Rare Disease Company Coalition established

By The Science Advisory Board staff writers

May 14, 2021 -- A coalition of pharmaceutical and biosciences companies has launched a new organization to educate policymakers on the challenges they face bringing therapies to the market for small patient populations.

The Rare Disease Company Coalition represents life science companies that have brought 22 treatments to the market and have more than 160 rare disease development programs, according to the group. In 2020, coalition members invested more than $4.1 billion in research and development, representing approximately 65% of their annual operating budgets.

Working with policymakers to establish long-term, consistent, equitable and sustainable research incentives for rare diseases will be among the coalition's objectives.

Founding members of the Rare Disease Company Coalition include Acceleron Pharma, Aeglea BioTherapeutics, Agios Pharmaceuticals, Alnylam Pharmaceuticals, Harmony Biosciences, Orchard Therapeutics, Orphazyme US, Sarepta Therapeutics, Taysha Gene Therapies, and Ultragenyx Pharmaceutical.

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