The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible.
HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner Martin Makary, MD, MPH, announced the new process, called the “plausible mechanism pathway” during a press conference on Monday. According to the draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, the FDA may approve or authorize