STRUCTURED ONCOLOGY LITERATURE SCREENING AS THE METHODOLOGICAL FOUNDATION FOR EU JOINT CLINICAL ASSESSMENT PREPARATION AND TARGET TRIAL EMULATION PLANNING
Author(s)
Ciara Coakley, MSc1, Xabier Garcia de Albeniz, PhD1, Mihaela Musat, PhD2, Anna Forsythe, MBA, MSc, PharmD2, Radek Wasiak, PhD1.
1Adigens Health Limited, Dublin, Ireland, 2Oncoscope, New York, NY, USA.
1Adigens Health Limited, Dublin, Ireland, 2Oncoscope, New York, NY, USA.
OBJECTIVES: The EU Joint Clinical Assessment (JCA) regulation mandates comparative clinical evidence at market authorisation, with comparator scope frequently outpacing available randomised data in oncology. Target trial framework (TTF) provides a guide for comparative effectiveness estimation from observational data, but valid design requires a priori knowledge of treatment patterns, outcome definitions, eligibility criteria, and covariate availability. This study applies Oncoscope, a curated multi-indication oncology platform to demonstrate this approach in chronic lymphocytic leukaemia (CLL).
METHODS: The CLL Oncoscope library was interrogated using a pre-specified extraction aligned to the seven target trial components (Hernán & Robins, 2016): eligibility criteria, treatment strategies, assignment procedures, follow-up rules, outcome definitions, causal contrast, and analytic approach. Extracted elements were mapped to JCA benefit domains (mortality, morbidity, HRQoL, and adverse events) to assess comparator feasibility and evidence gaps. Outputs were catalogued into a TTF design matrix and JCA readiness scorecard
RESULTS: The CLL library comprised 260 RWE studies (2020 onwards). OS was reported in 52% (n=136) and PFS in 34% (n=89); TTNT appeared in 15% (n=39). Safety reporting was uneven: AEs leading to discontinuation were captured in 20% (n=52), while overall AE reporting reached only 10% (n=25). Head-to-head comparisons were present in 23% (n=61). Subgroup data were sparse: age-based analyses in 10% (n=27), del(17p) in 5% (n=14), and frailty/performance status in 3% (n=9). Patient characteristics, treatment sequences, HCRU, and community versus academic setting data were not systematically captured, representing defined gaps for TTF covariate specification and JCA contextualisation.
CONCLUSIONS: Purpose-built oncology evidence platforms enable structured, replicable extraction of the data elements governing TTF design validity and JCA comparator feasibility. The CLL findings illustrate both the utility of this approach and the evidence gaps (particularly in treatment patterns and patient-level characteristics) that must be addressed before comparative effectiveness study protocols can be pre-specified with confidence. <!--EndFragment-->
METHODS: The CLL Oncoscope library was interrogated using a pre-specified extraction aligned to the seven target trial components (Hernán & Robins, 2016): eligibility criteria, treatment strategies, assignment procedures, follow-up rules, outcome definitions, causal contrast, and analytic approach. Extracted elements were mapped to JCA benefit domains (mortality, morbidity, HRQoL, and adverse events) to assess comparator feasibility and evidence gaps. Outputs were catalogued into a TTF design matrix and JCA readiness scorecard
RESULTS: The CLL library comprised 260 RWE studies (2020 onwards). OS was reported in 52% (n=136) and PFS in 34% (n=89); TTNT appeared in 15% (n=39). Safety reporting was uneven: AEs leading to discontinuation were captured in 20% (n=52), while overall AE reporting reached only 10% (n=25). Head-to-head comparisons were present in 23% (n=61). Subgroup data were sparse: age-based analyses in 10% (n=27), del(17p) in 5% (n=14), and frailty/performance status in 3% (n=9). Patient characteristics, treatment sequences, HCRU, and community versus academic setting data were not systematically captured, representing defined gaps for TTF covariate specification and JCA contextualisation.
CONCLUSIONS: Purpose-built oncology evidence platforms enable structured, replicable extraction of the data elements governing TTF design validity and JCA comparator feasibility. The CLL findings illustrate both the utility of this approach and the evidence gaps (particularly in treatment patterns and patient-level characteristics) that must be addressed before comparative effectiveness study protocols can be pre-specified with confidence. <!--EndFragment-->
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
MSR21
Topic
Health Technology Assessment, Methodological & Statistical Research, Real World Data & Information Systems
Topic Subcategory
Confounding, Selection Bias Correction, Causal Inference
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology