FDA Patient Input Opportunity - Achondroplasia
Date: March 01, 2018
On March 22, 2018, FDA is conducting a public advisory committee meeting. The purpose of this meeting to discuss the major objectives of a phase 3 drug development program indicated for the treatment of children with achondroplasia.
FDA very much values patient perspective on the development of pharmaceutical treatments for achondroplasia and is seeking to collect a rich and diverse set of patient perspectives on this topic.
This meeting presents individuals with achondroplasia and their families a valuable opportunity to provide FDA with input on important topics. These topics include the types of clinical trial endpoints that would have a clinically meaningful impact on patients’ functional or psychological well-being, and other considerations on the design of clinical trials involving people with achondroplasia.
The meeting’s open public session will take place on March 22, 2018, from 10:30 a.m. to 5:30 p.m. at our location in Silver Spring, MD, just outside of Washington, DC). Individuals, families, and others may provide testimony in person at this meeting’s open public comment. Anyone interested in providing public comment at the meeting should register with Marieann.Brill@fda.hhs.gov by March 7. Please note that there are a limited number of speaking slots. In addition, individuals, families, and others can submit a written comment on the topic of achondroplasia and development of potential treatments to the public docket. Additional details can be found in the federal register notice of the meeting.