment. There's no logic to a price so high which so few can afford.
1
@Flavius: This is just the beginning of the lifetime medical costs of the affected babies.
2
Part 2 -- After 16 weeks, Burroughs Wellcome stopped the trial because there was strong evidence that the compound appeared to be working. Those results were heralded as “the light at the end of the tunnel” by the company, and pushed the FDA approve AZT in a record 20 months with many questions left unanswered: How long did the apparent benefits last? Could people who weren’t sick yet still benefit? Did they benefit more than those further along in their disease? In the end, prophylaxis against opportunistic infections due to HIV infection proved to prolong lives & HIV monotherapy accelerated HIV resistance to AZT. The drug’s approval remains controversial to this day. After AZT’s approval, activists & public health officials raised concerns about the price of the drug; $8K/yr > $17K in today’s dollars) cost prohibitive to many uninsured patients and AIDS advocates accused Burroughs Wellcome of exploiting an already vulnerable patient population. Sound familiar? And the Band Played On.
2
@jmc....But you might add that new AIDS drugs were discovered and developed; so today what once was a death sentence is a treatable disease.
1
@W.A. Spitzer You missed the whole point. Unfortunately many people still die from HIV infection and HART has many side effects (kidney issues, cardio-vascular issues, liver issues, I could add many more). So I find your qualifying HIV as a "treatable disease" to be obtuse.
1
Corporate greed at it's worst. Who cares about the babies who might be able to live or doomed for whatever few years they live. Something akin to assault weapons, NRA and the increasing number of mass killings. And our Government (Republicans and Democrats) want to aid these corporations with incentives and tax breaks! The worst part is, we Americans have elected the Government we have. It sure makes us accessories too!
2
This is why I'm voting for Bernie.
1
@JT...Not sure Bernie knows how to discover new life saving drugs.
2
Drug prices should be based on the value they create for society. What is the cost of having an untreated DMD patient? No mention of that number here, but undoubtably it's also a shocking number. If the drug can alleviate a life time of suffering, create a productive, tax-paying citizen AND save insurers a great deal of money, then the company deserves a substantial fraction of those savings, even if the number is huge. Companies have to make their money while the drug is still on patent; after that they essentially become free forever. My very effective, generic blood pressure medicine costs a few pennies a day. A big 'thank you' to the scientists who devoted their careers, the company that invented it, and the US patent office.
29
Nonsense! What's the value of penicillin for a child who might otherwise die of pneumonia? Astronomical. What's the cost? Not much. Pricing by value means that the medical industry would reap huge profits for providing routine service.
21
@Greg Have to disagree with the notion of "value for society". A child may never grow up to be a fully tax paying citizen, but we can't deny the child's parents or the child the right to live, even with a reduced quality of life.
9
@Greg: Whatever the government pays to invent should be licensed to manufacturers, not given away to them.
12
If a drug costs $2 million that means rich people get to live and poor people have to die. Is that OK?
46
@Craig Willison That is already happening with diabetics who can't afford their insulin and die as a result of trying to ration it or switch types which aren't effective. Their only option is to drive to Canada to get affordable insulin if that is a possibility. Elderly people are also dying because they can't afford their medications.
14
@Craig Willison
If the price of future drugs is capped, they won't get created in the first place. Is that OK?
9
@Craig Willison
It's not true that capping prices leads to non-invention of future drugs. It leads to capping unnecessary windfalls for stockholders. The burden of proof is on those who would raise prices, not on we who call for caps, since we're the payers, whether through insurance premiums or directly when we get sick.
In a larger context, this is one more example of how unfettered capitalism doesn't exist. The article points out that the government is creating the conditions for the drug's existence and then abdicating its responsibility to control the drug delivery process, including prices.
8
Producing this cure is really dumb on all accounts. The drug company will have a very limited market, the number of families that can afford 2M a pop, and have a child that is afflicted, is probably very small. It's efficacy is apparently not known for sure. Society can not afford to pay the two million if the condition is wide spread. Yes, this means rationing treatment.
2
When our tax dollars pays for the majority of the research for these new drugs why don't we have government owned labs to finish the process?
Charge medicare and hospitals what it actually costs not the amount these crooked greedy companies need to get filthy rich on people pain.
They are blackmailing sick people for all of their money.
Is there a lower form of life than this?
2
How can the editors run this piece without asking how much this drug costs to produce? Never mind the years of R&D—purchasing the technology from whichever lab developed it, putting it through toxicity studies and then clinical trials—but simple manufacture: how much does it cost to make that single dose? Because in this case, it’s not like making Advil. It costs hundreds of thousands of dollars. If you don’t start with the baseline product price, you cannot have a good-faith conversation about what this drug should cost. This is apples and oranges to Martin Shkreli-type thuggery, where a drug that costs very little to manufacture, and is already approved for patient use, is purchased and its price jacked up beyond heart or reason. By all means drug prices are a problem, but be responsible and report on the difference between a “biologic” drug, say, and gene therapy. As gene therapy explodes into clinical use, this issue of pricing is going to sharpen. You’ll need better reporting to cover it effectively.
1
What is a lot of people missed in the comments is that even if you pay 2.1 million, it may not work!! Worse yet, there maybe harms to it.
The data was "manipulated or mishandled" because it DOESN"T WORK. And this is Pharma for you.
2
@Anoop: There is no guarantee that some other genetic defect will not become a costly medical issue, even if this one is fixed. This just opens the door to playing with the whole genome with the same technology.
@Anoop
You are entitled to your own opinions, but lets get the facts straight. According to the FDA, the drug should stay on the market and FDA affirmed the safety and efficacy of the drug. It does indeed work -- extremely well. The manipulated data -- which I do not in any way condone or defend -- was in a mouse experiment and has no bearing on whether the drug is safe and effective in people.
@Fly Over Flora
Good point. But for me that just shows that there are so many things that might have gone wrong that never comes out. If they can do it in mouse data, what is preventing them in doing in human trials?
I watched a video of Bernie Sanders traveling to Canada with a group of people in need of medicine, in that case insulin. It was so cheap over there compare to here. I am reading this and thinking about his promises.. I hope he wins.. And if he doesn't because people are afraid of getting what they deserve (health care, cheaper medicine, lower taxes), whoever wins takes on consideration this problem.
Another good argument for single-payer health care. Only a government led by a Democrat-controlled Senate and someone sane in the White House can curb the excesses of Big Pharma and ensure that the drugs provided to the American people are both safe and affordable.
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So, I think I have this straight. The NewYork Times which rails about the Right’s undercutting of the population’s trust in our institutions runs an Editorial Board piece questioning the entire integrity of the Food and Drug Administration when it says the drug is safe and efficacious. I understand compartmentalization but my expectations of the Times have just fallen. Why beyond your desire for me to do so should I trust you or as you say ‘how can ( I ) know for sure?”
1
Your money or your life. That's as immoral as you can get. Prepare for a genuine hold-up at your local pharmacy.
2
Pharma was given license to charge exorbitant prices for drugs beginning in the 80's with the AIDS crisis. AZT (azidothymidine), developed in the 60s by a US researcher to thwart cancer; it didn’t work when tested in mice & put aside. HIV emerged as new infectious disease & Burroughs Wellcome, known for antiviral drugs, began a massive test of potential anti-HIV agents. Among the things tested, Compound S, a re-made version of AZT. When thrown into a dish with animal cells infected with HIV, it seemed to block the virus’ activity. To test the compound’s effectiveness, a controversial trial was launched with nearly 300 people who had been diagnosed with AIDS. -- continued in 2nd post ---
1
Around 30 years ago when I interviewed for a position with a biotech I recall having asked about how new drugs are priced. The reply was something to the effect that we raise the price until we get to the "puke" point. I actually had to asked what that meant. (Didn't get the job.)
3
I had not idea that the charge of $1 million for a drug would be considered common pricing. Regardless of the efforts of RnD, what is the rational behind telling the average person/parent that there is a drug that "might" help you/child and the cost of that drug is $1 million, think about it and let's see if you want to go forward
How much does Zolgensma cost in Canada? Or Norway? Or Spain?
Seriously, why do your readers have to tell you what the obvious questions are?
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If bringing a drug from research to market was so expensive and such a gamble as drug companies claim, occasionally you should see one fail. Instead, the profitability of these companies, in every case, can only be described as excessive, if not obscene. Most people can’t afford these drugs, so disability pays, which means taxpayers. But having the headquarters of these companies off-shore means that taxpayers are simply making these companies and the stockholders richer. To top it off, any gov regulatory agencies are headed and staffed by industry insiders who either come from high paying jobs and/or leave for them.
One day, a lot of people are going to die or be damaged beyond repair by some drug that slid by, and then taxpayers will pay for that too. If the world doesn’t eat itself, someday we will look back on publicly-owned, for-profit transnational corporations (big-pharma simply being one) as one of the greatest mistake’s society ever let exist: An innately corrupt, (poorly) self-policing, closed system making the rich richer, the stock market being the 1%’s Monte Carlo which taxpayers regularly bail out, (and likely soon will again). All take, no give, sucking our society and the middle class’ quality of life and standard of living until nothing is left, including the planet. I don’t even see how (outside of something like the French Revolution) the situation is fixable, they are entirely entrenched.
1
@SMcStormy: "If bringing a drug from research to market was so expensive and such a gamble as drug companies claim, occasionally you should see one fail."
Actually, more than 90% of drugs fail.
https://blogs.sciencemag.org/pipeline/archives/2019/05/09/the-latest-on-drug-failure-and-approval-rates
1
No one is paying 2.1 million for this drug—most insurances cover it. Further, in the long run, 2.1 million is less than it costs to caring for these kids while they’re sick, and less than their expected long term productivity assuming successful treatment. The risk/cost is currently spread across the system by insurance covering the drug—e.g., exactly what the Medicare for all crowd advocates for. I don’t see how anyone has a complaint here. The people who need the drug have access without going bankrupt, and there’s is still a significant enough profit motive to entice a company to go through all the regulatory hoopla and failed trials necessary to create a life-saving drug for such a small subset of the population.
This new-wave expectation on the progressive left that folks must strive to be Mother Theresa and totally selfless in their professional endeavors is honestly just bizarre. There’s nothing wrong with the fact that folks want to be rich, and if we are able to harness that desire in a way that spurs innovation, helps society, and helps individual patients while ensuring that even the least fortunate have access, then power to the pharma industry.
Perhaps the outrage mob should learn the distinction between compensation for new innovation and the price gouging that goes in with generic drugs, and then perhaps consider redirecting their anger. But, that would require nuanced understanding, which society unfortunately seems to severely discourage of late.
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@Jon
*applause*
Well said. All of it, especially the last sentence.
3
@Jon Maybe it's your understanding that isn't sufficiently "nuanced." In many cases, insurance will NOT cover this drug, and many people in this rich country still do not have insurance at all. Many of those same uninsured people won't make two million dollars in their entire lives. How is letting some children die simply because their parents are poor while letting other children live simply because their parents are wealthy morally acceptable to you? Where is the line between a free market economy and genocide? Perhaps the mob are outraged at the inability of people like you to see the reality of their day to day lives and your total lack of empathy for their suffering, even when they lose their children.
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@Jon So it turns out that you're a socialist! Look at your argument: 'No one is paying 2.1 for this drug - most insurances cover it.' Well where do you imagine the dollars hat flow through the insurance companies come from -- that's everyone else. There are only two consistent positions on the financing of medical care -- socializing the costs (insurance systems based on annualized policy models are both inefficient and create perverse incentives) or not socialized it (in which case, only patients that can afford highway robbery will survive rare serious diseases). Market fundamentalists need to admit that their market fantasies only work when they eliminate the moral hazards either by closing the ERs to people without money. Their only alternative is to admit that markets don't function well when the demand for life is inelastic.
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Spinal muscular atrophy affects approximately 400 children per year. That means a drug company can only sell 400 doses of the drug per year. How are they supposed to recoup years of development if they are for instance limited to $200 per dose? its preposterous.
Its also preposterous that a company would charge $2 Million per dose, but it is their drug, they can charge what they like for it. If we don't incentivize drug companies to find better drugs, then they won't do it, and we won't have new and innovative treatments.
Furthermore, under current law, drug companies have a patent for 20 years. After 20 years anyone can sell the drug, presumably more cheaply. We all get the benefit of the innovation after 20 years. Thousands of drugs that were once lifesaving and expensive are now cheap, because of this law. Second, and more importantly, is that we don't have unlimited health care dollars. Spending $2M to save one child is probably not the best way to spend healthcare dollars, when that money might be better spent for, instance vaccinating 100,000 children. We are not used to making healthcare decision based on cost, but it simply isn't feasible to give unlimited healthcare to everyone...otherwise we might someday have the entire baby boom generation on respirators. The point is that limiting drug prices is a very shortsighted approach that will stifle innovation and cost many more lives in the long run than it saves in the short run.
1
Agreed. Drug costs are too expensive. But your article has all types of inconsistencies. If FDA shouldn't have expedited reviews then the costs of testing is way too prohibitive. It takes 5-10 years for FDA approval with massive oversight. You can't have it both ways. Lowers costs and longer delays. I wish NYTimes pays attention to details. The issue is about limiting meaningful profits for the risks taken.
Big Pharma is up there with the NRA and GAFA for the coveted title of most nefarious capitalist institution. Sorry, I forgot Monsanto.
Drugs for children that cost this much put their parents in a horrible bind. They want their new born child to thrive and prosper and will do anything to achieve that, but if they some how do find the resources it takes away from their ability as a family to thrive and prosper in the future. Medical care should never suck the life blood out of people and companies that charge this much are like kidnappers to hold children hostage.
Bernie Sanders is right that these pharma companies are greedy and selfish. How many families have $2 million lying around?
The cost of these drugs is outrageous beyond anything....like those golf outings we tax payers subsidize for Trump. Maybe we could stop making those Navy destroyers that cost billions and billions and funnel that money into subsidizing the cost of these drugs to the humans that need them.
Everyone always talks about the huge costs of RnD for creating new drugs, but no one talks about the fact that pharmaceutical companies spend a TITANIC 30-50 percent of their revenue on marketing and advertising.
With a Medicare for All system, the government could guarantee that it would purchase vast quantities of drugs from companies at greatly reduced prices. Although the pharmaceutical companies would make less per individual drug sale, they would still have plenty of room left over for profit since they would be able to virtually eliminate their advertising budgets. Couple this system with tax credits and government R&D funding for companies that create life-saving (yet unprofitable) drugs, and we will be able to preserve innovation while increasing affordability.
Plus, pharmaceutical advertising is inefficient for the economy. Why should anyone be making a medical decision based off a 30 commercial?
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@John Louis
Do all the commenters here know that CONGRESS in 2003 (GW Bush) made a LAW that Medicare could not negotiate drug prices?
Again, it's blame Congress. :Impeachment hearings not vacation, now.)
14
@John Louis: Of course they spend so much on advertising: it works. And we let them, because we support private sector development of drugs. We also support medical practice as private businesses. The result is that pharma uses a two-pronged approach to advertising: make patients believe that a new, expensive drug will make their lives much better, and simultaneously market to physicians so that they're ready to make their patients happy by prescribing these drugs.
Secondly, I will just say that Big Pharma does relatively little in the way of innovative research. The more typical path to innovation starts with academic research, then is spun out to small companies to translate academic breakthroughs into potential therapeutics, and then goes to Big Pharma, usually in the form of highly profitable acquisitions, for clinical trials, regulatory approval and post-market sales and manufacturing.
There is government funding for the earlier stages of this process--academic research and early-stage companies. But there's the 'Valley of Death' between promising research and demonstrated clinical efficacy, that is currently bridged by equity investments. This is where the profit motive takes over. Find a way to cross that valley without VCs and the scenario will change.
10
@John Louis
And much of the research is funded by the government, which then allows the companies to get the patents.
8
The (U.S.) National Inst. of Health (NIH) spends nearly $40B every year on biopharma research candidates... and billions more for the CDC to do pure research. U.S. taxpayers fund the FDA to ensure the safety and effectiveness of drugs. In addition, taxpayers subsidize clinical trial and commercialization costs to the tune of tens-of-billions more. Then, U.S. patients (and/or their insurers) pay billions more for the actual drug... in fact, much more than ANY other country that didn't invest a nickel for the drug's development... and we can't even negotiate the price LOL!
How about the ROI for the U.S. taxpayer? Why doesn't the NIH license their patents? Why doesn't the FDA set fees for drug approvals? Why have this opaque, disjointed, public-private "partnership" at all? The obvious answer is that the biopharma has become the most profitable sector, and one of the largest/most influential D.C. lobbies.
If the U.S. taxpayer stopped funding/subsidizing biopharma development tomorrow, how many new treatments would be brought to market? If Novartis et. al. want bankrupt tomorrow, what would be the market impact by comparison?
18
@Apathycrat:
* The NIH does license their patents, and universities funded by NIH grants aggressively patent and then seek royalty-bearing licences, at market terms, for any IP that results or could result in a treatment.
* The FDA does set fees for drug approvals. Look up PDUFA, the act that sets those fees, paid for by industry...
2
@Apathycrat
And the U.S. govt doesn't pay for the millions in clinical trials that companies must spend to get their drugs approved by the FDA.
1
@Apathycrat
Because stupidly, the US Government cannot patent or copyright anything. It's in the Constitution. The entire class of tech wealth is built upon technology and infrastructure invented by the US taxpayer. And every time some lawmaker wants to tax internet revenue, the tech companies con people into believing that this is an assault on internet freedom rather than restitution for freeloading. And people keep falling for it. Our country is dumb.
1
At some point there is a cost/benefit to society. It's amazing that research has delivered cures for very rare diseases, but the reality is these drugs help society very little compared to what we could do with better drugs for more common diseases and issues. Sure, it's $2 million to save your child, but there are hundreds of thousands of families struggling to pay for simpler drugs.
I think we need to take a hard look at who benefits the most from medical spending in terms of longevity, balanced with quality of life, and focus our spending there. I think part of what makes medical such a growing expense is that we have gotten better at keeping people alive who 15 years ago would be dead..... but that extension of life generally lacks quality. My mom got $250,000 of chemo drugs to extend her life a painful, tired and sad 9 months. To me, that's the wrong type of expenditure. Meanwhile, my ex had $150,000 worth of brain surgery and has thrived ever since.
What's the long term for these kids on a $2 million drug? Does the drug fix them wholly or does it leave them as dependents until something else in their weakened body fails?
@MC I posted this elsewhere:
You bring up a problem that has a relatively simple economic solution, but is almost a political impossibility: optimal allocation of health care resources.
If we assumed a fixed amount of our GDP we're willing to spend on health care, and we optimized it for the best outcomes vis-a-vis increased quality and quantity of life, we'd have a vastly different system. We'd spend more on preventative care, lifestyle choices, and much less on hugely expensive treatments to add, in many cases, just a few additional months of life. Even funding of basic research would look very different.
The problem is that no politician will tell her constituency that she's not going to support funding for rare diseases, and will deny expensive treatments that add little additional months of life. Even if the odds are slim that an expensive treatment will do anything more than prolong the inevitable, it offers hope. Hope is powerful, and elected officials that deny hope do so at their peril.
There is understandable concern that blanket federal price controls over pharmaceutical companies would stifle some innovations. But why does that control need to be total? Why can't there be compromise on pricing for these orphan drugs specifically targeting rare illnesses?
I understand that some will argue, still, that innovation would be stifled (unless additional subsidies were developed for this class of drugs?). Some will just argue that anything that smacks of federal regulation is no good.
The problems noted in this op-ed are not confined to the USA: https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0927-y ("The European challenges of funding orphan medicinal products," Nov 2018).
The idea that drug companies should maintain such high profitability is open to serious question: https://www.the-scientist.com/features/how-orphan-drugs-became-a-highly-profitable-industry-64278 ("How Orphan Drugs Became a Highly Profitable Industry," May 2018).
Whatever direction we take, we should revise (if Congress can do anything with respect to health care) the Orphan Drug Act: https://catalyst.nejm.org/time-reform-orphan-drug-act/ (Dec 2018). There are a number of reasoned suggestions for how to do this. Like so much else, it will require a change in the administrative attitude of our existing federal government.
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@JSK..."The idea that drug companies should maintain such high profitability is open to serious question:".....The pharmaceutical companies are in competition for investment dollars everyday on Wall Street with companies like Disney and PepsiCo. That competition is driven by profitability. If the profitability of the drug companies goes down investors to a certain measure will switch their allegiance. When it comes to pharmaceutical profits the question is how important is it to have fewer new drugs and more movies and soda pop?
@W.A. Spitzer
Thanks for your comment. I would encourage people to read the articles linked in my original remarks above. There is no simple answer, but letting the drug firms control their own pricing has all sorts of problems.
One major difficulty is that because some of the drugs being discussed will never face serious competition, this effectively creates indefinite monopoly power that by many standards would be considered unreasonable. Should those drug companies have that power forever? A lot of people (who know about the problems) would say no.
1
This is ransom. Novartis is guilty of extortion (or something close to that - I'm not a lawyer) and our own government is complicit. The choice not to tie price controls to all the breaks these companies get to develop these medicines was exactly that, a choice, and the lawmakers who made it are killing people. Vote. Them. Out. This is just the sort of thing Elizabeth Warren is trying to stand up against. Only in America is this sort of thing permitted. Ask yourself why and who is served by this? Then follow the money and vote out the lawmakers who work for big pharma, big chemical, big oil, the NRA, or anyone else besides We The People.
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Efficacy? Do these basically untested drugs work?
Just was watching on Amazon Prime a movie about muscular dystrophy "Living Proof" about people trying various drugs -- all of which seemed to fail sooner or later often immediately-- and then the Stanford University medical center WITHDRAWING a treatment that did work at least in one person -- using stem cells isolated/harvested from a person's own body fat! -- that seemed to have positive results fearing reprisals from rapacious drug companies who wanted to produce a much more expensive kind of one size fits all version of the same. (The drugs here sound similar to many of the MD meds -- long term useless- the placebo effect can be very convincing.)
Greed. Raise personal income tax brackets: when you are taxed at 90% on the last ten million or so in income, just maybe you won't bother with it. No one should spend 450 million on a reputed Leonardo painting and not pay 1 dime in FEDERAL tax on it. (Thanks, Bill Clinton and Congress, for getting rid of the 10% luxury tax!) Tax all capital gains at the income level. #How to generate extra income for things Americans need
Now to read the article about the rise in STDs!
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@Auntie Mame. Yes. So we can Live! Live! Live!
Nowhere in this article does it state that Novartis is going to make a profit on this new drug and how much profit. It is just assumed that they will not only make a profit, but a huge profit. The average drug costs a company over $1.5 billion to develop and get to market. Given that there are so few patients that would ever need this $2.1 million drug, I have to question, how much money is Novartis losing on this drug? And yes, drug companies often never recoup their investment on a drug line, and even on an expensive drug, especially one that treats so few patients. This is why so few drug companies have a rare disease program.
Well, but what can you do? We don't want to be socialists. We need to have a capitalistic system. Profits and corporations are way more important than a few people. Indeed, it seems that profits are more important than the entire human species. It is clear that the billionaires we have now are the peak of human achievement. It is this that we have been striving for. If the serfs didn't agree they would elect politicians who are not owned by corporations.
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@Chris....The value of anything (please note I said value) is determined by the law of supply and the demand. This is a truth whether you are a capitalist a socialist or anything in between. You can't change that fact by changing your politics. Whatever political system you prefer, if you want to be successful, you had better work with that fact.
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@W.A. Spitzer. "He who controls the supply, controls the demand."
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@W.A. Spitzer Now I understand why hospitals price gouge people for band aids and cotton gauze. The sick people need them 500 times more! /s
2
And why do all drugs cost so much less in other countries? Why are Americans the ones who have to shoulder the cost to drug companies of research and development? If - and it's a big if - new drugs are really so expensive to create, the cost should be spread out worldwide, not just in the U.S.A. A long time ago I worked in the hotel business, and witnessed firsthand the lavish banquets and suites Big Pharma treated itself to. All billed to "Research & Development".
2
@Roberta..."A long time ago I worked in the hotel business, and witnessed firsthand the lavish banquets and suites Big Pharma treated itself to." Having worked in drug research for a major pharma for 30 years , I can assure that lavish banquets and such only occur when the CEOs and executive officers are involved. For meetings which just involve the scientists, I can assure you that the fare is far more ordinary.
@W.A. Spitzer And I can assure you of the contrary because I was organizing the events myself! And I witnessed a pharmaceutical company book THIRTY LIMOS to just sit in front of our NYC hotel just in case one of their guests decided they wanted to go somewhere, and of course most of them sat there unused. Such waste can not be justified and never in my 15 years in the business did I see any other industry engage in it. And they were all treating their executives and CEO's very well too.
This price tag is unacceptable. The FDA should get involved
2
In this case, Zolgensma is a complicated gene therapy and not a cheap to manufacture small molecule. It is prohibitively expensive to manufacture gene therapies at industrial scale with the safety levels required to treat patients. On top of that, it is extremely expensive to deliver the therapy as only a few centers of excellence have the staff and skill necessary. In addition, maybe only 10,000 children at the most would be eligible for this treatment. Even if the company sold it at cost it would be too expensive for most patients. A socialist system would never even offer this treatment as an option!
@Stefan then why is the public funding the research t start with if in the end it is unavailable?
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@pat cannon it takes a while for the price for new technology to come down. If have to start selling at a high price before costs drop.
Here is the deal. The people who run this drug company sat down in a room and decided TWO MILLION DOLLARS is what they will charge to cure a child who suffers from a devastating illness. Who are these people? How does this happen? How do they get away with it?
In my minds eye I picture a child suffering. His or her parents heartbroken. My minds eye also pictures the people who set the price at TWO MILLION DOLLARS. What if they had a child who was suffering and the drug the child needed was set at TWENTY FIVE BILLION DOLARS by some other group of people.
Novartis is evil. Simply evil.
@Bret
No, just a for-profit company. They must recoup their costs or else declare bankruptcy, shoot themselves in the head.
Like all economic systems, capitalism has its downsides. This is one of many.
If only someone made rich by capitalism would pay for its shortcomings! But, no, it's a one-way street: Once the cash is in my hands, it's there to stay unless I need a fifth mansion in some exotic location. Gotta be comfortable, you know.
Dostoevsky could have written a story based on this editorial.
A poor family, good people but, through no fault of their own, impoverished, know of a special drug, an injection of chemicals, that has a good chance to cure their sick child of a deadly disease but the drug costs two million dollars.
Two million dollars! What are they to do?
Their plotting becomes extreme. They think of robbing a bank. They ponder going to the heirs of the Walmart fortune, all billionaires many times over, but to beg for money, even to cure their child of its disease, is beneath their dignity. They hope the government will help them out but its wheels turn slowly.
As they look at their sick child, they wonder why it is, in America, the wealthiest country on earth, that only the rich (and often the famous) get to use expensive specialty drugs to cure their diseases, to save them from death.
Money! Money!
Money, or, the lack of money, allows some to live and others to die. It is a dark question, this one about money and health-care, who gets to live and who is handed off to death. A Dostoevsky needs to write this story with all the feeling and passion, with all the life and death, it deserves.
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I have a vested interest in Adult Stem Cell Regeneration LLC. That being said, adultstemcellregeneration.com shows that our health and longevity can be be improved by taking the adolesce pill. It shows almost certain promise that new protein, and subquently new tissue and rejuvenated organs are a resultof following a few months regime of taking the Adolesce pill and taking cae of oneself. We can, and should, assist our bodies to regain full body homeostasis: full body rejuvenation as if it were young again.
Wake up people. What the world needs is not 2 million dollar drugs to save children. It needs 2 trillion dollars worth of contraceptives to prevent children. The past authoritarian abuses of India and China are irrelevant. Do it right. Do it benignly. Offer incentives attractive enough to induce voluntary acceptance. Rocket science it is not.
1
Only government can take on and control the obscene prices charged by the health care system. The United States is the only advanced country in the world where individual citizens have to fend for themselves or suffer and die.
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I wonder waht thr drug will cost in Canada. Most pharmaceuticals are a fraction of their cost in Canada and other countries compared to their cost in the U.S. Why is that allowed to happen. If you wonde why health care is so unaffordable for so many in the U.S., you might start by comparing drug prices. Thus does not require Medicare for All, just common sense limits on the price of mecications.
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As horrific as this will read, at what cost is a life not viable? A $2 Million drug? I think the answer is clear. It’s God’s will.
No matter what they look like, no matter what they call or think of themselves, no matter what the flow of money may dictate, people who do not provide life-saving medicine when they have the ability to do so are monsters.
I wonder what the modern version of the snake oil salesman and his wagon will look like when it starts rolling through the streets? Oh, we have had a preview with the direct ad programs on television promoting drugs so that we can all run to our doctors and demand the latest, greatest. All they need to do is get rid of those pesky warning in the ads. I'm sure the Republican Party will see it Big Pharma's way.
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Demanding one to two million dollars for a fast tracked drug for desperately ill patients is nothing short of unconscionable extortion.
It is amazing the number of folks who commented here who seem to think that companies should develop drugs and then just give them away for free.
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@James what a ridiculous comment to make. just make the drug affordable, to all. that's all. besides, it was developed using taxpayer funds. this price was green-lit by the FDA, under the present administration. gotta wonder how much novartis is contributing to trump's reelction campaign.
@buskat
Sorry, not free but affordable, whatever that mean to you. Usually when folks use that term it really mean a price that causes the company that created it to lose money.
Here's to the system that has given us environmental destruction, global poverty, unsecure employment, racism, and predatory healthcare: capitalism. There is always the charade of democracy that we could vote for a humane system.
1
Towards the end of the second Bush presidency, President Bush heard about the fact that AIDS was running rampant in many African nations. Another mass wave of death were reeling the continent, and people were on the edge of pandemonium.
In an effort to address the issue, in the final days of his presidency, President Bush, authorized sending a relatively inexpensive vaccine be sent in mass, to countries plagued by increasing infection and death.
In the months following, Obama took office.
A few months into office, Obama took a trip to Africa, where he was greeting by politicians an locals, to arousing applause!
Many in the press touting the reaction of those people in parliament, to Obama, being the first African-American President to grace African soil. This was only
Shortly after, Obama was awarded the Nobel Prize. President rode off into the sunset, with neither a mention of his good need, not a word of slander against Obama.
This is what is missing in American politics.Politicians willing to rise to the occasion, to show compassion and put their egos aside to simply do the right thing.
I miss the days when people come together for the common good of all people.
Let’s hope it’s not too late, to make a comeback in 2020. If that were to happen, somewhere John Kennedy and George Bush will be looking down and surely smiling.
Many Americans from both sides of the political aisle will again be able to work together for a greater good This is what America is really about
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There is no such thing as a not for profit Pharmaceutical company. One need only to look at the Sackler family to see how craven such companies really are. The Sacklers lied about Oxycodone, which gave rise to the opioid crisis in our country. According to this editorial, Novartis may have manipulated the data -- Novartis may also have lied. Do you see a pattern here? Lying for profit. Rather like our current administration.
I understand the huge costs associated with the development of new medications, but if companies like Novartis received government incentives to produce new drugs, their costs are borne by the American public. It is a slap in the face to our citizens to then turn around and charge millions to ensure a profit. It is obscene.
The FDA also bears responsibility. The approval process for new medications was significantly relaxed at the beginning of the AIDS crisis. The process has devolved dramatically since the 1980's. It is much easier now than before to get drugs approved.
And the pharma companies take great advantage of this approval process. I am alternately bemused and horrified by television commercials that tout new drugs -- often for minor conditions -- wherein the side effects outnumber the benefits.
The FDA must return to tighter strictures and more thorough vetting of new medications. At this point, I no more trust the FDA than I trust pharmaceutical companies whose only motive is profit.
How do you balance a return to stricter FDA approval with the fact that millions of people are alive today who would not be, because the cancer fighting drug, Keytruda, was FDA fast tracked approved just a few years ago? Without fast track approval, Keytruda would still be awaiting approval, and those millions would be dead. I understand your concerns and anger, but we must never lose sight that saving lives comes first. A fast track drug that is ineffective is one thing, but a drug that is not fast tracked but would have saved lives lost is something that should never happen.
@Sparky
I understand your point, however fast-tracked medications often have side effects that cannot be measured quickly. I believe more care must be taken.
I am diabetic. My doctor prescribed Trulicity, which costs $800 for 2 ml. It was FDA approved after a 52 week test on 800 subjects -- not long by FDA standards. Trulicity caused me to become so badly dehydrated after one injection that testing showed I was in the third stage of renal failure. Which is to say it nearly killed me. Keytruda has similar life-threatening side effects. I personally wish they had taken more time to test these drugs.
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Pharmaceutical companies aren't allowed to advertise on Canadian television. So when we watch a US broadcast, usually CBS Sunday morning, we find the amount of pharmaceutical advertising amazing.
Sometimes that program's *only* advertisers are drug companies whose ads all show vibrantly healthy and active people pursuing lives of perfect taste and balance as a result of ingesting unpronounceable chemical substances with lists of possible side effects that sometimes include the phrase "may result in death".
America's drug culture is all pervasive.
4
As suggested by bigreenie, another solution would be to increase the NIH budget and remove legal impediments to allow that agency to undertake drug R&D, thus awarding such patents to the federal government. Presumably this would result in lower drug pricing.
Jasper
1
But the Government can not produce drugs, so it sells the patents to companies who then have freedom of pricing.
Expediting approval for experimental medicines is great if the patients are told about the potential dangers and benefits of the drug. I would not mind being experimented upon if this was my only chance to survive. Medicine is not about dispensing aspirin and platitudes. It is an experimental science that will eventually extend our lives beyond the current limit and cure the indignities of old age, not to mention all the multitudes of diseases that natural selection has inflicted upon humanity. And this costs money. All those complaining about the high cost of pharmaceuticals should read about the discovery of sulfa drugs that saved millions of lives and cost millions of dollars to develop. Or would you rather go back to the time when a scratch could be a death sentence? Incidentally, since muscular dystrophy is a genetic disease, wouldn’t it be cheaper to diagnose it in utero and abort defective fetuses?
1
Actually, sulfa drugs are pretty cheap, that's why they are so popular in the developing countries.
Seems that big pharma gets approvals far easier, and with less and less evidence, than were required historically. The marketing of drugs that require pages and pages of warnings, side effects etc. seem like little more than protections from prosecution when things don't work as advertised. On the other hand, the amount of effort and fear tactics against any sort of Marijuana based treatment makes it quite clear that big pharma has some influence,(political donations), that influence any sort of research that will not enrich them. IMO
@zorroplata..."The marketing of drugs that require pages and pages of warnings, side effects etc. seem like little more than protections from prosecution."....This assumption is incorrect. The warning label on drugs is mandated and approved by the FDA and includes everything that was identified in the clinical trial, even things that were very likely not caused by the drug.
"big pharma gets approvals far easier, and with less and less evidence, than were required historically."...In medicine the measure of a drug is possible risk verses potential benefit. It follows that a new blood pressure lowering drug where good drugs are already available that might be used to treat tens of millions of people should require very extensive and conclusive studies. Where as a potential life saving drug used to treat a rare disease for which no other treatment is available should require less scrutiny for initial approval.
There was a time when the National Institutes of Health were far more active than now in the development of novel drug therapies. Costs to patients could be better contained. At some point, this role was limited through legislation to the advantage of the pharmaceutical industry. That is reflected in this case, where a life-saving and new form of treatment is priced at 2.1 million.
6
@blgreenie The regulatory hurdles are so much more onerous today than 20 years ago that it is virtually impossible for any academic researcher with NIH grant funding to bring a new therapy to clinical trials let alone FDA approval. More often than not, intellectual property for new therapies gets licensed to big companies who can afford the testing, marketing, branding etc. required to get a new drug on the market. This results in less innovation and fewer new drugs for rare diseases (or, astronomical costs for those drugs).
Where is the evidence that this drug saves lives? There isn't any, and I strongly object to the headline.
According to the FDA 'the safety and effectiveness of Zolgensma is based on an ongoing clinical trial and a completed clinical trial involving a total of 36 pediatric patients with infantile-onset SMA between the ages of approximately 2 weeks and 8 months at study entry. The primary evidence of effectiveness is based on results from the 21 patients treated with Zolgensma in the ongoing clinical trial. In this trial, there are 19 remaining patients, who range in age from 9.4 to 18.5 months; 13 of these 19 patients are at least 14 months of age. Compared to the natural history of patients with infantile-onset SMA, patients treated with Zolgensma also demonstrated significant improvement in their ability to reach developmental motor milestones (e.g., head control and the ability to sit without support)."
This is extortion, pure and sinple
2
@Sallie...."This is extortion, pure and simple"....No, it is research. If no one bothered to discover and develop the drug, then it would not matter how much it cost because you couldn't buy it no matter how much money you had. If the drug works, others will follow, lives will be saved, and the cost will come down. And if you think it is so easy and so much of a rip off, then why don't you do it?
SO CHILDREN'S LIVES WILL BE SAVED Save for the $2 million cost. And it has been alleged that the manufacturer submitted a report to the FDA where some of the data is questionable. And the clock is ticking for those children born with this genetic mutation. What I say is, put the matter to a vote among the stockholders. Do they wish to contribute a part of their earnings so that children in need can receive life-saving treatment? In fact, I believe that stockholders are entitled to submit proposals to the board and to have them announced. It's time to bring those who profit the most into the loop: stockholders. To whom much is given, much is expected. Old fashioned--the idea of noblesse oblige. But it's not such a bad idea, giving stockholders a chance to participate in philanthropy. That said, Novartis must get its act together, along with the FDA. There is too much politics and not enough scientific rigor in the process.
2
I would be very interested to see an analysis by the NYT of how profitable the Biotech and Pharmaceutical sector is when compared to other parts of the economy. If, after accounting for the (many) failures which go out of business, the sector is making more profit per $ invested that makes a compelling case that protections favor the companies and with little cost to our future medicine supply we can demand more of drug companies (because the money won't go somewhere else).
I want cheap medicine which is easy to access, safe and effective to be on hand when I or a loved one gets sick. I also want my kids and grandkids to have better medicine than I ever did when they get to my age and I worry that the 'free lunch' everyone feels is there for the taking with tighter rules comes at the price that my grandkids get access to the same treatment I have...
Please, use the data to convince me I'm wrong!
3
@Adam. I believe that the tech sector (Apple, Google, etc.) has higher profit margins that the biotech/pharmaceutical sector.
Jasper
The cautions you raise are legit.
However, we must consider the health benefit on the person side of the equation. We have sympathy and want to help - that's a given. But, there's a dollars-and-cents benefit as well.
Most of the children suffering - quite literally - from these diseases depend on advanced medical technology for their entire lives.
Even when those lives are cut short, the annual cost of care generally exceeds the annual cost of the medication. In addition, most of these unfortunate little ones will end up on Medicaid.
Like you, I will be shocked and demand payback if the drug companies cooked the books on the benefits.
That said, the literature I have read indicates some of these cruel maladies are cured when identified early - often via public health screening for at birth for a raft of inherited diseases.
As a pediatrician, I've seen the bad outcomes - if the promise holds true, we should all be willing to pay the price to give a baby a chance at a normal life.
1
@Hugo Furst
Your sentiment that we should be willing to pay the price to give a baby a chance at a normal life is laudable from a human perspective. However, is there ever a price-point for a drug where we have to question its use? Since the cost for these treatments is born by the community at large, we also need a debate around the use of those dollars for pediatric benefits that might be available to a much larger group of kids than a single individual with equally compelling outcomes but for a larger group.
I also note that the focus on orphan drugs is a very deliberate strategy by life sciences companies to get a drug into the market faster and cheaper, and use that scenario to broaden the use of that drug for the treatment of other diseases.
@Hans In the case of this drug, it's a gene therapy for a very specific disease--spinal muscular atrophy--it will not benefit any other condition.
@Hugo Furst....Yes, the cost is outrageous; but does it work? And if it works, the medical community will have learned something and other better less expensive drugs will follow. I would remind you that the human genome project completed in 2003 took thirteen years and cost $2.7 billion dollars. Today, 16 years later, you can do the same thing in about two weeks and the cost is around $20,000 dollars.
Cap prices for unproven medications... so the Editorial Board would be fine with prices skyrocketing once a medication is proven to work? The early beneficiaries get it cheap, and then later patients have to pay much more?
Sometimes the NYT editors reveal that they don't know how economics work. Or, more accurately, that they don't care.
This piece would benefit from an estimate of the patient population, the cost that's gone into the drug development, an allowable rate of return on that investment especially considering failed efforts in other programs, etc. None of that appears to be considered.
4
A better approach on drug pricing, if the government wants to control pricing and yet incentivize drug discovery and cure, would be to payout bonuses to drug companies on drugs that are subsequently proven to be effective. Why not pay a drug company a huge bounty or bonus for a major game changing drug discovery by lowering the pricing on the front end with a potential of a huge payoff if the drug is effective?
For example, a safe and effective drug that would wipeout obesity would save society trillions and trillions of dollars in medical care alone over just a few years. I would have no problem in paying a huge bonus to a drug company for that drug. I do not want to reward drug companies for ineffective drugs, but we must reward drug companies handsomely for drugs that really work, save lives, and significantly reduce or even eliminate down line medical and other costs. For example, the new hepatitis c drugs that actually cure hep c, so that the patient doesn’t need a $400,000 liver transplant and remain on expensive anti rejection drugs, and yet, still has a much lower life expectancy. Those drugs are expensive, yet a fraction of the cost of the alternative, and the patient will have normal life expectancy without further treatment, and keep their own properly functioning liver. Lower pricing, but with a bonus incentive could be an effective approach.
"Today there is an increasing number who can’t see a fat man standing beside a thin one without automatically coming to the conclusion the fat man got that way by taking advantage of the thin one. So they would seek the answer to all the problems of human need through government." -- Ronald Reagan, 1964.
Seems to capture the general view of the NY Times Editorial Board. They propose more regulation, more government control, and now price controls. How about less government and more freedom? Where I live, we actually believe that doctors and patients can decide if the evidence is sufficient for a drug to be prescribed and taken. We don't need government officials in some far off capitol who never see patients interfering. And we actually believe that private insurance companies can make good decisions about what medicines they will cover in their policies. We don't need need government officials in some far-distance capitol deciding that either.
We also actually believe that pharmaceutical companies are a force for good in society and have done wonders to improve our lives. This new drug from Novartis cures a lethal disease. Maybe one of these government officials -- or members of the Editorial Board -- wants to go sit with the mother of such a child and tell her they are making the decision for her -- and they have decided her child should die.
1
@Fly Over Flora. Pharmaceutical companies, like all companies, continue to operate because they make a profit. I can only assume that they figured they could extort the insurance companies or the government to pay for this drug because of the sympathy level and the age of the sufferers. Life can be extremely painful in its cruelties, but sometimes the cure (maybe a cure?) just doesn’t add up for anyone — including the sick.
Well, I guess you, guys, should pay for these expensive drugs that could or could not save lives, especially while you are figuring out how effective these drugs are. Why should I pay for overpriced drugs of questionable efficiency through my ever-increasing insurance premiums? At such rate, soon I will have no access to cheap drugs that save millions of lives.
@Fly Over Flora Do you remember Thalidomide or are you too young?
The "fox and the grapes" classic with a moral comes to mind here. What we can't reach probably isn't good for us, anyway.
Any "cure" or remediation that's sent through our gut doesn't affect just what ails us. It can mess up our gut, and poison us with its side-effects.
Bloodstream injections save the gut, but who knows for sure what happens to other cells on the way to those bad ones.
We still await a perfectly-aimed bad cell-fixing. One so focused that the good ones are left alone.
It's not just children's lives. It's all our lives. As for the price, government tax incentives for pharmaceutical pricing is needed.
What good is a cure or remedication that can't be administered? Probably sour anyway.....
1
The US has to realize that there is no real market in healthcare. You cannot depend on the goodwill of CEOs. People need to wake up. In all other democratic countries there is some sort of public control of drug prices. It is also about time to stop dreaming about the supposed superiority of the private sector over government programs. Why not having federal institutes doing the research and making the drugs patents available for free?
7
It would be helpful to know how and upon what bases drug companies make pricing decisions? Is that process overseen? regulated? Other than monopolistic greed how is it legitimate to raise the price of an existing, proven, and long-on-the-market drug 400% and claim research as cost recovery? If this overblown pricing represents the capitalist way then it's time to regulate with a harsh hand.
3
Will patients get a refund if they develop ALS as adults? The price should be the cost of producing the treatment until it has been shown to be effective (thru adulthood).
@A J SMA is not the same as ALS.
@WJ SMA treated as a child may very well 'present' as ALS as an adult. How long do you think the (one) treatment can be effective? We don't know....I know what SMA is.
I have to say that the announcement by Novartis that this drug will cost $2.1 million per treatment sounds like an advertising campaign to me. Novartis must know that only a handful of parents of babies born with this affliction would ever be able to afford this cost.
Obviously new drugs are never developed for just a few dozen patients. Pharmaceutical companies would go bust very quickly if that was the case.
I suspect that Novartis received all of the three government incentives mentioned and probably others that are better left unmentioned.
Added to this is the advantage of being the company that has brought only the second gene therapy to be approved by the FDA to market. The announcement of the price tag only piles more attention on Novartis as the pioneering white knight come to the rescue of the suffering few. I am sure that the 'cost' will diminish greatly in the near future.
1
@RHR: Like the cost of Epipens?
It is important that the government get a just percentage of the profits from drugs to which we have contributed. This money could be used to improve health programs or to subsidize the purchase of expensive drugs. There are dozens of businesses that receive substantial subvention from the government while politicians decry the help that are given to citizens.
5
@Frank Casa
Do you feel that way about Silicon Valley companies and their profit making from the government created internet? Go for it!
Isn't the most important thing to provide essential care and medicines to all?
If so, we have to make sure there is a way to pay for that.
It is WRONG to focus on how to provide for the care of rare disorders and ignore the gaping holes in disease care coverage that affect many in our country.
Stephen Rinsler, MD
4
@Stephen Rinsler
Why should children born with devastating inherited genetic diseases suffer because society is unable to fix the "gaping holes in disease care coverage" for the masses? And many of these children suffer dramatically more (before they die) than patients with acquired diseases such as diabetes or cardiac disease. Should the private drug companies be forced to stop working to save children's lives because society is upset at the system in place or the high cost of generating these therapies?
Believe me, if we could develop a gene therapy that would make people eat less, exercise and stop smoking (certainly the best way to improve care for the masses), we would. But in the meantime, I'm proud of the work that gene therapists are doing.
and incidentally, @nytimes, why is all the coverage we see focused on the inscrupulousness and "bad behavior" of the drug companies instead of the incredibly positive stories of the families of children whose lives are being saved? All this talk of coming together and unity in this country, when what you really want to publish is content that allows people to be upset at each other... I don't get it.
Nick Weber, PhD
1
I tell you why. The pool of money is limited. Say you have 2mln dollars, and 100 sick people to care. You can spend 2 mln to cure 1 person, or to cure 50 people for same amounts of money, what will be more humane? Don't pretend that money is falling from the sky, it comes from our pockets making drugs for more common diseases less accessible. Let's save one child with rare disease, and let thousands children die from common ones.
@yulia
The pool of money is limited because of priorities and greed. Don't be naive in saying that there is not enough money in this country to save people with rare diseases. It's about priorities and nothing else. Does the public want to spend money on healthcare for their neighbors or does the public want to spend money on Netflix?
Big Pharma is not doing anything illegal. They are simply taking advantage of a political system that has degenerated into quid quo pro capitalism.
Our founding fathers never envisioned a political ruling class that remained in office indefinitely. Rather, serving in government was supposed to be a privilege and a public service.
The only way to stop this abuse of power is to enact finite term limits for all Federal elected officials so that they do not get too "cozy" with industry lobbyists, ban all political contributions and publicly fund all election campaigns with a set amount of campaign dollars that levels the playing field.
Our founding fathers are spinning in their graves at this abuse of power.
20
@Tony
I agree absolutely and I am sure that a large majority of Americans feel the same way. And this begs the question as to why this system that does so much harm to the people, who are America and who do not want to be conned and robbed, been thrown out?
1
@Tony I totally reject being denied the opportunity to vote for someone because they have previously been elected. If you want to limit someone's term, don't vote for them.
2
Realistically, the number of people who have this disease and could afford a $2.1 Million price tag is incredibly small.
They must be counting on insurance or some other government agency to foot the bill, otherwise they would have never invested the up front money to develop it. Corporate welfare on display
Drug prices took off immediately after Medicare started paying for prescriptions- if you want to lower drug prices get Medicare out. It will decimate the profits of the pharma industry but they won't suddenly stop selling drugs because we won't pay medicare prices. In a business you can only charge prices that your customers can afford to pay. 99% of Americans could not afford to pay the prices charged so the Pharma industry does the next best thing, get the government involved.
Drugs are expensive because everyone says they are so it must be. That is not the case in a true free market
8
@Hugh G Actually, drug prices took off once health insurance became the norm. Why not keep Medicare but remove the grotesque prohibition against negotiating drug prices? Most countries with universal health care successfully negotiate much lower prices.
And there is no such thing as a free market when someone is facing a life-threatening disease.
5
@Terry My memory may be fuzzy but it seems back in the 80's when we had to buy amoxicillin for ear infections, we had to pay the $40 out of pocket. $40 was a lot of money for sure but it was worth it to get your children to stop screaming and sleep. If they would have set the price at $200 they wouldn't have sold any and parents would have been pushing their pediatricians for something cheaper.
It wasn't until later that insurance started to cover prescriptions.
Another recommendation: audit the drug companies and cap their profits from any one medication. It's obscene to rake in *excess* profits on the back of sick people or babies who will not see their 3rd birthday.
A reasonable profit would keep those drug companies in comfort, rather than being the one of most profitable industries --- all because of the market distortions caused by sick people with no other options.
4
Make no mistake. Many pharmaceutical companies are publicly traded companies. Their fiduciary responsibility is to the shareholders so maximization of profit is the goal, not developing drugs that work to benefit patients. In the case of "boutique" pharma firms, the company is often beholden to its private investors, who want X return on their investment monies. We like to attribute altruistic motives to drug companies, but that is a fallacy. Follow the money, always, if you want to know the truth.
35
@Karen K
Exactly. And not just pharma. Any publicly traded company is only beholden to their shareholders, not the general public and greater societal good. There is nothing wrong with making a profit, but if we want things like this case to change we have to have a cultural shift where we ask - how much is enough? Is it more important for me, the shareholder, to get additional dividends or that the company balance profits with actually walking the talk of their marketing materials.
1
In theory public companies are beholden to their shareholders. In reality, they serve top management with their hefty compensation packages. Capitalism has a new definition - socialize risk and privatize profit - and senior executives of pharma companies are the largest beneficiaries of this.
8
@Karen K And pharma companies make the bulk of their profit in the US, greatly funded by trillion dollar deficits.
The only difference between democrats and republicans is that the democrats at least admit they are sponging off of the government.
1
There are important trade offs involved when developing a drug for a rare disease. The patient pool for trials is small, for some of the diseases listed, the need is great. But, the issue can extend to other therapeutic areas.
Use drug resistant bacteria as an example. The rule for antimicrobial drugs is that “if you use it, you will lose it.” And the more you use it , the sooner you will lose it. The NIH had an outbreak of drug-resistant klebsiella that killed at least 9 patients. There is an urgent need for anti bacterial s that work through a novel mechanism in order to get past resistance. Yet, when such a drug is developed, it will be important to hold in reserve and use only when absolutely needed. How does the developer of the drug make a profit?
And then do you know how drugs are priced? That is done by a young stock analyst at Lloyd’s. It seemed like every new drug for MS was priced at $1000,000/year wholesale. Why? There are about 500,000 patients: if the manufacturer can capture 10,000 patients and that will generate $1,000,000,000 in sales.
Well before a drug is approved, stock analysts predict what sales a drug will generate and the stock price will already have those sales factored in when the drug gets approved. That is why you do not see much rise when a drug approval is announced and a big drop when an approval is denied or a Phase 3 trial bombs.
That is our system. How do you change it?
8
There are important trade offs involved when developing a drug for a rare disease. The patient pool for trials is small, for some of the diseases listed, the need is great. But, the issue can extend to other therapeutic areas.
Use drug resistant bacteria as an example. The rule for antimicrobial drugs is that “if you use it, you will lose it.” And the more you use it , the sooner you will lose it. The NIH had an outbreak of drug-resistant klebsiella that killed at least 9 patients. There is an urgent need for anti bacterial s that work through a novel mechanism in order to get past resistance. Yet, when such a drug is developed, it will be important to hold in reserve and use only when absolutely needed. How does the developer of the drug make a profit?
And then do you know how drugs are priced? That is done by a young stock analyst at Lloyd’s. It seemed like every new drug for MS was priced at $1000,000/year wholesale. Why? There are about 500,000 patients: if the manufacturer can capture 10,000 patients and that will generate $1,000,000,000 in sales.
Well before a drug is approved, stock analysts predict what sales a drug will generate and the stock price will already have those sales factored in when the drug gets approved. That is why you do not see much rise when a drug approval is announced and a big drop when an approval is denied or a Phase 3 trial bombs.
That is our system. How do you change it?
1
@Profbam the same way every other industrialized country has done.
Creating a drug -- any drug -- that sells for two million dollars for one dose, is creating nothing anyone can ever use.
Savage corporatism will lunge towards grotesque levels of profit, claiming their "costs" justify the price.
Extreme profiteering is self-defeating. It is not sustainable. But this doesn't stop casino-player moves on the part of entrepreneurs operating in the so-called "free market". It isn't free, and it's not a level-playing field. It's the Wild West.
The land of opportunity is closing the noose on the poor, on low-wage earners, and on people who used to be part of what we called the middle class that no longer exists.
oz.
7
It escapes me why a specific drug should "pay entirely for its development", as if this was the only product a company was making. As in other fields, it would appear logical to invest part of the (huge) profits from "simple" drugs in research (although investing in research/long term benefits has become rather less popular in recent short-term-focused times).
1
Pharmaceutical pricing has become an obscene caricature of US-style robber-baron capitalism (and a little Swiss, too). The entire industry is built on a public/non-profit infrastructure of medical schools, the National Institutes of Health and a regulatory system which was built to prevent thalidomide-type tragedies.
8
It is unfortunate the Times opines on a subject it little understands. For example, capping prices for unproven medications. It is not feasible for a company to spend the typical billion or more over 10 years to gain an approval only to then see its ability to recover its investment artificially limited. My company has spent $1.1 billion over 18 years to develop a best in class product that had first year sales of $6M and will take five years to get to about $200M. The Times makes profits every day!
4
@No
Your argument might make some sense in the context of another drug. The example here of an accelerated approval with little data though is the Sarepta DMD drug. Look into the approval process for this drug--it's all available on the web. The FDA review division said no to accelerated approval, the independent Peripheral and Central Nervous System advisory committee said no, and a dispute resolution panel convened by FDA's Acting Chief Scientist said no. The FDA Division Director overrode all of that to give accelerated approval. Maybe the NYT writer knows all of this. Shouldn't the company be required to do the required follow-up studies very quickly and as an experimental drug (no cost to patients) since, as agreed to by nearly all win the review process, there was little to no evidence that the drug works??
@No:
i understand little of how "big pharma" works.
but i often hear that quite a number of companies
spend n times more on advertising than on R&D,
presumably bc of commercial competition.
i do think people's [good] health,
clean air, clean water, safe food and housing, i.e.
their decent livelihoods, should not be left to the
"free" market - which is never free.
it's high time for an overhaul of "democracy",
unless we want to leave what's left of it to the
cannibal capitalists who couldn't care less ...
@Stephanie Wood:
... and do EVERYTHING to distract people's
attention away from them and pretend it's all
the fault of "illegal immigrants".
while the top brass' tax legislation has all the
loopholes written into it for them to be able to
gleefully claim: "what we do is all legal!"
I am no fan of big pharma - they are a central evil of capitalism, socializing costs and privatizing profits and government funding.
But the article neglects to pass the fairness test by simply not reporting on the number of people who'd take the drug - a single time - at the ridiculous cost. Is is 10 or 10 million? The answer makes a difference.
I am tired of pharma companies getting away pricing things at "what the market will bear" and believe the market should bear a whole lot less. I believe that our government should apply laws which cover fair pricing - Robinson-Pattman for instance -and develop a few new ones.
But if you cannot show that at a given price the company can recoup its investment quickly and the rest is gouging, you haven't made an argument.
We cannot afford to make weak, emotional arguments. The propaganda machine of big business is well funded and wide spread. By all means expose the Robber Barons. Expose them more with hard data.
3
@Cathy
But that's the whole point: there is no "market" for these sorts of drugs. How many people have $1 or $2 million lying around? Without passing these costs to someone else, either through taxes or insurance premiums, these sorts of drugs are beyond the reach of even the most ardent patient or parent. And that doesn't even scratch the surface of whether these costs are justified in the terms of the best use of scarce resources. One or two million dollars buys a lot of preventive healthcare that would benefit thousands of people, not just one.
