Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care.

Gye, A; De Abreu Lourenco, R; Goodall, S

Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care.

Gye, A De Abreu Lourenco, R Goodall, S

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Publisher:: Springer Nature
Publication Type:: Journal Article
Citation:: Pharmacoeconomics, 2024, pp. 1-13
Issue Date:: 2024-09-07

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Field	Value	Language
dc.contributor.author	Gye, A
dc.contributor.author	De Abreu Lourenco, R
dc.contributor.author	Goodall, S
dc.date.accessioned	2024-09-25T04:10:51Z
dc.date.available	2024-08-18
dc.date.available	2024-09-25T04:10:51Z
dc.date.issued	2024-09-07
dc.identifier.citation	Pharmacoeconomics, 2024, pp. 1-13
dc.identifier.issn	1170-7690
dc.identifier.issn	1179-2027
dc.identifier.uri	http://hdl.handle.net/10453/180968
dc.description.abstract	OBJECTIVE: Chimeric antigen-receptor T-cell therapy (CAR-T) is characterised by early phase data at the time of registration, high upfront cost and a complex manufacturing and administration process compared with standard therapies. Our objective was to compare the performance of different models to assess the cost effectiveness of CAR-T using a state-transition model (STM), partitioned survival model (PSM) and discrete event simulation (DES). METHODS: Individual data for tisagenlecleucel for the treatment of young patients with acute lymphoblastic leukaemia (ALL) were used to populate the models. Costs and benefits were measured over a lifetime to generate a cost per quality-adjusted life-year (QALY). Model performance was compared quantitatively on the outcomes generated and a checklist developed summarising the components captured by each model type relevant to assessing cost effectiveness of CAR-T. RESULTS: Models generated similar results with base-case analyses ranging from an incremental cost per QALY of $96,074-$99,625. DES was the only model to specifically capture CAR-T wait time, demonstrating a substantial loss of benefit of CAR-T with increased wait time. CONCLUSION: Although model type did not meaningfully impact base-case results, the ability to incorporate an outcome-based payment arrangement (OBA) and wait time are important elements to consider when selecting a model for CAR-T. DES provided greater flexibility compared with STM and PSM approaches to deal with the complex manufacturing and administration process that can lead to extended wait times and substantially reduce the benefit of CAR-T. This is an important consideration when selecting a model type for CAR-T, so major drivers of uncertainty are considered in funding decisions.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	Springer Nature
dc.relation.ispartof	Pharmacoeconomics
dc.relation.isbasedon	10.1007/s40273-024-01430-7
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	11 Medical and Health Sciences, 14 Economics
dc.subject.classification	Health Policy & Services
dc.subject.classification	3214 Pharmacology and pharmaceutical sciences
dc.subject.classification	3801 Applied economics
dc.subject.classification	4203 Health services and systems
dc.title	Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care.
dc.type	Journal Article
utslib.location.activity	New Zealand
utslib.for	11 Medical and Health Sciences
utslib.for	14 Economics
pubs.organisational-group	University of Technology Sydney
pubs.organisational-group	University of Technology Sydney/Faculty of Health
pubs.organisational-group	University of Technology Sydney/Faculty of Health/Research Centres/Centre for Health Economics Research and Evaluation
pubs.organisational-group	University of Technology Sydney/Faculty of Health/Research Centres
utslib.copyright.status	open_access	*
dc.rights.license	This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0). To view a copy of this license, visit https://creativecommons.org/licenses/by-nc/4.0/
dc.date.updated	2024-09-25T04:10:49Z
pubs.publication-status	Published online

Abstract:

OBJECTIVE: Chimeric antigen-receptor T-cell therapy (CAR-T) is characterised by early phase data at the time of registration, high upfront cost and a complex manufacturing and administration process compared with standard therapies. Our objective was to compare the performance of different models to assess the cost effectiveness of CAR-T using a state-transition model (STM), partitioned survival model (PSM) and discrete event simulation (DES). METHODS: Individual data for tisagenlecleucel for the treatment of young patients with acute lymphoblastic leukaemia (ALL) were used to populate the models. Costs and benefits were measured over a lifetime to generate a cost per quality-adjusted life-year (QALY). Model performance was compared quantitatively on the outcomes generated and a checklist developed summarising the components captured by each model type relevant to assessing cost effectiveness of CAR-T. RESULTS: Models generated similar results with base-case analyses ranging from an incremental cost per QALY of $96,074-$99,625. DES was the only model to specifically capture CAR-T wait time, demonstrating a substantial loss of benefit of CAR-T with increased wait time. CONCLUSION: Although model type did not meaningfully impact base-case results, the ability to incorporate an outcome-based payment arrangement (OBA) and wait time are important elements to consider when selecting a model for CAR-T. DES provided greater flexibility compared with STM and PSM approaches to deal with the complex manufacturing and administration process that can lead to extended wait times and substantially reduce the benefit of CAR-T. This is an important consideration when selecting a model type for CAR-T, so major drivers of uncertainty are considered in funding decisions.

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