REAL-WORLD EVIDENCE (RWE) READINESS FOR EU JOINT CLINICAL ASSESSMENT: GAP ANALYSIS BETWEEN CURRENT RWE STUDIES AND EU HTA EXPECTATIONS
Author(s)
Neil R. Brett, PhD1, Garthiga Manickam, PhD2, Deborah Tallarigo, PhD3, Jack Ishak, PhD3, Susan A. Oliveria, MPH, ScD4, Roy Mootoosamy, PhD5.
1Research Scientist, Thermo Fisher Scientific, Saint Laurent, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, Montreal, QC, Canada, 4PPD™ Observational Studies, Thermo Fisher Scientific, New York, NY, USA, 5PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom.
1Research Scientist, Thermo Fisher Scientific, Saint Laurent, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, Montreal, QC, Canada, 4PPD™ Observational Studies, Thermo Fisher Scientific, New York, NY, USA, 5PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom.
OBJECTIVES: EU Joint Clinical Assessment (JCA; started January 2025) requires clinical evidence on relative effects of new technologies on health outcomes, with Population, Intervention, Comparator, and Outcome-driven scope setting emphasis on direct and indirect comparisons. We assessed alignment of recent RWE studies with a JCA-readiness framework reflecting EU health technology assessment (HTA) requirements and Member State Coordination Group on HTA (HTACG) guidance.
METHODS: A practical JCA-readiness framework was derived from key topics in EU HTA regulations (Regulation 2021/2282) and HTACG guidance. Domains assessed: Comparator design/relevance, patient-relevant outcomes, internal validity/robustness, outcome ascertainment validity, statistical precision/uncertainty reporting, and cross-country transferability. A targeted literature review (Embase/MEDLINE) identified comparative RWE oncology studies (2023-May 2026) evaluating treatment outcomes, reflecting evidence situations likely relevant to early JCA implementation. Studies were descriptively rated "high" alignment (strong adherence to domain-specific criteria), "moderate" alignment (partial fulfillment with some methodological limitations), and "low" alignment (substantial gaps or inconsistencies).
RESULTS: Of 225 screened studies, 52 were included/reviewed (pharmacologic treatments, with full articles accessible). The strongest domains were comparator design/relevance and outcomes ascertainment validity (42/52, 41/52 studies respectively with high alignment). Internal validity and statistical precision domains had moderate alignment for 34/52 and 31/52 respectively, because studies had inconsistent use of statistical adjustment/matching techniques. No studies collected patient-reported outcomes (PROs), but consistently collecting effectiveness, safety and treatment patterns led to moderate alignment for 50/52 studies. Cross-country transferability had low alignment for 26/52 studies due to many single-country studies outside of the EU, with small site numbers. Additional potential framework domains that could not be assessed without specific JCA scope, include data source fitness for JCA-relevant questions, alignment with JCA scope, and study transparency/pre-specification of design, outcomes and analyses.
CONCLUSIONS: Future comparative oncology RWE JCA-readiness may be enhanced by stronger comparative methods, PROs inclusion and improved cross-country applicability. This framework may support early planning/design of JCA-relevant studies.
METHODS: A practical JCA-readiness framework was derived from key topics in EU HTA regulations (Regulation 2021/2282) and HTACG guidance. Domains assessed: Comparator design/relevance, patient-relevant outcomes, internal validity/robustness, outcome ascertainment validity, statistical precision/uncertainty reporting, and cross-country transferability. A targeted literature review (Embase/MEDLINE) identified comparative RWE oncology studies (2023-May 2026) evaluating treatment outcomes, reflecting evidence situations likely relevant to early JCA implementation. Studies were descriptively rated "high" alignment (strong adherence to domain-specific criteria), "moderate" alignment (partial fulfillment with some methodological limitations), and "low" alignment (substantial gaps or inconsistencies).
RESULTS: Of 225 screened studies, 52 were included/reviewed (pharmacologic treatments, with full articles accessible). The strongest domains were comparator design/relevance and outcomes ascertainment validity (42/52, 41/52 studies respectively with high alignment). Internal validity and statistical precision domains had moderate alignment for 34/52 and 31/52 respectively, because studies had inconsistent use of statistical adjustment/matching techniques. No studies collected patient-reported outcomes (PROs), but consistently collecting effectiveness, safety and treatment patterns led to moderate alignment for 50/52 studies. Cross-country transferability had low alignment for 26/52 studies due to many single-country studies outside of the EU, with small site numbers. Additional potential framework domains that could not be assessed without specific JCA scope, include data source fitness for JCA-relevant questions, alignment with JCA scope, and study transparency/pre-specification of design, outcomes and analyses.
CONCLUSIONS: Future comparative oncology RWE JCA-readiness may be enhanced by stronger comparative methods, PROs inclusion and improved cross-country applicability. This framework may support early planning/design of JCA-relevant studies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA349
Topic
Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Oncology