Rare Disease Patients Face Barriers in Access and Care

People with Friedreich's ataxia face debilitating symptoms, too few medical specialists and too many health insurance barriers, physicians explain in new policy paper

WASHINGTON, Feb. 5, 2025 /PRNewswire/ -- An FDA-approved medication can now treat Friedreich's ataxia, but health plan barriers and formulary exclusions keep many patients from receiving it.

In a white paper by the Alliance for Patient Access and the Friedreich's Ataxia Research Alliance, titled "Friedreich's Ataxia: A Rare Condition Facing Common Access Challenges," physician authors outline the disease's debilitating symptoms as well as the treatment challenges that accompany it. The rare genetic condition often begins in childhood and affects the brain, heart and neurological system. 

After years with no dedicated treatment option, the FDA approved omaveloxolone in 2023. Hailed as "an exciting step forward for the FA community," the medication remains out of reach for many patients. Some health plans have excluded the new drug from their formulary. Others cover it but only with onerous prior authorization and step therapy protocols, which delay care for patients already struggling with the disease's symptoms.

These barriers compound existing challenges. Finding a specialist or clinician who can diagnose Friedreich's ataxia, given its rarity, remains difficult. Clinician knowledge gaps and transportation challenges often mean delayed diagnosis or misdiagnosis.

"Due to the progressive nature of Friedreich's ataxia, people living with the condition cannot afford delays in access to knowledgeable healthcare professionals or treatment," says Kellyn Madden, patient engagement manager for the Friedreich's Ataxia Research Alliance. "The FA community has partnered for many years with researchers to bring treatments forward. This white paper marks a significant step in raising awareness and advocating for policies that facilitate access to this care."

The white paper proposes several potential policy solutions, including:

• Limits on health plan utilization management
• Expanded coverage for telehealth
• Support for innovation and more opportunities for children to participate in clinical trials

STATEMENT FROM JOSIE COOPER, EXECUTIVE DIRECTOR OF THE ALLIANCE FOR PATIENT ACCESS: "Patients with Friedreich's ataxia deserve access to innovative treatments and patient-centered care. With health plan barriers negatively impacting these patients' physical and mental health, policy change is critical."

Learn more in "Friedreich's Ataxia: The Rare Condition Facing Common Access Challenges."

About the Alliance for Patient Access
The Alliance for Patient Access is a network of policy-minded health care providers who advocate for patient-centered care.

About the Friedreich's Ataxia Research Alliance
The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), nonprofit, organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich's ataxia.

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SOURCE Alliance for Patient Access