Rare Daily Staff

A group of rare disease drug developers have formed a coalition saying that the “one-size-fits-all” approach to healthcare policy and regulations is detrimental to continued innovation needed to address rare diseases.

The Rare Disease Company Coalition said it will be a “unified voice of life science companies committed to discovering, developing and delivering rare disease treatments.” The organization will inform and help educate policymakers on the unique circumstances facing life science companies when developing and bringing to market therapies for very small and differentiated patient populations. The Coalition will also advocate for policies and regulations that enable cost-effective and more timely delivery of treatments by recognizing these differences.

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

Collectively, the Rare Disease Company Coalition represents life science companies that have brought to market 22 treatments and are working on more than 160 rare disease development programs, many of which would be first-to-market therapies if approved. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development.

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