The FDA is preparing for a surge in cell and gene therapy submissions, with up to 10 regulatory decisions expected this year alone. The establishment of the Office of Therapeutic Products (OTP) is part of the FDA's response to the growing demand for regulatory review of these therapies. However, experts have warned that modernization of both the regulatory and access and reimbursement frameworks for cell and gene therapies is necessary to ensure they reach patients. Standardization, chemistry, manufacturing and controls (CMC), collaboration, and expertise are among areas highlighted as requiring attention.
Keywords: FDA, cell and gene therapy submissions, Office of Therapeutic Products (OTP), regulatory framework, standardization
Additional sentences: The number of investigational new drug (IND) applications for cell and gene therapies has more than doubled since 2017. The rapid growth in the development of these products has resulted in a “continuous and sustained increase in the number of applications and meeting requests for a variety of diverse and complex products.” While the new super office with its sizeable staff is a good start, it is likely to be insufficient in the long run.