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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, 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.