Rare Disease Patients Are Not Consistently Heard in Medicaid Coverage Reviews, Study Shows
- 6 days ago
- 3 min read
Let My Doctors Decide Action Network (LMDDAN) was launched to address access barriers in autoimmune disease, documenting the prior authorization requirements, step therapy protocols, and coverage denials that insurers used to delay or redirect patients away from physician-prescribed therapy. That work revealed a consistent pattern: Administrative tools presented as cost management measures were functioning as barriers to care, often without clinical justification.
With a new study, LMDDAN has begun utilizing this experience to research barriers in rare disease.
As rare disease therapies are developed, it’s important for state Medicaid programs to keep pace in covering the treatments physicians prescribe. LMDDAN’s nine-state study paints a troubling picture of how geography shapes medical access in America today, underscoring the urgent need for federal standards that establish consistent, transparent decision-making procedures nationwide.
While all states reviewed have a Drug Utilization Review (DUR) and/or Pharmacy & Therapeutics (P&T) Committee, there is little uniformity in how these committees operate, how treatments are evaluated, or how coverage decisions are made. Meeting frequency, public comment procedures, and meeting agenda release timelines differ widely, making it challenging for patients, advocates and medical professionals to effectively participate in coverage decision-making.
LMDDAN believes Congress and the Centers for Medicare & Medicaid Services have a role to play, establishing baseline standards that would ensure more consistent access nationwide.
"Our study confirms what many people with rare diseases and their families already know from lived experience: Where you live in America can determine whether you have access to the treatment your doctor prescribes," said Brett McReynolds, policy advisor for LMDDAN. "The inconsistencies revealed in this report underscore the critical need for greater transparency, standardization and patient-centered approaches in state Medicaid coverage determinations.”
The report included the following recommendations for procedural improvement:
Require rare disease expertise to inform drug coverage reviews and policy development
Invite a rare disease physician expert to offer their clinical expertise to inform disease background and treatment approach when developing a coverage policy for a rare disease drug
Offer an opportunity for the drug innovator to present clinical and scientific data
Improve transparency and stakeholder input in Medicaid P&T and DUR meetings
Publish a step-by-step explanation of the DUR/P&T Committee process
Ensure DUR/P&T Committee members do not have conflict of interests
Provide at least 30 days’ advance notice of meeting date, specific agenda topics (i.e. drugs to be reviewed), and draft coverage policies for discussion
Offer virtual interactive access to DUR/P&T committee meetings, or similar body, to encourage and allow diverse stakeholder participation, including clinician, patient, caregiver, patient advocate and manufacturer
Allow public testimony during each individual drug review, and prior to DUR/P&T vote on coverage policy
Allow adequate time for individual public comment (i.e. five minutes), and do not limit the number of stakeholders
Publish meeting minutes and drug coverage policies within 14 days of meeting
Align drug coverage policies to the FDA-approved, medically accepted indication
Publish coverage policies to a central repository on the state’s Medicaid webpage
Share coverage policies with providers and with the state’s contracted managed care organizations (MCOs) to ensure consistent patient access within the state
Collectively, adoption of these best practices will ensure a more transparent and accessible platform for all stakeholders to inform the development of drug specific coverage policies in the best interest of rare disease patients.
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