Multicriteria Decision Analysis (MCDA) to Elicit Criteria for CAR-T Cell Health Technology Assessment (HTA) in Brazilian Private Healthcare System

OBJECTIVES: To identify and analyze priority criteria for HTA of CAR-T cell therapy for relapsed or refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy using a MCDA approach.

METHODS: A literature review identified a set of relevant criteria validated in a 3-round web-Delphi composed of three groups of stakeholders (n=3 each): medical specialists, representatives of patient associations, and private healthcare managers. Decision conferences were then conducted, one per group, to weight the final set of criteria using the MACBETH’s qualitative swing weighting protocol.

RESULTS: The final set included 13 criteria related to efficacy, safety, economics, quality of life and evidence level. During the decision conference, the managers selected only seven criteria for weighting. Budget impact was deemed most important (40.59%) followed by overall survival (OS) (19.80%), objective response rate (ORR) (11.88%), adverse event (AE) grade ³ 3 (9.9%), cytokine release syndrome (CRS) (7.92%), neurotoxicity (6.93%) and evidence level (2.97%). The same criteria set was defined by patients, but the total weight assigned showed CRS as the main criterion (30.34%), while budget impact was weighted least (4.49%). ORR and OS had 15.73% each, followed by AE³3 (13.48%), neurotoxicity (12.36%) and evidence level (7.87%). Medical specialists selected CR as the main criterion (33.33%) followed by budget impact (24.73%), OS (17.20%), ORR (15.05%), neurotoxicity (6.45%) and CRS (3.23%). They excluded evidence level and AE>3 as less important because they judged CRS and neurotoxicity to be sufficient for measuring safety.

CONCLUSIONS: The above differences in relative weights reflect the different priorities and perceptions of CAR-T cell characteristics. Economic impact was identified as an important criterion for managers and medical experts, while patients showed concerns related to safety. The results indicate that all groups recognized the importance of efficacy of this new technology.