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Modelling the Cost Effectiveness of Disease-Modifying Treatments for Multiple Sclerosis

Issues to Consider

PharmacoEconomics volume 31, pages 455–469 (2013)Cite this article

Abstract

Several cost-effectiveness models of disease-modifying treatments (DMTs) for multiple sclerosis (MS) have been developed for different populations and different countries. Vast differences in the approaches and discrepancies in the results give rise to heated discussions and limit the use of these models. Our main objective is to discuss the methodological challenges in modelling the cost effectiveness of treatments for MS. We conducted a review of published models to describe the approaches taken to date, to identify the key parameters that influence the cost effectiveness of DMTs, and to point out major areas of weakness and uncertainty. Thirty-six published models and analyses were identified. The greatest source of uncertainty is the absence of head-to-head randomized clinical trials. Modellers have used various techniques to compensate, including utilizing extension trials. The use of large observational cohorts in recent studies aids in identifying population-based, ‘real-world’ treatment effects. Major drivers of results include the time horizon modelled and DMT acquisition costs. Model endpoints must target either policy makers (using cost-utility analysis) or clinicians (conducting cost-effectiveness analyses). Lastly, the cost effectiveness of DMTs outside North America and Europe is currently unknown, with the lack of country-specific data as the major limiting factor. We suggest that limited data should not preclude analyses, as models may be built and updated in the future as data become available. Disclosure of modelling methods and assumptions could improve the transferability and applicability of models designed to reflect different healthcare systems.

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Acknowledgments

The authors would like to express sincere gratitude and appreciation to Dr. Matthew Bellizzi for his clinical consultations and thoughtful suggestions. After publishing a risk-benefit assessment of natalizumab [55], Joel Thompson and Katia Noyes received a χresearch grant from Biogen Idec to update the model (unpublished).

Funding

Contract HC 0103 from the National Multiple Sclerosis Society (Programme Officer: Nicholas LaRocca, PhD); Clinical and Translational Science Award (CTSA) [UL1 RR024160] from the National Center for Research Resources (NCRR), a component of the National Institutes of Health (NIH) and the NIH Roadmap for Medical Research.

Author Contributions

Joel Thompson contributed to the concept and design, the acquisition of data, the analysis and interpretation of data, drafting the manuscript, critical revision of the manuscript, and study supervision. Amir Abdolahi contributed to the acquisition of data, drafting the manuscript, and critical revision of the manuscript. Katia Noyes contributed to the concept and design, the acquisition of data, the analysis and interpretation of data, drafting the manuscript, critical revision of the manuscript, and study supervision, and obtained funding for the study. Joel Thompson acts as guarantor for the overall content of this article.

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Affiliations

  1. Department of Medicine, University of Rochester Medical Center, 601 Elmwood Ave, Box 648, Rochester, NY, 14642, USA

    Joel P. Thompson

  2. Community and Preventive Medicine, University of Rochester Medical Center, Rochester, NY, USA

    Amir Abdolahi & Katia Noyes

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  1. Joel P. Thompson
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Correspondence to Joel P. Thompson.

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Thompson, J.P., Abdolahi, A. & Noyes, K. Modelling the Cost Effectiveness of Disease-Modifying Treatments for Multiple Sclerosis. PharmacoEconomics 31, 455–469 (2013). https://doi.org/10.1007/s40273-013-0063-4

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Keywords

  • Multiple Sclerosis
  • Multiple Sclerosis Patient
  • Expand Disability Status Scale
  • Natalizumab
  • Glatiramer Acetate