Advocacy
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.
Related Materials
- Task Force Celebrates ORPHAN Cures Becoming Law
- Task Force Statement of Support for Senate Introduction of ORPHAN Cures
- Task Force Statement of Support for ORPHAN Cures in Reconciliation
- Senators Barrasso and Heinrich Introduce ORPHAN Cures Act
- Task Force ORPHAN Cures Act One-Pager
- Task Force ORPHAN Cures Act Support Letter
- House Legislation (H.R. 946 ORPHAN Cures Act)
- Biotechnology Industry Organization: BIO Applauds Introduction of Bipartisan ORPHAN Cures Act
- California Life Sciences: California Life Sciences Supports the ORPHAN Cures Act to Advance Rare Disease Research
- Rare Disease Company Coalition: New Bill Protects Continued Development for Rare Disease Medicines
- Global Liver Institute: The ORPHAN Cures Act.
Congressional Cosponsors
Thanks to the bipartisan, bicameral cosponsors who championed ORPHAN Cures on Capitol Hill.
- John Barrasso [R-WY]
- Martin Heinrich [D-NM]
- John Joyce [R-PA-13]
- Donald G. Davis [D-NC-1]
- Lloyd Smucker [R-PA-11]
- Janelle Bynum [D-OR-5]
- Thomas H. Kean [R-NJ-7]
- Dina Titus [D-NV-1]
- Kevin Hern [R-OK-1]
- Scott H. Peters [D-CA-50]
- Mariannette Miller-Meeks [R-IA-1]
- William R. Keating [D-MA-9]
- Richard Hudson [R-NC-9]
- Shri Thanedar [D-MI-13]
- Gus M. Bilirakis [R-FL-12]
- Josh Gottheimer [D-NJ-5]
- Andrea Salinas [D-OR-6]
- Neal P. Dunn [R-FL-2]
- Adam Gray [D-CA-13]
- Dan Crenshaw [R-TX-2]
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.
The Scale of the Problem
The Urgency of Rare Diseases
An estimated 30 million Americans have a rare disease, which is a disease affecting 200,000 Americans or less. Today, less than 10 percent of Americans with a rare disease have access to an FDA-approved treatment. That means most Americans with a rare disease have no treatment specifically designed to treat their disease.
According to the FDA:
- “Drug…development in rare diseases is challenging for many reasons, including the complex biology and the lack of understanding of the natural history of many rare diseases. The inherently small population of patients with a rare disease can also make conducting clinical trials difficult.”
- Because of the challenges in rare disease research and development, Congress passed the Orphan Drug Act in 1983 to provide incentives that increased rare disease research. Those incentives have resulted in a 1,576% increase in the number of FDA-approved orphan drugs – from 38 to 600+ therapies to treat more than 1,100 indications. However, a new policy in the Medicare Drug Price Negotiation Program affects those incentives, which are still urgently needed to foster rare disease treatment innovation and address critical unmet needs.