Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review

Abstract

Objective

The aim of this review was to summarize all available evidence on the cost effectiveness of potentially curative gene therapies and identify challenges that economic evaluations face in this area.

Methods

We conducted a systematic review of four databases (PubMed/MEDLINE, Embase, CINAHL, EconLit) and grey literature sources. We conducted the search on August 23, 2019 and updated it on November 26, 2020. We included all English, French and Spanish language studies that addressed a gene therapy that had received regulatory approval or had entered a phase III trial, and also reported on costs related to the therapy. Critical appraisal was conducted to assess quality of reporting in included studies.

Results

Fifty-six studies were identified. Of the 42 full economic evaluations, 71% (n = 30) evaluated chimeric antigen receptor T-cell therapies, most used either a Markov model (n = 17, 40%) and/or a partitioned survival model (n = 17, 40%), and 76% (n = 32) adopted a public or private payer perspective. The model characteristics with the greatest impact on cost effectiveness included assumptions about the efficacy of the treatment and the comparators used.

Conclusion

All gene therapies in this review were shown to be more effective than their comparators, although due to high costs not all were considered cost effective at standard cost-effectiveness thresholds. Despite their high cost, some gene therapies have the potential to dominate the alternatives in conditions with high mortality/disability. The choice of comparator and assumptions regarding long-term effectiveness had substantial impacts on cost-effectiveness estimates and need to be carefully considered. Both the quality of inputs and the quality of reporting were highly variable.

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Correspondence to Larry D. Lynd.

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Funding

This research was supported by unrestricted funds from Dr Larry Lynd’s research funding.

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The authors report no conflicts of interest or competing interests.

Availability of data and material

All included publications and grey literature were obtained through publically available sources. All data generated or analyzed during this study are included in this published article and its supplementary information files.

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Authors' contributions

All authors contributed to the study conception and design. Material preparation, data collection and analysis were performed by all authors. The first draft of the manuscript was written by JKH and KB and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

Supplementary Information

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40273_2021_1051_MOESM1_ESM.docx

Online Resource 1: Supplemental Materials and Methods This document contains additional information about the methods used to conduct the literature review including the full search strategy used for the MEDLINE search, keywords used for the grey literature search strategy, details about the inclusion and exclusion criteria, and additional details about the methods used for study selection, data extraction, and data analysis

40273_2021_1051_MOESM2_ESM.xlsx

Online Resource 2: Results of Critical Appraisal These tables outline the quality of reporting in the included studies. Economic evaluations were critically appraised using the CHEERS checklist and we used the AACODS checklist for those studies that were not economic evaluations

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Ho, J.K., Borle, K., Dragojlovic, N. et al. Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review. PharmacoEconomics (2021). https://doi.org/10.1007/s40273-021-01051-4

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