OBJECTIVES : Providing an initial indication of the differences in health technology assessment (HTA) evidence requirements for the assessment of oncology drugs between the European Medicines Agency (EMA) and HTA bodies and among HTA bodies themselves.

METHODS : A comparative analysis was conducted based on interviews with experts or representatives from EMA and six HTA jurisdictions (England, Italy, the Netherlands, Poland, Portugal, and Sweden). The interviews covered 5 evidence domains (identified by Shaping European Early Dialogues; population, comparator, clinical endpoints, trial design and data sources, and statistical analysis) divided into 19 subdomains to identify differences in evidence requirements. Recommendations for more efficiently dealing with potential differences were obtained. The data were analyzed for evidence acceptance (to what extent are different types of evidence accepted by the agency), alignment (to what extent are HTA bodies aligned with EMA and each other on evidence requirements), and predictability (how predictable is the acceptance of specific types of evidence).

RESULTS : HTA bodies and EMA were ‘aligned’ on 39% subdomains and ‘not aligned’ on 13%. Overall, HTA bodies were aligned on 16% of the subdomains and aligned with none of the other HTA bodies on 28% of the subdomains. The level of acceptance varied between 37% for Portugal and 79% for Poland. Predictability of accepting types of evidence was highest for England with 58% and lowest for Italy and Portugal with 32% in both countries. Recommendations for better aligning or dealing with the differences included joint early dialogues, intensified collaboration and exchange between countries, joint relative effectiveness assessment, and the use of adaptive pathways.

CONCLUSIONS : Differences were identified between both assessments by EMA and HTA bodies and among HTA bodies in important evidence requirements’ subdomains. Increased alignment between EMA and HTA bodies and among HTA bodies is needed and could be enhanced by following the recommendations provided.