SCAM NEEDS SHORT ! WHAT DO YOU THINK? 2 Patients out 12 without Primary endpoint - FDA approval or NDA ?
Unfortunately, great results from small trials have a history of not bearing out in larger studies. Even for rare disease drugs, this study was tiny. Worse, the Sarepta results only look good when two of the 12 patients are excluded – two boys were too sick to be helped by the drug. The FDA usually insists that clinical trials be presented in what is known as an “intent-to-treat” analysis, which means that if you even thought about treating a patient they need to be included when you do the math on the study’s results. This is intended to keep scientists from lying to themselves, convincing themselves that a drug works when it doesn’t. One biotech executive with a great deal of experience in rare diseases told me recently that this issue meant the data “would never fly” with the FDA. The recent failure of a similar drug from Prosensa and GlaxoSmithKline GSK -1.64% made the odds dimmer.
nd any trial seems likely to last 2 years. Seems to me that even if all goes well, approval would get pushed out much more than two years. They're going to spend 9 months arguing over study design and probably won't start enrolling until early 2015. Two-year trial plus filing and approval. Sounds like early 2018 approval at best.