On Jan. 15, 2019, Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks issued a statement proposing policies to support cell and gene therapy product development. According to Gottlieb and Marks, FDA is seeing a surge of cell and gene therapy products enter early stages of development, and the Agency expects a jump in product approvals in the near future. By 2020, FDA anticipates it will receive more than 200 Investigational New Drug (IND) Applications per year, and by 2025, 10-20 cell and gene therapy products will be approved annually. As a result, FDA announced new policies to advance development of cell and gene therapies. These policies build on existing therapy programs to provide new and more detailed information to sponsors and researchers about the Agency’s plans.
Maximize Use of Expedited Programs
FDA emphasized its commitment to working with sponsors to maximize and fully utilize expedited approval pathways, such as regenerative medicine advance therapy (RMAT) designation. Expedited and accelerated pathways offer a faster route to approval of new treatments without a reduction in safety standards, and also allow FDA to require postmarket studies to ensure products are durable and have minimal instances of off-target effects.
Active Product Development Guidance
FDA announced it would be issuing a series of guidance documents focusing on the following:
- Guidance relating to the development of gene therapy products for inherited blood disorders and for certain neurodegenerative diseases;
- Guidance addressing how the accelerated approval pathway may be used and when it should be used;
- Guidance outlining when to use more traditional drug development approaches in gene therapy advancement;
- Guidance recommending a framework for innovators to manufacture and promote CAR-T therapies without necessarily requiring costly new clinical investigations;
- Guidance proposing a new trial design that would allow individual researchers and small sponsors (including academic investigators, who may be too small to conduct clinical trials on their own) to pool their clinical data after following a common manufacturing protocol.
According to FDA, the purpose of these new policies is to promote a better understanding of the critical quality attributes and other factors related to product manufacturing, and to further advance the field of cell and gene therapy.
FDA to Hire New Reviewers
In addition to the policies discussed above, FDA plans to add 50 additional clinical reviewers to conduct clinical investigations, development, and review of cell and gene therapy products, as well has hold public meetings and work with stakeholders to further flesh out these policies.
As we have previously noted here and here, FDA once again issued a warning to cell and gene therapy manufactures operating in a noncompliant manner—the Agency plans to take additional enforcement action against these companies that fail to comply and are creating patient safety concerns.
The FDA has emphasized cell and gene therapy regulation and guidance over the past few years, and given the growth in this field, we expect this trend to increase. Sponsors should monitor this situation closely.