2023-25500. Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public meeting; request for comments.

SUMMARY:

The Food and Drug Administration (FDA, the Agency, or we) is announcing the following public meeting entitled “Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement.” Convened by the Duke-Robert J. Margolis, MD Center for Health Policy (Duke-Margolis) in collaboration with FDA and supported by a cooperative agreement between FDA and Duke-Margolis, the workshop will focus on how best to understand patients' experiences living with a rare disease and how to incorporate those experiences, as well as patients' priorities for treatment goals, throughout the drug development process.

DATES:

The public meeting will be held virtually on December 14, 2023, from 12 p.m. to 5 p.m. Eastern Time. Either electronic or written comments on this public meeting must be submitted by February 12, 2024. See the SUPPLEMENTARY INFORMATION section for registration date and information.

ADDRESSES:

The public meeting will be held virtually using the Zoom platform.

You may submit comments as follows. Please note that late, untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of February 12, 2024. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are received on or before that date.

Electronic Submissions

Submit electronic comments in the following way:

• Federal eRulemaking Portal:https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://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 https://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): Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

• For written/paper comments submitted to the Dockets Management Staff, 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–2023–N–4718 for “Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement.” Received comments, those filed in a timely manner (see ADDRESSES ), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240–402–7500.

• 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 https://www.regulations.gov. Submit both copies to the Dockets Management Staff. 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: https://www.govinfo.gov/​content/​pkg/​FR-2015-09-18/​pdf/​2015-23389.pdf.

Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the “Search” box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500.

FOR FURTHER INFORMATION CONTACT:

Stuti Ganatra, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave, Silver Spring, MD 20993, 301–796–8112, [e-mail].

SUPPLEMENTARY INFORMATION:

I. Background

This public meeting is intended to facilitate improvements in the treatment of rare diseases and conditions, consistent with the requirements under section 3202 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). Section 3202 of FDORA requires FDA to conduct a number of activities related to improving the treatment of rare diseases and conditions, including the convening of one or more public meetings to address increasing and improving engagement with rare disease patients, rare disease patient groups, and experts on small population studies, in order to improve the understanding of patient burden, treatment options, and the side effects of treatments (see section 3202(d)(2) of FDORA).

II. Topics for Discussion at the Public Meeting

The purpose of this public meeting is to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence for rare disease drug approvals. The meeting will address approaches to increasing and improving engagement with rare disease patients, groups representing such patients, rare disease experts, and experts on small population studies, to improve the understanding of how to best understand patients' experiences living with a rare disease and how to incorporate those experiences and priorities throughout the drug development process. This includes understanding patient perspectives on the burden of their condition and any existing treatment options, as well as how their current health status and risk of disease progression may impact willingness to accept risks from treatment side effects.

Meeting updates, the agenda, and background materials (if any) will also be made available at https://duke.is/​4/​7yuu prior to the meeting.

III. Participating in the Public Meeting

Registration: To register for the public meeting, please visit the following website: https://duke.is/​4/​7yuu. Please provide complete contact information for each attendee, including name, title, affiliation, address, email, and telephone. Registration will end at 11:59 p.m. Eastern Time on December 13, 2023.

Registration is free and persons interested in attending this public meeting must register to receive a link to the meeting. Registrants will receive a confirmation email after they register.

If you need special accommodations due to a disability, please contact [e-mail] no later than November 30, 2023. Please note, closed captioning will be available automatically.

Transcripts: Please be advised that as soon as a transcript of the public meeting is available, it will be accessible at https://duke.is/​4/​7yuu. The transcript will also be available at https://www.regulations.gov and may be viewed at the Dockets Management Staff (see ADDRESSES ).

Dated: November 14, 2023.

Lauren K. Roth,

Associate Commissioner for Policy.