- November 15, 2022
- 12:00 PM - 4:30 PM ET
The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) hosted a virtual patient-focused drug development listening meeting to better understand patient perspectives on gene therapy products, including cell-mediated gene therapies. 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).
Patients and their advocates possess the unique, first-hand perspective of what it is like to live with or care for an individual with a disease, as well as the impact of available treatments on daily life. Patients and caregivers can provide valuable input into the discussion of investigational therapies by describing their experience with a disease or condition, and by defining meaningful change in terms of their specific disease and the risks they are willing to accept. With the potential for more gene therapies to become available to patients in the future, it is important to understand patient and caregiver perspectives on these products.
This virtual listening meeting was an opportunity for patients, caregivers, patient advocates, and other important stakeholders to share their understanding and expectations regarding gene therapy risks and benefits and involvement in clinical study design and execution for these products.
Submitting Electronic or Written Comments
The FDA is also collecting feedback and comments via written and electronic comments. These comments must be submitted by December 15, 2022.
To electronically submit comments to the docket, visit Regulations.gov and type FDA-2022-N-2394 in the search box. For assistance in submitting electronic comments, please see the Regulations.gov FAQ page. Comments submitted electronically, including attachments, will be posted to the docket unchanged. 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 a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comment, that information will be posted on Regulations.gov.
Due to the COVID-19 pandemic, Dockets Management Staff is receiving U.S. Postal Service mail intermittently, so electronic submissions are preferred. To submit comments to the docket by mail, use the following address: Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Be sure to include “Docket Number FDA-2022-N-2394” on each page of your written comments. Comments received by mail will be considered timely if they are postmarked on or before December 15. 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.
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.” The Agency will review this copy, including the claimed confidential information, in its consideration of the 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 “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 the comments in the Federal Register from September 18, 2015, or access the information at the Dockets Management page.
For access to the docket and to read the electronic and written/paper comments received, go to Regulations.gov and insert the docket number, FDA-2022-N-2394, into the Search box and follow the prompts.
About This Meeting
Under the Prescription Drug and User Fee Act (PDUFA) VII, by the end of FY 2023, FDA will convene a public patient-focused drug development meeting with key stakeholders, including patients and patient advocacy organizations, to better understand patient perspectives on gene therapy products, including cell-mediated gene therapy. There will be a docket for the public comment period, which will be open prior to and for 30 days following the meeting. Within 6 months of the public meeting, FDA will issue a report summarizing the views expressed at the meeting and the comments from the docket.
OTAT oversees development for a wide variety of biological products, including gene and cell therapies, tissues and tissue engineering products, xenogeneic products, and more. OTAT strives to lead all regulatory decisions with data, impartiality, and compassion and always welcomes the participation of patients and their advocates in formal meetings related to the development of investigational products. Learn more about OTAT and view our available resources.