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  4. Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement - 12/14/2023
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Event Title
Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement
December 14, 2023

December 14, 2023
12:00 PM - 5:00 PM ET

On December 14th, 2023, the FDA in collaboration with the Duke-Robert J. Margolis, MD Center for Health Policy (Duke-Margolis) and supported by a cooperative agreement between FDA and Duke-Margolis, will host a virtual public meeting titled “Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement.” 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 to support rare disease drug approvals. This meeting will also fulfill the requirement for a public meeting as discussed in Section 3202 of the Food and Drug Omnibus Reform Act of 2022 (FDORA).

This public meeting will discuss approaches and opportunities for engaging patients, patient groups, rare disease or condition experts, and experts on small population studies during the drug development process for rare diseases. The meeting will focus on 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.

Attendees are encouraged to submit questions when they register. Moderated discussions will incorporate attendee questions submitted in advance through the registration site, and live via the webinar’s Q&A function.

This website will be updated as meeting materials are developed.

Submit a Comment to the Docket

We encourage stakeholders to submit written comments on advancing the development of therapeutics through rare disease patient community engagement to the docket: FDA-2023-N-4718 or go to www.regulations.gov and search for: FDA-2023-N-4718. This docket will remain open until February 12th, 2024.

Meeting Details

Date: December 14th, 2023

Time: 12 – 5 p.m. ET

Location: Zoom

Meeting Materials

Registration Link: https://duke.is/4/7yuu

Related Information on Rare Diseases at FDA: https://www.fda.gov/patients/rare-diseases-fda

Federal Register Notice: https://www.federalregister.gov/documents/2023/11/20/2023-25500/advancing-the-development-of-therapeutics-through-rare-disease-patient-community-engagement

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