Presentation (CTI)

OBJECTIVES: Both the European HTA bodies and the European Society for Medical Oncology (ESMO) rate the added benefit of EMA-authorised oncology medicines. As these ratings could ultimately affect the access to novel drugs and the treatment of cancer patients accross European countries, their concordance is potentially interesting. In this study, we compared the ratings provided by the Italian Medicines Agency (AIFA), the French Haute Autorité de Santé (HAS) and the Gemeinsamer Bundesausschuss (G-BA), alongside the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS) scores, on a dataset of oncology medicines and their indications.
METHODS: The study dataset included 76 oncology medicines and 166 indications that were assessed for 'innovativeness' by AIFA Italy between 2017 and 2025. For each indication, the corresponding rates from the HAS and the G-BA were collected, together with ESMO-MCBS scores. The inter-agency concordance was evaluated using percentage agreement, Cohen’s kappa, and the Prevalence-Adjusted Bias-Adjusted Kappa (PABAK).
RESULTS: Concordance among the HTA bodies ranged from high-to-moderate (Cohen’s Kappa from 0.48 to 0.66). In contrast, concordance between ESMO-MCBS scores and HTA assessments was generally poor (PABAK from -0.01 to -0.10). The highest concordance was established between ESMO-MCBS scores and the SMR ratings from HAS (PABAK 0.63). Furthermore, in the analysis of quality of clinical evidence assessed by AIFA, about half of the evaluated outcomes underwent at least one downgrade in evidence certainty, highlighting uncertainty within the available clinical evidence.
CONCLUSIONS: The substantial discordance on the added benefit of novel oncology medicine between the final decision of European HTA bodies and the ESMO-MCBS suggests a misalignment in the interpretation of the evidence generated to support access to the market and patient care.