Gene Therapies Need Long-Term Follow-Up Plan From Outset Or Risk Clinical Hold
Although a sponsor may conclude that a long-term follow-up protocol is not necessary for a gene therapy, FDA may have a different interpretation, and the agency won't let studies start if that's the case, draft gudiance states.
You may also be interested in...
Clinical development programs for human gene therapy products that incorporate genome editing should address the potential risks of off-target editing as well as unintended consequences of on-target effects, agency says in new draft guidance.
FDA continues to emphasize the routine and the positive, even though strains on staff and sponsors increase as shutdown grinds on. CBER's new clinical reviewers would oversee the clinical investigation, development and review of cell and gene therapies, although a timeline on the hiring is unclear.
CBER Director Peter Marks says the "half-life" for the existing six gene therapy guidances likely is two to three years due to the fast pace of scientific discovery.