New Study Reveals Wide Variance in State Medicaid Review of Rare Disease Treatments

Nine-State Analysis Shows Significant Differences in Review Procedures, Meaningful Patient Advocacy Opportunities, and Availability of Published Coverage Policies for Life-Altering Therapies

WASHINGTON, D.C. (April 21, 2026) — Today, Let My Doctors Decide Action Network (LMDDAN) released a study that explores how state Medicaid programs make decisions about coverage for new therapies to treat rare diseases. The analysis revealed significant inconsistencies in publicly available information, review procedures, and opportunities for patients and clinicians to meaningfully contribute their expertise to inform coverage decisions. These findings reflect a common frustration within the U.S. rare disease community, where coverage of new therapies can vary dramatically based on state of residence.

LMDDAN, an advocacy organization dedicated to advancing patient access to physician-prescribed treatments, examined Medicaid coverage decision processes for four recently FDA-approved rare disease treatments in nine states: Colorado, Georgia, Illinois, Kansas, Massachusetts, Minnesota, Mississippi, Oregon and Virginia. The four therapies evaluated were Skyclarys for Friedreich's ataxia, Livdelzi for primary biliary cholangitis, Elevidys for Duchenne muscular dystrophy, and Qalsody for amyotrophic lateral sclerosis (ALS).

The report contains targeted policy recommendations to strengthen state review processes by incorporating rare disease expertise, establishing consistent transparency standards, and ensuring patient and physician input precedes adoption of coverage policies.

Key Findings:

• While all states analyzed have a Drug Utilization Review (DUR) and/or Pharmacy & Therapeutics (P&T) Committee, there is little uniformity in how these committees operate, which treatments are evaluated, or how coverage policies are developed.

• DUR and P&T Committee meeting frequency, public comment procedures, and advance notice of review agendas differ widely among states, making it challenging for patient advocates and medical professionals to effectively participate in coverage decision-making processes.

• Opportunities for drug manufacturers to present clinical information and for patients, physicians, and advocates to share perspectives vary significantly by state. Some states offer robust engagement mechanisms while others provide minimal opportunities for meaningful stakeholder input. For example, one state's DUR Committee held in-person public comment after board members already voted to adopt coverage policies.

"This 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 urgent need for greater transparency, standardization and patient-centered approaches in state Medicaid coverage decisions.

“By documenting these differences in committee procedures, stakeholder engagement mechanisms and timeline frameworks, we hope to provide a foundation for identifying practices that can enhance transparency and accessibility across state programs. Congress and the Centers for Medicare & Medicaid Services have an opportunity to establish baseline standards that would ensure more equitable access nationwide."

LMDDAN based its analysis on data from state Medicaid websites, personal observations of Pharmacy & Therapeutics (P&T) Committee and Drug Utilization Review (DUR) Board meetings, and other public information.

Additional Study Findings:

• For the four therapies evaluated, the time from FDA approval to a drug's review by a P&T/DUR Committee varied dramatically across the nine states, ranging from 1 to 18 months post-approval, with most common reviews occurring eight to 12 months after FDA approval. Oregon and Massachusetts were the only states to publish a coverage policy for all four therapies analyzed.

• The nine states studied did not uniformly review all four rare disease drugs through their P&T/DUR Committee processes. For example, Qalsody, for the treatment of Amyotrophic Lateral Sclerosis (ALS), was reviewed by only two states. Elevidys, for the treatment of Duchenne muscular dystrophy, was reviewed by eight of the nine states. Massachusetts did not review any of the four drugs through a public P&T/DUR committee process.

• Meeting materials were not consistently published with advance notice. While approximately half of the surveyed states publish detailed agendas 30 days before meetings, some provided as little as 2 days' notice. Some states released draft drug coverage policies prior to a public review (ranging from 1 to 60 days advance notice), but several states did not publish in advance, including Colorado, Illinois and Virginia.

• Six of the nine states offered a virtual participation option at their meetings. Georgia, Mississippi, and Virginia held meetings exclusively in-person, restricting the ability of patients, many of whom live with conditions that limit their mobility, to engage in coverage decision-making.

• Notably, three states demonstrated innovative approaches to incorporating clinical expertise. Minnesota and Oregon allowed rare disease subject matter experts to serve as P&T Committee members during drug reviews. Georgia offered manufacturers the opportunity to participate in clinical reviews weeks prior to P&T meetings, ensuring coverage policies are informed by comprehensive scientific data.

The recommendations detailed in LMDDAN’s report call for requiring rare disease expertise to inform drug coverage reviews and policy development, improving transparency and stakeholder input in Medicaid P&T and DUR meetings, and aligning drug coverage policies to the FDA-approved, medically accepted indication.

The study emphasizes that all stakeholders — state Medicaid programs, patients and families, caregivers, patient advocates, treating physicians, drug manufacturers, and policy organizations — share an interest in improving health outcomes for patients with rare diseases. Understanding state-by-state variations in coverage review processes represents an important step toward optimizing treatment access and enabling improvements in state coverage infrastructure.

Let My Doctors Decide Action Network

LMDDAN is a 501(c)(4) organization uniting patient advocates, healthcare providers, and policy experts to advance meaningful reforms that eliminate barriers to essential care.