Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement

On December 14th, 2023, the FDA in collaboration with the Duke-Robert J. Margolis, MD Center for Health Policy (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 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.

Registration is open, click here for more info and details.

Agenda is also available.