Orphan-only drug exclusion marks encouraging progress for rare disease patients and innovation as the community awaits additional pricing model rulemaking.
Washington, D.C. — The Rare Disease Company Coalition (RDCC) today commended the Centers for Medicare & Medicaid Services (CMS) for ensuring that medicines approved exclusively for one or more rare diseases or conditions will be excluded from the agency’s latest pricing model. CMS’s final Global Benchmark for Efficient Drug Pricing (GLOBE) Model ensures that orphan-only drugs covered under Medicare Part B will not be subject to the model’s international pricing benchmark or associated manufacturer rebates.
The exclusion will be critical for protecting timely patient access to treatments and sustained investment in rare disease research. With approximately 95 percent of rare diseases still lacking an FDA-approved treatment, policies aimed at achieving greater affordability must preserve the incentives necessary to develop new therapies and bring them to patients.
“RDCC commends the adoption of this important protection for rare disease patients and the innovators working to develop new treatments. We also thank our congressional champions who advocated on behalf of the rare disease community and helped elevate concerns about the GLOBE Model’s potential impact on rare disease patients and investment. Further, we appreciate CMS’ implicit acknowledgment of the licensing intricacies of the small biotechs who are the backbone of rare disease innovation, and we will continue to educate on these challenges. While RDCC continues to have concerns about the broader implications of international reference pricing, we appreciate the CMS’ decision to exclude orphan-only therapies from the final model.
“We are grateful to the RDCC members and partners who joined our advocacy efforts with Congress and the administration on this issue. As we await a final decision on the related Guarding U.S. Medicare Against Rising Drug Costs (GUARD) Model, we urge CMS to extend the same protection to orphan-only drugs covered under Medicare Part D.”
The five-year GLOBE Model will begin January 1, 2027, and extend through March 31, 2032.
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