ORPHAN Cures Act
The Save Rare Treatments Task Force is committed to helping Congress understand the importance of robust research incentives that provide hope for new treatment options to the rare disease community.
The ORPHAN Cures Act:
Thanks to Congress for Passing into Law
Law Restores Incentives for Research & Development of Rare Disease Treatments
The ORPHAN Cures Act ensures that drugs used to treat one or more rare diseases will not be subject to government price setting under the Medicare Drug Price Negotiation Program and clarifies the timeline used to determine when an orphan drug may become eligible for negotiation, further incentivizing the innovation of necessary rare disease treatments.
Defending ORPHAN Cures Against Repeal
Since the ORPHAN Cures Act became law on July 4, 2025, several bills have been introduced that would roll back its protections. These include H.R. 5094, S. 2447, and similar legislation. The Task Force is actively working with bipartisan members of Congress to defend this law and ensure that rare disease research incentives remain intact.
Repealing ORPHAN Cures would re-introduce the unintended consequences that discouraged pharmaceutical companies from pursuing additional rare disease indications — directly harming the patients who rely on continued innovation. The Task Force urges Congress to reject efforts to repeal or weaken the ORPHAN Cures Act.
The Orphan Cures Act Addressed an Overly Narrow Exclusion in Statute
The Medicare Drug Price Negotiation Program
In 2022, Congress created the “Medicare Drug Price Negotiation Program”. This program requires CMS to set prices for certain drugs covered under Medicare Part B (physician-administered drugs) and Part D (retail prescription drugs), starting with 10 high-spending, single-source drugs for 2026 and increasing to 20 per year by 2029.
The Program excluded orphan drugs treating rare diseases from CMS negotiation eligibility. However, this exclusion was limited to drugs treating a single rare disease. This narrow exclusion discouraged research and development of treatments for other rare diseases. The passage of ORPHAN Cures addressed this unintended consequence of the Program, allowing for rare disease drugs to receive the exemption and protecting research incentives for subsequent rare disease indications.