The Legislation

Save Rare Treatments Task Force members are committed to helping Congress understand the importance of passing a critical piece of legislation for the rare disease community.

The ORPHAN Cures Act:
Bipartisan, Bicameral Legislation

Legislation Restores Incentives for Research & Development of Rare Disease Treatments

The ORPHAN Cures Act (H.R. 946)  would ensure that drugs used to treat one or more rare diseases will not be subject to government price setting under the Inflation Reduction Act and clarify the timeline used to determine when an orphan drug may become eligible for negotiation, further incentivizing the innovation of necessary rare disease treatment. Polling from The Biotechnology Innovation Organization indicates strong voter support for the ORPHAN Cures Act. The survey found that 94% of American voters prioritize research for rare disease treatments, and nearly 70% would support their representatives voting for the bill.

Related Materials

Congressional Cosponsors

See our list of growing cosponsors championing this legislation on Capitol Hill

  • John Joyce [R-PA-13]
  • Donald G. Davis [D-NC-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]

An Overly Narrow Exclusion in Statute

The Medicare Drug Price Negotiation Program

In 2022, Congress created the “Medicare Drug Price Negotiation Program” (MDPNP). 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 excludes orphan drugs treating rare diseases from MDPNP CMS negotiation eligibility. However, this exclusion is limited to drugs treating a single rare disease. This narrow exclusion discourages research and development of treatments for other rare diseases.

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.