Non-mandatory clinical outcome assessments in health technology assessment reviews in England, France, and Germany: exploratory associations with benefit ratings, pricing, and sales

Abstract Background The importance of clinical outcome assessments (COA) in healthcare decision making is increasingly recognised. However, evidence on associations between non-mandatory COA inclusion, health technology assessment (HTA) decision making, pricing, and sales of medicinal products is scarce. This study aimed to generate new insights into these associations. Methodology HTA reports published in 2012–2022 by bodies in Germany (G-BA), France (HAS), and England (NICE) were retrieved and combined with pricing and sales data. Multiclassification machine learning models were built to analyse the associations between non-mandatory COA inclusion and HTA-related decision making and sales against other relevant features. For Germany, the association with pricing was analysed in addition. Results Of 1506 identified HTA reports, 40.5% included non-mandatory COAs. In Germany and France, beneficial ratings were observed more often among HTA reports with non-mandatory COA (Germany: 47%, France: 42%) than among HTA reports without such COAs (12% and 24%, respectively). Non-mandatory COA inclusion was unrelated to HTA ratings in England. In Germany, rebates and achieved list price premiums did not differ significantly between HTA reports with and without non-mandatory COAs. Non-mandatory COA inclusion appeared among the model features in Germany and England, but not France. Given the low accuracy of the sales models, the sales findings should be regarded as exploratory only. Conclusions In France and Germany, positive HTA ratings were more likely for medicinal products with HTA reports that included non-mandatory COAs. In Germany, achieved list price premiums were numerically higher for products with HTA reports that included non-mandatory COAs, although the difference was not statistically significant. In Germany and England, pricing and/or sales were numerically, but statistically insignificantly, higher. Sales models linked COA-related features to higher predicted sales categories in Germany and England. Exploratory sales models linked COA-related features to higher predicted sales categories in Germany and England. Given the low accuracy of the sales models, findings related to sales should be regarded as exploratory.