Altoona Times

Congressman John Joyce, M.D. from Pennsylvania and Congressman Don Davis from North Carolina have put forward H.R. 946, known as the Optimizing Research Progress Hope And New (ORPHAN) Cures Act. This legislation aims to speed up the development of new treatments for rare diseases and provide hope to millions of Americans affected by these conditions.

Currently, under federal law, a drug approved by the U.S. Food and Drug Administration (FDA) to treat a single rare disease—termed an "orphan drug"—is eligible for certain incentives, including exemption from Medicare's drug negotiation program. However, if an orphan drug is approved to treat multiple rare diseases, these incentives do not apply. This situation has inadvertently discouraged research into whether such drugs could address additional rare diseases.

The ORPHAN Cures Act seeks to address this issue by restoring incentives intended by the Orphan Drug Act of 1983, thereby encouraging further discovery of cures for rare diseases.

"Over 30 million Americans are affected by nearly 10,000 rare diseases– yet 95% of these rare diseases lack an FDA-approved treatment. We need to be doing more – not less – to bring new FDA-approved treatments to market for rare disease patients," stated Congressman John Joyce, M.D.

Congressman Don Davis emphasized the importance of supporting innovators: "We must empower our innovators to continue developing lifesaving rare disease treatments."

Christopher P. Molineaux, President and CEO of Life Sciences Pennsylvania, expressed support for the act: “Life Sciences Pennsylvania applauds Congressman Joyce on the introduction of the ORPHAN CURES Act in the 119th Congress.”

Maimah Karmo from Tigerlily Foundation also endorsed the legislation: "At Tigerlily Foundation, we stand firmly in support of the ORPHAN Cures Act."

Kelly Brazzo from CureLGMD2i Foundation highlighted its significance: "On behalf of our LGMD2I/R9 community, CureLGMD2i fully supports the Orphan Cures Act (OCA)."

Sarah Jones from Eosinophilic & Rare Disease Cooperative noted the urgency: “Eosinophilic & Rare Disease Cooperative strongly support the passage of the Orphan Cures Act.”

Lisa Schlager from FORCE emphasized its necessity: “Passage of the ORPHAN Cures Act is essential to encourage therapeutic innovation for these patients.”

Finally, Save Rare Treatments Task Force praised Congressmen Joyce and Davis for their leadership: “This vital legislation corrects an unintended consequence in law.”