Last month, 125 rare disease patient organizations sent a letter to Medicaid directors from individual states highlighting the importance of Medicaid access for rare disease patients.
“As organizations representing millions of Americans with rare diseases, we are writing to you about the importance of preserving patient access to orphan therapies in your Medicaid program. In sending this letter, we hope to foster a dialogue with you on the best way to engage with patient organizations and other rare disease experts to improve patient access to innovative new medicine.”
The letter is appealing to state leaders to consider how patients with rare diseases will be affected by likely cuts and reductions.
States are under pressure to control healthcare costs, but patient leaders worry that the rare disease community will get the short end of the stick.
“We believe that these decisions disproportionately affect rare disease patients because they are not suffering from a more prevalent condition even though they are no less deserving of treatment options.”
The letter outlines three direct calls to action that patient leaders hope can have a great impact for the rare disease community:
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Reconsider legislative actions that could prevent drug access to patients. The letter specifically calls out waivers that seek an exemption to Section 1927 of the Social Security Act, which may harm patients seeking coverage for new medications that provide an enhanced clinical benefit over existing treatment options.
“Excluding coverage for drugs that utilize FDA’s expedited programs like accelerated approval could rob rare disease patients, many of whom are children, of access to FDA-approved medicines that may be their only treatment option.”
2. Open up the channels of communication between health policymakers and the rare disease community. The letter even announces Tim Boyd of the National Organization for Rare Disorders (NORD) as a liaison of sorts to discuss the issues raised in the letter or as a patient representative for engagement on any issues that need patient perspectives.
3. Lastly, given the federal prioritization of innovative orphan product development, the letter proposes that states provide additional assistance to cover these products for Medicaid beneficiaries.
“We would appreciate feedback from your state on the necessity and potential structure of such assistance, and on other opportunities to innovate when it comes to meeting the needs of the rare disease community.”
