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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., Competing Interests: Declarations Conflict of interest Amy Gye is an employee of Novartis Pharmaceuticals. Stephen Goodall and Richard De Abreu Lourenco have no conflicts to disclose. Author contributions Concept and design: Gye, Goodall, De Abreu Lourenco; acquisition of data: Gye; analysis and interpretation of data: Gye; drafting of the manuscript: Gye, Goodall, De Abreu Lourenco; critical revision of the paper for important intellectual content: Goodall, De Abreu Lourenco; supervision: Goodall, De Abreu Lourenco. Data availability statement Individual data used to support this study are not publicly available for ethical and legal reasons. All other sources used in this research are referenced and publicly available. Funding Open Access funding enabled and organized by CAUL and its Member Institutions. This research is part of an Industry Doctorate Program at the Centre for Health Economics Research and Evaluation, University of Technology Sydney in collaboration with Novartis Pharmaceuticals Australia. Novartis Pharmaceuticals Australia provided a Research Support Fee. Role of the Funder/Sponsor The funder had no role in the design and conduct of the study; collection, management, analysis and interpretation of the data; or preparation of the manuscript. The manuscript was reviewed by Novartis prior to submitting for publication. The views expressed are those of the authors and not necessarily those of Novartis Pharmaceuticals or the Centre for Health Economics Research and Evaluation (CHERE)., (© 2024. The Author(s).)

Objective: This research assesses the impact of an outcome-based payment arrangement (OBA) linking complete remission (CR) to survival as a means of maintaining cost-effectiveness for a chimeric antigen receptor T cell (CAR-T) therapy in young patients with acute lymphoblastic leukemia (ALL)., Methods: A partitioned survival model (PSM) was used to model the cost-effectiveness of tisagenlecleucel versus blinatumomab in ALL from the Australian healthcare system perspective. A decision tree modeled different OBAs by funneling patients into a series of PSMs based on response. Outcomes were informed by individual patient data, while costs followed Australian treatment practices. Costs and quality-adjusted life years (QALYs) were combined to calculate a single incremental cost-effectiveness ratio (ICER), reported in US dollars (2022) at a discount rate of 5% on costs and outcomes., Results: For the base case, incremental costs and benefit were $379,595 and 4.27 QALYs, giving an ICER of $88,979. The ICER was most sensitive to discount rate ($57,660-$75,081), "cure point" ($62,718-$116,206) and extrapolation method ($76,018-$94,049). OBAs had a modest effect on the ICER when response rates varied. A responder-only payment was the most effective arrangement for maintaining the ICER ($88,249-$89,434), although this option was associated with the greatest financial uncertainty. A split payment arrangement (payment on infusion followed by payment on response) reduced variability in the ICER ($82,650-$99,154) compared with a single, upfront payment ($77,599-$107,273)., Conclusion: OBAs had a modest impact on reducing cost-effectiveness uncertainty. The value of OBAs should be weighed against the additional resources needed to administer such arrangements, and importantly overall cost to government., (© 2022. Crown.)

Objective: This review explores the evidence from the literature regarding how meta-health effects (effects other than health resulting from the consumption of health care) are valued for use in economic evaluations., Methods: A systematic review of the published literature (the EMBASE, MEDLINE, PsycINFO, CINAHL, EconLit and SocINDEX databases were searched for publications in March 2016, plus manual searching) investigated the associations between study methods and the resulting values for meta-health effects estimated for use in economic evaluations. The review considered which meta-health effects were being valued and how this differed by evaluation approach, intervention investigated, source of funds and year of publication. Detailed reasons for differences observed between values for comparable meta-health effects were explored, accounting for the method of valuation., Results: The search of the literature revealed 71 studies of interest; 35% involved drug interventions, with convenience, information and process of care the three meta-health effects most often investigated. Key associations with the meta-health effects were the evaluation method, the intervention, and the source of funds. Relative values for meta-health effects ranged from 0.9% to 68% of the overall value reported in a study. For a given meta-health effect, the magnitude of the effect evaluated and how the meta-health effect was described and framed relative to overall health explained the differences in relative values., Conclusions: Evidence from the literature shows variability in how meta-health effects are being measured for use in economic evaluations. Understanding the sources of that variability is important if decision makers are to have confidence in how meta-health effects are valued.

Lower back pain is one of the most prevalent musculoskeletal conditions in the developed world and accounts for significant health services use. The American College of Physicians and the American Pain Society have published a joint clinical guideline that recommends providing patients with information on prognosis and self-management, the use of medications with proven benefits and, for those who do not improve, consideration be given to the use of spinal manipulation (for acute lower back pain only), interdisciplinary rehabilitation, exercise, acupuncture, massage, yoga, cognitive behavioural therapy or relaxation. The purpose of this review was to evaluate published economic evaluations of pharmacological management for chronic lower back pain. A total of seven studies were eligible for inclusion in there view. The quality of the economic evaluations undertaken in the included studies was not high. This was primarily because of the nature of the underlying clinical evidence, most of which did not come from rigorous randomised controlled trials (RCTs), and the manner in which it was incorporated into the economic evaluations. All studies provided reasonable information about what aspects of healthcare and other resource use were identified, measured and valued. However, the reporting of total costs was not uniform across studies. Measures of pain and disability were the most commonly collected outcomes measures. Two studies collected information on quality of life directly from participants while two studies modelled this information based on the literature. Future economic evaluations of interventions for chronic lower back pain, including pharmacological interventions, should be based on the results of well-conducted RCTs where the measurement of costs and outcomes such as quality of life and quality-adjusted life-years is included in the trial protocol, and which have a follow-up period sufficient to capture meaningful changes in both costs and outcomes. In the absence of RCT data, economic models should be used to estimate future costs and outcomes using robust methods.