Approved doesn’t always mean accessible. This rings especially true for patients with a rare condition known as Friedreich’s ataxia.
As a new clinician-authored policy paper from the Alliance for Patient Access and Friedreich’s Ataxia Research Alliance explains, health plan barriers can complicate access to treatment. That includes the first FDA-approved medication specifically for the genetic disease. “Friedreich’s Ataxia: A Rare Condition Facing Common Access Challenges” explores the challenges that patients face and outlines several policy solutions.
Undergoing the Diagnostic Odyssey
Before seeking treatment, patients must first put a name to their struggle. It isn’t easy.
Like many rare disease patients, people with Friedreich’s ataxia often struggle to receive a proper diagnosis. Symptoms of Friedreich’s ataxia typically begin in childhood, but many pediatricians don’t recognize the signs or misinterpret them. Patients are often misdiagnosed with other neurological conditions such as multiple sclerosis.
This long and difficult process is known as the “diagnostic odyssey.”
Overcoming Treatment Hurdles
Once patients are diagnosed, they may face barriers to treatment.
The Friedreich’s ataxia community welcomed the approval of omaveloxolone, the first FDA-approved medication for the rare disease, in 2023. But health plans’ red tape prevents some patients from receiving the drug.
Prior authorization and step therapy, for example, can delay care and burden providers and staff with excessive paperwork requirements. These tactics are often imposed by pharmacy benefit managers, who can also forego coverage of a medication altogether. In fact, the Friedreich’s ataxia community currently faces a formulary exclusion, with Express Scripts completely removing omaveloxolone from plan coverage.
Medication access issues are compounded by barriers to care. There are only a few Friedreich’s ataxia specialists in the United States. Finding a specialist is a challenge and can require patients to travel long distances. Virtual appointments could reduce the burden of travel and inaccessibility to specialists, but some health plans do not cover telehealth visits. In other instances, restrictions on treatment across state lines limits patients’ abilities to access the closest and most knowledgeable provider.
These barriers make it difficult to access treatment and weigh on patients who are already under physical and mental strain.
Improving Treatment Access
Policymakers can help reduce these limitations.
Decisionmakers can drive change and improve the life of the Friedreich’s ataxia community, specifically by:
- Restricting utilization management to ensure that patients receive timely and effective treatment
- Expanding telehealth coverage to allow more patients access to the specialty care they need
- Encouraging innovation, resulting in more approved treatments for Friedrich’s ataxia patients in the years to come
The FDA’s approval of the first Friedreich’s ataxia medication marked an important milestone for the patient community. With sound policy decisions to support them, these patients can connect with the treatment, the specialty care and the quality of life that they deserve.
Read “Friedreich’s Ataxia: The Rare Condition Facing Common Access Challenges” to learn more.
