Defining value criteria for outcomes-based managed entry agreements in CAR-T therapy for relapsed/refractory multiple myeloma: a multi-criteria decision analysis study.
María Victoria Mateos Manteca, José Luis Poveda Andrés, Albert Oriol Rocafiguera, Vicente Escudero-Vilaplana, José Luis Trillo Mata, Pedro Gómez Pajuelo, Alberto Arribas Ruiz, Teresa Domínguez Lubillo, Neus Vidal-Vilar, Asís Ariznavarreta Martín
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Abstract
Background: This study aimed to identify and prioritize criteria for designing and monitoring outcomes-based managed entry agreements (MEAs) for chimeric antigen receptor T-cell (CAR-T) therapy in relapsed/refractory multiple myeloma (RRMM), from the perspective of healthcare professionals and decision-makers.
Research design and methods: A structured MCDA-informed expert weighting exercise was conducted using an adapted EVIDEM framework. The study comprised two phases: a) selection and structuring of criteria through a targeted literature review and scientific committee (SC) consensus; and b) nonhierarchical weighting of criteria and sub-criteria by a multidisciplinary expert group (n = 19) of hospital pharmacists, physicians, and evaluators/decision-makers.
Results: Twelve criteria were identified. Following SC discussion, five criteria were selected for weighting: efficacy, safety, patient-reported outcomes, treatment cost, and other direct medical costs. Among the weighted sub-criteria, overall survival (OS) and progression-free survival (PFS) achieved the highest scores, with mean values of 4.21 (95% CI: 3.68-4.74) and 4.05 (95% CI: 3.52-4.58), respectively.
Conclusions: In this structured MCDA-informed expert weighting exercise, OS and PFS were identified as the highest-priority value criteria for designing and monitoring outcomes-based MEAs for CAR-T therapies in RRMM. These findings may support feasible, evidence-based reimbursement models but require validation in larger and more diverse stakeholder groups.
期刊介绍：
Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review.
The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections:
Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results
Article Highlights – an executive summary of the author’s most critical points.
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