Biologics

Comments on Gene, Cell Therapy Surveillance

David McFarland

— 1 min read
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The National Organization for Rare Disorders (NORD) says it applauds FDA efforts to seek extensive comments and community engagement on post-market surveillance for cell and gene therapy products, and greatly appreciated the April public listening session on the topic. The NORD comment letter discusses these topics with recommendations:

  • post-market surveillance should be designed to answer the key questions and concerns of rare disease patients, caregivers, families considering these products as therapeutic options;
  • leverage FDA’s new congressional authority and establish a “platform” program for post-market surveillance registries for cell and gene products;
  • build capacity to ensure the patient communities can be integral partners in the registry program;
  • ensure patients are fully invested in and benefit from the registry program to make long-term follow-up possible;
  • develop today’s post-market surveillance systems so they can help generate data for tomorrow’s cell and gene therapies; and
  • leverage all available data streams and intentionally try to build in redundancies to future-prove surveillance systems.

In its comment, Pharmaceutical Research and Manufacturers of America (PhRMA) praises FDA for taking early steps to fulfill its PDUFA 7 commitment to hold a public meeting on cell and gene therapies. PhRMA says it believes that existing methods for long-term follow-up of patients receiving cell and gene therapy products in clinical trials and post-approval studies can be refined and enhanced. It encourages the agency to be flexible and open to using platform studies and to provide guidance on how sponsors can use a platform study approach.

Finally, BioMarin says that while there has been significant progress in gene therapy over the past decade, there remain significant gaps regarding the long-term safety and efficacy of gene therapy products, specifically relating to the variability and durability of their effectiveness. It asks FDA to address in future guidance outstanding uncertainties related to long-term safety and effectiveness. It also asks the agency to clearly articulate the requirement for post-approval efficacy data to support label expansion and specify whether data from voluntary long-term follow-up studies may support label expansion. It further recommends that FDA clarify the scope of required post-approval efficacy data to include data from confirmatory trials for products receiving accelerated approval.

PhRMA and BioMarin also submitted specific line-by-line comments.

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