OBJECTIVES

: With more advanced health technologies (HTs) entering the healthcare market, also procedures for health technology assessment (HTA) require changes. Therefore, the aim of this study was to assess which HTs are perceived as complex and which assessment issues are considered challenging by European HTA organizations, to gain understanding of the needs for new HTA methods and requirements for advanced HTs.

METHODS

: To identify complex HTs and challenges in HTA, a questionnaire was send out to European HTA organizations. The questionnaire used relevant case studies and was validated and tested for reliability by an expert panel and it was pilot tested before dissemination.

RESULTS

: Twenty-two organizations completed the questionnaire. ATMPs were considered as the most challenging types of therapy for HTA. Out of seven prespecified case studies, the histology independent therapy was reported as most challenging. Additionally reported complex HTs by the organizations were mostly pharmaceuticals, oncology products, orphan medicines, personalized treatments and combinations of treatments.

For the case studies, most challenging issues were reported to occur in the relative effectiveness assessment (REA) and cost-effectiveness assessment (CEA). In the open questions, however, organizations reported data challenges most often, including absent, insufficient, immature or low-quality data. These initial data issues are subsequently expressed during the REA and CEA and ultimately complicate decision-making, according to 13 organizations. Policy and organizational challenges, as well as societal or political pressure, were reported by respectively 8 and 4 organizations. Direct modelling issues were least often reported.

CONCLUSIONS

: Challenges in HTA of complex HTs mainly root in data insufficiencies and result in outcome uncertainties in the REA and parameter uncertainty in the CEA, ultimately complicating decision making. Complex HTs, e.g. gene therapies, sometimes inherently cause data insufficiencies, making some complex HTs more challenging for HTA. These results highlight the importance of the methodological work done in the HTx project.