Differences in evidentiary requirements for oncology drug assessments across health technology assessment bodies in Germany, Spain, and France, the European Medicines Agency, and the EU Health Technology assessment regulation.

Abstract

Background: This study compares evidentiary requirements for oncology drugs across the European Medicines Agency (EMA), the EU Health Technology Assessment Regulation (HTAR), and national Health Technology Assessment (HTA) practices. Given the heterogeneity in evidentiary requirements across EU Member States, identifying these differences is critical for navigating the EU HTAR.

Research design and methods: EMA guidelines and EU HTA guidance documents were reviewed, followed by interviews with HTA experts from Germany, Spain, and France to identify differences in oncology drug assessments and anticipated EU HTAR impact. Six recent oncology drug assessments conducted by these three HTA bodies were reviewed to substantiate the interview findings. A comparative analysis of evidence acceptability and potential alignment between organizations was conducted.

Results: The EMA and EU HTAR guidance showed higher acceptability of available evidence than national HTA bodies. The German HTA body demonstrated the highest level of acceptability, followed by Spain and France. The six assessments supported expert insights by demonstrating national variability.

Conclusion: Differences in the acceptability of evidence across the EMA, EU HTAR, and HTA bodies were identified, highlighting the need for increased alignment and shared understanding of evidentiary requirements. Recommendations to reduce or manage variability include international collaborations, harmonization, and early dialogues.