Dear Friends,

As many of you may know, the passage of the Brown-Brownback Amendment (Pub. L. 111-80, Section 740) was a great step for the rare disease community. This Amendment tasks the Commissioner of the U.S. Food and Drug Administration (FDA) with establishing review groups within the agency to “recommend…appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of rare diseases,” both domestically and in the developing world.

FDA has called a public meeting and seeks comment (F.R. 2010-10079 http://edocket.access.gpo.gov/2010/2010-10079.htm) to better understand how to go about implementing and managing these new review groups. Genetic Alliance will give oral and written testimony to FDA at a Public Meeting on June 29-30, regarding orphan product development.

The FDA has asked for comments on the orphan drug marketing application process, as well as the Humanitarian Use Device and Humanitarian Device Process Exemption process. FDA has also asked for comment regarding communication between FDA and rare disease stakeholders. To inform our response to these questions, we’d like to get your input.

Please take this short survey so that we understand your thoughts:
http://www.zoomerang.com/Survey/WEB22AR9388A9C