Washington, D.C., June 27, 2018 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, released the following statement regarding this week’s actions by the Centers for Medicare and Medicaid Services (CMS) to protect patient access to medication within Medicaid formularies:
“NORD lauds the Centers for Medicare and Medicaid Services (CMS) for its actions this week to protect patient access to therapies approved by the Food and Drug Administration (FDA) within the Medicaid program.
In today’s notice, CMS reaffirmed that medications approved through FDA’s Accelerated Approval Program must be accessible to patients in the Medicaid program because these therapies are covered under existing Medicaid Drug Rebate agreements. CMS further reaffirmed that therapies approved via the Accelerated Approval Program are subject to the same evidentiary standards as any other FDA-approved medicines.
Without the Accelerated Approval Program, many individuals with rare diseases would be left with few, if any, treatments because traditional clinical trials would be nearly impossible to conduct. NORD believes it is crucial that FDA continue to utilize these tools, and CMS continue to support access to these therapies once approved.
This week, CMS also rejected elements of Massachusetts’ section 1115(a) Medicaid demonstration request, otherwise known as ‘MassHealth,’ that would have restricted access to new breakthrough therapies for Medicaid beneficiaries. Massachusetts proposed to implement a closed formulary within their Medicaid program and specifically proposed to limit coverage of drugs approved through the Accelerated Approval Program. If states choose to pursue alternative permissible demonstration projects, NORD strongly requests that anything that would limit patient access to medically-necessary treatment would not be pursued.
In February, NORD joined over 100 patient organizations in sending a letter to Medicaid Directors across the country highlighting the importance of formulary access for rare disease patients and seeking opportunities for collaboration. Additionally, NORD and its RareMA advocates opposed the implementation of Massachusetts’ waiver in comments sent to both the state and to CMS. We are incredibly grateful that CMS took the voice of the patient community into consideration, and we look forward to continuing to work with CMS and various states in guaranteeing access to medically-necessary treatment for all rare disease patients.”