Is it time to revisit orphan drug policies?

BMJ (Online)

Volumn 341, Issue 7774, 2010, Pages 614-

  McCabe, Christopher ^a   Stafinski, Tania ^b   Menon, Devidas ^b  

^a UNIVERSITY OF LEEDS   (United Kingdom)

^b UNIVERSITY OF ALBERTA   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ENZYME; ORPHAN DRUG;

CLINICAL PRACTICE; DRUG CONTROL; DRUG MARKETING; EDITORIAL; ENZYME REPLACEMENT; HEALTH CARE ACCESS; HEALTH CARE COST; HEALTH CARE SYSTEM; MEDICAL DECISION MAKING; MEDICAL RESEARCH; PRIORITY JOURNAL; RARE DISEASE; TREATMENT OUTCOME; UNITED STATES;

HEALTH POLICY; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 77957114931     PISSN: None     EISSN: 17561833     Source Type: Journal    
DOI: 10.1136/bmj.c4777     Document Type: Editorial

References (12)

1. US Food and Drug Administration. Orphan drug act (as amended). www.fda.gov/orphan/oda.htm.
2. Regulation (EC) No 141/2000 of the European Parliament and of the Council of December 16, 1999 on orphan medicinal products. Official Journal of the European Communities 2000;43(L18):1-5.
3. Miles KA, Packer C, Stevens A. Quantifying emerging drugs for rare conditions. QJM 2007;100:291-5. [Abstract/FREE Full text] (Pubitemid 47062906)
4. Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 2007;23:36-42. [Medline] [Web of Science] (Pubitemid 46648298)
5. National Institute for Health and Clinical Excellence. Citizens council. Report on ultra-orphan drugs. 2004. www.nice.org.uk/niceMedia/pdf/Citizens- Council-Ultraorphan.pdf.
6. Desser AS, Gyrd-Hansen D, Olsen JA, Grepperud S, Kristiansen IS. Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. BMJ 2010;341:c4715. [Abstract/FREE Full text]
7. Department of Health. Guidelines for the management of paediatric Gaucher disease. 2005. www.dh.gov.uk/prod-consum-dh/groups/dh-digitalassets/@dh/@en/ documents/digitalasset/dh-4118411.pdf.
8. GenEngNews. Big pharma steps into orphan drugs market. 2010. www.genengnews.com/gen-articles/big-pharma-steps-into-orphan-drug-market/3329.
9. Grace C. Developing new technologies to address neglected diseases: the role of product development partnerships and advanced marked commitments. DFID Health Resource Centre, 2006. www.dfidhealthrc.org/publications/PDPs-and-AMCs- presentation-06.pdf.
10. Thomas H. Fighting for herceptin. BBC Stoke and Staffordshire, 2005. www.bbc.co.uk/stoke/content/articles/2005/09/26/herceptin-intro-2005-feature. shtml.
11. BBC News. Woman denied "sight-save drugs". 2008. http://news.bbc.co.uk/1/hi/england/london/7288326.stm.
12. Osley R. Condemned to die...the patients being refused a life-saving cancer treatment drug. Camden New Journal, 2007. www.thecnj.com/camden/2007/ 101807/news101807-09.html.