Optimizing Regulation for CGT Trials: American Solutions Advocated

In an extraordinary roundtable attended by leaders in the field of cell and gene therapy (CGT), a renewed call to action was launched urging the U.S. Food and Drug Administration (FDA) to place their trust in American solutions to advance the field. Attendees of this roundtable included molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates. These stakeholders brought their personal stories and policy recommendations to the table, advocating for a shift in the current regulatory paradigm.

The roundtable was held face-to-face with recently appointed top officials of the FDA, including director Martin A. Makary, MD, and Vinayak (Vinay) Kashyap Prasad, MD, director of the Center for Biologics Evaluation and Research. They were joined by three other top healthcare leaders of President Donald Trump’s administration, Robert F. Kennedy, Jr., Secretary of the U.S. Department of Health and Human Services (HHS), the new NIH director Jayanta (Jay) Bhattacharya, MD, PhD, and Mehmet Oz, MD, administrator for the Centers for Medicare & Medicaid Services.

Dr. June, a key voice in the roundtable, proposed a two-tier regulatory model, emphasizing the need for more efficient processes for autologous products like hemopoietic cells. He highlighted the sluggish and costly nature of the current U.S. system, pointing to promising trials overseas and the emergence of innovative treatments abroad, particularly in China. This shift signals a pivotal moment in the CGT landscape, urging regulatory adaptability and speed to keep pace with global advancements.

Dr. June’s call for FDA trust in American solutions underscores the urgency for streamlined regulations in CGT trials. As biotech companies seek opportunities abroad and significant CAR-T trials originate outside the U.S., there is a growing concern about the U.S. falling behind in the development of cutting-edge therapies. To maintain competitiveness and foster innovation domestically, regulatory frameworks must evolve to support timely and cost-effective development of novel treatments.

Kennedy, in his address, challenged the experts to identify regulations that should be removed, highlighting the almost “providential power” that they possess. He also expressed his excitement over the regulatory progress made to cover sickle cell disease patients around the country, following the 2023 approval of Casgevy® (“exa-cel” or exagamglogene autotemcel), the first CRISPR gene editing therapy authorized by the FDA.

The FDA, as Makary noted in his opening remarks, stands for “gold-standard science and common sense”. This assertion emphasizes the agency’s commitment to support the scalable and affordable development of innovative treatments. Embracing this shift towards global collaboration and innovation can position the U.S. as a leader in the rapidly expanding field of gene and cell therapy.

In conclusion, this roundtable discussion underscored the urgency for the FDA to adapt and evolve its regulatory frameworks to foster domestic innovation and keep pace with global advancements in the field of cell and gene therapy. The success of this endeavor will hinge on the ability of the FDA and other healthcare leaders to listen, challenge deeply held assumptions, and commit to support the development of cost-effective and scalable treatments.