OBJECTIVES: The aim of this study is to establish, agree and weigh the most appropriate criteria for the design and monitoring of a outcomes-based managed entry agreement (MEA) for a CAR-T therapy in patients with multiple myeloma who have progressed after a proteosome inhibitor (PI), immunomodulator (IMiD) and anti-CD-38 antibodies, from the point of view of healthcare professionals and decision-makers.

METHODS: Multi-criteria Decision Analysis (MCDA) technique was used based on the EVIDEM framework. All the study was assisted by a multidisciplinary scientific committee (SC). The study was divided in two phases: a) selection and structuring criteria through a literature review and consensus with the SC and b) weighting of criteria and sub-criteria by a multidisciplinary group (n=21), composed of different profiles (9 hospital pharmacists, 8 physicians and 4 evaluators/decision-makers). The participants of the multidisciplinary group were selected based on their experience in RRMM or in outcomes-based MEAs or CAR-T therapies and/or MCDA.

RESULTS: As a result of the literature review, 12 criteria were identified, of which 5 were selected for the weighting of these (efficacy, safety, Patient Reported Outcomes [PRO], cost of treatment and other direct medical costs of treatment) and their corresponding sub-criteria. The highest score for the design and monitoring of an outcomes-based MEA for CAR-Ts in RRMM was achieved by two efficacy sub-criteria: overall survival (OS) (4.21 ± 0.53) and progression-free survival (PFS) (4.05 ± 0.53).

CONCLUSIONS: Based on reflective MCDA methodology and SC’s experience, OS and PFS are the most relevant value criteria for the design and monitoring of an outcomes-based MEA for CAR-T therapies in MMRR. These findings provide valuable insights for developing effective and evidence-based MEAs in the context of CAR-T therapy for RRMM patients.