Morning Briefing
Summaries of health policy coverage from major news organizations
FDA To Fast Track Certain Gene Therapies In Effort To Keep Pace With Fast-Growing Field
Stat: FDA Plans To Speed Path To Approval For Some Gene Therapies
The Food and Drug Administration will soon be alerting companies that certain gene therapies in development can qualify for less arduous review at the agency, Commissioner Scott Gottlieb said Tuesday. Specifically, gene therapies for hemophilia, a rare disease in which blood doesn’t clot properly because it lacks certain proteins, could be evaluated based on whether therapy increases those proteins in the blood, regardless of whether the therapy actually causes the patient to bleed less. (Swetlitz, 5/22)
Bloomberg: Gene Therapies That Could Transform Diseases Get Easier FDA Path
Unlike traditional drugs, gene therapies are intended to be given once, transform the inner workings of the body and last for a lifetime. For regulators, the challenge is to find ways to get the new therapies to desperate patients while balancing the need to monitor their long-term safety for years after approval, FDA Commissioner Scott Gottlieb said Tuesday. “For some of these products, there’s going to be some uncertainty, even at the time of approval,” Gottlieb said in a speech at the Alliance for Regenerative Medicine’s annual board meeting. “But these products are initially being aimed at devastating diseases, many of which are fatal and lack available therapy. In these settings, we’ve traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies.” (Cortez, 5/22)
In other pharmaceutical news —
Stat: The Drug Industry's Next Big Idea To Treat Alzheimer's Faces Fresh Doubts
After a generation of failures, the drug industry has pivoted to its next-best idea for treating Alzheimer’s disease. But a troubling string of clinical trial results suggests that the latest approach to the devastating disease might go the way of its forebears, continuing decades of frustration for patients and scientists. Over the past year, three clinical trials involving two similar medicines have been halted early, two because a drug was clearly not working and a third because the treatment came with worrying safety concerns. (Garde, 5/23)
