FDA Gene Therapy Office Chief Prefers Flexibility With Accelerated Approval Confirmatory Trials
Ideally, the studies would be underway at the time of approval, as the US FDA’s new authority allows, but Office of Therapeutic Products Director Nicole Verdun sys there will be exceptions.
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Oncology Center of Excellence Director Richard Pazdur also says that he will not grant an accelerated approval if the confirmatory trial is not underway, even if the product shows safety and efficacy.
Some worries about heritable genetic modifications are subsiding and Center for Biologics Evaluation and Research Director Peter Marks said sponsors should consider the US for regulatory advice and clinical trials in the space.
OTP Director Nicole Verdun acknowledged the agency may need help to determine how to ensure 15 years of patient follow-up is completed despite physician retirements, company closures and other issues that could impact the post-market commitments for gene therapies.