Use of real-world evidence in economic assessments of pharmaceuticals in the United States.

Despite the increasing interest in expanding the use of real-world evidence (RWE) in economic assessments of pharmaceuticals, decision makers face uncertainty about how RWE should be used. To assess the use of RWE in economic assessments of drugs by the Institute for Clinical and Economic Review (ICER). We reviewed cost-effectiveness and budget impact analyses in final evidence reports of pharmaceuticals published by ICER. We calculated the total number of RWE uses and the proportion of model inputs informed by RWE per report. We classified model inputs into 15 categories based on their attributes and then examined what category each RWE informed to classify the reason for RWE use. Finally, we characterized RWE by study design, data source, and sponsor type. We identified 33 reports, all of which used RWE; the mean RWE use per report was 12 (range = 4-26). The average proportion of model inputs informed by RWE per report was 32.7%, but this proportion had a wide range (range = 4.1%-76.9%). RWE was most commonly used for disease progression inputs (28.7%) and health care resource utilization and costs (21.1%), but was rarely used for drug-specific clinical outcomes such as effectiveness (1.5%), adverse drug event rates (0.5%), and discontinuation rates (1.2%). The most frequently used study design was a retrospective cohort (56.6%), and the most frequently used data source was registry data (41.4%). About a third (30.2%) of RWE was industry-sponsored. RWE has been commonly used to inform pharmaceutical value assessments conducted by ICER. However, there has been relatively limited use of RWE to inform drug-specific effectiveness, despite calls for greater inclusion of RWE in value assessments for real-world drug effectiveness. This study was funded by the University of Washington School of Pharmacy Corporate Advisory Board Health Tech Fund (HTF). The funder had no role in management, analysis, and interpretation of the data; preparation, review, and approval of the manuscript; and the decision to submit the manuscript for publication. All authors were employed by the CHOICE Institute, University of Washington School of Pharmacy, at the time of the study. Carlson reports grants from the Institute for Clinical and Economic Review during the conduct of the study and personal fees from Bayer, Adaptive Biotechnologies, Allergan, Galderma, and ViFor Pharma, unrelated to this study. Veenstra reports personal fees from several manufacturers unrelated to this study. The other authors have nothing to disclose.