from the false-sense-of-hope dept.
Eteplirsen received approval for use as a Duchenne muscular dystrophy therapy despite the FDA review team concluding that the treatment was unlikely to show any benefit for patients.
Dr. Janet Woodcock's (Director of the Center for Drug Evaluation and Research) decision was heavily influenced by the "parading diseased children in front of the cameras" and was made before the FDA's review team completed their analysis.
Part of Dr. Woodcock's rational for approval included the stock price of Sarepta (the pharmaceutical company responsible for eteplirsen):
She opined that Sarepta in particular "needed to be capitalized." She noted that [Sarepta's] stock went down after the AC meeting and went up after FDA sent the June 3, 2016 letter. Dr. Woodcock cautioned that, if Sarepta did not receive accelerated approval for eteplirsen, it would have insufficient funding to continue to study eteplirsen and the other similar drugs in its pipeline.
FDA Commissioner Dr. Robert Califf, Acting Chief Scientist Dr. Luciana Borio, and Dr. Ellis Unger, the Director of the Office of Drug Evaluation, all opposed the approval but Dr. Califf declined to overrule Dr. Woodcock's decision.
Dr. Unger argued that the approval was unethical and counterproductive:
By allowing the marketing of an ineffective drug, essentially a scientifically elegant placebo, thousands of patients and their families would be given false hope in exchange for hardship and risk.
Dr. Borio argues:
Granting accelerated approval here on the basis of the data submitted could make matters worse for patients with no existing meaningful therapies — both by discouraging others from developing effective therapies for DMD and by encouraging other developers to seek approval for serious conditions before they have invested the time and research necessary to establish whether a product is likely to confer clinical benefit.
[...] [Sarepta] has exhibited serious irresponsibility by playing a role in publishing and promoting selective data during the development of this product. Not only was there a misleading published article with respect to the results of Study 201/202147 –which has never been retracted—but Sarepta also issued a press release relying on the misleading article and its findings.
Dr. Derek Lowe, from In The Pipeline, agrees with Dr. Unger and Dr. Borio that the drug is "unlikely to provide much benefit, and is reasonably likely to provide none at all" and that the drug "may well be [$300,000 per year] worth of placebo".
Note: Bold was added by the submitter.
As the world knows, we face an emerging virus threat in the Wuhan coronavirus (2019-nCoV) outbreak. The problem is, right now there are several important things that we don't know about the situation. The mortality rate, the ease of human-human transmission, the rate of mutation of the virus (and how many strains we might be dealing with – all of these need more clarity. Unfortunately, we've already gone past the MERS outbreak in severity (which until now was the most recent new coronavirus to make the jump into humans). If we're fortunate, though, we'll still have something that will be worrisome, but not as bad as (say) the usual flu numbers (many people don't realize that influenza kills tens of thousands of people in the US each year). The worst case, though, is something like 1918, and we really, really don't need that.
[Ed note: The linked story is by Derek Lowe who writes a "commentary on drug discovery and the pharma industry". He is perhaps best known for his "Things I Won't Work With" blog entries which are as hilarious as they are... eye opening. I have found him to be a no-nonsense writer who "tells things as they are", holding no punches. The whole story is worth reading as he clearly explains what a coronavirus is, about the current one that reportedly originated in Wuhan, China, what could be done about it, how long that would likely take, and what can be done for those who have already been infected. --martyb]
Previous Stories Referencing Derek Lowe:
Machine Learning Comes to Biochemistry
Ignition! The Funniest, Most Accessible Book on Rocket Science is Being Reissued
Another Failed Alzheimer's Disease Therapy
Marathon Pharmaceuticals is Part of the Problem
Lobbying Results in FDA Approval for Controversial Drug
"Right to Try" New Experimental Medicine and the Value of Experts
Cancer Hazard vs. Risk - Glyphosate
A Terrific Paper on the Problems of Drug Discovery
Things I Won't Work With