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Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review

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Abstract

Objective

Decision-makers need to resolve constraints on delivering cell and gene therapies to patients as these treatments move into routine care. This study aimed to investigate if, and how, constraints that affect the expected cost and health consequences of cell and gene therapies have been included in published examples of cost-effectiveness analyses (CEAs).

Method

A systematic review identified CEAs of cell and gene therapies. Studies were identified from previous systematic reviews and by searching Medline and Embase until 21 January 2022. Constraints described qualitatively were categorised by theme and summarised by a narrative synthesis. Constraints evaluated in quantitative scenario analyses were appraised by whether they changed the decision to recommend treatment.

Results

Thirty-two CEAs of cell (n = 20) and gene therapies (n = 12) were included. Twenty-one studies described constraints qualitatively (70% cell therapy CEAs; 58% gene therapy CEAs). Qualitative constraints were categorised by four themes: single payment models; long-term affordability; delivery by providers; manufacturing capability. Thirteen studies assessed constraints quantitatively (60% cell therapy CEAs; 8% gene therapy CEAs). Two types of constraint were assessed quantitatively across four jurisdictions (USA, Canada, Singapore, The Netherlands): alternatives to single payment models (n = 9 scenario analyses); improving manufacturing (n = 12 scenario analyses). The impact on decision-making was determined by whether the estimated incremental cost-effectiveness ratios crossed a relevant cost-effectiveness threshold for each jurisdiction (outcome-based payment models: n = 25 threshold comparisons made, 28% decisions changed; improving manufacturing: n = 24 threshold comparisons made, 4% decisions changed).

Conclusion

The net health impact of constraints is vital evidence to help decision-makers scale up the delivery of cell and gene therapies as patient volume increases and more advanced therapy medicinal products are launched. CEAs will be essential to quantify how constraints affect the cost-effectiveness of care, prioritise constraints to be resolved, and establish the value of strategies to implement cell and gene therapies by accounting for their health opportunity cost.

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Authors and Affiliations

  1. Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Oxford Road, Manchester, M13 9PL, UK

    Sean P. Gavan, Stuart J. Wright & Katherine Payne

  2. Division of Cancer Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, M13 9PL, UK

    Fiona Thistlethwaite

  3. Department of Medical Oncology, The Christie NHS Foundation Trust, Wilmslow Road, Manchester, M20 4BX, UK

    Fiona Thistlethwaite

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  1. Sean P. Gavan
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All authors contributed to the study conception and design. Material preparation, data collection, and analysis were performed by SPG, SJW, and KP. The first draft of the manuscript was written by SPG, and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

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Correspondence to Sean P. Gavan.

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Conflict of interest

Sean P. Gavan and Katherine Payne declare that they have no conflict of interest. Stuart J. Wright reports consultancy fees for acting on a steering group for a project investigating the impact of including supply constraints in economic evaluations for PHMR on a project funded by Roche. Fiona Thistlethwaite reports institutional research grant income from Instil Bio; honoraria from GSK, Bristol Myers Squibb, Janssen, and Pfizer; advisory committee membership for Immatics; and speaker fees from Kite/Gilead; is principal investigator for commercial ATMP trials (Adaptimmune, GSK, T-knife Therapeutics, Instil Bio), but does not receive personal payment; and is director of iMATCH (Innovate Manchester Advanced Therapy Centre Hub), supported by Innovate UK.

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This work was supported by Innovate UK (grant number 104234). For the purpose of open access, the authors have applied a Creative Commons Attribution (CC BY) licence to any Author Accepted Manuscript version arising.

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Gavan, S.P., Wright, S.J., Thistlethwaite, F. et al. Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review. PharmacoEconomics 41, 675–692 (2023). https://doi.org/10.1007/s40273-022-01234-7

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