Skip Navigation


QJM Advance Access originally published online on October 3, 2005
QJM 2005 98(11):829-836; doi:10.1093/qjmed/hci128
This Article
Right arrow Full Text Freely available
Right arrow FREE Full Text (PDF) Freely available
Right arrow All Versions of this Article:
98/11/829    most recent
hci128v1
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in ISI Web of Science
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Add to My Personal Archive
Right arrow Download to citation manager
Right arrow Search for citing articles in:
ISI Web of Science (20)
Right arrowRequest Permissions
Right arrow Disclaimer
Google Scholar
Right arrow Articles by Hughes, D.A.
Right arrow Articles by Yeo, S.T.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Hughes, D.A.
Right arrow Articles by Yeo, S.T.
Social Bookmarking
Add to CiteULike   Add to Connotea   Add to Del.icio.us  
What's this?

© The Author 2005. Published by Oxford University Press on behalf of the Association of Physicians. All rights reserved. For Permissions, please email: journals.permissions@oxfordjournals.org

Commentary

Drugs for exceptionally rare diseases: do they deserve special status for funding?

D.A. Hughes, B. Tunnage and S.T. Yeo

From the Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, University of Wales, Bangor, UK

Address correspondence to Dr D.A. Hughes, Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, Dean Street, University of Wales, Bangor, Gwynedd LL57 1UT. email: d.a.hughes{at}bangor.ac.uk

Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, some EU healthcare systems assess the cost-effectiveness of therapies when deciding if they should be funded. As ultra-orphan drugs are invariably cost-ineffective, factors in addition to cost-effectiveness need to be considered if ultra-orphan drugs are to be provided by public health services. Health service funding of ultra-orphan drugs, which varies across the EU and within the UK, has led to geographical inequities in patients' access to treatment. In some instances, support for these drugs would appear to have been approved on the basis that diseases that are rare and severe are a special case. We explore whether ultra-orphan drugs merit special status by considering efficiency, effectiveness and equity criteria. Mechanisms are discussed for creating a policy that would reduce geographical inequalities in provision across Europe.


Add to CiteULike CiteULike   Add to Connotea Connotea   Add to Del.icio.us Del.icio.us    What's this?


This article has been cited by other articles:


Home page
 J. Med. EthicsHome page
M Schlander
The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process
J. Med. Ethics, July 1, 2008; 34(7): 534 - 539.
[Abstract] [Full Text] [PDF]

Home page
 Journal of Health Politics, Policy and LawHome page
J. P. Cohen, E. Stolk, and M. Niezen
Role of Budget Impact in Drug Reimbursement Decisions
Journal of Health Politics Policy and Law, January 1, 2008; 33(2): 225 - 247.
[Abstract] [PDF]

Home page
 ASH Education BookHome page
P. Hillmen
The Role of Complement Inhibition in PNH
Hematology, January 1, 2008; 2008(1): 116 - 123.
[Abstract] [Full Text] [PDF]

Home page
 J. Am. Soc. Nephrol.Home page
R. J.H. Smith, J. Alexander, P. N. Barlow, M. Botto, T. L. Cassavant, H. T. Cook, S. R. de Cordoba, G. S. Hageman, T. S. Jokiranta, W. J. Kimberling, et al.
New Approaches to the Treatment of Dense Deposit Disease
J. Am. Soc. Nephrol., September 1, 2007; 18(9): 2447 - 2456.
[Abstract] [Full Text] [PDF]


Disclaimer: Please note that abstracts for content published before 1996 were created through digital scanning and may therefore not exactly replicate the text of the original print issues. All efforts have been made to ensure accuracy, but the Publisher will not be held responsible for any remaining inaccuracies. If you require any further clarification, please contact our Customer Services Department.