Authors' objectives:

To assess the clinical effectiveness and cost-effectiveness of riluzole for the treatment of motor neurone disease (MND).

Authors' results and conclusions: Median follow-up in all trials was 18 months with most patients having follow-up of between 16 and 21 months. Combined results favoured riluzole with a hazard ratio for tracheostomy-free survival of 0.88 (95% confidence interval (CI), 0.75 to 1.02). There was no evidence that the effectiveness of the treatment differed by site of onset. There was also no significant difference in effectiveness in daily dosages of between 50 and 200 mg. There was, however, some evidence of statistical heterogeneity (p = 0.09) and, if this is not due to chance, there is no clear explanation as to why this may have arisen. Riluzole does not improve symptoms. When data on functional status were combined, a small reduction in the rate of deterioration of functional status was observed, although it was not clear whether this was clinically significant. A large proportion of patients in both groups reported adverse events, but there was little overall difference between riluzole and placebo. There was no evidence available on treatment outcomes beyond 18 months. All placebo patients were offered riluzole at the end of follow-up, and no longer-term comparative data will thus be available from any of these trials.

Authors' recommendations: There is limited evidence of a modest benefit in tracheostomy-free survival for patients taking riluzole. However, the evidence is restricted and uncertainty remains as to the true benefit of riluzole; the CI is wide and compatible with little or no difference between riluzole and placebo. When costs and the health economic impact are considered when extrapolating survival beyond that observed in trials, the uncertainty about whether the benefits are worth the costs is magnified. Even under the most optimistic assumptions, riluzole at best only postpones death for a few months, and does not preclude the need for supportive care and practical help. If riluzole were to be made available to all patients in whom it is not contraindicated, the annual cost to the NHS would be about 8.4 million GBP, assuming all these patients wish to take it. Many patients, given accurate information about the benefits and effects of riluzole, may choose not to. Patients should be made aware that riluzole does not cure amyotrophic lateral sclerosis (ALS); accurate patient information is essential.

Authors' methods: Systematic review, Cost study

Project Status: Completed

URL for project: http://www.hta.ac.uk/1174

Year Published: 2001

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Health Technology Assessment programme

Contact Address: NIHR Journals Library, National Institute for Health and Care Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK

Contact Name: journals.library@nihr.ac.uk

Contact Email: journals.library@nihr.ac.uk

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