Background and objectives: To compare the utilization and costs (total and out-of-pocket) of new-to-market neurologic medications with existing guideline-supported neurologic medications over time.
Methods: We used a healthcare pharmaceutical claims database (from 2001-2019) to identify patients with both a diagnosis of one of 11 separate neurologic conditions and either a new-to-market medication or an existing guideline-supported medication for that condition. Neurologic conditions included orthostatic hypotension, spinal muscular atrophy, Duchenne's disease, Parkinson's disease, Multiple sclerosis, amyotrophic lateral sclerosis, myasthenia gravis, Huntington's disease, tardive dyskinesia, transthyretin amyloidosis, and migraine. New-to-market medications were defined as all neurologic medications approved by the FDA between 2014 and 2018. In each year, we determined the median out-of-pocket and standardized total cost for a 30-day supply of each medication. We also measured the proportion of patients receiving new-to-market medications compared with all medications specific for the relevant condition.
Results: We found that the utilization of most new-to-market medications was small (<20% in all but one condition), compared to existing, guideline-supported medications. The out-of-pocket and standardized total costs were substantially larger for new-to-market medications. The median (25th percentile, 75th percentile) out-of-pocket costs for a 30-day supply in 2019 were largest for edaravone ($712.8 ($59.8, $802.0)) and eculizumab ($91.1 ($3.0, $3,216.4)). For new-to-market medications, the distribution of out-of-pocket costs were highly variable and the trends over time were unpredictable compared to existing guideline-supported medications.
Discussion: Despite the increasing number of FDA-approved neurologic medications, utilization of newly approved medications in the privately insured population remains small. Given the high-costs and similar efficacy for most of the new medications, limited utilization may be appropriate. However, for new medications with greater efficacy, future studies are needed to determine if high costs are a barrier to utilization.
© 2022 American Academy of Neurology.