METHODOLOGICAL AND STATISTICAL LEARNINGS FROM INITIAL JOINT CLINICAL ASSESSMENT REPORTS UNDER THE EU HTA REGULATION: CROSS-COMPANY PERSPECTIVES
Author(s)
Rossella Belleli, MA, MPH1, Sarah Boehme, PhD2, Orlando Dohring, BSc, MPhil, MSc3, Sandro Gsteiger, PhD4, Min-Hua Jen, PhD5, Eva Kleine, MSc6, Annett Kucka, PhD7, Rachid MASSAAD, PhD8, Laura Walton, PhD9.
1Hoffman La Roche, Basel, Switzerland, 2Pfizer Pharma GmbH, Berlin, Germany, 3Novartis, London, United Kingdom, 4Roche, Basel, Switzerland, 5Eli Lilly, Uxbridge, United Kingdom, 6Boehringer Ingelheim Pharma GmbH & Co KG, Ingelheim am Rhein, Germany, 7SERVIER, München, Germany, 8MSD Europe Belgium BV, Brussels, Belgium, 9MSD (UK) Ltd, London, United Kingdom.
1Hoffman La Roche, Basel, Switzerland, 2Pfizer Pharma GmbH, Berlin, Germany, 3Novartis, London, United Kingdom, 4Roche, Basel, Switzerland, 5Eli Lilly, Uxbridge, United Kingdom, 6Boehringer Ingelheim Pharma GmbH & Co KG, Ingelheim am Rhein, Germany, 7SERVIER, München, Germany, 8MSD Europe Belgium BV, Brussels, Belgium, 9MSD (UK) Ltd, London, United Kingdom.
OBJECTIVES: The EU Health Technology Assessment Regulation introduced the Joint Clinical Assessment (JCA) as a new centralized clinical evaluation process. To date, practical experience regarding how methodological guidance is interpreted and applied remains limited. This poster summarizes early learnings from the first published JCA reports, based on insights from the JCA Exchange subgroup, a cross‑company working group of industry statisticians and evidence synthesis experts.
METHODS: The JCA Exchange subgroup meets regularly to discuss challenges associated with JCA implementation and to monitor evolving guidance, templates, and procedures. This poster synthesizes qualitative insights from subgroup discussions and a structured review of publicly available JCA reports, which are currently restricted to advanced therapy medicinal products (ATMPs) and oncology therapies.
RESULTS: The poster focuses on early observations from the first JCA reports related to the interpretation of pre‑specification requirements and documentation standards; the handling of multiplicity and subgroup analyses; expectations for evidence synthesis and indirect treatment comparisons; statistical reporting as outlined by guidance, including treatment effect measures, data cut-offs handling, and presentation of efficacy results; and approaches to assessing and summarizing the certainty of evidence. Early observations are anticipated to highlight both areas of alignment with existing guidance and areas where interpretation remains uncertain.
CONCLUSIONS: Available JCA reports provide an important opportunity to understand how the EU HTA framework is applied in practice. Cross‑company reflection may support more consistent interpretation and facilitate informed methodological dialogue as the JCA process evolves. Ultimately, this can help to inform the evaluation of the JCA process in 2028 foreseen under the EU HTA regulation.
Acknowledgements
The authors acknowledge the valuable contributions of the remaining members of the JCA Exchange subgroup, a cross-company working group of industry statisticians and evidence synthesis experts, whose discussions and insights informed this work.
METHODS: The JCA Exchange subgroup meets regularly to discuss challenges associated with JCA implementation and to monitor evolving guidance, templates, and procedures. This poster synthesizes qualitative insights from subgroup discussions and a structured review of publicly available JCA reports, which are currently restricted to advanced therapy medicinal products (ATMPs) and oncology therapies.
RESULTS: The poster focuses on early observations from the first JCA reports related to the interpretation of pre‑specification requirements and documentation standards; the handling of multiplicity and subgroup analyses; expectations for evidence synthesis and indirect treatment comparisons; statistical reporting as outlined by guidance, including treatment effect measures, data cut-offs handling, and presentation of efficacy results; and approaches to assessing and summarizing the certainty of evidence. Early observations are anticipated to highlight both areas of alignment with existing guidance and areas where interpretation remains uncertain.
CONCLUSIONS: Available JCA reports provide an important opportunity to understand how the EU HTA framework is applied in practice. Cross‑company reflection may support more consistent interpretation and facilitate informed methodological dialogue as the JCA process evolves. Ultimately, this can help to inform the evaluation of the JCA process in 2028 foreseen under the EU HTA regulation.
Acknowledgements
The authors acknowledge the valuable contributions of the remaining members of the JCA Exchange subgroup, a cross-company working group of industry statisticians and evidence synthesis experts, whose discussions and insights informed this work.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA75
Topic
Clinical Outcomes, Health Policy & Regulatory, Health Technology Assessment
Disease
No Additional Disease & Conditions/Specialized Treatment Areas