6
Sounds to me this is The Boeing Max all over again. The difference being that the people killed by the MAX were not expected to die while using the product, but patients receiving last hope drugs are already far gone and if a drug "that has the potential to cure (spinal muscular atrophy)" is ineffective, that will never be noticed. There's a major difference between "has the potential" and is proven effective (and safe) when the price is $2million per patient.
1
The point is that both in Europe and America the public, democratic authorities have mostly reduced and/or given up their power of control and rigorous verification of the big pharma drugs manufacturing. They substantially trust the the pharma producers to act well and in the interest of the patients.
It is sad that the public drugs agencies have insufficient staff and limited resources to fulfill at best their tasks.
1
@renarapa:
it is sad, yes.
but could there be "method in't" as well?
@°julia eden
Maybe!
There are many instances of the abuses here. Beyond the price, we now hear of manipulated data. It is time for the FDA to change the control of data so that all data is received by the FDA from the beginning of clinical testing. This will be cumbersome, but we were wrong to rely in the drug manufacturers to report honestly. (So side effect, unfavorable results etc.)
3
It is a double edge sword: if you delay the approval for more testing and data, you deny access to those in need who cannot wait. The cost to development, testing and the approval process is expensive and for every successful drug there are many that fail. The economics are what they are.
4
I keep wondering about not only the survivability of these children when no one talks about long term prognosis of children with what seems like lifelong continuing disability. No mention of that. When we or our family make the cost of preserving just life without any concerns for disability or what the future is likely to be yet are willing to spend any amount for same, we clearly make ourselves vulnerable to the for profit treatments of any kind, drugs, surgery, etc. This is not just a dollars question but a moral question. Instead of spending millions to save a child with this disability maybe it should be spent to discover what causes happened to create it then a real problem would be solved.
6
Well I see it this way. You have 600 kids born with this each year. If it truly was a cure, 2 million is doable. You easily spend 100K per year on 1.7 million people in LTC beds and don't blink an eye. These are people with zero chance of improvement. I'm all for the "common good." Take my LTC money, I will choose medical aid in dying, my time is done, "to everything there is a season". Only 20 people like me covers 1 kid. You spend over 1 million on people with pancreatic cancer with an abysmal cure rate, so just to get 1 extra year of survival. No, 2 million is really a drop in the bucket.
4
@Detalumis
OK, please make your contact information available to families who need the drug (assuming it really works, which is a big assumption) and provide the $2.1 million to people who ask.
The point is - most individuals don't have that kind of money - it has to come from the government. And our current government isn't concerned about providing decent living conditions for children in detention, let alone a small number of kids with a rare genetic disease. This issue is a matter of national priorities. I'm no socialist but it should be abundantly clear in every way imaginable that our current representatives have little interest in pubic health - especially when it might limit corporate profits
3
@CSD:
getting hold of a tax evader
or money launderer, or two,
should be mega-easy to do.
big tech could donate pocket money.
if there were a will there'd be a way.
But the fundamental information is not presented: how much money did it take to develop the drug; how much to produce it; and how much revenue will be generated at the $2.1M price tag.
Yes $2.1M is a a huge amount of money ... and emotionally the image of a sick child versus this number is very upsetting...
But with the information presented there is no way to conclude if this is reasonable or unreasonable...there is the implication of impropriety based on the eye popping number $2.1M but no direct proof of impropriety...
This type of therapy is at this point the highest of high tech...applied to a small enough group of patients...it could be very costly...
12
@Mdb288
The "proof of impropriety' can be found in the soaring stock prices and eye-popping CEO salaries in the drug industry. Over and over again, the claim that the money is needed to fund research has been proven false. It's greed, pure and simple.
2
And all new drugs benefit directly or indirectly from medical research funded by NIH and other agencies of various governments.
16
@Kevin Cahill, excatly. It is simply impossible to tease apart company from tax payer in many hot fields of biomedical research. Therefore, the number 2.1 as well as claims from the other side that this should be less because of heavy subsidies from federal agencies during the R&D phase will remain murky at best.
And Zolgensma is just the beginning in gene therapy ...
1
"One of the top explanations that drug companies give for the high cost of their products is that it’s expensive to get drugs through clinical trials and across the regulatory finish line."
They also complain about the financial impossibility of bringing a drug to market at a low list price when the supply chain is jam-packed with people making money off higher list prices. No mention here of the arcane system of getting drugs to actual patients; nothing about "pharmacy benefit managers"; nary a word about rebates.
The list price is not really the issue. Manufacturers paid $166 billion in discounts and rebates last year, according to Adam J. Fein, writing in the WSJ. "PBMs pass the bulk of the rebates to the health plans they represent. Plans are then able to offset part of the cost of nondrug treatments, and reduce their premiums.
"Plans could choose to use these rebates to lower the premiums of the sickest patients, whose frequent purchases of expensive drugs allow manufacturers to give rebates in the first place. Instead health plans prefer to apply the rebates to the price of average plans, hoping to compete for business through lower premiums."
This is a situation where the sick subsidize the healthy. Action to force redirection of rebates, or to clean up the supply chain and rid it of bad incentives, could begin fixing this. But with lobbyists at their throats, I'm not sure Congress can do that. Legislatures require SOME privacy -- many sunshine laws need repealing.
3
In a normal society no patient should ever need to know, let alone pay for, the cost of a life saving or any other drug needed to combat disease. A reasonable maximum out-of-pocket cost contracted with the insurance should be all an individual patient needed to care about.
9
@InNorCal
In what "normal society" do you think this is the case?
In reality, the cost has always been and will always be a part of the consideration, in combination with the benefits provided.
It maybe that this is considered collectively, as in countries with universal health care,as opposed to individually, but the value of the drug (or procedure) is always evaluated.
And this doesn't mean that prices should not be negotiated, or in some places controlled, It doesn't mean drug companies aren;t out to maximize profits. It just means that there is always some reckoning as costs rise.
How severe does the cap need to be on the price of a drug when uncapped the price is $2.1 million -- even when treatment is a single dose? 10%? 50%? At 90% the price for Zolgensma would still be $210,000. The same question can be asked for life-sustaining drugs that must be taken for a life time.
Limiting expedited drug approvals, capping prices for unproven medications (and why not for expensive, proven medications?), and demanding more and more timely post-market studies are all smart ideas generally. But for a drug like Zolgensma or for that matter, something like HIV medications, a price cap and more data as a pre-requisite for lifting the cap is not the solution.
There are times when the government should use its power of eminent domain to take ownership of the drug/s. Compensate the company for the investment and a one-time reasonable profit. The government can require the manufacture to continue to produce the drug or make another arrangement (even selling the rights to produce and market the drug to another company) and make it available at a price that doesn't make a require defining "affordable" in a way no one understands.
8
For rare drugs like this, what would it cost for the government to simply purchase the rights to the drug? Take the development cost, multiply it by a factor of 2 or 3, for the onetime purchase price. The company makes a profit of 100-200% on their profit. The company retains research knowledge and experience to develop new drugs. Individuals get drugs they can in no way afford themselves, or through their health insurance.
8
FACTS
1. Orphan drugs would not be developed without a price per patient expected to create gross revenue roughly equivalent to that expected from a non Orphan drug.
2. The data problem w/Zolgensma related to preclinical experiments that did no involve the final drug The FDA is making an example of Novartis for having run afoul of standard, expected process. Careful not to impugn the drug.
3. The FDA has its biases and power agendas, as industry has its profit motives. Novartis should have reported immediately. Because the problems were not related to the drug, Novartis took a calculated risk, to not slow it's getting to SMA kids. Maybe the FDA would have delayed, maybe they wouldn't have. It is a certainty that parents and caregivers of kids who got the drug, without delay, view Novartis as heroes.
4. Whoever wrote that Novartis picked Zolgensma up for a song must pay a lot for his/her concert tickets: they acquired the company that made it for $9 billion.
7
Jonas Salk developed the polio vaccine in the 50s and never patented the drug nor earned any profits from it. He was a true hero.
Contrast that to our own ghastly time and the horrors of Big Pharma. It is like a different universe we inhabit now.
59
Does anyone actually believe the fairytale spun by pharmaceutical industry that medical research in general and pharmacology research in particular would grind to a halt if they weren't allowed to make truly obsene profits?
19
Corporate patents on life saving drugs are abominable. If the AIDS research in Wisconsin had been corporate instead of non for profit, millions would be dead from the disease. There should be a two to five year limit for any drug and then a generic should be allowed to be produced. Our for profit medical establishment is a shame on our nation.
26
@Thankful68
Without a patent no company would produce drugs in the first place. Sorry but you can't separate out profit.
2
@Thankful68
Actually, I think just the opposite would work better: Instead of a time limit for a patent--which, by definition, forces a company to maximize profit over the short term, resulting in maximum cost per patient--why not allow a patent to continue until the company recoups a REASONABLE profit? No more orphan drugs.
@Terry
Then tell us what a "reasonable" profit is and whose standards we use to determine it. Frankly I can't think of a more subjective term.
1
A businessman with unlimited resources will invest in every project that promises a positive return, but when resources are limited he rationally allocates the available resources to the projects with the highest returns on investment,
Our medical care's financing 'system," a disorganized mess of public and private mechanism for collecting and distributing money to pay for medical products and services, has limited resources. When treatments are covered by insurance they are socialized by definition, which means that the humongous prices paid for the therapy described here is paid for by everyone. So the cruel but rationale question: is this the best ROI for $2.1 M available among the many alternatives ?
3
Why shouldn't saving lives make you a billionaire? Because it's morally and ethically reprehensible, that's why. Making a billion off something that people don't need to survive is fine by me. But, when it's something they do, then the profit motive becomes as abhorrent as it is degenerate.
The argument that the manufacturing of drugs for obscure diseases wouldn't happen unless people made a mint off it, is pure, unmitigated hogwash. Because it implies that scientific advancement isn't possible without a massive profit motive. Tell it to the Russian who discovered the Periodic Table.
This profit argument is as spurious as it is unfounded when one considers how many life changing discoveries have been made without it.
So, what's the solution? Like, healthcare, or anything else that is a necessity for survival - get the private sector out of it - period.
This kind of medical research and development should be the sole purview of the government. Funded by tax-payers for the benefit of all.
If you want to get rich, fine. But, don't expect to do it on the pain and misery of other people's lives. Particularly children.
To do so, violates the primary ethic of the medical profession.
You want a lot of money? Then go make it somewhere else.
38
@Chicago Guy What about making a billion dollars selling something you do need, such as food? Is that okay? What about making a billion dollars making cars? Is that a need too? 'Need' cannot be consistently defined, and that is the crux of the problem. It's subjective and always will be.
2
@RS
You're obfuscating. If there was one only one kind of food, my argument would apply. Since there isn't, it doesn't. People can live without a car, so that doesn't apply either.
A "need" is exactly that. Something you can't live without. The children who "need" this drug will die without it. That's the point.
There is nothing subjective about it.
1
Gene editing or other gene therapies are the perfect example why we need socialized healthcare.
While I believe that $2M for a gene therapy is too much (more on that later), economically it somewhat makes sense. NICE, the British agency that determines economic feasibility of drugs, sets the price for a Quality Life Year Earned at 50,000 British Pounds. Multiply that by 30 and you are at the $2M. (Economic feasibility of rare disease drugs are calculated differently by NICE, but the concept somewhat still holds true).
Only socialized medicine has an incentive to pay this much money because it reaps the long term benefit from it.
The economy for Exondys 51 is different, and besides, this drug is probably the biggest hoax in recent drug development approvals.
