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Economic Studies in Motor Neurone Disease: A Systematic Methodological Review

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Abstract

Background

Motor neurone disease (MND) is a devastating condition which greatly diminishes patients’ quality of life and limits life expectancy. Health technology appraisals of future interventions in MND need robust data on costs and utilities. Existing economic evaluations have been noted to be limited and fraught with challenges.

Objective

The aim of this study was to identify and critique methodological aspects of all published economic evaluations, cost studies, and utility studies in MND.

Methods

We systematically reviewed all relevant published studies in English from 1946 until January 2016, searching the databases of Medline, EMBASE, Econlit, NHS Economic Evaluation Database (NHS EED) and the Health Economics Evaluation Database (HEED). Key data were extracted and synthesised narratively.

Results

A total of 1830 articles were identified, of which 15 economic evaluations, 23 cost and 3 utility studies were included. Most economic studies focused on riluzole (n = 9). Six studies modelled the progressive decline in motor function using a Markov design but did not include mutually exclusive health states. Cost estimates for a number of evaluations were based on expert opinion and were hampered by high variability and location-specific characteristics. Few cost studies reported disease-stage-specific costs (n = 3) or fully captured indirect costs. Utilities in three studies of MND patients used the EuroQol EQ-5D questionnaire or standard gamble, but included potentially unrepresentative cohorts and did not consider any health impacts on caregivers.

Conclusion

Economic evaluations in MND suffer from significant methodological issues such as a lack of data, uncertainty with the disease course and use of inappropriate modelling framework. Limitations may be addressed through the collection of detailed and representative data from large cohorts of patients.

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Data Availability Statement

Data sharing not applicable to this article as no datasets were generated or analysed during the current study.

Acknowledgements

The authors would like to thank the Motor Neurone Disease Association UK for their funding.

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Authors and Affiliations

  1. Centre for Health Economics and Medicines Evaluation, Bangor University, Ardudwy, Holyhead Road, Bangor, LL57 2PZ, UK

    Alan Moore & Dyfrig A. Hughes

  2. The Walton Centre NHS Trust, Liverpool, UK

    Carolyn A. Young

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Correspondence to Dyfrig A. Hughes.

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AM, CAY and DH declare that they have no conflict of interest.

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AM, CAY and DH contributed substantially to the conception and design of the work. All authors made contributions to the acquisition, analysis, or interpretation of data. AM drafted the paper and all authors revised it critically for important intellectual content, and gave their final approval of the version to be published. All authors agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

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Moore, A., Young, C.A. & Hughes, D.A. Economic Studies in Motor Neurone Disease: A Systematic Methodological Review. PharmacoEconomics 35, 397–413 (2017). https://doi.org/10.1007/s40273-016-0478-9

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