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  4. Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products - 04/27/2023
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Event Title
Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products
April 27, 2023

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) is hosting 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.

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. The FDA is seeking 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. 

Meeting Logistics and Registration

  • Date: Thursday, April 27, 2023
  • Time: 12:00 p.m.–4:30 p.m. ET
  • Location: This is a virtual public listening meeting and will be held via Zoom.
  • Registration: This meeting is free and open to the public; however, registration is required. Early registration is recommended. Please register for the event now.

Registration will close on Wednesday, April 26, 2023 at 11:59 p.m. ET. A recording and other relevant meeting materials will be posted online following the event. 

Request to Speak or Present

During the registration process, you may indicate if you wish to make an oral presentation during the meeting. Topic areas include:

  1. Development and establishment of product-based and/or disease-based registries
  2. Real-world data collected in clinical settings, through digital health technologies, electronic health records (EHR), insurance claims databases, and other administrative databases, and population based data sources
  3. Alternative study designs, including decentralized studies
  4. Determination of specific safety or efficacy outcomes (e.g., development of malignancies, effects on fertility, or confirmation of benefits) for which collection of post-approval safety or efficacy data may be necessary for cell or gene therapies

Registrants can speak to one or multiple topics during their allotted time; if interested in speaking about multiple topics, please select your primary topic during registration.

We will do our best to accommodate all speaking requests. Individuals and organizations with common interests are urged to consolidate or coordinate their presentations and request time for a joint presentation or to submit requests for designated representatives to participate. All requests to make oral presentations must be received by 11:59 p.m. ET on March 30, 2023 ET

Based on the number of presentation requests, we will determine the amount of time allotted to each presenter and the approximate start time for each session. We will notify participants shortly after March 20, 2023 with additional instructions and deadlines. Please note that speakers who opt to present during this meeting can use slides, but slides are not mandatory. 

Submitting Electronic or Written Comments

In addition to holding the virtual public listening meeting, FDA will open a docket and accept electronic or written comments. Electronic or written comments on this listening session must be submitted no later than 11:59 p.m. ET on Friday, May 26, 2023.   

Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are postmarked or the delivery service acceptance receipt is on or before May 26. Due to the COVID-19 pandemic, Dockets Management Staff is receiving U.S. Postal Service mail intermittently, so electronic submissions are preferred.

Electronic Submissions

Submit electronic comments in the following way:

  • Visit the Federal eRulemaking Portal at Regulations.gov and type FDA-2023-N-0398 in the search box. For assistance in submitting electronic comments, please see the Frequently Asked Questions page
  • Comments submitted electronically to Regulations.gov will be posted to the docket unchanged, including attachments. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as specifics of a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on Regulations.gov.
  • To submit any comment with confidential information that you do not wish to be made available to the public, submit the comment as a paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

Written/Paper Submissions

Submit written/paper submissions as follows:

  • Send mail/hand delivery/courier (for written/paper submissions) to Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Room 1061, Rockville, MD 20852.
  • For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.”
  • Instructions: All submissions must include the Docket No. FDA-2023-N-0398 for “Methods and approaches for capturing post-approval safety and efficacy data on cell and gene therapy products, Public Listening Meeting; Request for Comments.” Received comments will be placed in the docket and, except for those submitted as “Confidential Submissions,” will be publicly viewable at Regulations.gov or at the Division of Dockets Management between 9 a.m. and 4 p.m. ET, Monday through Friday.
  • Confidential submissions: To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential, with a heading or cover note that states “THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.” FDA will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on Regulations.gov. Submit both copies to the Division of Dockets Management. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments, and you must identify this information as “confidential.” Any information marked as “confidential” will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access this information at FDA’s Dockets Management website.

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.

About OTP

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.

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