The argument against $2M for Zogensma is not only that Novartis got the drug in an early development stage for practically free. Neither is it that the approval process is faster, which is also true. The fact is, that rare disease drugs trials are cheaper to conduct. While it may be difficult to recruit 10 or 20 subjects for such a disease (after all it is rare), only such a small number is needed. The patients typically have not had any other drugs or treatment that could confound the clinical trial outcome results.
An approach to make this more affordable would be to amortize the payments to the pharmaceutical companies and make those payments dependable on clinical outcome for each patient.
5
@TT Your analysis makes sense when resources are available for any beneficial treatment -- in this case you've argued that that the drug's current price of $2M breaks even when it results in 30 years of high quality life.
But what if alternative uses of $2M dollars, eg vaccination, produces hundreds or thousands of years of high quality life. Which project do you pick when you have to pick?
1
@Eben
That is exactly the reasoning behind a Quality Life Year Earned. For the British system, where medical services are socialized and paid from the same pot of money as, say defense or road improvements, the question is: Should I spend $50,000 on medicine or $50,000 to build a traffic light. Up to $50,000 medicine wins, after that it might now.
Please understand, the real system is somewhat more complicated (i studied it in detail in 2010). But the underlying logic is about right.
@Eben
That is exactly the reasoning behind a Quality Life Year Earned. For the British system, where medical services are socialized and paid from the same pot of money as, say defense or road improvements, the question is: Should I spend $50,000 on medicine or $50,000 to build a traffic light. Up to $50,000 medicine wins, after that it might now.
Please understand, the real system is somewhat more complicated (i studied it in detail in 2010). But the underlying logic is about right.
By the way, I got the term wrong. The term used is Quality Adjust Life Year (QALY), not Quality Life Year Earned). Memory sometimes is deceiving.
First you battle the doctors for months to get them to prescribe the drug you believe might save a life you value when that life has no particular value to a medical profession that needs to move on to the next case and the next insurance payment. Then you find out a price of $2 million has been placed on this life, no guarantees? At least I got to see my friend finally get her first dose of the immunotherapy drug that might have helped if she could have gotten it sooner. She was so weak by then that it only hastened her death. And at least those who loved her didn't have to also go crazy imagining $2 million could have saved her.
4
It's pretty simple. The United States health care delivery system, which includes the price of pharmaceuticals, is just glorified and legal extortion. "Your money or your life."
26
good old capitalism - it wonderful for everyone.
2
Your money - all of it - or your life. What a business model!
Adding insult to injury - even to those who generously donated to find a cure for Cancer or Alzheimer's, the drug companies will profit handsomely off of that research and the donors that made it happen don't even get a discount as patients in spite of their role in financing the research.
A new ad campaign requesting donations to fight Alzheimer's highlights the idea that the first person to be cured is out there. Indeed, he/she will likely also be the first one to die because he/she couldn't afford the drug after all - in spite of all the money he/she donated to the cause of finding the cure. If he/she does get the treatmet and survive, he/she will be so broke ...
4
Some of these drugs, treating diseases that afflict one in a million, are now routinely advertised on TV channels that used to pitch nothing more expensive than garden hoses and acne cream. So much programming is now sponsored Big Pharma, so indiscriminately, that the only conclusions are that these companies are swimming in cash and that the return on even a single course of treatment is a major ROI.
4
Many basic scientists dedicate their careers to the work on which these drugs are based, but their motives are underminded by this kind of exorbitant pricing....the recent artlcle about Banting and Best and their selfless contribution of their discovery of insulin was a beautiful example, and their are many working with the same ethical committment today.
5
This feels like ransoming of pharmaceuticals, which seems to be the rule rather than the exception when new drugs come to market these days. It is important to realize that the costs are not born just by patients or even insurance companies, but ultimately by everyone who pays insurance premiums, of which one form is taxes for Medicare and Medicaid. It also translates into higher co-pays and deductibles as insurance companies struggle to stay solvent in the face of extortionate pharmaceutical pricing. The beneficiaries are the shareholders of Novartis, who at some point may wish to consider whether any ethical person would be a part of an enterprise that does this.
A lot has been written on how this can be stopped, but the political system remains paralyzed. The headlines are about Presidential embrace of Clinton conspiracy theories, not about creating solutions to these problems.
So it will go on. There will be episodic eruptions of outrage, and then the pharmaceutical companies will realize that is it is just headlines; another summer squall of angry words. The people who need the medications and their insurers will be stuck having to pay up. Money will flow from the average people struggling to pay taxes and insurance premiums to the investors, devastating the economy, the competitiveness of our industry and, worst of all, our basic belief that government is there to protect people from this type of attack on the well being of its citizens.
7
For $2.1 million, you could buy a lot of insulin (which is quickly becoming unafordable for many patients, and they are dying because they can’t pay for it). Or you could buy a lot of anti-malaria drugs; malaria kills 3,000 children per day.
I’d probably feel differently if it was my child who was ill with spinal muscular atrophy or another rare disease, but unfortunately no resources are infinite, and health care resources are particularly limited.
13
Yes let's do save lives with knowledge not everyone can be saved. When we demand save every life we put ourselves at the mercy of the pharmaceutical greed or any other supposedly life saving treatment.
Also, drugs that are researched with FEDERAL (or State) dollars must have their prices set in coordination with the funding governments. It is unfair to help pay for the research and get socked in the head with an unreasonable price tag. And it's immaterial whether an insurance company ponies up for the cost or not, high insurance payouts mean higher premiums for everyone. We must stop being responsible for funding pharmaceuticals without having a say in their cost.
10
@BillG basic research is paid for by the federal government. The real cost is taking basic research and translating that to a therapy through clinical trials. That is paid for by companies. Your argument is a red herring.
6
This is exactly why private enterprises should have no place in the medical arena. If you can't afford your dream Ferrari, that's too bad, but ultimately inconsequential. Access to life saving medicine though should not rely on the depth of your pockets.
33
@Nima
Maybe in a fantasy world with unlimited resources but we don't live in that world.
1
There is nothing new under the sun, at least for as long as I have been involved within the medical field as a retired hospital RN and now a wife of a man with advanced PD. The first fundamental truth is the manipulation that drug companies have over the public. Perhaps just as much if not more than health insurance companies, they wield powerful control. Where is the ethics and honor if drug companies create life-saving medications, and they are unaffordable? You think insurance companies will foot the bulk of the cost? No way, it falls to the consumer, the desperately ill patient, who will be left without, left to die, or in the poor house after losing all assets. This op ed writes at the end something that can not be overstated in its importance: Demand more data. There it is. I would wager that there is not one reader who has not found out the hard way that the "prescribed" drug either did zilch or actually produced adverse side effects which led to more problems than before. We have a long way to go, this nation of ours when it comes to health care. I find that shameful.
26
$2 million is a fair price. As long as a reasonable, long term repayment plan could be worked out - with insurance footing part of the bill, parents covering another part, and the person who benefits paying the rest back when they reach adulthood (like a mortgage), it would work. If I had SMA - or a similarly devastating disease, such as ALS - I’d consider a cure to be a man-made miracle and would gladly spend years repaying the cost .... and not begrudge a single penny of it.
6
@GBR Where is the next Richard Nixon when we are in desperate need of price controls.
It is obscene that a drug meant to save children's lives will be so prohibitively expensive that most will not be saved.
It is obscene to even suggest such an outlandish price - who can afford even one dose?
It is an obscene truth that drug company profits take precedence over the life of a child.
37
These drugs do not come out of a vacuum. Drug companies benefit from taxpayer funded basic science research. Therefore since they are just standing on the backs of others they should not be permitted to charge obscene prices.
44
Not quite - mostly it’s just kernels of knowledge, but most funding comes from VC’s and the drug companies themselves.
1
Drug approval and pricing is such a swamp of uncertainty, it's impossible to make definitive statements about the process. Yet, the price tag of $2 million per case seems hard to justify. Is the standard "whatever the market will bear?" It seems to be cruel barter for the life of a child.
10
Makes sense. Read Marcia Angell's book "The Truth About the Drug Companies."
5
Drug companies are the railroad monopolies of our times.
6
With a Medicare for All system, the government could guarantee that it would purchase vast quantities of drugs from companies at greatly reduced prices. Although the pharmaceutical companies would make less per individual drug sale, they would still have plenty of room left over for profit since they would be able to virtually eliminate their advertising budgets. Couple this system with tax credits and government R&D funding for companies that create life-saving (yet unprofitable) drugs, and we will be able to preserve innovation while increasing affordability.
Plus, pharmaceutical advertising is inefficient for the economy. Why should anyone be making a medical decision based off a 30 commercial?
7
Everyone always talks about the huge costs of RnD for creating new drugs, but no one talks about the fact that pharmaceutical companies spend a TITANIC 30-50 percent of their revenue on marketing and advertising.
9
@John Louis
A fantasy. There is no point making "vast quantities" of drugs for which there is only a small need. The most expensive drugs treat rare diseases, and have no other uses. They are expensive because the R&D costs must be spread over a tiny market. Insurance then spreads the cost over the general public, who might (but very likely won't) need them themselves.
4
At $2 million, this drug is an incredible bargain. Kids with devastating neuromuscular problems could easily run up $2 million in in medical bills before succumbing in misery at a very young age. Now they will (hopefully) be normal, become productively employed, and dedicate their lives to paying taxes and insurance premiums.
14
@Troglotia DuBoeuf Bargain for who exactly? If private insurance doesn't cover it, patients have to pay on their own. I don't know many middle class families who could afford this drug. If you mean it's a bargain for the government as far as Medicaid patients are concerned, you may be correct. But I'll bet the company that developed this drug could still survive if it were $1 million a treatment.
6
And how do you know that will be the outcome for these children with no proof and assume no long term disability which may make them less than able to contribute to society?
1
Well, the people receiving the drug will certainly consider it a bargain! How much would you pay to live a normal life instead of being confined to a wheelchair until you suffocate or die from inhaling your own vomit? But nobody cares about that--it's the $5 co-pay that really irks.
What’s the point of developing drugs to cure or prevent diseases if a person in need can’t afford the drug?
78
You say hundreds of people will benefit. That is the whole issue. Like all drugs, this drug likely cost 9 or 10 figures to produce. But there will be very few buyers. So, the cost must be extremely high, or it would be uneconomical to produce it or research similar drugs for rare diseases in the future. It's a tough situation! We obviously want cures for all diseases, but someone has to make the drugs to cure for rare diseases. And no one will do so if they cannot make money. Hence the high cost.
9
It does not cost 9 or 10 figures to produce..... but the salaries of the executive suite members and TV ads do.....
It is perfectly appropriate for pharmaceutical companies to make a profit. And 20 years ago, I would have come down strongly in favor of drug companies. But making a realistic profit (having taken a risk on developing a drug) is distinct from pure unmitigated greed, which is what the pharmaceutical companies have been demonstrating in recent years. Perhaps greed and unsavory business practices were there all along but I am increasingly disgusted by the indiscriminate marketing (of opioids and direct to consumer marketing), skyrocketing costs of existing generic drugs (that have no development costs) and unending shortages of multiple drugs (ranging from dirt cheap generics to sterile salt water). Deceit about data is nothing new either. Other trials in the past have been designed to shine the best possible light on data but some data now is almost impossible to interpret (e.g. the recent approval of esketamine). Unfortunately, once a drug is approved, few patients are willing to risk being randomly assigned to placebo or an older comparison drug. And companies have no interest in post-approval studies that may show less (or no) benefit. Without more regulatory constraints on drug companies, patient safety and scientific knowledge will continue to be compromised and patients and taxpayers will foot the bill for drug company executives, stockholders and lobbyists. Enough is enough!
