Meeting 2: Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases
The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on enrollment of rare disease patients into gene therapy clinical trials in the pre-symptomatic or early symptomatic stages of their disease.
Some rare genetic diseases can be detected in infancy or childhood, early adulthood, or later in a persons’ life, but visible decline in health and effects on quality of life may not be apparent for years or decades. However, even among patients with the same genetic mutation, the rate of progression or severity of symptoms can vary widely and often cannot be predicted. Caregivers for children in pre-symptomatic or early stages of a disease or adults who are in the pre-symptomatic or early stages of a disease or may be interested in participating in a gene therapy clinical trial to determine if an investigational gene therapy might prevent or meaningfully slow the progression or delay the onset of a genetic disease.
The objective of this meeting is to understand what patients, and caregivers of patients in the pre-symptomatic or early stages of disease take into consideration when deciding whether to enroll in a gene therapy clinical trial and potentially receive an investigational gene therapy product.
Meeting Logistics and Registration
- Date: December 4, 2024
- Time: 11:00 a.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 Tuesday, December 3, at 11:59 p.m. ET. A recording and other relevant meeting materials will be posted online following the event.
Requests to Speak or Present
During the registration process, you may indicate if you wish to speak and/or present during the public listening meeting. Speakers should indicate the session in which they would like speak or present during the meeting. If interested in speaking about both topics, please select the session which represents your primary topic of interest during the registration process.
Key Questions
• If you were to consider enrollment in a gene therapy trial for early-stage or pre-symptomatic disease for your child, what would you want to know?
• What would you think about regarding disease stage of progression when considering enrollment in a gene therapy trial?
Key Questions
• If you were to consider enrollment in a gene therapy trial for early-stage or pre-symptomatic disease for yourself, what would you want to know?
• What would you think about regarding disease stage of progression when considering enrollment in a gene therapy trial?
We will do our best to accommodate all requests. Individuals and organizations with common interests are urged to consolidate or coordinate their presentations, and request time for a joint presentation, or submit requests for designated representatives to participate. All requests to make oral presentations must have been received by November 5, 2024, at 11:59 p.m. 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 presentation. We will notify participants shortly after November 1 with additional instructions and deadlines. Please note, speakers who opt to present during this meeting can share slides, but slides are not mandatory.
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 February 3, 2025.
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.
Electronic Submissions
Submit electronic comments in the following way:
- Federal eRulemaking Portal: visit http://www.regulations.gov and type FDA-2024-N-4605 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-4605 for “Patient Perspective on Safety Consideration 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-4605, 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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