BioCentury
ARTICLE | Regulation

Action in amyloidosis

What amyloidosis patients hope to accomplish by writing draft guidance

December 7, 2015 8:00 AM UTC

The Amyloidosis Research Consortium is following in the footsteps of another well-organized rare disease community by drafting a proposed FDA guidance that will reflect patients' priorities. The advocacy group also plans to qualify a cardiac biomarker as a surrogate endpoint that could shorten trials and speed development of therapies for one subtype of amyloidosis.

Last month, ARC convened a policy meeting with senior FDA officials, patients, clinical researchers and drug developers to continue a public conversation aimed at reaching a consensus on developing therapies for systemic amyloidoses. A September meeting focused on assessing what needed to be done, and charting a path for the consortium's work...