Simulating PICOs to Predict Evidence Requirement Implications of the EU Joint Clinical Assessment: Learnings From 24 Case Studies
Author(s)
Savvas Dimiou, MSc, PhD1, Edel Falla, MSc1, Sian Tanner, BA, PhD2, Lise AMOURA, MSc3, Oliver Curtis, MSc1, Pascaline Faivre, PharmD4, Nabila Faour Slika, MSc1, Natalie Green, PhD1, Ansa Ibok, MSc1, Fani Kotseva, MSc5, Robert Friedrich Krüger, MSc6, João Leite, MSc7, Ana Lisica, PhD1, Benjamin Mynors-Wallis, MSc8, Daniel Matthew Newsome, BSc1, Corinna Oswald, MSc1, Alexandre Quequet--Leyssieux, MSc3, Xenia Sitavu-Radu, PhD1, Benedetta Spadaro, MPhil9, Anastasia Stamoulou, MSc10, Paula Skowron, MSc11, Anke van Engen, MSc2, Jessica Walters, MSc1.
1IQVIA, London, United Kingdom, 2IQVIA, Amsterdam, Netherlands, 3IQVIA, Paris, France, 4IQVIA, Bron, France, 5IQVIA, Sofia, Bulgaria, 6IQVIA, Bad Homburg, Germany, 7IQVIA, Oeiras, Portugal, 8IQVIA, Munich, Germany, 9IQVIA, Milan, Italy, 10IQVIA, Athens, Greece, 11IQVIA, Kraków, Poland.
1IQVIA, London, United Kingdom, 2IQVIA, Amsterdam, Netherlands, 3IQVIA, Paris, France, 4IQVIA, Bron, France, 5IQVIA, Sofia, Bulgaria, 6IQVIA, Bad Homburg, Germany, 7IQVIA, Oeiras, Portugal, 8IQVIA, Munich, Germany, 9IQVIA, Milan, Italy, 10IQVIA, Athens, Greece, 11IQVIA, Kraków, Poland.
OBJECTIVES: As of 12th January 2025, all new oncology medicines and ATMPs are subject to Joint Clinical Assessment (JCA). As the final scope will only be shared 60-100 days ahead of the JCA dossier deadline, Health Technology Developers (HTDs) need to anticipate the JCA scope.
METHODS: The JCA scoping process was simulated for 24 case studies between January 2022-April 2025 (in 14 distinct indications). European treatment guidelines and the competitive landscape were used to anticipate the Assessor PICO (Population, Intervention, Comparator, Outcomes) proposal. Local treatment guidelines, analogues and local stakeholder input was used to anticipate Member State (MS) responses for a selection of EU MS. The latest Methodological and Practical Guidance (MPG) on the JCA scoping process was followed to consolidate PICOs and predict the JCA scope.
RESULTS: PICOs were simulated for 21 oncology products (with 2 products investigated in 2 different indications) and 1 genetic disorder; 2 of these were ATMPs and 2 also had orphan drug status. The median number of PICOs was 9 (range 5 to 21) with 67% of PICOs requiring ITCs, while worst-case scenarios showed that up to 88 PICOs could be expected. The minimum number of anticipated analyses ranged between 175-720, pending ITC feasibility.
CONCLUSIONS: Simulating the JCA scoping process often results in a significant number of PICOs, especially in therapeutic areas where treatment patterns vary significantly across MS. Our approach yielded comparable number of PICOs to the exercises of the JCA subgroup for medicinal products (range 7-13), demonstrating the robustness of our analysis. Many of these PICOs require ITCs or objection handling if not feasible, thus imposing a significant burden on HTDs to deliver comprehensive evidence within tight timelines. HTDs should anticipate JCA PICO requirements early in the development process and iteratively refine the PICO prediction to account for changes in treatment landscape and broader asset strategy.
METHODS: The JCA scoping process was simulated for 24 case studies between January 2022-April 2025 (in 14 distinct indications). European treatment guidelines and the competitive landscape were used to anticipate the Assessor PICO (Population, Intervention, Comparator, Outcomes) proposal. Local treatment guidelines, analogues and local stakeholder input was used to anticipate Member State (MS) responses for a selection of EU MS. The latest Methodological and Practical Guidance (MPG) on the JCA scoping process was followed to consolidate PICOs and predict the JCA scope.
RESULTS: PICOs were simulated for 21 oncology products (with 2 products investigated in 2 different indications) and 1 genetic disorder; 2 of these were ATMPs and 2 also had orphan drug status. The median number of PICOs was 9 (range 5 to 21) with 67% of PICOs requiring ITCs, while worst-case scenarios showed that up to 88 PICOs could be expected. The minimum number of anticipated analyses ranged between 175-720, pending ITC feasibility.
CONCLUSIONS: Simulating the JCA scoping process often results in a significant number of PICOs, especially in therapeutic areas where treatment patterns vary significantly across MS. Our approach yielded comparable number of PICOs to the exercises of the JCA subgroup for medicinal products (range 7-13), demonstrating the robustness of our analysis. Many of these PICOs require ITCs or objection handling if not feasible, thus imposing a significant burden on HTDs to deliver comprehensive evidence within tight timelines. HTDs should anticipate JCA PICO requirements early in the development process and iteratively refine the PICO prediction to account for changes in treatment landscape and broader asset strategy.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA300
Topic
Clinical Outcomes, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Oncology, Rare & Orphan Diseases