One in 10 Americans lives with a rare disease. Whether it’s a family member, friend, colleague, classmate, neighbor, or even you, rare disease may touch your life. Yet only 5% of the roughly 10,000 known rare diseases have an FDA-approved therapy.
The Rare Disease Company Coalition (RDCC) represents life sciences companies dedicated to advancing treatments for these patients. Our members are highly innovative, investing on average 70% of annual revenue back into R&D; that’s over $25 billion each year. Together we have delivered over 100 treatments to patients, with hundreds more in development.
This progress is driven by scientific advancements spurred by smart, tailored policy. Before passage of the Orphan Drug Act of 1982, only 10 rare disease therapies were FDA-approved. Today, there are more than 650. Recent policies reaffirm that rare diseases remain a bipartisan issue, including the ORPHAN Cures Act, Mikaela Naylon Give Kids a Chance Act, and Accelerating Kids’ Access to Care Act. These bills are critical pieces of legislation that spur continued development of rare disease therapies, especially for children. As children make up 1 out of every 2 rare disease diagnoses, and a third of those children won’t live to see their fifth birthday, this progress is urgent and necessary.
But this ecosystem is fragile.
Rare disease drug development is uniquely risky, costly, and complex. Most rare disease therapies are developed by small companies, either precommercial or with just a handful of therapies on the market. Often, a single clinical program represents a company’s entire portfolio, and one setback can jeopardize a company’s survival.
Shifts in regulatory approaches, incentives, tax policy, or pricing frameworks can destabilize programs with long timelines and high upfront costs. Rare disease innovation depends on a predictable policy environment that recognizes the scientific complexity, financial risk, and urgency of developing treatments for rare and underserved patient populations.
In recognition of these needs, the RDCC calls for policymakers to:
- Recognize the significant unintended consequences of Most Favored Nation policies on rare disease innovation and access, and advocate for an exclusion of orphan drugs from any MFN policy proposals, including the GLOBE and GUARD models.
- Support a predictable and consistent regulatory journey for rare disease drug developers and ensure a fit-for-purpose regulatory approach to rare disease by harnessing FDA’s existing rare disease “toolkit.”
- Strengthen the rare disease innovation ecosystem by restoring the Orphan Drug Tax Credit via H.R. 1414, Cameron’s Law.
For millions of families waiting for their first treatment, the stakes could not be higher, and there is no time to lose.
To learn more, visit rarecoalition.com
