News & Events

Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities

The Food and Drug Administration (FDA or the Agency) is announcing a public meeting and an opportunity for public comment on “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.” This public 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 heterogenous 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.

Date and Location

April 29, 2019 
1:00 - 5:00PM EST.

The meeting will be held at:

FDA White Oak Campus
10903 New Hampshire Avenue
Building 31 Conference Center, Great Room C
Silver Spring, MD 20993-0002

The entrance for public meeting participants (non-FDA employees or contractors) is through Building 1, where routine security check procedures will be performed.

For parking, security and other visitor information, please refer to Public Meetings at the FDA White Oak Campus.

Meeting Information

A recorded webcast will be posted to this page approximately 1 week after the event.

Registration

Please register to attend the event in-person or via webcast at Eventbritedisclaimer icon. If you need special accommodations, please contact Eleanor Dixon-Terry at 301-796-7634 or Eleanor.Dixon-Terry@fda.hhs.gov by April 15, 2019.

Submitting Comments

Regardless of attendance at the public meeting, you can submit electronic or written comments to the public docket by May 30, 2019. Submit electronic comments to Regulations.gov. Submit written comments to the Division of Docket’s Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. 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.

All comments must be identified with the docket number FDA-2019-N-0077. The FDA may post, without change, all comments received to Regulations.gov, including any personal information provided.

Questions

If you have questions about registering for the meeting, contact Eleanor Dixon-Terry, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993, phone: 301-348-1591, or email OOPDOrphanEvents@fda.hhs.gov.

 

Page Last Updated: 04/16/2019
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