PharmacoEconomics · 2026
Methodological Challenges and Preferred Approaches for the Economic Evaluation of CAR T Therapies: Evidence Review and Expert Consensus from NICE Appraisals.
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Yousefi Mehdi, Mehrabian Amin, O'Reilly Maeve A, Roddie Claire, Auguste Peter, Lee Dawn, Parsons Jo, Gallacher Daniel
Аннотация
BACKGROUND: Chimeric antigen receptor T cell (CAR T) therapies pose challenges for economic evaluation because of complex treatment pathways and uncertainty around long-term outcomes. Within health technology assessment (HTA) bodies such as the National Institute for Health and Care Excellence (NICE), these features have led to recurring methodological debates.
OBJECTIVES: To identify methodological challenges specific to the HTA of CAR T therapies within NICE technology appraisals and to develop Delphi-informed recommendations to support more consistent modelling practice.
METHODS: A three-phase mixed-methods study was conducted. Phase 1 involved qualitative content analysis of published NICE appraisals of CAR T therapies to identify methodological issues and solutions. Phase 2 used a modified Delphi to determine which issues were specific to CAR T therapies and to agree upon preferred methodological approaches. Phase 3 involved clinical sense-checking of the preferred methodological approaches.
RESULTS: Phase 1 reviewed nine NICE appraisals, identifying 16 issues around survival modelling, cure assumptions, costing and long-term outcomes. Ten issues were specified in relation to CAR T. Phase 2 identified one further relevant issue. The preferred approaches included modelling from leukapheresis (85%), applying cure assumptions consistently across treatment arms (95%) and applying modest utility decrements for long-term survivors (94%). For delivery costs, 61% preferred the most recent NHS England CAR T tariff with targeted supplements, while 28% preferred the most transparent tariff choice, indicating less uniform agreement. UK-specific real-world data were preferred to inform assumptions on treatment delivery, resource use and subsequent therapies. Phase 3 provided a clinical sense-check of these preferred approaches and identified important additional considerations for future appraisals.
CONCLUSIONS: Methodological challenges in the economic evaluation of CAR T therapies recur across NICE appraisals and materially influence cost-effectiveness results. Drawing on evidence from NICE appraisals combined with Delphi findings and clinical sense-checking, this study provides practical recommendations relating to model start-point, cure modelling and long-term survivor utility decrements supporting more consistent and transparent economic evaluation within the NICE framework.
OBJECTIVES: To identify methodological challenges specific to the HTA of CAR T therapies within NICE technology appraisals and to develop Delphi-informed recommendations to support more consistent modelling practice.
METHODS: A three-phase mixed-methods study was conducted. Phase 1 involved qualitative content analysis of published NICE appraisals of CAR T therapies to identify methodological issues and solutions. Phase 2 used a modified Delphi to determine which issues were specific to CAR T therapies and to agree upon preferred methodological approaches. Phase 3 involved clinical sense-checking of the preferred methodological approaches.
RESULTS: Phase 1 reviewed nine NICE appraisals, identifying 16 issues around survival modelling, cure assumptions, costing and long-term outcomes. Ten issues were specified in relation to CAR T. Phase 2 identified one further relevant issue. The preferred approaches included modelling from leukapheresis (85%), applying cure assumptions consistently across treatment arms (95%) and applying modest utility decrements for long-term survivors (94%). For delivery costs, 61% preferred the most recent NHS England CAR T tariff with targeted supplements, while 28% preferred the most transparent tariff choice, indicating less uniform agreement. UK-specific real-world data were preferred to inform assumptions on treatment delivery, resource use and subsequent therapies. Phase 3 provided a clinical sense-check of these preferred approaches and identified important additional considerations for future appraisals.
CONCLUSIONS: Methodological challenges in the economic evaluation of CAR T therapies recur across NICE appraisals and materially influence cost-effectiveness results. Drawing on evidence from NICE appraisals combined with Delphi findings and clinical sense-checking, this study provides practical recommendations relating to model start-point, cure modelling and long-term survivor utility decrements supporting more consistent and transparent economic evaluation within the NICE framework.