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Review
. 2005 Oct 29;331(7523):1016-9.
doi: 10.1136/bmj.331.7523.1016.

Orphan drugs and the NHS: should we value rarity?

Christopher McCabe  1 , Karl Claxton, Aki Tsuchiya
Affiliations
Review

Orphan drugs and the NHS: should we value rarity?

Christopher McCabe et al. BMJ. .

Abstract

The growing number and costs of drugs for rare diseases are straining healthcare budgets. Decisions on funding these treatments need to be made on a sound basis

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Figures

Figure 1
Figure 1
Special status for drugs for rare diseases such as neurofibromatosis may be unsustainable Credit: PENNY TWEEDIE/SPL
See this image and copyright information in PMC

Comment in

References

    1. National Institute for Clinical Excellence. NHS should consider paying premium prices for drugs to treat patients with very rare diseases says NICE Citizen's Council. Press release 26 Jan. www.nice.org.uk/pdf/2005_002_CitizensCouncilreport.pdf (accessed 21 Feb 2005).
    1. Department of Health. National designation and funding of treatment for patients with lysosomal storage disorders. www.dh.gov.uk/assetRoot/04/09/22/10/04092210.pdf (accessed 22 Feb 2005).
    1. Rawlins M. Managing rare diseases. London: Royal College of Physicians, 2004.
    1. National Institute for Clinical Excellence. Final appraisal determination: imatinib for the treatment of unresectable and/or metastatic gastro-intestinal stromal tumours. London: NICE, 2004 www.nice.org.uk/page.aspx?o=216331 (accessed 18 Aug 2005).
    1. National Institute for Clinical Excellence. Guide to the methods of health technology appraisal. London: NICE, 2003.