Assessment of the Level of Satisfaction and Unmet Data Needs for Specialty Drug Formulary Decisions in the United States

Formulary management within a limited budget is critical, especially for specialty drugs, which are used for serious medical conditions and are very expensive. Despite attempts to summarize the pertinent evidence, it is uncertain whether data needs of formulary decision makers for specialty drugs are satisfied. To assess the level of satisfaction of specialty drug formulary decision makers with regards to the strength of current available data sources and unmet needs regarding clinical, economic, and unpublished evidence. This study targeted pharmacists and physicians involved with formulary decision making at health plans or pharmacy benefit management companies at the national, large regional, and local levels. 95 individuals were invited to participate (without compensation) in a 21-item, web-based survey (Qualtrics), which was open from June 14 to July 31, 2014. The responses were coded for descriptive and statistical analysis. Statistical analyses included the Kruskal-Wallis test, analysis of variance, and the Mann-Whitney-Wilcoxon test. Of 95 pharmacists or physicians, 40 respondents initiated the survey, and 33 respondents completed the survey (response rate = 34.7%). Drug formulary decision makers infrequently rated data evidence strength (17.1% "always"). Clinical data evidence strength was rated highest with published randomized controlled trials (RCTs; mean [SD] = 4.06 [0.87] of 5.0), while participant organizations' internal data were rated highest for economic data evidence strength (mean [SD] = 3.91 [1.07] of 5.0). Decision makers rated the highest unmet need as more data generated from head-to-head RCTs (mean [SD] = 2.94 [0.25] of 3.0) and cost-effectiveness analyses (mean [SD] = 2.53 [0.67] of 3.0). The participants believed manufacturers might be in the best position to satisfy their desire for head-to-head RCTs (mean [SD] = 4.31 [1.09] of 5.0). Despite a variety of data sources, drug formulary decision makers continue to rely on published RCTs or internal economic analyses as having the strongest evidence strength. The study respondents believed that pharmaceutical manufacturers would be best able to satisfy the greatest clinical data unmet need, that is, head-to-head RCTs in specialty drug formulary decisions. This study was not funded by any company or pharmaceutical manufacturer. Navarro has worked as a consultant for Biogen, Purdue Pharma, and Novartis and has offered expert testimony on behalf of AstraZeneca. The authors declare no o