Current Policy Priority

Protect Orphan Drug Therapy Investments and Access

An estimated 30 million Americans have a rare disease. A rare disease is a condition affecting 200,000 Americans or less. PARP inhibitors, targeted therapies for ovarian and pancreatic cancer patients with BRCA mutations, are considered orphan drugs because the number of people affected is under the 200,000 threshold. As more genetic mutations are discovered, additional “rare” cancer communities may benefit from orphan drugs/therapies.

Passed in 1983, the Orphan Drug Act incentivizes the development of treatments for these small disease populations by providing. This law changed the face of treatment for rare cancers and conditions, spurring never-before-seen investments in rare disease research and development, resulting in an exponential increase in the number of FDA-approved orphan drugs for cancers like less common cancers or subsets of the cancer community.

Under the new Medicare Drug Price Negotiation Program’s narrow Orphan Drug Exclusion provision, an orphan drug is excluded from price negotiation only if it is approved to treat one rare disease. Much of the rare cancer development pipeline and the progress over the last 40 years has relied on identifying additional uses for existing drugs that could address the unmet needs of patients living with rare cancers or conditions.

H.R. 946, the Optimizing Research Progress Hope and New Cures Act (ORPHAN Cures Act) will: 

• Improve the Orphan Drug Exclusion, ensuring that protections remain to incentivize research into multiple rare diseases. This modification would allow orphan products to remain excluded from price negotiation as long as their FDA-approved uses are exclusively for rare conditions affecting fewer than 200,000 people.
• Clarify that the negotiation eligibility timeline for an orphan product starts once it loses the exclusion due to approval to treat a non-rare disease.