Patients’ access to rare neuromuscular disease therapies varies across US private insurers

Nikoletta M. Margaretos, Komal Bawa, Natalie J. Engmann & James D. Chambers; Orphanet J Rare Dis 17, 36 (2022). doi:10.1186/s13023-022-02182-3

The high cost of providing coverage for rare disease therapies remains a key challenge for health insurers. This challenge will increase as regulatory agencies continue to approve increasingly large numbers of rare disease therapies and healthcare payers have to balance providing access with budget constraints. The evaluated set of large US private insurers tended to apply coverage restrictions beyond the FDA label indication in their coverage policies for a set of rare NMD DMTs. Plans rarely applied the same criteria in their coverage policies for the same products. Inconsistent coverage criteria mean that patients with different insurers have variable access to the same therapies, which may have important consequences for patients who move from one plan to another.