Meeting 1: Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases September 20, 2024

Meeting | Other

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• Date: September 20, 2024
• Time: 11:00 AM - 4:30 PM ET

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The FDA Center for Biologics Evaluation and Research (CBER) hosted a public patient listening meeting and opened a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases.

The objectives of this listening meeting were to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes.  The meeting was divided into two sessions and addressed the following questions:

Session 1: Patient and Caregiver Safety Considerations in Decision-making About Gene Therapy

1. What risks or uncertainties did you or would you consider when determining whether to receive an approved gene therapy?
2. What do you believe patients and care partners making decisions on behalf of patients should know about gene therapy?
3. In a scenario where more than one treatment option is available to treat a disease or condition, what information would you want to know?

Session 2: Partnering with Patients and Caregivers on Long-term Studies After Receiving a Gene Therapy

1. What knowledge or factors might positively or negatively impact a patient’s decision to participate in a gene therapy registry or other long-term study that captures gene therapy outcomes over a long period? 
2. What is important to patients and their care partners about how a potential registry or other long-term study that includes gene therapy outcomes would be designed, operated, and managed?   
3. What information about gene therapy outcomes in the long term is important to be communicated to patients?  

View the recording for the FDA CBER Patient Listening Meeting on Patient Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases.

Polling results from the FDA CBER Patient Listening Meeting on Patient Perspectives on Safety Considerations for Approved Gene Therapy are available in docket FDA-2024-N-3208 at Regulations.gov.

Submitting Electronic or Written Comments

In addition to holding the virtual public listening meeting, FDA has opened a docket on September 20, 2024 and is accepting electronic or written comments. Electronic or written comments on this listening session must be submitted no later than 11:59 p.m. ET on November 19, 2024. 

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 that date.

Submitting Electronic or Written Comments

In addition to holding the virtual public listening meeting, FDA opened a docket on September 20, 2024 and is accepting electronic or written comments. Electronic or written comments on this listening session must be submitted no later than 11:59 p.m. ET on November 19, 2024. 

Electronic Submissions

Submit electronic comments in the following way:

• Federal eRulemaking Portal: visit http://www.regulations.gov and type FDA-2024-N-3208 in the search box. For assistance in submitting electronic comments, please see regulations.gov/help. Comments submitted electronically, including attachments, to http://www.regulations.gov 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 comments, that information will be posted on http://www.regulations.gov.
• If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

Written/Paper Submissions

Submit written/paper submissions as follows:

• Mail/Hand delivery/Courier (for written/paper submissions):  Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 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 received must include the Docket No. FDA-2024-N-3208 for- “Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases.” Received comments will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at http://www.regulations.gov or at the Division of Dockets Management between 9 a.m. and 4 p.m., 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.”  The Agency 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 http://www.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 the information at:  http://www.fda.gov/regulatoryinformation/dockets/default.htm.

For access to the docket to read background documents or the electronic and written/paper comments received, go to http://www.regulations.gov and insert the docket number, FDA-2024-N-3208, into the “Search” box and follow the prompts and/or go to the Division of Dockets Management, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.

About FDA CBER 

Vision: CBER uses sound science and regulatory expertise to:

• Protect and improve public and individual health in the United States and, where feasible, globally;
• Facilitate the development, approval of, and access to safe and effective products and promising new technologies; and
• Strengthen CBER as a preeminent regulatory organization for biologics.

Mission: CBER’s mission is to ensure the safety, purity, potency, and effectiveness of biological products including vaccines, allergenics, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions, or injury. Through our mission, we also seek to protect the public against the threats of emerging infectious diseases and bioterrorism.

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