Speedy Drug Approvals Have Become the Rule, Not the Exception

If this same 'fast track' were based on merit, no matter what the treatment, bloodletting, siple blood donation, would replace most drugs.
Iron deprivation/iron reduction/phlebotomy/bloodletting, whatever one calls it, has been shown to be recommended for more diseases than any treatment in history. Merit. Based on merit alone.
One in three of us is slated to manifest metabolic syndrome.
"Bloodletting Makes Comeback for Metabolic Syndrome"

I guess I should not be surprised that the NYT readers all favor bigger, more intrusive government. But, these commenters all seem to think that new drugs are ineffective, higher priced, and inadequately tested. However, if they were the ones waiting for a drug to treat a terminal illness, they would prefer fast to safe. My complaint is the FDA likely kills more people by delay than they save by exhaustive testing.

What is worse is that the FDA and its bureaucracy is trained to say "No" or "Wait" because the risk to them is in approving a drug, not in delaying a drug. On top of that, there is no immunity from a lawsuit after the drug passes the FDA. When all is said and done, the FDA provides little value add and much delay to the process. Milton Friedman was complaining about that decades ago.

Multiple sclerosis drugs cost upwards of $50,000/year/patient and not a single one has adequate data to show that it works at all. NO drug has ever been shown to slow the pace of disability in MS. But thanks to marketing there is some widespread belief that these drugs work and we spend hundreds of BILLIONS of dollars per year on them. That is a broken system - not just wasting money on things that don't work but removing the incentive to create drugs that actually do. My husband's doctor, a leading researcher in MS at a major medical institution, finally admitted what I as a scientist have observed from these drugs study- there is no evidence any of these drugs work!

The FDA is a farce....an embarrassment to the medical community! Horrendous decision making! No patient safety concern! Drug after drug, approved with mind boggling adverse sideaffects! No different than selling snake oil out of a covered wagon! Shake your head, "no way", but this is not the USS Enterprise....everybody has been brainwashed into believing drugs are as good as the Tricorder!

Example: Hypnotics such as zolpidem cause accidents and huge workups for nonspecific complaints! I have seen many patients get MRI's of the head, CT scans of the head, chest, abdomen and pelvis, an exploratory abdominal laproscopy, and a cardiac cathertization.....all because of zolpidem! No kidding!

Lots of snake oil out there!

Unfortunately, there are too many financial conflicts of interest between medicine and the medical device and drug companies. The FDA evaluates drugs based on the information that the drug company chooses to give them. Side affects and adverse reactions are too often minimized. As a patient, I would refuse to try a new drug until it had been on the market for 2 years - unless I was terminally ill.

The most important breakthrough policy change required is at the FDA! There is no point in approving targeted drugs based on surrogate endpoints when the overall survival of the patients remains equivalent to the older drugs except the cost has now increased considerably!!! The recent approval of so called Breakthrough drug” (Palbocilib) in combination (not by itself) with older approved drug letrozole, for breast cancer showed that PFS (progression free survival-a surrogate endpoint) doubled compared to the older drug. However, the approval is not based on improved survival and on top of that the hematological toxicity increased considerably!! For example, Rates of grade 3/4 neutropenia were increased from 1 to 51 percent and leukopenia from 0 to 14 percent. In the study, 71 percent of the patients required a dose interruption and 35 percent required a dose reduction; adverse events led to 10 percent of discontinuations.
Neutropenia is a low neutrophil count. Should the neutrophil count falls below a certain level, a patient becomes immunocompromised and faces the risk of serious infections resulting in early death. The older hormone drug therapy alone (letrozole) has always been considered a relatively benign treatment, however the addition of new drug makes hormone therapy a lot less benign. This doesn’t look like a transformative drug as the FDA claims for a so called “breakthrough Designated drug to be…..

"The popularity of the programs is no sign that drug companies are doing anything wrong."

I would assume, quite legitimately, the default should be the opposite. Just common sense.

Fast tracking drugs has little to do with pain, illness or concern for patients. It is, in the main, a way for drug companies to make big money and use real humans as a guinea pigs in human experiments. The FDA has been turned into Big Pharma's lap dog by Congress members, mainly GOP but not exclusively.

What a sad commentary on an agency that used to be the Gold Standard of food and drug safety.,

Fast Track is great until one of these Fast tracked drugs causes someone harm or doesn't work at great expense. Then the cries will begin Who missed this ?who was asleep at the switch?

As long as drug companies can keep introducing new drugs, they can control their stock price ... so it doesn't matter much if the products actually help anyone.

That is the tipping point at which the "free market" actually becomes monopolistic, and begins to suppress the creation of better products.

Clinical trials cost a lot of money, so the possibility of qualifying for a smaller study, and fewer phases is appealing to Pharma. The FDA imposes the same technical standards for any study, and they are quite high.

The standards were raised in the 80's and approvals stretched out to 12-18 years. You could view expedited Approval as a return to the middle. The reality is that even a Phase III trial with 2,000 patients is not going to find all the side effects and problems. It will only show that the drug works.

It's the Postmarketing surveillance phase that finds the issues with a drug in the general population. I would argue that this is where the focus of the FDA should be.

Antibiotics need more incentives and even faster paths to approval than current Expedited Pathways allow for -- no new class of ABX has been invented in over 3o years... The SuperBugs seem to be winning, ie, killing more of us. Hopefully Congress will get its Act together and pass some meaningful legislation.

16,000 bleedout every year in the US from OTC NSAID's ....considered safe and effective
98,000 dead from avoidable medical errors every year in US
How many from adverse reactions to properly prescribed and taken Rx drugs?

I don't believe that even the "slow track" system for FDA drug approval does enough testing on particular drugs to determine whether their advantages outweigh their negative side effects. I have refused to take many meds that were lauded as wonder drugs (or drug combinations, such as hormone replacement therapy, which doctors prescribed for menopausal women for years and which are now believed to cause more harm than they prevent). Often when I read drug package inserts, I find, as I recently did, that a particular drug was tested on, perhaps (and this is a large number) 3 or 400 subjects (many of whom, you have to read the fine print to find out, dropped out of the study because of negative side effects) for 6 months or even a year. At the same time, doctors have had me on one such med for 14 years (before another doctor instructed me to stop it) and another for 11 (with horrible long-term side effects that other doctors did not recognize as such and proceeded to try to prescribe yet other, relatively untested, meds (with their own side effects) to counteract. It's not that I am against taking all meds. I have taken some for many, many years. But I am finding, as I age, that more and more are being prescribed 1) before they have been tested adequately over the long term, and 2) for conditions for which they are not needed (such as the "new" guidelines which would have something like 75% of all people over 65 on statins.

The FDA is just an appendage of big pharma and cannot be trusted to regulate pharmaceuticals.

Breakthrough Therapy designation is clearly a boon for pharma, as it guarantees a dedicated response team at the FDA. But it comes at a cost, literally. Each BTD team at the FDA is largely unavailable to review any other drugs. The FDA needs more resources.

Fast tracking some drugs makes absolute sense (especially given the lack of foresight of pharmaceutical companies to much beyond their bottom line).
I was appalled at the recent column of shortages of even routine medications (antibiotics) at hospitals. To see commercials (why do we allow advertising pills?) for new medicines to treat dry eye and other ailments and know that these are money makers for our profit driven companies...well, it makes one a bit cynical. Add to that, personal experience. A drug that went from $45 to $292 in two years. Another, in 5 years: from $5 to approximately $110.