- April 27, 2023
- 12:00 PM - 4:30 PM ET
The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) hosted a virtual public listening meeting to solicit input on methods and approaches (e.g., use of real-world evidence (RWE), registries) for capturing post approval safety and efficacy data for cell and gene therapy products. This event was held to meet an FDA commitment that is part of the seventh authorization of the Prescription Drug User Fee Act (PDUFA VII).
The rapid pace of innovation for cell and gene therapy products holds promise for transforming medicine and creating treatment options for patients living with difficult, rare, and often incurable diseases. With an increasing number of cell and gene therapy products in development and the potential for more of these treatments to become available to patients in the future, it is important to understand the full spectrum of long-term effects and collect accurate, timely, and comprehensive data to ensure these products remain safe, effective, and of high quality. This virtual listening meeting was an opportunity for stakeholders to provide input on methods, approaches, logistics, privacy concerns, and other aspects related to efficacy and safety data collection in the post-approval setting for cell and gene therapies.
In addition to holding the virtual public listening meeting, FDA opened a docket, which closed on Friday, May 26, 2023. For access to the docket, to read background documents or the electronic and written/paper comments, go to Regulations.gov and insert the docket number, FDA-2023-N-0398, into the “Search” box and follow the prompts, and/or go to the Division of Dockets Management at 5630 Fishers Lane, Room 1061, Rockville, MD 20852.
About the OTP Public Listening Meeting
Under the Prescription Drug User Fee Act (PDUFA) VII, by the end of fiscal year 2024, FDA is required to convene a meeting to solicit input on methods and approaches (e.g., use of RWE, Deve) for capturing post-approval safety and efficacy data for cell and gene therapy products. There will be a docket for the public comment period, which will be open for 30 days following the meeting. Within six months after the public meeting, FDA will issue a summary report or a transcript of the meeting.
OTP, previously known as OTAT, oversees development for a wide variety of biological products, including gene and cell therapies, tissues and tissue-engineered products, xenotransplantation products, and more. OTP strives to lead all regulatory decisions with data, impartiality, and compassion and welcomes the participation of patients and their advocates in formal meetings related to the development of investigational products. Learn more about OTP and view our available resources.
|FDA CBER OTP Listening Meeting: Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products||pdf (681.99 KB)|