Earlier this month, the EveryLife Foundation for Rare Diseases submitted comments to the Centers for Medicare and Medicaid Services (CMS), urging them to reject a proposal contained within Oregon’s 1115 Waiver Application that excludes therapies approved using the accelerated approval pathway from Medicaid coverage.
If approved by CMS, the Oregon 1115 Waiver would allow the Oregon Health Authority (OHA) to alter the state Medicaid program to restrict access to therapies approved using the accelerated approval pathway. It would also allow OHA to establish their own review process to assess the clinical safety and efficacy of these therapies. If CMS approves this waiver, it will set a precedent for other states to apply for similar waivers, potentially restricting access to accelerated approval therapies for rare disease patients across the nation.
An 1115 Waiver refers to the section of the Social Security Act that gives authority to the Secretary of Health and Human Services to waive certain provisions of Medicaid law, giving flexibility to state programs.
In urging CMS to reject the portion of Oregon’s application related to the accelerated approval pathway, EveryLife stressed the importance of upholding the U.S. Food & Drug Administration’s (FDA) role in determining the safety and efficacy of medical products. EveryLife asserted that Oregon’s proposal undermines the FDA’s statutory authority, their expertise and understanding of the complexities involved in bringing therapies to the market for complex rare diseases, and the high level of rigor in the FDA regulatory review process.
The accelerated approval pathway allows FDA to use a surrogate endpoint that is reasonably likely to predict clinical benefit to evaluate the safety and efficacy of therapies for serious conditions with unmet needs, enabling earlier access to therapies while ongoing research is conducted to verify the expected clinical benefit. In its comments, EveryLife identified numerous misperceptions about the accelerated approval pathway evident in OHA’s rationale for its drastic change to Medicaid drug coverage policy. EveryLife cited recently released resources on the pathway and early data from an ongoing EveryLife study that shows nearly three out of four therapies brought to market using the pathway prior to 2020 have been converted to traditional approval with more studies under way. The comments also pointed to data from the Partnership to Fight Chronic Disease, showing these therapies account for just a .5% increase in the Medicaid spending growth in Oregon, highlighting their minimal impact on the state budget.
Read EveryLife’s full comment letter to CMS.
Read EveryLife’s comment letter to OHA earlier this year.
EveryLife, along with its Community Congress partners, continues to monitor and actively engage in addressing proposals that could threaten access to safe and effective therapies such as those approved via the accelerated approval pathway, while working with all stakeholders to identify long-term policy solutions.
