Rare Disease Company Coalition Forms to Educate Policymakers on Rare Disease Business Model, Experience Bringing Treatments to Market

Date: May 14, 2021 | Country: UNITED STATES | Region: NORTH AMERICA | Type: Policy | 
Keywords: #acceleron #aeglea #agios #alnylam #harmony #innovation #marketaccess #orchard 
#orphandrug #orphazyme #r&d #raredisease #rarediseasecompanycoalition 
#sarepta #taysha #ultragenyx

PRICENTRIC BRIEF:

  • The Rare Disease Company Coalition has launched with the aim of informing and educating policymakers on the rare disease company business model, specifically the unique circumstances facing life science companies when developing and bringing to market therapies intended for extremely small and differentiated patient populations
  • The Coalition’s additional goals include advocating for public policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space; working with policymakers to establish long-term, consistent, equitable and sustainable research incentives for rare diseases and access to the resulting innovation; and increasing awareness of the innovation happening within rare disease companies
  • Representing life science companies that brought 22 treatments to market and have 160 rare disease development programs, the Coalition is comprised of founding members Acceleron Pharma, Aeglea BioTherapeutics, Agios Pharmaceuticals, Alnylam Pharmaceuticals, Harmony Biosciences, Orchard Therapeutics, Orphazyme U.S., Inc., Sarepta Therapeutics, Taysha Gene Therapies, and Ultragenyx Pharmaceutical

THE DETAILS

WASHINGTON, D.C., United States – The Rare Disease Company Coalition has launched with the aim of informing and educating policymakers on the rare disease company business model, specifically the unique circumstances facing life science companies when developing and bringing to market therapies intended for extremely small and differentiated patient populations.

Representing life science companies that brought 22 treatments to market and have 160 rare disease development programs, the Coalition is comprised of founding members Acceleron Pharma, Aeglea BioTherapeutics, Agios Pharmaceuticals, Alnylam Pharmaceuticals, Harmony Biosciences, Orchard Therapeutics, Orphazyme U.S., Inc., Sarepta Therapeutics, Taysha Gene Therapies, and Ultragenyx Pharmaceutical.

The education and advocacy-focused group underscored the work accomplished by companies focused on rare diseases—in 2020 alone, Coalition members invested more than $1.4 billion in research and development (R&D), representing about 65% of their annual operating budgets. Further, these companies stated they spend more annually on R&D than revenues generated.

Along with educating policymakers on the experience of bringing orphan drugs to market, the Coalition’s goals include advocating for public policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space; working with policymakers to establish long-term, consistent, equitable and sustainable research incentives for rare diseases and access to the resulting innovation; and increasing awareness of the innovation happening within rare disease companies.

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