Presentation (CTI)

OBJECTIVES: Neuromuscular diseases (NMDs) comprise a heterogeneous group of disorders affecting nerve and muscle function, often resulting in immobility and requiring long-term treatment. In their drug coverage policies, many health plans include explicit criteria that patients must meet to qualify for treatment continuation. In this study, we examined the extent to which health plans’ reauthorization criteria for NMD therapies align with the endpoints included in the therapies’ registration studies.
METHODS: We identified reauthorization coverage criteria for 18 NMD therapies issued by 18 large US commercial health plans from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database. Decisions were current as of December 2024. We categorized reauthorization criteria as requiring “general/adequate response”, e.g., evidence of a positive treatment response, or “specific clinical outcomes”, e.g., a symptom score below a defined threshold on a disease-specific scale. For decisions requiring specific clinical outcomes, we compared these requirements with registration clinical trial endpoints and categorized them as (1) consistent with the clinical trial endpoint, (2) partially consistent, i.e., the plan used the same measure but imposed a different threshold, or (3) inconsistent, i.e., using measures not assessed in clinical trials.
RESULTS: Our sample included 401 NMD coverage decisions. Health plans included reauthorization criteria in 75.6% (303/401) policies (plan-level range, 34.6%-92.0%). Among these, 74 (24.4%) required evidence of a general/adequate response, and 229 (75.6%) required specific clinical outcomes. Of the policies requiring specific clinical outcomes, 66 (28.8%) were categorized as consistent with registration-study endpoints (plan-level range, 0%-61.5%), 140 (61.1%) as partially consistent (28.6%-82.3%); and 23 (10.0%) as inconsistent (0%-28.6%).
CONCLUSIONS: Reauthorization criteria for NMD therapies vary widely across US commercial health plans and frequently diverge from clinical trial endpoints. Although most plans require specific clinical outcomes for continued coverage, these requirements typically do not fully align with clinical trial endpoints.