Save Rare Treatments Task Force

Because Every Person With a Disease Deserves Treatment Options

Join our mission to protect bipartisan Orphan Drug Act incentives that have made rare disease therapies a reality.

The Scale of the Problem

The Urgency of Rare Diseases

An estimated 30 million Americans have a rare disease. A rare disease is a disease affecting 200,000 Americans or less. Of the estimated 10,000 rare diseases, 95 percent lack 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.”
“Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases.” It is critically important to protect the important incentives of the Orphan Drug Act.

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.

Watch: Drugs Treating Rare Disease & Medicare Drug Price Negotiation

Paul Kim, an attorney with more than 30 years of experience in federal health policy, including at the FDA, explains the problem with the narrow exclusion for orphan drugs in the Medicare Drug Price Negotiation Program.

The overly narrow exclusion undermines longstanding incentives for medical innovation to treat rare diseases and thereby jeopardizes access to future treatments for persons living with rare diseases.

Work with Congress and the Administration to ensure that the Orphan Drug Exclusion from eligibility for negotiation under the Medicare Drug Price Negotiation Program allows products to remain excluded so long as their FDA approved uses are exclusively for rare diseases.

Restore the spirit of the research and development incentives in the Orphan Drug Act that have succeeded for more than 40 years so more persons living with rare diseases can have treatment options.

The Task Force is a growing, multi-sector public policy and advocacy collaboration of organizations representing persons living with rare diseases, biopharmaceutical innovators, and other health care stakeholders.