WHAT DO THE FIRST EU JOINT CLINICAL ASSESSMENT COHORTS REVEAL ABOUT FUTURE EVIDENCE EXPECTATIONS? EARLY INSIGHTS FOR MANUFACTURERS AND HTA STAKEHOLDERS
Author(s)
Alexandra Guyenet, BA, MA1, Neha Pethad, MSc, Pharm D2, Kate Burslem, BSc, MSc2.
1Manager, Value Evidence, AESARA Europe GmbH, Zug, Switzerland, 2AESARA Inc., London, United Kingdom.
1Manager, Value Evidence, AESARA Europe GmbH, Zug, Switzerland, 2AESARA Inc., London, United Kingdom.
OBJECTIVES: The first products entering the European Union (EU) Joint Clinical Assessment (JCA) offer early insight into the evidence packages manufacturers are bringing into the new EU health technology assessment (HTA) system. This study characterized the evidence profiles of the initial JCA cohorts and explored how these may translate into methodological challenges through review of the first published JCA.
METHODS: Publicly available information was extracted for the first 15 active JCA products, including therapeutic area, pivotal study design, comparator strategy, endpoint selection, orphan status, advanced therapy medicinal product (ATMP) designation, and biomarker-defined populations. Products meeting ≥3 predefined criteria (single-arm study, surrogate endpoint, orphan designation, ATMP, biomarker-defined population) were descriptively classified as complex evidence profiles. The published JCA report for tovorafenib was reviewed to illustrate how these characteristics were reflected during assessment
RESULTS: Among the 15 products, 80.0% (12/15) were oncology therapies, 46.7% (7/15) were orphan medicines, 26.7% (4/15) were ATMPs, and 33.3% (5/15) targeted biomarker- or genetically defined populations. 53.3% (8/15)were supported by single-arm studies, while 46.7% (7/15) relied on randomized controlled trials. For 91.6% (11/12) of oncology products, surrogate or intermediate endpoints were the primary efficacy outcomes, whereas only 25.0% (3/12) used overall survival. Nearly half of all products (46.7%; 7/15) were classified as complex evidence profiles. The first published JCA demonstrated that these profiles translated into challenges related to comparative evidence, indirect treatment comparisons, methodological transparency, and residual uncertainty.
CONCLUSIONS: Early JCA implementation is evaluating a heterogeneous mix of conventional and complex evidence profiles. The first published assessment suggests that products supported by more complex evidence require greater attention to comparator strategy, evidence synthesis, and methodological transparency. These findings indicate that successful JCA readiness depends not only on generating clinical evidence, but on designing evidence strategies that anticipate PICO requirements and enable robust comparative assessment.
METHODS: Publicly available information was extracted for the first 15 active JCA products, including therapeutic area, pivotal study design, comparator strategy, endpoint selection, orphan status, advanced therapy medicinal product (ATMP) designation, and biomarker-defined populations. Products meeting ≥3 predefined criteria (single-arm study, surrogate endpoint, orphan designation, ATMP, biomarker-defined population) were descriptively classified as complex evidence profiles. The published JCA report for tovorafenib was reviewed to illustrate how these characteristics were reflected during assessment
RESULTS: Among the 15 products, 80.0% (12/15) were oncology therapies, 46.7% (7/15) were orphan medicines, 26.7% (4/15) were ATMPs, and 33.3% (5/15) targeted biomarker- or genetically defined populations. 53.3% (8/15)were supported by single-arm studies, while 46.7% (7/15) relied on randomized controlled trials. For 91.6% (11/12) of oncology products, surrogate or intermediate endpoints were the primary efficacy outcomes, whereas only 25.0% (3/12) used overall survival. Nearly half of all products (46.7%; 7/15) were classified as complex evidence profiles. The first published JCA demonstrated that these profiles translated into challenges related to comparative evidence, indirect treatment comparisons, methodological transparency, and residual uncertainty.
CONCLUSIONS: Early JCA implementation is evaluating a heterogeneous mix of conventional and complex evidence profiles. The first published assessment suggests that products supported by more complex evidence require greater attention to comparator strategy, evidence synthesis, and methodological transparency. These findings indicate that successful JCA readiness depends not only on generating clinical evidence, but on designing evidence strategies that anticipate PICO requirements and enable robust comparative assessment.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA399
Topic
Health Policy & Regulatory, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas