In a monumental victory for the rare disease community, the Centers for Medicare and Medicaid Services (CMS) approved the Oregon Health Plan’s 1115 Demonstration Waiver WITHOUT the provision that would have permitted the state to exclude coverage for prescription drugs approved using the accelerated approval pathway.
The approved waiver provides several expanded health services for the residents of Oregon, including continuous enrollment in Medicaid to limit the disruption of services for some children and adults and expanded programs to address social determinants of health within the state.
The CMS approval letter references the more than 100 public comments received by the agency, many of which opposed the planned changes to Oregon’s prescription drug benefit. The letter goes on to note that the state has withdrawn the proposed prescription drug benefit changes, thereby protecting access to safe and effective therapies that are approved using the accelerated approval pathway.
The accelerated approval pathway allows FDA to use a surrogate endpoint (also called a biomarker) to evaluate the safety and efficacy of therapies for serious conditions with unmet needs. By evaluating a surrogate endpoint that is reasonably likely to predict clinical benefit, the accelerated approval pathway allows patients to access treatments at the earliest possible moment while ongoing research is conducted to verify the expected clinical benefit.
If the waiver had been approved with the prescription drug provision, Oregon’s proposal would have caused ripple effects across the country. Other states likely would have requested and been granted similar permissions, creating inequities in access to therapies approved using the accelerated approval pathway and crippling innovation for the 95% of rare diseases that still lack an approved therapy.
This victory would not have been possible without the rare disease organizations and advocates who submitted comments to CMS in the spring to raise concerns about the harmful effects of Oregon’s proposal for the rare disease community. Oregon’s decision to drop the drug benefit changes is a direct testament to the impact of community engagement and the value of data driven policy efforts.
For more information on CMS’s response to the Oregon 1115 Waiver application, read the letter of approval.
