Sarepta gene therapy shows flaws in evidence generation, regulatory processes 

The plight of boys stricken with Duchenne muscular dystrophy has again led a top FDA official to grant a controversial accelerated approval in the face of near-unanimous opposition from the agency’s review staff.

The situation does more than evoke a sense of déjà vu; it reveals flaws in the ways evidence about rare disease therapies is generated and the ways FDA makes decisions. Those flaws are creating unacceptable and unnecessary dilemmas for patients, regulators and payers. ...