US FDA Struggling With Long-Term Follow-Up Requirements For Gene Therapies

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.

long-term
Many data collection hurdles could arise in the 15-year follow-up period for gene therapy patients. • Source: Shutterstock

Ensuring that long-term follow-up agreements for gene therapy patients remain unencumbered despite economic or personnel changes is proving to be a question that may require multi-stakeholder creativity to answer, US Food and Drug Administration officials acknowledged.

Because gene therapies still are new and their long-term effects unknown, the agency wants sponsors to track recipients for up to

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