Long-Term Postmarket Studies For Gene Therapies May Need To Be Combined, Standardized
Running multiple product-specific postmarket trials for 15 years or longer would not be cost-effective and one industry stakeholder suggests workstreams eventually could merge.
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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.
Commercial scale-up during Phase III trials could create labeling issues and force additional clinical trials, US FDA officials said.
Former OTAT Director Wilson Bryan says Peter Marks’ order to file Sarepta’s DMD gene therapy application was mainly a public relations move, which does not seem to have worked in the FDA’s favor. Upcoming advisory committee could now involve as much scrutiny of the agency as the application itself.