By Marites T. Woon, Associate Director, Nonclinical, and Jessica Lee, Principal, Global Head, Cell and Gene Therapy
Cell and gene therapies (CGT) represent a growing subset of products that do not follow a traditional development journey, fraught with various challenges from inception to manufacturing and further on to commercialization. And yet, the curative potential these products hold continues to drive interest and investment in seeing their development through to fruition. It is, therefore, unsurprising that as the demand for these innovative products increases, with the FDA anticipating the approval of 10 to 20 CGT products per year by 2025 1 , so must the resources needed to assess their efficacy and safety (including, but not limited to, acute, subacute, delayed toxicity and long-term safety).
Change in Organizational Structure
In Q3 of 2022, the FDA announced a change in the organizational structure within the Center for Biologics Evaluation and Research (CBER)’s Office of Tissues and Advanced Therapies (OTAT) 2 . OTAT will be retitled as the Office of Therapeutic Products (OTP) and elevated to a Super Office 2 . This move comes at a critical time given the sustained growth in innovation within the CGT space and in consideration of potential risks to patients receiving these products.
In recent years, submissions to CBER through the Electronic Submissions Gateway (ESG) grew exponentially, with the center receiving at least twofold higher ESG submissions than the Center for Drug Evaluation and Research (CDER) for the year 2021 to 2022 3 (Figure 1). While these numbers do not specify the type of documents or regulatory submissions, they underscore the sheer volume of transmissions the FDA receives. Note that there are already several highly anticipated regulatory decisions expected this year for CGT products targeting various indications 4 .
As such, maintaining the same resources and workforce to meet continued growth in CGT is untenable. And with the FDA’s latest move, a key benefit to sponsors lies in one of the Agency’s long-term visions — recruiting and retaining high-quality staff2.
What Comes Next
With innovation continuing to evolve and CGT products becoming less and less “pie in the sky” type ideas, it stands to reason that those tasked with regulating such complex products be experts in the field and continually develop their expertise over time. What remains to be seen, however, is how OTP will address the loss of experienced staff and, consequently, the loss of historical knowledge and expertise6.
While few small and mid-sized biotechs are able to have experienced regulatory experts among their internal staff, it is possible to utilize expertise outside your internal resources by forming a partnership with a trusted clinical research organization like Allucent.
For support navigating the regulatory complexities of cell and gene therapy product development, contact Allucent’s regulatory team for expert guidance.
Allucent brings new therapies to light by solving the distinct challenges of small and mid-sized biotech companies. We are a global provider of comprehensive drug development solutions, including consulting, clinical operations, biometrics, and clinical pharmacology across a variety of therapeutic areas. Our individualized partnership approach provides experience-driven insights and expertise to assist clients in successfully navigating the complexities of delivering novel treatments to patients. Learn more about how the A-Team can support your drug development program.
- U.S. Food & Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. Jan. 15, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics.
- Statement of Organization, Functions, and Delegations of Authority, 87 Fed. Reg. 58806 (Sept. 28, 2022) https://www.federalregister.gov/documents/2022/09/28/2022-20997/statement-of-organization-functions-and-delegations-of-authority.
- U.S. Food & Drug Administration. Submission Statistics. Content current as of Apr. 11, 2023. https://www.fda.gov/industry/about-esg/submission-statistics.
- Hunt, T. The Cell and Gene Therapy Sector in 2023: A Wave is Coming – Are We Ready? In Vivo Pharma Intelligence. 09 Jan. 2023, https://invivo.pharmaintelligence.informa.com/IV146781/The-Cell-And-Gene-Therapy-Sector-In-2023-A-Wave-Is-Coming–Are-We-Ready. Accessed 17 Jan 2023.
- U.S. Food & Drug Administration. Submission Statistics Archives. Content current as of Feb. 15, 2023. https://www.fda.gov/industry/about-esg/submission-statistics-archives.
- Eglovitch, JS. FDA Elevates OTAT to “Super Office” Within CBER. Regulatory Affairs Professionals Society. 30, Sept. 2022, https://www.raps.org/news-and-articles/news-articles/2022/9/fda-elevates-otat-to-super-office-within-cber.