PharmacoEconomics

, Volume 20, Issue 6, pp 367–387 | Cite as

Cost Effectiveness of Treatments for Amyotrophic Lateral Sclerosis

A Review of the Literature
  • Gary Ginsberg
  • Serena Lowe
Review Article

Abstract

Amyotrophic lateral sclerosis (ALS) is a difficult to diagnose, fatal, progressive degenerative disease with an average survival time of 2 to 5 years. Percutaneous endoscopic gastrotomy (PEG) and bi-level intermittent positive pressure (BIPAP) ventilation may be the major interventions leading to longer survival of patients with ALS.

Riluzole has been shown to have modest effects on survival (as opposed to functional) gains and is currently the only drug approved for the treatment of ALS. There is conflicting evidence with regard to the ability of recombinant human insulin-like growth factor (rhIGF-I) to retard ALS progression. Mechanical ventilation (via a tracheostomy tube) is expensive, but is widely used in later stage patients with ALS in the US. A review of nine cost-effectiveness studies of riluzole and one of rhIGF-I found the following: drug costs and survival gains are the major drivers of cost effectiveness; survival gains are estimated from truncated databases with a high degree of uncertainty; more accurate stage-specific utility weights based on patients who agreed to treatment are needed; case incidence-based evaluations should be carried out; cost-effectiveness ratios are insensitive to discount rates; employment and caregiver issues or externalities have been widely ignored; threshold acceptance cost-effectiveness values are ill-defined and evaluations are not generalisable to other countries because of cost and treatment style differences.

On account of the high degree of uncertainty pertaining to survival gains and the relatively high costs per life years or quality-adjusted life-years gained, and while acknowledging that not every therapy has to be cost effective (e.g. orphan drugs), it is still inconclusive as to whether or not riluzole or rhIGF-1 can be considered as cost-effective therapies for ALS.

Keywords

Amyotrophic Lateral Sclerosis Percutaneous Endoscopic Gastrostomy Riluzole Gross National Product Utility Weight 

Notes

Acknowledgements

The authors would like to thank Dr Anthony Stewart of the West Midlands Development and Evaluation Service, University of Birmingham for sharing information sources. Dr Ginsberg has in the past delivered two manufacturer-sponsored lectures on riluzole to general practitioners in the UK. No external funding sources were used in the preparation of this article.

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Copyright information

© Adis International Limited 2002

Authors and Affiliations

  • Gary Ginsberg
    • 1
  • Serena Lowe
    • 2
  1. 1.Department of Medical Technology AssessmentMinistry of HealthJerusalemIsrael
  2. 2.Health and Disabilities Practice, Sagamore AssociatesWashington, DCUSA

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