The continued growth in the development of cell and gene therapies (CGTs) has led to an exponential increase in the workload of the organisations involved. As ClinicalTrials.gov. indicates, there will be more than 1,000 ongoing clinical trials for CGTs registered in 20221. The US Food and Drug Administration (FDA) expects that they will receive more than 200 investigational new drugs (INDs) per year, building on the more than 800 active cell therapy or directly administered gene therapy INDs currently submitted to the FDA2. The diverse and complex nature of CGTs can often make regulatory affairs matters more intricate than traditional pharmaceutical products.
To meet the demand for advanced medicines, the FDA announced in the Federal Register that the Office of Tissue and Advanced Therapies (OTAT) in the Center for Biologics Research and Evaluation (CBER) has been changed to the Office of Therapeutic Products (OTP) and upgraded to a “Super Office” that should significantly improve the efficiency of the workflow for reviewing regulatory submissions. This will include therapeutic tissue-engineered products, human cell and tissue products and combination products, as well as gene therapies that result in persistent cellular modifications.
This initiative has also received significant financial support from the US Congress. More products will be able to be reviewed and supported by the knowledge of more scientific experts, which should help companies to launch their products in a timely manner. Advanced medicines such as CGTs are often intended to meet unmet medical needs and the steps taken by the FDA are a positive move to ensure patients receive life-changing treatments.
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- Young CM, Quinn C, Trusheim MR – September 2021 – Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues – https://www.sciencedirect.com/science/article/pii/S1359644621003901
- S. Food & Drug Administration (FDA) – January 2019 – https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics
- The Science Advisory Board – October 2022 – https://www.scienceboard.net/index.aspx?sec=ser&sub=def&pag=dis&ItemID=4803