Apparently, it's not uncommon for pharma to coach the families that comment to the committee either... And in another orphan drug case with small sample size,

Apparently, it's not uncommon for pharma to coach the families that comment to the committee either... And in another orphan drug case with small sample size, that of Sarepta/eteplirsen, in which several evaluating scientists seem confused and the majority were overridden (for approval) by the FDA director in charge. Comments included, “serious irresponsibility” (by selectively publishing data during the whole process) and saying that the drug was a “scientifically elegant placebo”. http://blogs.sciencemag.org/pipeline/archives/2016/09/20/sarepta-gets-an-approval-unfortunately http://blogs.sciencemag.org/pipeline/archives/2016/09/20/sar... This new leukemia treatment is a really interesting, and perhaps the effect size is large enough for the small trial size, but we seem to have some problems in the review process (ignoring data) and incentive structure (coaching parents) for these "orphan drugs". http://blogs.sciencemag.org/pipeline/archives/2017/05/19/im-surprised-that-theyre-surprised http://blogs.sciencemag.org/pipeline/archives/2017/05/19/im-...