Guidance for Real-World Evidence Generation for the Joint Clinical Assessment Process and Its Implications for Country-Specific HTA Body Acceptability
Author(s)
Georgios Papadakis, PhD1, Ansa Ibok, MSc2, Spyros Kolovos, PhD1, Jennifer Gaultney, PhD, MPH2, Edel Falla, MSc2.
1IQVIA, Athens, Greece, 2IQVIA, London, United Kingdom.
1IQVIA, Athens, Greece, 2IQVIA, London, United Kingdom.
OBJECTIVES: Real-world evidence (RWE) is increasingly used to support decision-making in health technology assessments (HTA). With the implementation of the Joint Clinical Assessment (JCA) under the EU HTA Regulation in January 2025, health technology developers (HTDs) must consider JCA and country-specific HTA body requirements for designing transferable RWE studies. This study assessed the feasibility of conducting RWE studies acceptable for JCA and select European HTA bodies.
METHODS: We reviewed guidance from the EU HTA Coordination Group (2024), the French Haute Autorité de Santé (HAS, 2021), the German Institüt für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG, 2025), and the UK’s National Institute for Health and Care Excellence (NICE, 2025) to compare RWE study requirements.
RESULTS: The JCA guidance includes limited references to RWE, primarily within the guidance for indirect treatment comparisons (ITCs). The latter aims at supporting assessor review of anchored and unanchored ITCs using aggregate and patient-level data, with brief mention of unanchored ITCs using non-randomized evidence to form external controls. However, without an RWE framework, the utility and acceptance of RWE studies for JCA remains unclear. In contrast, NICE, HAS, and IQWiG provide varying levels of detail for HTDs on RWE study design and analysis requirements. HAS and NICE extend their guidance beyond ITCs to include long-term efficacy and safety estimates and treatment landscape characterization, with NICE most clearly outlining expectations for RWE studies and methods to minimize bias. These findings were further considered alongside perspectives from broader multi-stakeholder initiatives.
CONCLUSIONS: Given the complexity of the JCA scoping process, RWE studies may be necessary to characterize treatment patterns and comparative effectiveness, especially for HTDs with single-arm trial evidence for orphan technologies. However, without a clear JCA RWE framework, harmonizing RWE study transferability and expectations across European HTA bodies remains challenging, as HTDs must rely on country-specific frameworks, including those of non-EU member states.
METHODS: We reviewed guidance from the EU HTA Coordination Group (2024), the French Haute Autorité de Santé (HAS, 2021), the German Institüt für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG, 2025), and the UK’s National Institute for Health and Care Excellence (NICE, 2025) to compare RWE study requirements.
RESULTS: The JCA guidance includes limited references to RWE, primarily within the guidance for indirect treatment comparisons (ITCs). The latter aims at supporting assessor review of anchored and unanchored ITCs using aggregate and patient-level data, with brief mention of unanchored ITCs using non-randomized evidence to form external controls. However, without an RWE framework, the utility and acceptance of RWE studies for JCA remains unclear. In contrast, NICE, HAS, and IQWiG provide varying levels of detail for HTDs on RWE study design and analysis requirements. HAS and NICE extend their guidance beyond ITCs to include long-term efficacy and safety estimates and treatment landscape characterization, with NICE most clearly outlining expectations for RWE studies and methods to minimize bias. These findings were further considered alongside perspectives from broader multi-stakeholder initiatives.
CONCLUSIONS: Given the complexity of the JCA scoping process, RWE studies may be necessary to characterize treatment patterns and comparative effectiveness, especially for HTDs with single-arm trial evidence for orphan technologies. However, without a clear JCA RWE framework, harmonizing RWE study transferability and expectations across European HTA bodies remains challenging, as HTDs must rely on country-specific frameworks, including those of non-EU member states.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA166
Topic
Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas