Action in amyloidosis

What amyloidosis patients hope to accomplish by writing draft guidance

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.

"What we're seeing is a number of clinical trials are struggling, and it's not to do with the therapies companies are developing. It's to do with the complexity of the process and the disease," said ARC President and CEO Isabelle Lousada. "That was really where we thought we needed to come in and help define what actually is meaningful in clinical trials for patients."

Systemic amyloidoses are rare diseases characterized by the production of misfolded proteins that accumulate in tissues,

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