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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 Not only was there a misleading published article with respect to the results of Study 201/202147 –which has never been retracted—but Sarepta also issued a press release relying on the misleading article and its findings.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.
http://endpts.com/senior-fda-officials-warned-that-eteplirsen-ok-would-lower-fda-standards/
http://blogs.sciencemag.org/pipeline/archives/2016/09/20/sarepta-gets-an-approval-unfortunately
https://en.wikipedia.org/wiki/Eteplirsen
(Score: 3, Insightful) by Fluffeh on Thursday September 22 2016, @12:30AM
Wow, this is really scraping the bottom of a barrel here. Allowing a drug like this to go ahead purely because the company needs cash? What happened to the Oath and all that?
(Score: 3, Interesting) by bzipitidoo on Thursday September 22 2016, @01:20AM
If Donald Trump can really shoot someone and not lose any voters, this ever more brazen profiteering is certainly not going to get on the radar.
This MD drug is just like our current Alzheimer's drugs. They don't cure Alzheimer's. They don't even slow down the progression. All they might do, for some patients, is improve daily functioning, a little.
Drugs for high blood pressure and high cholesterol actually work in controlling the condition, but, they share the highly profitable property of not actually curing anything. They'd have you believe you have to take the medication for the rest of your life. Doctors might make some noises about changing your diet and getting more exercise, but for many patients the magic pill is easier.
(Score: 4, Insightful) by Some call me Tim on Thursday September 22 2016, @03:25AM
If Donald Trump can really shoot someone and not lose any voters
Oh for crying out loud. Hillary could slaughter a school bus full of children on the horns of the Wall Street bull, in broad daylight, and the media would spin it as a right wing anti-abortion protest.
Answering absurdity with absurdity.
Questioning science is how you do science!
(Score: 4, Informative) by skater on Thursday September 22 2016, @12:50PM
Err...you know he actually said that, right?
(Score: 2) by Reziac on Friday September 23 2016, @03:48PM
Saying isn't doing.
And there is no Alkibiades to come back and save us from ourselves.
(Score: 2) by Magic Oddball on Thursday September 22 2016, @09:36AM
Drugs for high blood pressure and high cholesterol actually work in controlling the condition, but, they share the highly profitable property of not actually curing anything. They'd have you believe you have to take the medication for the rest of your life. Doctors might make some noises about changing your diet and getting more exercise, but for many patients the magic pill is easier.
High cholesterol is caused by either a mixture of genetic & environmental factors, disorders like hypothyroidism, or medical treatments like dialysis. To quote Wikipedia:
"Diet has an effect on blood cholesterol, but the size of this effect varies substantially between individuals. Moreover, when dietary cholesterol intake goes down, production (principally by the liver) typically increases, though not always with complete compensation, so that reductions in blood cholesterol can be modest. Genetic abnormalities are in some cases completely responsible for hypercholesterolemia, such as in familial hypercholesterolemia [which] affects about one in five hundred people."
Regarding high blood pressure, 90-95% of cases involve inherited susceptibility to risk factors (age, weight, salt intake, etc.), but no definitive cause. So different people's cases will be due to different causes, what works great for one person might have no impact on the next, and for the people that don't have any of the 'lifestyle' factors, medication may be necessary to really bring their BP under control.
(The other 5-10% have hypertension due to a condition like renal insufficiency, and in those cases medication's typically needed unless the underlying disorder can be cured.)
(Score: 2) by Reziac on Friday September 23 2016, @04:01PM
In fact, hypothyroidism causes high blood pressure two different ways -- by causing calcium to be deposited in arterial walls, and by changing the tone of blood vessels so they're less flexible. I expect its effect on calcium (sucks it out of the blood and deposits it in arteries and joints) also has an effect on potassium balance, which in turn affects BP.
Also has a direct effect on whether cells can uptake cholesterol (when they can't, more is left in the blood).
Yet another reason why a complete thyroid workup should be the first thing done, not the last resort.
And there is no Alkibiades to come back and save us from ourselves.
(Score: 4, Insightful) by edIII on Thursday September 22 2016, @01:36AM
The medical industry has nothing to do with patient well-being, patient's rights, or ethics. Or even morals.
I'm not surprised when "more profit" is the sole incentive in this country to do anything. That doctor got paid off, patients will suffer, the disease still rages, and profits keep funneling to a few parasites.
Standard.Operating.Procedure.
Just waiting for civil war to start so we can kill all the Pharma execs with guillotines.
Technically, lunchtime is at any moment. It's just a wave function.
(Score: 0) by Anonymous Coward on Thursday September 22 2016, @10:28AM
You're just a shill of the guillotine industry! :-)
(Score: 2) by davester666 on Thursday September 22 2016, @06:09PM
"I Promise To Do Anything and Everything To Make A Buck For Myself" - oath of a CEO. The "For Myself" part is normally not spoken aloud, just understood by everyone involved.