Challenges in Predicting the Joint Clinical Assessment (JCA) Scope From a Stakeholder Perspective: A Case Study in Relapsed/Refractory Multiple Myeloma
Author(s)
Kaisa Miikkulainen, MSc1, Maria victoria Mateos, PhD2, Caroline Delaitre-Bonnin, PharmD3, Almudena Olid Gonzalez, MA4, Milon Waththuhewa, PharmD5, Aleksandra Zapala-Szufel, MPharm6, Benedikte Lensberg, MSc7, Brett Doble, PhD8, Damla Kilic, MD8.
1Gilead Sciences Sweden, Solna, Sweden, 2University Hospital of Salamanca, Salamanca, Spain, 3Thermo Fisher Scientific, Ivry-sur-Seine, France, 4Thermo Fischer Scientific, Waltham, MA, USA, 5Thermo Fisher Scientific, Bethesda, MD, USA, 6Thermo Fisher Scientific, Alicante, Spain, 7Avilon AS, Bergen, Norway, 8Kite, a Gilead Company, Santa Monica, CA, USA.
1Gilead Sciences Sweden, Solna, Sweden, 2University Hospital of Salamanca, Salamanca, Spain, 3Thermo Fisher Scientific, Ivry-sur-Seine, France, 4Thermo Fischer Scientific, Waltham, MA, USA, 5Thermo Fisher Scientific, Bethesda, MD, USA, 6Thermo Fisher Scientific, Alicante, Spain, 7Avilon AS, Bergen, Norway, 8Kite, a Gilead Company, Santa Monica, CA, USA.
OBJECTIVES: The Joint Clinical Assessment (JCA) guidance defines that the assessment scope should reflect policy questions from the European Union (EU) Member States and is not evidence-driven. The Population, Intervention, Comparator and Outcome (PICO) framework is used to define the assessment scope. We performed a PICO prediction for a new therapy in relapsed/refractory multiple myeloma (r/r MM), with the aim of exploring the feasibility of using a policy-based approach.
METHODS: A policy-based PICO prediction for 11 EU Member States was conducted by reviewing clinical guidelines and recent Health Technology Assessment appraisals. The identified PICOs were validated via e-survey and semi-structured interviews (n=19) with clinical and HTA experts. Qualitative responses were analysed to identify common themes and potential challenges.
RESULTS: Over 60 PICOs were initially predicted using the policy approach. The following recurring themes were identified: challenges related to JCA’s policy approach to predict a relevant scope and the need to significantly modify PICOs to meet local Member State needs. Specifically, HTA experts were hesitant to define PICOs without clinical trial evidence. It was expressed that local HTA bodies will likely rely on evidence-driven scoping approaches. Finally, a key challenge was the resulting high number of PICOs and misalignment on populations. This is partly driven by the policy-approach of the prediction, and by the breadth of available therapies in r/r MM across EU.
CONCLUSIONS: In a disease area such as r/r MM, the policy-based approach presents challenges to HTA bodies, clinical experts and Health Technology Developers (HTDs) to predict and assess the JCA scope. An unintentional consequence of the policy-based approach may be that HTDs cannot adequately respond to the requested scope which may result in further requests at Member State level and duplication of efforts.
METHODS: A policy-based PICO prediction for 11 EU Member States was conducted by reviewing clinical guidelines and recent Health Technology Assessment appraisals. The identified PICOs were validated via e-survey and semi-structured interviews (n=19) with clinical and HTA experts. Qualitative responses were analysed to identify common themes and potential challenges.
RESULTS: Over 60 PICOs were initially predicted using the policy approach. The following recurring themes were identified: challenges related to JCA’s policy approach to predict a relevant scope and the need to significantly modify PICOs to meet local Member State needs. Specifically, HTA experts were hesitant to define PICOs without clinical trial evidence. It was expressed that local HTA bodies will likely rely on evidence-driven scoping approaches. Finally, a key challenge was the resulting high number of PICOs and misalignment on populations. This is partly driven by the policy-approach of the prediction, and by the breadth of available therapies in r/r MM across EU.
CONCLUSIONS: In a disease area such as r/r MM, the policy-based approach presents challenges to HTA bodies, clinical experts and Health Technology Developers (HTDs) to predict and assess the JCA scope. An unintentional consequence of the policy-based approach may be that HTDs cannot adequately respond to the requested scope which may result in further requests at Member State level and duplication of efforts.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA72
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
Oncology