As Prescribed


Human cell and gene therapy research has advanced dramatically in recent years and opened the door to potential treatments for diseases once considered incurable. On January 15, FDA Commissioner Scott Gottlieb, M.D., and Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER), issued a joint statement announcing plans to keep pace with the rapidly growing and evolving field through new policy guidance and other assistance. According to the statement, FDA is turning its attention and additional resources toward these therapies in 2019 due to a “large upswing” in the number of cell and gene therapy investigational new drug (IND) applications. Based on an assessment of the more than 800 cell-based and gene therapy INDs current on file with the agency, FDA projects that it will receive more than 200 cell and gene therapy INDs per year by 2020, and will approve 10 to 20 such products per year by 2025.

To accommodate the uptick and to ensure regulation of firms that may be operating outside of regulatory compliance, the statement sets forth FDA’s planned actions to support cell and gene therapy product development in 2019:

  • Expanded review group. According to the statement, the agency is currently working to expand its review group dedicated to evaluating cell and gene therapy INDs, with a goal of adding about 50 additional clinical reviewers this year.
  • Expedited programs. FDA plans to work with sponsors to utilize expedited programs, such as the regenerative medicine advanced therapy (RMAT) designation and accelerated approval. Products that receive an RMAT designation may be entitled to rolling and priority review, as well as the opportunity to have frequent meetings with FDA to discuss issues such as study design. Accelerated approval allows for earlier approval of investigational products that treat serious conditions and fill an unmet medical need based on a surrogate endpoint. While the accelerated approval pathway may offer a faster route to approval, it also permits FDA to require postmarket follow-up studies on the treatments.
  • Clinical guidance documents. Following its issuance of six draft guidance documents in July 2018 on gene therapies, the statement proposes additional clinical guidance documents related to different areas of active product development. The plan calls for guidance on products for specific disorders and products, such as inherited blood disorders (FDA previously issued a draft guidance for hemophilia drug development in the July 2018 set), neurodegenerative diseases, and cell-based regenerative medicine products. Another guidance is to address manufacturing of cell-based therapies, such as CAR-T cells. Still another guidance that FDA intends to issue will permit certain sponsors of cell-based regenerative medicines to pool clinical data after following a common manufacturing protocol.

The joint statement unveils FDA’s plan to provide significant support toward the development and approval of cell and gene therapy products and to help expedite the agency’s review—which is a major boon for sponsors of such products. Yet, sponsors should also be aware that the additional support will likely lead to increased regulatory requirements and oversight, and a need for quicker turnaround in connection with reviews and inquiries from FDA.