We are the Rare Disease Company Coalition. We represent life science companies committed to discovering, developing, and delivering rare disease treatments for the patients we serve. Our goal is to inform policymakers of the unique challenges—and promises—we face in taking these rare disease drugs from research through development, approval, and manufacturing.
Rare diseases and growing – Yet a quarter of patients wait over four years to be diagnosed, while 50% of patients must live with no accurate diagnosis.
Progress in motion – 70% of the nearly 3,000 therapies in phase III clinical trials are from small pharmaceutical companies.
The need is critical – Only 7% of rare diseases have an FDA-approved treatment.
Rare is not that rare – One in ten people lives with a rare disease and half are children.
We are invested – On average, 65% of our annual expenditures go to research and development.
Hope is there – Our members collectively have 160 rare disease development programs in progress.
The Rare Disease Company Coalition will help educate key policy stakeholders on the unique considerations of life science companies when developing and bringing to market therapies for small and differentiated patient populations. If you are a life science company dedicated to rare diseases and interested in joining us, please contact email@example.com.
If you are a member of the media interested in learning more about the Rare Disease Company Coalition, please contact us at firstname.lastname@example.org.