Qualitative research on the acceptance of evolving evidence for HTA body approval of innovative health technologies in selected European countries.

Objective: With increasing efforts of streamlining market access of innovative drugs, the current acceptance of health technology assessment (HTA) bodies on non-traditional trial designs is still unclear. The aim of this study was to assess the perspective of drug developers on the acceptance of non-traditional trial designs by HTA bodies in Europe.

Methods: Eleven semi-structured interviews were conducted with experts to gain insights on their perception of non-traditional trial design acceptance by HTA bodies from the Netherlands, Germany, United Kingdom, Sweden, and France. Future perspectives on and recommendations for facilitating the acceptance of non-traditional trials were provided.

Results: All experts highlighted the HTA bodies' preference for randomized controlled trials over non-traditional trial designs. The degree of acceptance varied between countries. Experts recommended multistakeholder discussions between drug developers and HTA bodies for early scientific advice and reevaluation of evidence requirements. The EU-HTA regulation was viewed as both a potential opportunity for harmonization and limiting innovative approach.

Conclusion: The results of this study highlighted significant variations in the acceptance of non-traditional trial designs by HTA bodies across Europe. Actions are needed to facilitate a more progressive view on non-traditional clinical trial designs to ensure fast patient access to highly innovative treatments.