19
Most patients would benefit from placebo as much or more (no side effects....) than from drugs. And if you look at the productivity of the industry it is pathetic. Most large companies create 1-2 drugs themselves and survive by acquisitions.... this is an industry run amok spending millions on lobbying rather than worrying about patients
It would require TWO THOUSAND eople to not get sick for a year and each pay a completely UNAFFORDABLE $1,000 a month (give an take after overhead, etc) in healthcare premiums for ONE patient to be afforded this medication.This is clearly unsustainable --such prices can only live for a few years before insurers go bankrupt.
Is a healthy patient worth $2M. Yes, but the price cannot be imposed on healthcare plans. At this level of cost only the Federal Government can afford them.
Yet along Federal dollars should come regulations and transparency. Pharma then should choose behind ultra-expensive medications under federal scrutiny, or reasonably priced medications under a health plan's scrutiny.
6
@Yoandel "At this level of cost only the Federal Government can afford them." So in your view the current national debt of 22 trillion is not high enough? (smile)
@Ludwig
2 million is approximately 0.0000001% of 22 trillion. Not much of a factor.
The one recommendation I don't agree with: "Reserving these programs for companies without a record of bad behavior would go a long way toward accomplishing that goal." This sounds arbitrary and subject to favoritism. Any limits should be based on scientific and/or medical criteria.
4
Full disclosure: I'm chronically ill with a slowly progressing neuromuscular disease, currently called mitochondrial dysfunction.
The challenges of navigating life with a progressive disease are way more complex than understanding that those of us who are ill simply can not afford to pay for treatment due to lost income from disability.
So why bother to develop these drugs?
22
Drug companies never seem to want to share with the public what it really costs to develop a drug or a therapy. In many instances, those costs are enormous and the company can justify the price it expects to charge. In others, e.g. insulin, they are not and the huge price increases are merely profit-taking. Drug companies want something from the government - patent protection. Why not tie the time they are protected by a patent to some formula which takes cost, price, etc. into account?
10
@Mark
They get more than patent protection. They get money from our taxes so we get to pay for the R&D and for the drugs. It is way past time for the Big Pharma to get regulated. $1,000,000 for an unproven drug?
25
@Mark
A large share of the money goes to marketing. Only two countries permit obscene direct- to- consumer drug marketing: the U.S. and New Zealand.
29
What happens if some years down the road the disease recurs or more treatment is needed? Will the patient have to pay again? 2 million dollars is not cheap even if it's spread over 5 years. That's more money than most of us will ever see in our entire lives. Is that money going to be reinvested to improve the treatment, to follow the patients, to cover them if the treatment results in complications later on?
I think that the costs are outrageous no matter what. In fact there's a name for this: highway robbery.
20
@hen3ry: Or, in terms of the 19th-Century railroading, "whatever the traffic will bear."
We have socialized medicine, but privatized profits. What we need is a larger risk pool and a set of price controls that will extend the benefits to all, along with the costs.
4
Where are the billionaire one percenters? Certainly whatever their field of interest they could have a go-fund-me group to help these children, members of which would be restricted to fellow billionaires. A million dollar contribution from each should cover this and any other "rich orphan" medication for years to come.
8
@Joan In California: It's sad how often I see go-fund-me efforts to pay medical expenses. Funny, but I don't see these in Sweden or Denmark. (And yes, I've looked.)
36
@hammond That's true, but cutting-edge drugs are generally also not available in these European countries until years down the line. With universal healthcare always comes some form of cost-rationing.
However, since we have no idea whether some of these drugs even work, why should anyone take them? The drug companies should pay for all doses until their efficacy is actually proven.
4
@gus: "The drug companies should pay for all doses until their efficacy is actually proven."
I like this idea. I mean, I really like it! We'd have to agree on the metric required to show efficacy, but I think there's something there.
I need to look into your other claim about what's covered in European countries. It's important and I don't know much about it.
1
Full disclosure: I have a small oncology company that develops new cancer therapeutics.
The challenges are way more complex that this editorial suggests. Sure, let's require companies that get fast-track approval to continue with clinical studies, or risk de-approval. Sure, let's make sure that the entire approval process is governed by scientists and clinicians who have absolutely no conflicts of interest.
The costs of these new drugs, and how to contain them, is vexing. One option, of course, is to have all new development funded and staffed by government institutions, thereby eliminating the profit motive. If this happens, I, and many others in the field, will find other lines of work. It's hard to exaggerate the huge differences in the environments of government labs vs. agile and highly creative small companies.
Okay, so maybe we can cap prices. Fine. But then many investors will move to other, more profitable sectors; or at least lower the risks of biopharma investments by supporting incremental improvements of existing therapeutics over new, first-in-class drugs. No one puts money in this space without the expectation of a significant return.
I don't know the answer, I just know it's not simple.
57
@hammond As you know, many if not most medical developments already are funded by the government, through university research labs and grants to private institutions. "Agile and highly creative small companies" do not, for the most part, do this work, as you surely also know.
What line of work would you pursue instead?
76
@Law Feminist As a scientist who worked for years in academia doing NIH-funded research, and now having moved to doing drug development in biotech, I cannot agree that the business of drug discovery and development is being done using mostly govt funding. The currency of academia is publications and further grant funding, not drug approvals. The laser focus and efficiency, not to mention the orders of magnitude higher levels of private funding, allows small biotech to pursue actual drugs at a pace that far outstrips any academic research. As @hammond mentioned, this private funding comes with strings attached, and at the end of the day they are investors looking for a return. The scientists stuck in the middle working on this problems are not the ones making the big bucks, and there's not much to be done since the small biotech system is really beginning to churn out very interesting drug candidates that WILL be efficacious and safe - and when they do, they often get licensed or bought out by large pharma who can sell for whatever they wish...
46
@Law Feminist: You are repeating a common argument. I paraphrase: The NIH already pays for the development of these drugs, so why do private companies get the profits?
This claim is false. It is true that much of the basic scientific research in the disease biology is done in academia. But the long, very expensive and risky path to an approved drug is largely done in the private sector. One study assessed capitalized and out-of-pocket costs of drug development at $1.8 billion and $870 million, respectively[1]. I know of no academic lab that is funded at anything close to that level. None.
Regarding work, I'd go back to the tech sector or clinical medicine before I'd take a job in a large government lab.
1. Paul, Steven M.; Mytelka, Daniel S.; Dunwiddie, Christopher T.; Persinger, Charles C.; Munos, Bernard H.; Lindborg, Stacy R.; Schacht, Aaron L. (2010). "How to improve R&D productivity: The pharmaceutical industry's grand challenge". Nature Reviews Drug Discovery. 9: 203–14.
23
Eandbee is correct that the manufacturer is charging $2 million; that is not the "cost." It's like when a Beverly Hills mansion or Manhattan condo goes on the market for $100 million. That's not what it's worth; it's worth what someone decides to pay for it.
But there is a cost to these posted prices. Imagine you work for a modest-sized company with 1,000 employees enrolled in its health plan. Imagine the plan is self-insured, as many are. And one of the employees has the horrible fortune of having the one-in-6,000 baby with spinal muscular atrophy.
The next year, all 1,000 employees share the cost of treatment: $2,100 each in increased premiums.
This already happens with cancer and other treatments. I've worked for a company where someone well-known and well-liked gets a terrible illness... and a year later, premiums go up because of that.
I think the main problem here is the extravagant cost of so many medicines and treatments, but the second problem is the small size of so many insurance pools. I'm not advocating Medicare-for-all, I don't think it's the right time for it, but this is an example of why some version of universal coverage is needed.
4
@George Judson You are doing the same thing which universities do -- they talk about 'how to pay the tag', and not the underlying 'reduce the cost'. Novartis should be held to account, and just like gun control, laws must be enacted to hold costs AND price under control
15
@George Judson
Not that this makes situation much better, but the cost of Zolgensma (it is a one-time treatment, not really a drug) is spread over 5 years, so the annual cost to the plan is less. The problem to the plan is pretty similar to the one with say the cost of neonatal care for the babies born very early, or someone going through the multiple bone marrow transplants etc. In the end it is the society that bears these costs - but for the plan in question, paying a moderate premium for the reinsurance is worth it.
2
@Tamza
Not at all. As I said, the main problem is the extravagant costs, and extravagant costs should be reduced.
I don't like the title of this editorial. There is no evidence offered to objectively state that the drug 'costs' $2 million. More accurately, you should say that the manufacturer is 'charging' $2 million. There is a big difference between 'cost' and 'charge,' and that is a big reason why we are where we are with our 'health care' system.
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@eandbee
I don't like the title because it is unknown whether this treatment will save lives.
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@eandbee At the moment, no insurance plan would cover this treatment, so the cost is effectively $2 million out of pocket. Once it is covered, this cost is rolled into health care premiums - then we all bear the 'cost' of this treatment. A drug costs all of society precisely as much as the company sells it for at first.
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@gus What they're trying to say is that using the word "cost" is misleading because it could mean that it takes 2 million dollars to actually make the drug. However, cost, price, charge are all synonyms.
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As an employee of Michigan Medicaid, the public insurance plan is mandated to cover these types of drugs, whereas most commercial insurances exclude them from coverage. This basically shunts the costs of clinical trials to the public and the risks of unproven drugs on to the poor.
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@Eric
You are absolutely right. The poor ( often children) become Big Pharma guinea pigs. There’s a reason Public Citizen’s Health Research Group says to wait seven years before trying a new drug- it takes that long for the real side effects profile to become clear.
3
The orphan drug law should be repealed altogether. It's the reason why colchicine, which has been used safely and effectively against gout (for which there is no other effective treatment, for many people) for hundreds of years, costs 3 cents a pill everywhere in the world except the US, where it costs $5 a pill.
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@Brian Harvey
Sorry to disappoint, but Colchicine is a generic medication not having much to do with orphan drugs. As there is currently only one generic supplier in the U.S., apparently the expected sales volumes even at $5 per capsule (not a pill...) don't cover the expense of regulatory filings and setting up the production.
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@cobbler
But it used to cost only pennies per pill in the US. Now the company is price gouging because our US politicians permit it.
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@cobbler That makes no sense. It's been sold w/out FDA approval for years, apparently easily paid for by the cheaper price. Why should it cost more to produce it under a new name? Who's paying for "regulatory filings"? The FDA reviewed & approved it under its own initiative to inspect a lot of unapproved drugs.
Simple solution to this pharmaceutical company and FDA problem, Medicare for All.
This is what Bernie Sanders, Elizabeth Warren, and other Democratic leaders have been advocating for years. More control by the government over the entire healthcare industry. And no for profit business enterprises in this sector.
That would eliminate the price gouging, and a host of other maladies currently in place. It will be hard convincing the public, particularly those with company insurance plans, but put Democrats in charge and we have a chance.
Vote 2020
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@Dr. Trey
And some other people will still work hard, be paid a reasonable salary and enjoy their work and knowing that they are benefiting humanity because they aren't in it just to get rich. Don't worry, I'm sure you'll still be able to hang out at the yacht club and the polo grounds and figure things out.
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@Dr. Trey your response is quite callous and you forgot to mention that the scientists working at most of these companies or academic institutions do not make fortunes if they make discoveries that are useful. They have signed away their rights as part of their contract with the company or institution. The money goes to the employer not the employee unless it comes back as stock if it's a company that shares.
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@Dr. Trey Thankfully, that was not how Frederick Banting, James Collip, and Charles Best thought, so people in Canada (or those who travel there) can get inexpensive insulin treatments. There are plenty of people who want to make their community or the world better rather than rake in obscene profits. If these drugs are only available to those who can pay $2.1 million, is it really any different than not developing them at all?
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