FDA announces suite of guidance documents to facilitating development and approval of gene therapies

Marc Döring

On 22 May 2018, FDA Commissioner Scott Gottleib gave an address at the Annual Board Meeting of the Alliance for Regenerative Medicine.  The wide ranging speech focused on the promise of cell and gene therapies and the challenge that such new technologies present to the existing regulatory frameworks for development and approval.

The key takeaways from the speech are:

  • The pace of research in the gene therapy field is increasing – FDA has more than 500 active IND applications for gene therapy products, over 100 of which were received in the last year.
  • Unlike traditional drug reviews, in which 80% is clinically focused and 20% concerns manufacturing, cell and gene therapies almost completely invert those numbers – the technical complexity of such products means that manufacturing is the primary concern, whereas clinical efficacy is typically established at an early stage based on small numbers of patients
  • FDA recognises the promise of cell and gene therapies and wants to provide accelerated approval pathways for such treatments where they are highly promising and target unmet needs, and are committed to developing specific regulatory mechanisms to strike a careful balance between facilitating timely access and managing any uncertainty around the safety or benefits.
  • FDA intends to release a suite of guidance documents setting out a framework for the manufacturing and clinical development of gene therapy products, to facilitate the development of such products.
  • The documents will include guidance in respect of potential accelerated approval endpoints for certain classes of gene therapy products, which will be taken as surrogates for clinical efficacy. The first of these will be for haemophilia therapies, where factor production may serve as a surrogate for reduction in bleeding rates
  • FDA will require significant post-marketing follow-up and monitoring commitments for gene therapy products, in order to establish durability of response and, where approval is granted based on a surrogate measure, actual therapeutic benefit.
  • Consistent with its recognition of the importance of robust post-marketing activity and real world evidence for cell and gene therapies, FDA is seeking additional resources to expand their capabilities for collecting post-market data, with a view to increased real-time surveillance of safety issues.
  • FDA are working with sponsors and other partners to help address limitations in manufacturing capability that inhibit cell and gene therapy R&D

These remarks are entirely consistent with the focus on facilitating access to highly innovative therapies that address unmet needs and proactive approach which are characteristic of the FDA under Dr Gottleib.  This announcement of a gene therapy framework comes not long after the launch of the FDA’s regenerative medicine framework in November 2017, including the Regenerative Medicine Advanced Therapy designation, which may also apply to gene therapies that produce durable effects.  Life sciences companies developing cell and gene therapies will no doubt welcome this continuing commitment from the FDA to facilitating the development of innovative and life changing therapies, which Dr Gottleib expects “will soon become the mainstay of how we treat a wide range of illness”.  We eagerly await further details of the new framework.

The full text of the speech is available on the FDA website here.

This post was originally co-authored by Daniel Lim.

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