Is there a need for a different methodological approach to modelling the cost-effectiveness of cell and gene therapies?

Gye, Amy

Is there a need for a different methodological approach to modelling the cost-effectiveness of cell and gene therapies?

Gye, Amy

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Publication Type:: Thesis
Issue Date:: 2024

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Field	Value	Language
dc.contributor.author	Gye, Amy
dc.date.accessioned	2025-01-21T01:40:26Z
dc.date.available	2025-01-21T01:40:26Z
dc.date.issued	2024
dc.identifier.uri	http://hdl.handle.net/10453/183908
dc.description	University of Technology Sydney. Faculty of Health.	en_US.UTF-8
dc.description.abstract	Chimeric antigen receptor T-cell therapies (CAR-Ts) were the first cell and gene therapies registered globally and considered for public funding via a health technology assessment (HTA) process. Access to CAR-T provides a potential cure for patients with certain blood cancers where previously there were limited alternatives and survival outcomes were poor. At the same time, these novel therapies have disrupted health care systems and challenged public funding decisions. While many of the issues in assessing the value of CAR-Ts are similar other medicines, particularly in oncology, complex manufacturing, potential for cure and high up-front costs make CAR-T unique. These elements combined have made assessing their cost-effectiveness for HTA purposes particularly challenging. Using the CAR-T, tisagenlecleucel, for the treatment of young patients with relapsed or refractory (r/r) acute lymphoblastic leukaemia (ALL) as a case-study, the research objectives were: (1) identify sources of uncertainty in modelled economic evaluations of CAR-Ts considered by HTA agencies, (2) assess the utility of outcome-based payment arrangements (OBAs) as a mechanism for managing long-term clinical and economic uncertainty using a de novo modelling approach, (3) explicitly model the CAR-T treatment pathway to capture the impact of CAR-T infusion wait-time using discrete event simulation (DES), and (4) compare different modelling techniques to assess the impact of structural uncertainty on cost-effectiveness. In addressing each of these objectives, this research demonstrates the importance of designing flexible model structures to incorporate OBAs to determine a suitable payment structure, outcome selection, and contractual conditions for implementation. The importance of considering the process of CAR-T in model design is evident, as delay in infusion of CAR-T resulted in substantial reduction in benefit at a population level. Overall, this research highlights the need for a bespoke modelling approach for CAR-T to identify the main sources of uncertainty impacting cost-effectiveness to aid decision-making at the initial point of assessment and facilitate speed of access to patients.	en_US.UTF-8
dc.format	Thesis (PhD)
dc.language.iso	en_US	en_US.UTF-8
dc.relation	https://opus.lib.uts.edu.au/bitstream/10453/183908/1/thesis.pdf
dc.rights	info:eu-repo/semantics/openAccess
dc.rights	The author owns the copyright in this thesis including all reproduction and reuse rights for the work. The work may not be altered without the permission of the copyright owner. Attribution is essential when quoting or paraphrasing from this thesis.
dc.rights	© 2024 Amy Gye
dc.rights	au.edu.uts.lib/cph
dc.title	Is there a need for a different methodological approach to modelling the cost-effectiveness of cell and gene therapies?	en_US.UTF-8
dc.type	Thesis
utslib.copyright.status	open_access	*

Abstract:

Chimeric antigen receptor T-cell therapies (CAR-Ts) were the first cell and gene therapies registered globally and considered for public funding via a health technology assessment (HTA) process. Access to CAR-T provides a potential cure for patients with certain blood cancers where previously there were limited alternatives and survival outcomes were poor. At the same time, these novel therapies have disrupted health care systems and challenged public funding decisions. While many of the issues in assessing the value of CAR-Ts are similar other medicines, particularly in oncology, complex manufacturing, potential for cure and high up-front costs make CAR-T unique. These elements combined have made assessing their cost-effectiveness for HTA purposes particularly challenging. Using the CAR-T, tisagenlecleucel, for the treatment of young patients with relapsed or refractory (r/r) acute lymphoblastic leukaemia (ALL) as a case-study, the research objectives were: (1) identify sources of uncertainty in modelled economic evaluations of CAR-Ts considered by HTA agencies, (2) assess the utility of outcome-based payment arrangements (OBAs) as a mechanism for managing long-term clinical and economic uncertainty using a de novo modelling approach, (3) explicitly model the CAR-T treatment pathway to capture the impact of CAR-T infusion wait-time using discrete event simulation (DES), and (4) compare different modelling techniques to assess the impact of structural uncertainty on cost-effectiveness. In addressing each of these objectives, this research demonstrates the importance of designing flexible model structures to incorporate OBAs to determine a suitable payment structure, outcome selection, and contractual conditions for implementation. The importance of considering the process of CAR-T in model design is evident, as delay in infusion of CAR-T resulted in substantial reduction in benefit at a population level. Overall, this research highlights the need for a bespoke modelling approach for CAR-T to identify the main sources of uncertainty impacting cost-effectiveness to aid decision-making at the initial point of assessment and facilitate speed of access to patients.

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http://hdl.handle.net/10453/183908