NEC Endorses Legislation to Encourage New Treatments for Rare Diseases
On May 9, 2024, the New England Council sent a letter to the New England congressional delegation expressing support for S. 3131 and H.R. 5539, the Optimizing Research Progress Hope and New (ORPHAN) Cures Act. Orphan drugs are drugs that target rare diseases, which are defined as diseases that affect fewer than 200,000 people in the United States. Currently, fewer than 5 percent of rare diseases have an FDA-approved treatment. Due to the nature of orphan drugs benefiting small patient populations, incentives to invest in these treatments are limited.
Currently, under the Inflation Reduction Act, orphan drugs are exempt from government price negotiations, but only if the drugs are approved for a single indication. Ultimately, this provision removes the incentive for drug manufacturers to test whether an orphan product can treat other conditions. Of the more than 280 orphan drugs approved since 2003, nearly a quarter were later approved to treat additional diseases. The ORPHAN Cures Act seeks to boost rare disease drug development to ensure that patients have access to innovative therapies by ensuring that the Medicare Drug Price Negotiation Program’s provide an exemption for orphan drugs from negotiation eligibility so long as the product is used exclusively for rare diseases.
Additionally, April 23, 2024, the New England Council hosted a program focused on challenges and opportunities for continued innovation to treat rare diseases. The ORPHAN Cures Act was mentioned repeatedly as a key tool in developing and treating rare diseases, and the New England Council is proud to support this important legislation. The importance of this legislation was a key topic of discussion at the Council’s New England Innovates: Tackling Rare Diseases event. You can watch the program HERE.
For more information on the NEC Healthcare Committee, please contact Sean Malone.