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A U.S. Food and Drug Administration advisory panel has recommended against the approval of Sarepta's eteplirsen, a drug intended to treat Duchenne muscular dystrophy (DMD). Two other experimental DMD drugs have already been rejected. The recommendation came despite the emotional testimony of children who had apparently seen improvements due to the experimental treatment. Representative Mike Fitzpatrick, R-Pa. also spoke on behalf of a constituent with DMD. However, concerns were raised about the small sample size of the trial:
"I am very sorry to say that approval of eteplirsen based on today's data would set a dangerously low bar for all drugs in the future," said Gottschalk, a senior fellow at the National Center for Health Research, a nonprofit in Washington, D.C. "Treatments for rare diseases can be proven on small samples, but not based on 12 patients in a poorly designed study with ambiguous results. These boys and their families deserve better."
The problem, FDA scientists said earlier in the day, is that, due to its small size and design, the study Sarepta submitted to the agency cannot prove that eteplirsen deserves credit for the boys' ability to remain on their feet. Although the company says dozens more boys are now taking the drug, its case with the FDA rests on only the study involving the 12 boys in the orange t-shirts. Except for the first 24 weeks, all of the boys in that study have been taking the drug. With no long-term placebo group, Sarepta chose to compare them to untreated boys from a registry of DMD patients. These types of studies tend to have more favorable results than studies that randomly assign participants to the active treatment or a placebo, Dr. Robert Temple, deputy director of the FDA office that evaluates nervous system drugs, told the advisory committee.
The advisory panel's 7-3 recommendation (with three abstentions) is not the final word on eteplirsen, but the FDA generally follows such recommendations. Sarepta's shares plummeted on the news.
(Score: 3, Insightful) by anubi on Thursday April 28 2016, @09:52AM
Sure seems like a small sample to me, too.
I would have a problem *denying* treatment using this if the patient wanted it this way, but I would have a really hard time vouching for it as an effective treatment based on so little evidence.
What I saw looked like a witch doctor or faith healer could make like statistics - as well as have equivalent testimonials.
Our populace looks to our government to screen out generic snake oils from our pharmaceuticals. I am not saying the treatment is useless, rather I don't see enough evidence to persuade me that it is truly effective. This kinda research is really tricky when humans are involved as we have a huge placebo effect - and in this case, the hawthorne effect [wikipedia.org] is in play as well.
Based on what I read, I think they acted prudently - I would have done the same.
"Prove all things; hold fast that which is good." [KJV: I Thessalonians 5:21]
(Score: 2) by sjames on Thursday April 28 2016, @11:18AM
The problem is that the FDA has forgotten it's proper mission of assuring safety. I'm not opposed to a secondary mission of ensuring safety but would urge that it be kept separate.
That is, a requirement for safety being all that is required for limited marketing, and a secondary efficacy endorsement. Selling can happen if it is found safe OR if it is found effective enough that a risk is warranted.
I would also suggest that if an M.D. prescribes it, it may be taken regardless of the FDA. Or perhaps even if a customer signs a suitably dire warning, they can buy and take whatever they want.
(Score: 0) by Anonymous Coward on Thursday April 28 2016, @12:09PM
With the direct marketing to patients and hypochondriacs, I prefer the FDA to require efficacy. Other countries ban direct marketing and just regulate safety of new drugs.
Luckily, the US is a large enough market that companies will try to meet the higher standard even if they are already approved in other countries.
(Score: 2) by sjames on Thursday April 28 2016, @12:30PM
They don't have to permit marketing of drugs that haven't fully met the higher standard. It's a matter of not denying people the natural right to attempt to improve their condition.
(Score: 1, Informative) by Anonymous Coward on Thursday April 28 2016, @02:51PM
That is already covered under "compassionate use", which allows patients to take drugs currently under clinical trials even if they aren't enrolled, as long as the pharmaceutical company allows it. Doctors can also prescribe medicine off-label if they have been approved for other indications.
(Score: 3, Insightful) by ledow on Thursday April 28 2016, @12:29PM
Wonder what Michael Jackson, his family and lawyers would say about that?
You can't just let someone prescribe ANYTHING, based on a patient's wishes. That would basically turn doctors into legal drug-dealers, bought-out advertisers, and unaccountable murderers overnight (literally, the doctors are the people who judge whether someone is of sound mind or not, and then if they get them to write out or forge a bit of paper, they can kill off the patient with no legal comeback).
(Score: 2) by Thexalon on Thursday April 28 2016, @02:15PM
Wait, are you saying that they should "assure" safety without actually "ensuring" safety? As in, tell everybody that everything's OK, whether or not it is? The IT equivalent would be an account manager carefully assuring you that the software is completely secure even though you know that it's vulnerable to a simple SQL injection attack.
Also, efficacy is absolutely relevant to the safety question: Medical treatments typically have side effects, which makes an ineffective treatment worse than no treatment. In addition, an ineffective treatment that doctors and patients believed would work might cause them to forego the use of other treatments that would be more effective.
The only thing that stops a bad guy with a compiler is a good guy with a compiler.
(Score: 2) by sjames on Thursday April 28 2016, @04:06PM
Clearly I meant I don't see a problem with a secondary mission of assuring efficacy.
(Score: 3, Informative) by physicsmajor on Thursday April 28 2016, @04:13PM
You misunderstand, I think - there is a difference between assuring something is safe, and ensuring it actually is effective.
The FDA is tasked to assure drugs are safe to administer, that what the label says is actually what the medicine contains, and to tell providers and patients what the possible effects/reactions are.
They are also tasked to evaluate what "indications" a drug can be "labeled" for. This is important, because generally labeled usage is reimbursable by Medicare/Medicaid while off-label use is more difficult to get paid for.
In this story, the drug appears to be safe from the perspective of significant adverse effects, when dosed appropriately. That's the former thing - assuring safety. The potential rejection is due to low statistical power/poor study design in order to label this drug for the indication of DMD. This is the latter task - labeling a drug as effective for a given condition.
The main question is if this compound should remain available for off-label or compassionate use, despite unclear evidence if it truly helps DMD patients, given that there is no current therapy... Or should it be rejected entirely.
(Score: 1) by Francis on Thursday April 28 2016, @02:00PM
There are people who will go to all sorts of lengths for a treatment or a cure. And I can't blame people with serious illnesses like ALS or Parkinson's to be willing to go way beyond what would normally be considered as ethical in search of a cure, those conditions are pretty nightmarish.
But, people do die and we do have to have some standards in place to ensure that we don't wind up conducting the sort of "research" that the Nazis and Japanese were conducting in WWII.
Unfortunately, that means that there's medications that don't receive the sort of testing that they probably ought to.