Presentation (CTI)

OBJECTIVES: The importance of clinical outcome assessments (COA) in healthcare decision making is increasingly recognized. However, evidence on associations between non-mandatory COA inclusion in Health Technology Assessment submissions (i.e., COAs not directly requested or explicitly expected to be included by HTA bodies), HTA decision making, pricing, and sales of medicinal products is scarce. This study aimed to generate new insights into these associations.
METHODS: HTAs 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 AIML models were built to analyse the relationship 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 HTAs, 40.5% included non-mandatory COAs. In Germany and France, HTAs with non-mandatory COA were more likely to receive beneficial ratings (Germany: 47%, France: 42%), compared to HTAs without (12% and 24%, respectively). Non-mandatory COA inclusion was unrelated to HTA ratings in England. In Germany, non-mandatory COA inclusion in HTAs was associated with a 4% lower rebate and a 27% higher premium, yet these trends were non-significant. Predictive performance of the AIML models tentatively suggested that non-mandatory COA inclusion may be associated with higher sales in Germany and England, but not in France.
CONCLUSIONS: In France and Germany, positive HTA ratings were more likely for medicinal products with HTAs that included non-mandatory COAs. In Germany and England, trends suggested a relation with higher pricing and/or sales. Further research is needed to corroborate the findings.