Feedback on FDA's Platform Technology Designation Program Includes Addressing Post-approval Changes
Andrew Liermann and Caitlin McCombs, MBA - September 23, 2024
In feedback to FDA on its Platform Technology Designation Program, ASGCT highlighted several key areas where additional clarity and adjustments are needed, including address post-approval changes to platforms across products.
The Platform Technology Designation Program, introduced as part of the Food and Drug Omnibus Reform Act (FDORA) of 2022, is designed to reduce the burden on developers and regulators by encouraging the use of standardized platforms across drug development programs. This approach is crucial as the pipeline for cell, gene, and RNA therapies continues to expand, with over 4,000 therapies currently in development. ASGCT is supportive of standardized platforms which can facilitate efficient scaling and streamlining of development and manufacturing processes, ultimately benefiting patients through quicker access to new treatments.
ASGCT submitted comments on FDA's draft guidance on the "Platform Technology Designation Program for Drug Development." ASGCT's feedback highlighted several key areas where additional clarity and adjustments are needed to maximize the program's impact. One of the recommendations is to address post-approval changes to platforms across products. The current requirements, developed for small molecule drugs, do not adequately address the unique needs of CGT manufacturing, as process improvements in CGT often occur throughout product development. A single application for major CMC changes would provide a streamlined process for developers. As multiple gene therapies come to market on designated platforms, this program can enable the latest CMC learnings to be applied across products to ensure timely patient access to these transformative therapies.
Another critical issue is the utilization of Drug Master Files (DMFs). The existing rule restricts BLAs from incorporating information about drug substances, intermediates, or products via DMFs, diminishing the value of platform designation. ASGCT suggests revisiting this rule in the context of CGTs to enhance regulatory efficiency by avoiding redundant submissions and reviews.
Considering many CGT developers are small entities (start-ups, small biotech companies, or academic researchers) without any currently approved products, comments also requested clarity on how to apply "Designated Platform Technologies" principles in their initial programs. This could help streamline the designation process after approval for subsequent products.
ASGCT appreciates FDA's efforts in developing the Platform Technology Designation Program (and the forthcoming draft guidance on platform technologies for gene editing). By incorporating ASGCT's recommendations, FDA can further enhance the program's effectiveness and reduce the regulatory burden on CGT developers. The Society looks forward to continued collaboration with FDA and other stakeholders to ensure the regulatory environment supports innovation while maintaining the highest standards of patient safety. For more detailed information on ASGCT’s comments, please refer to our full letter.
Andrew is ASGCT's Regulatory Affairs Manager and Caitlin is ASGCT's Senior Advocacy Program Specialist.
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