Importance: Prescription drug spending in the US requires policy intervention to control costs and improve the value obtained from pharmaceutical spending. One such intervention is to apply cost-effectiveness evidence to decisions regarding drug coverage and pricing, but this intervention depends on the existence of such evidence to guide decisions.
Objective: To characterize the availability and quality of cost-effectiveness studies for prescription drugs with the greatest Medicare Part D spending.
Design, setting, and participants: In this national cross-sectional analysis, publicly available 2016 Medicare drug spending records were merged with 2016 US Food & Drug Administration Orange Book data and the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry. All studies published through 2015 that evaluated the cost-effectiveness of the 250 drugs for which Medicare Part D spending was the greatest in US-based adult patient populations were included. Data were analyzed from September 2018 to June 2020.
Main outcomes and measures: The presence and quality of published cost-effectiveness analyses for the 250 drugs for which Medicare Part D spending was greatest in 2016 were assessed based on the inclusion of key cost-effectiveness analysis elements and global ratings by independent reviewers for the Tufts CEA Registry.
Results: Medicare Part D spending on the 250 drugs in the sample totaled $122.8 billion in 2016 (84.1% of total spending). Of these 250 drugs, 91 (36.4%) had a generic equivalent and 159 (63.6%) retained some patent exclusivity. There were 280 unique cost-effectiveness analyses for these drugs, representing data on 135 (54.0%) of the 250 drugs included and 67.0% of Part D spending on the top 250 drugs. The 115 drugs (46.0%) without cost-effectiveness studies accounted for 33.0% of Part D spending on the top 250 drugs. Of the 280 available studies, 128 (45.7%) were industry sponsored. A large proportion of the studies (250 [89.3%]) did not meet the minimum quality requirements.
Conclusions and relevance: In this cross-sectional study, a substantial proportion of 2016 Medicare Part D spending was for drugs with absent or low-quality cost-effectiveness analyses. The lack of quality analyses may present a challenge in efforts to develop policies addressing drug spending in terms of value.