Presentation (CTI)

OBJECTIVES: The 2022 Inflation Reduction Act (IRA), criticized for hindering drug research and development (R&D) sets Maximum Fair Prices (MFP) for 10 drugs 2026 onwards. Although orphan drugs are exempt, the criteria create financial challenges for manufacturers. This study explores the IRA's impact on rare disease research.
METHODS: We reviewed policy papers, academic databases, Professional Society for Health Economics and Outcomes Research (ISPOR) conference abstracts, and company press releases to assess the IRA's potential impact on rare diseases.
RESULTS: The Food and Drug Administration (FDA) reports over 7,000 rare diseases affecting 30 million Americans, most without approved treatments. The IRA exempts orphan drugs from price negotiation only if they have a single indication, potentially leading to: Reduced research and development: Drug development for rare diseases faces challenges like small sample sizes, limited understanding, and complex FDA trial requirements. While the 1983 Orphan Drug Act incentivizes, IRA restrictions on multiple indications for exemptions reduce Return on Investment (RoI), prompting disinvestment. Reports indicate manufacturers are already halting rare disease projects. Delayed launch or non-availability: Manufacturers may prioritize launching orphan drugs in markets like Europe, and Japan, shifting away from the US-first approach and limiting access to innovative medicines for Americans. Alternatively, manufacturers might prioritize high burden disease areas as first indication to offset price negotiation. Reduction in subsequent indications: Between 2003-2022, 23% of the 282 FDA-approved orphan drugs received additional indications, requiring further R&D investment. However, IRA restriction now makes this financially unviable. Impact on indications in same disease: It remains unclear if line or age-specific indications will count as subsequent indications. The decision on treatments like Tagrisso, approved for multiple non-small cell lung cancer indications, needs to be observed.
CONCLUSIONS: The IRA's single-indication exclusion for rare disease may deter manufacturers from pursuing additional labels, limiting treatments, and potentially impacting global markets.