Quick Policy Note - 📝 Orphan Cures Act(rare-disease) in Big Beautiful Bill
H.R.1 makes key changes to Medicare’s drug price negotiation program, specifically expanding exemptions for orphan drugs those intended to treat rare diseases.
H.R.1 makes key changes to Medicare’s drug price negotiation program, specifically expanding exemptions for orphan drugs those intended to treat rare diseases.
Under current law, only orphan drugs approved for a single rare condition are exempt from negotiation. H.R.1 broadens this significantly:
Any period during which a drug held an FDA orphan designation (even if temporary) will no longer count toward the time needed before Medicare can negotiate its price.
Drugs approved for multiple rare diseases will now be fully exempt from negotiation. Previously, these could become eligible for price talks.
📌 What this could mean in practice:
Fewer drugs may be subject to Medicare negotiations.
Pharmaceutical companies may seek multiple orphan designations to bypass future pricing controls.
The provision could delay or limit cost savings for Medicare and by extension, for patients.
🏛️ Primary Sponsors
Rep. John Joyce (R‑PA) and Rep. Don Davis (D‑NC) introduced the Orphan Cures Act, aiming to expand protections for orphan drugs under Medicare’s price negotiation framework
📈 Industry & Advocacy Support
Biotech trade group BIO publicly backed the measure, praising it as a “pro‑patient, pro‑innovation proposal” to maintain incentives for rare-disease drug development
EveryLife Foundation, representing rare-disease patients and families, lobbied Congress to broaden the orphan-drug exclusion in the Inflation Reduction Act
✅ In Summary
Who: Reps. Joyce & Davis, backed by patient groups and biotech industry.
What they want: Extend Medicare’s orphan-drug exemption to cover any period of orphan designation and protect drugs with multiple rare-disease approvals.
Why: To maintain incentives for developing new treatments in the rare-disease space.