From European Union Joint Clinical Assessments to Local Health Technology Assessments: An Environmental Scan of Methodological Guidance Across Key European Markets

Speaker(s)

Vinals L1, Muir J2, Radhakrishnan A3, Sarri G4
1Cytel, Montreal, QC, Canada, 2Cytel, Toronto, ON, Canada, 3Cytel, Mississauga, ON, Canada, 4Cytel, London, LON, UK

Presentation Documents

OBJECTIVES: Six months before the European union (EU) health technology assessment regulation (HTAR) is implemented, health technology developers (HTDs) need to familiarize with the methodological requirements for EU joint clinical assessments (JCA) and their interplay with national, local HTA submissions. We conducted an environmental scan of methodological guidance documents across the EU JCA (including EUnetHTA) and key European HTA agencies (Germany, France, Spain and The Netherlands) to assess their commonalities and differences.

METHODS: The websites of the European Network for HTA (EUnetHTA), the European Commission (EC), the Institute for Quality and Efficiency in Health Care (IQWiG), Haute Autorité de Santé (HAS), Agencia Española de Medicamentos y Productos Sanitarios (AEMPS) and the Zorginstituut Nederland (ZIN) were searched in March 2024 and June 2024 for methodological guidance documents. Results were synthesized thematically to identify commonalities and differences.

RESULTS: Thirty documents were identified (EUnetHTA and EC [n=12], IQWiG [n=7], HAS [n=5], ZIN [n=4], AEMPS [n=2]). Guides consistently emphasized randomized controlled trials as the highest quality evidence; non-randomized studies to be considered in exceptional cases although HAS, ZIN and AEMPS provided a wider range of real-world evidence acceptability criteria than the other HTA bodies. Alignment was found on the importance and accurate reporting of clinically relevant endpoints (including validity, reliability and interpretability issues), and the need for appropriate use of statistical methods considering data properties. EU HTAR and IQWiG showed close alignment for Bucher methods in comparative effectiveness research and the use of prediction intervals for bias assessment. All agencies recommended assessing the quality of RCTs with validated tools.

CONCLUSIONS: This environmental scan highlights commonalities regarding evidence sources and quality assessment and differences in evidentiary requirements regarding comparative effectiveness between EU JCA and key European countries. HTDs need to anticipate the impact of these differences to assess additional evidence generation strategies and value stories for local submissions.

Code

PT29

Topic

Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Coverage with Evidence Development & Adaptive Pathways, Decision & Deliberative Processes, Reimbursement & Access Policy, Value Frameworks & Dossier Format

Disease

No Additional Disease & Conditions/Specialized Treatment Areas