FDA has recently increased its efforts to support the development and approval of cell, tissue, and gene therapy (CTGT) products. This is evidenced by the upswing in CTGT approvals in the last few years and the unprecedented approval, on the same day in 2023, of a novel gene editing product and another gene therapy product for sickle cell disease. Although FDA’s approval rate remains slower than the one to two applications that FDA receives each week for new gene therapy products, the agency continues to demonstrate commitment to the accelerated development of these products. The agency recently increased its repertoire of tools for accelerating the development of CTGT products by providing additional opportunities for early interactions with sponsors, as we have summarized below.
START program
Under FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (START) program, sponsors with active INDs for certain CTGT products may be able to seek more frequent advice and have more communication with FDA on issues such as clinical study design, choice of control group, and choice of population, if they meet certain eligibility criteria. As we previously summarized in greater detail online here, FDA intends to notify successful applicants of their acceptance into the pilot program by May 30, 2024. FDA may repeat the program, depending on stakeholder feedback.
Voluntary Consensus Standards (VCS) Recognition program
The VCS program permits sponsors to recommend novel scientific standards applicable to their products, if the standard meets certain criteria. Developed in response to a mandate under the 21st Century Cures Act and modeled after a similar program for medical devices, this program is especially helpful for regenerative therapies, including CTGT products, whose complex nature makes regulatory standards challenging and unpredictable. FDA has started accepting standard recognition requests and will maintain a public list of recognized standards.
Platform Technology Designation program
FDA’s Platform Technology Designation is intended to accelerate review of product applications, including supplement applications for sponsors who have multiple products that use a similar technology or platform, like adeno-associated viral vectors. Designation gives sponsors additional opportunities for early FDA interactions and may also permit a sponsor to reference data from the designated technology. FDA has indicated that the agency may use the platform approach even in the “absence of product approval” to leverage existing data available to the agency and facilitate quicker product development. FDA will soon issue a guidance with specific examples of drugs that can be manufactured with platform technologies as well as how the agency intends to implement the program.
Next steps
In a recent Food and Drug Law Institute (FDLI) annual conference, Dr. Nicole Verdun, Director of Office of Therapeutic Products (OTP), specifically referenced the START and the Platform Technology Designation Programs and underscored the importance of these programs in helping to facilitate rapid ad-hoc communication between sponsors and FDA and in accelerating the development of CTGT products.
Other recent efforts include FDA’s advocacy for greater use of the accelerated approval pathway under its Split Real Time Application Review (STAR) pilot program for new uses of approved therapies to address unmet medical need, as well as a Rare Disease Endpoint Advancement (RDEA) pilot program.
We anticipate that collectively, these FDA initiatives should lead to more CTGT approvals in the next few years.
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