RARE Daily

FDA to Hold Public Meeting on the Impact of Rare Diseases

March 6, 2019

Rare Daily Staff

The U.S. Food and Drug Administration will hold a public meeting on April 29 with an opportunity for public comment on “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.”

The meeting is intended to obtain patients’ and caregivers’ perspectives on impacts of rare diseases on daily life and to assess commonalities that may help the Agency and medical product developers further understand and advance the development of treatments for rare diseases.

Developing a treatment for a rare disease can present unique challenges, such as the small number of individuals affected and heterogeneous etiologies and manifestations. While the differences between rare diseases are critically important, it is also important to assess commonalities to synergize product development in rare diseases. The goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

FDA will provide a summary document from this public meeting. This meeting will include participants from FDA, the patient community, caregivers, and other interested stakeholders. The meeting will consist of panels of patients/caregivers and facilitated discussions. The aim of the meeting is to hear directly from patients with rare diseases and their caregivers and family members. The meeting will include patients with any rare disease and their caregivers and family members. It is not restricted to a specific rare disease or group of rare diseases.

The meeting will focus on several related topics including disease symptoms, treatment considerations, and factors relevant to participating in a clinical study or registry. The agency invites the public to register and participate in our panel discussions. A detailed agenda and meeting topics will be posted on the FDA website.

Registration is free and seating is limited. Patients and patient representatives who are interested in presenting comments as part of the initial panel discussions will be asked to indicate in their registration which topic(s) they wish to address.

FDA will hold an open public comment period to give the public an opportunity to comment. Registration for open public comment will occur in the meeting registration and at the registration desk on the day of the meeting on a first-come, first-served basis. Panelists and open public comment period speakers will be notified of their selection approximately one week before the public meeting.

The meeting will be held at the FDA White Oak Campus in Silver Spring, Maryland. Online registration to attend is open until April 15, and onsite registration on the day of the meeting will be based on space availability. For those unable to attend in person, FDA will provide a live webcast of the meeting.

Comment submissions, guidelines, and instructions on registration and transcripts are available on the Federal Register website.

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