FDA explains plans to bolster cell and gene therapy approvals through wider messaging

Regulatory NewsRegulatory News | 19 May 2022 |  By 

Updated 25 May 2022 to correct ASGCT's name.

Wilson Bryan director of the US Food and Drug Administration’s Office of Tissues and Advanced Therapies (OTA) in the agency’s Center for Biologics Evaluation and Research, told a 19 May meeting of the American Society of Gene and Cell Therapy (ASGCT) that the division is undertaking a series of internal measures to improve communications with sponsors and to widen its messaging.
These measures are being taken to compensate for a lack of individual attention to applications for cell and gene therapies as the result of the growing number of cell and gene therapies amidst staffing constraints.
These measures include the issuance of more “group communications” through guidance, workshops and webinars.
Bryan said that while OTAT received a slightly fewer number of investigational new drug applications (INDs) last year, he expects this number to increase. In 2021, OTAT received 299 INDS, which is a small drop from the 350 submitted in 2020, yet a huge jump from the 163 submitted in 2016. He attributes the drop to the pandemic and said “I think in the years ahead this trajectory will resume. We have lots of work coming in.”
The number of breakthrough (BT) and Regenerative Medicine Advanced Therapy Designation (RMAT) requests have similarly grown, which also includes applications for cell and gene therapies. Bryan said that the staff pays extra attention to RMAT requests as “these designations have some type of clinical evidence that addresses an unmet need.” He added that “staff cannot give as much attention to those applications that do not have RMAT designations.
Bryan pointed out that staffing levels have not kept up with the growing caseload nor the growing number of meetings with industry. He made a similar observation at a meeting last year (RELATED: Gene therapy developers can expect less hand-holding from OTAT, Regulatory Focus, 10 December 2021).
Doing things differently
Bryan said that because of these challenges, OTAT must change the way it works. “With the discrepancy between the increasing workload and the number of employees, it is more and more difficult to give individual attention” to cell and gene therapy applications. Instead, OTAT plans to issue more group communications via guidance, or through workshops and webinars.
Bryan said “OTAT is committed to patients and high-quality scientific exchange, we want to bring cell and gene therapy products forward. Every reviewer wants these [submissions] to be successful. We are all trying to help patients,” he said. He noted that over 90% of applications are for serious or life-threatening diseases, while 50% are for rare diseases. “There is just this huge need. The rapid growth in the development of cell and gene therapies has created challenges for us.”
ASGCT annual meeting



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Tags: and, cell, FDA, gene, OTAT, therapies

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