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Research

Multiple sclerosis risk sharing scheme: two year results of clinical cohort study with historical comparator

BMJ 2009; 339 doi: https://doi.org/10.1136/bmj.b4677 (Published 02 December 2009) Cite this as: BMJ 2009;339:b4677
  1. Mike Boggild, consultant neurologist1,
  2. Jackie Palace, consultant neurologist2,
  3. Pelham Barton, senior lecturer in mathematical modelling3,
  4. Yoav Ben-Shlomo, professor of clinical epidemiology4,
  5. Thomas Bregenzer, director, biostatistics5,
  6. Charles Dobson, senior projects officer6,
  7. Richard Gray, director7
  1. 1The Walton Centre, Liverpool L9 7LJ
  2. 2John Radcliffe Hospital, Oxford OX3 9DU
  3. 3Health Economics Unit, University of Birmingham, Birmingham B15 2TT
  4. 4Department of Social Medicine, Canynge Hall, Bristol BS8 2PS
  5. 5Biostatistics Unit, Parexel International, 14050 Berlin, Germany
  6. 6Department of Health, Quarry House, Leeds LS2 7UE
  7. 7University of Birmingham Clinical Trials Unit, Birmingham B15 2TT
  1. Correspondence to: M Boggild mike.boggild{at}thewaltoncentre.nhs.uk
  • Accepted 5 August 2009

Abstract

Objective To generate evidence on the longer term cost effectiveness of disease modifying treatments in patients with relapsing-remitting multiple sclerosis.

Design Prospective cohort study with historical comparator.

Setting Specialist multiple sclerosis clinics in 70 centres in the United Kingdom.

Participants Patients with relapsing-remitting multiple sclerosis who started treatment from May 2002 to April 2005 under the UK risk sharing scheme.

Interventions Treatment with interferon beta or glatiramer acetate in accordance with guidelines of the UK Association of British Neurologists.

Main outcome measures Observed utility weighted progression in disability at two years’ follow-up assessed on the expanded disability status scale (EDSS) compared with that expected by applying the progression rates in a comparator dataset, modified for patients receiving treatment by multiplying by the hazard ratio derived separately for each disease modifying treatment from the randomised trials.

Results In the primary per protocol analysis, progression in disability was worse than that predicted and worse than that in the untreated comparator dataset (“deviation score” of 113%; excess in mean disability status scale 0.28). In sensitivity analyses, however, the deviation score varied from −72% (using raw baseline disability status scale scores, rather than applying a “no improvement” algorithm) to 156% (imputing missing data for year two from progression rates for year one).

Conclusions It is too early to reach any conclusion about the cost effectiveness of disease modifying treatments from this first interim analysis. Important methodological issues, including the need for additional comparator datasets, the potential bias from missing data, and the impact of the “no improvement” rule, will need to be addressed and long term follow-up of all patients is essential to secure meaningful results. Future analyses of the cohort are likely to be more informative, not least because they will be less sensitive to short term fluctuations in disability.

Footnotes

  • We acknowledge the contribution of Cindy Cooper and Mark Pickin in relation to patient recruitment to the scheme

  • Contributors: CD was involved in the original negotiation of the risk sharing scheme and continues to advise the parties to the scheme on analytical aspects. MB and JP are the clinical leads for the project. MB, JP, PB, YB-S, TB, and RG helped design the primary analysis plan, interpreted the results, and suggested further sensitivity analyses. TB ran the analyses. All the authors have contributed to the final version of the paper. MB is guarantor.

  • Funding: The central costs of data collection and analysis were shared in five equal amounts between the Department of Health and the four pharmaceutical companies (Bayer Schering Pharma, Biogen, Merck Serono, Teva). Treatment costs (drug and staff) were largely met by local commissioners, with financial support for nursing and other staff from the companies and the MS Society. Analysis and interpretation of results and drafting of the manuscript were undertaken by a group independent of the study sponsors.

  • Competing interests: MB and JP have received support for attending international congresses from each of the four pharmaceutical companies funding the study. CD is an employee of the Department of Health and was involved in the negotiation of the original risk sharing scheme.

  • Ethical approval: This study was approved by the South East multicentre research ethics committee (02/10/78).

  • Data sharing: Data from the scheme may be available for collaborative studies, applications should be made to the scientific advisory group via the clinical leads (JP, MB).

This is an open-access article distributed under the terms of the Creative Commons Attribution Non-commercial License, which permits use, distribution, and reproduction in any medium, provided the original work is properly cited, the use is non commercial and is otherwise in compliance with the license. See: http://creativecommons.org/licenses/by-nc/2.0/ and http://creativecommons.org/licenses/by-nc/2.0/legalcode.

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