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Review
. 2005 Oct 29;331(7523):1019-21.
doi: 10.1136/bmj.331.7523.1019.

Commissioning for rare diseases: view from the frontline

Amanda Burls  1 , Daphne Austin, David Moore
Affiliations
Review

Commissioning for rare diseases: view from the frontline

Amanda Burls et al. BMJ. .

Abstract

Deciding whether to fund treatments that do good one by one tends to lead to a positive decision. However, this can cause wider harmful effects, as West Midlands' experience in the funding of enzyme replacement therapy for lysosomal storage diseases shows

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Figures

Figure 1
Figure 1
Spleen of patient with Gaucher's disease: enzyme replacement therapy can prevent accumulation of glucocerebroside Credit: CNRI/SPL
See this image and copyright information in PMC

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References

    1. McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331: 1016-9. - PMC - PubMed
    1. Department of Health. Guidance on commissioning arrangements for specialised services. London: DoH, 2003.
    1. Department of Health. National Specialist Commissioning Advisory Group (NSCAG). London: DoH, 2005.
    1. McIver S. The views of the public about funding enzyme replacement therapy. Birmingham: West Midlands Specialised Services Agency, 2004.
    1. Sheehan MP. Enzyme replacement therapy, resource allocation and distributive justice. Birmingham: West Midlands Specialised Services Agency, 2004.