Beyond Achondroplasia

Growing together with Clara

FDA Advisory Committee Meeting on Achondroplasia – Call for patients input

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On March 22, 2018, FDA, the US Food & Drug Administration, will conduct a public advisory committee meeting on achondroplasia. 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 values patient perspective on the development of pharmaceutical treatments for achondroplasia and 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 (patient organizations) may provide testimony in person at this meeting’s open public comment. Anyone interested in providing public comment at the meeting should register with by March 7.

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.

FDA is seeking to collect a rich and diverse set of patient perspectives on this topic.  We have developed a flyer, attached, with information relevant to this meeting and encourage you to disseminate it to any individuals or groups who may be interested in this meeting.

Preview of the information flyer. To read the flyer in full, click on the following link: AchondroplasiaAC

FDA link to the announcement


Beyond achondroplasia will participate in this call for contribution by sending a written comment.

The information here provided was kindly shared by Blake Bannister, Decision Support and Analysis Team, Center for Drug Evaluation and Research, FDA     

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