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Defining value criteria for outcomes-based managed entry agreements in CAR-T therapy for relapsed/refractory multiple
María Victoria Mateos Manteca1, José Luis Poveda Andrés2, Albert Oriol Rocafiguera3
1Hematology Department, University Hospital of Salamanca, CIBERONC and Institute of Biomedical Research of Salamanca (IBSAL), Salamanca, Spain.
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.
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