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Shorter Gene Therapy Postmarket Studies ‘On The Table’

 
• By Derrick Gingery

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.

Natural history or other data could help the FDA shorten gene therapy follow-up times. (Shutterstock)
Key Takeaways
  • Office of Therapeutic Products Director Nicole Verdun said upcoming gene therapy guidance on post-approval safety and efficacy data collection could include adjustments to the length of time patients are followed.
  • Verdun said the FDA wants to “right-size” postmarket follow-up, potentially using natural history or other data.
  • Guidance on accelerated approval for gene therapies in rare diseases also is being developed, which could further illustrate CBER’s flexibility in using the pathway.

Upcoming cell and gene therapy guidance could adjust the challenging requirement for up to 15 years of postmarket follow-up and potentially further streamline product development.

The US Food and Drug Administration’s lengthy follow-up time is intended to ensure long-term safety and efficacy. But stakeholders worry maintaining contact and gathering data from patients over the long...

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