Spending on Targeted Therapies for Duchenne Muscular Dystrophy

The US spent an estimated $3 billion on targeted therapies for Duchenne muscular dystrophy from 2016-2023, despite limited evidence of effectiveness and delayed confirmatory trials. More than $1 billion was paid by Medicaid and Medicare. Policymakers should compel the timely completion of confirmatory trials for these therapies and implement alternative payment models to minimize financial risk in case novel interventions prove ineffective.