Posted June 30, 2023
CMS today released revised guidance on the Inflation Reduction Act Medicare Drug Price Negotiation Program, clarifying that a drug with more than one designation for a rare disease or condition will not qualify for the Orphan Drug Exclusion, even if the drug has not been approved for any additional indications. Unfortunately, by making orphan products with a second designation eligible for drug price negotiation, this provision will disincentivize further investment in rare disease research and development.
Why it’s important: This policy will impact the rare disease drug development pipeline for decades to come, increasing health disparities for the 30 million Americans living with rare, debilitating diseases, 50% of which affect children.
Undermining the bipartisan Orphan Drug Act (ODA): The ODA was enacted to incentivize the development of drugs for rare diseases and has demonstrated overwhelming evidence of success.
The final word: The RDCC is disappointed in CMS’s revised guidance and remains committed to working with stakeholders in the Administration and Congress to find ways to mitigate the impact of this policy on Americans living with rare diseases.