The problem of orphan drugs

BMJ (Online)

Volumn 341, Issue 7782, 2010, Pages 1059-

  Ferner, Robin E ^a   Hughes, Dyfrig A ^b  

^a CITY HOSPITAL   (United Kingdom)

^b BANGOR UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

3,4 DIAMINOPYRIDINE; AMIFAMPRIDINE; ATOSIBAN; BEVACIZUMAB; HYDROXYUREA; IMATINIB; RANIBIZUMAB; UNCLASSIFIED DRUG;

DRUG APPROVAL; DRUG COST; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG MANUFACTURE; DRUG MARKETING; EDITORIAL; PRIORITY JOURNAL; RARE DISEASE;

DRUG APPROVAL; DRUG COSTS; EUROPEAN UNION; HUMANS; LEGISLATION, DRUG; ORPHAN DRUG PRODUCTION;

EID: 78549264507     PISSN: None     EISSN: 17561833     Source Type: Journal    
DOI: 10.1136/bmj.c6456     Document Type: Editorial

References (12)

1. Waxman HA. The history and development of the Orphan Drugs Act. In: Scheinberg IH, Walshe JM, eds. Orphan drugs and orphan diseases. Manchester University Press/Fulbright Commission. 1986.
2. Regulation (EC) No 141/2000 of The European Parliament and of the council of 16 December 1999 on orphan medicinal products. http://eur-lex.europa.eu/ LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF.
3. European Medicines Agency. European public assessment report. Firdapse. 2009. www.ema.europa.eu/docs/en-GB/document-library/EPAR---Public-assessment- report/human/001032/WC500069918.pdf.
4. Braun MM, Farag-El-Massah S, Xu K, Coté TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov 2020; online 7 June; doi:10.1038/nrd3160.
5. General Medical Council. Supplementary guidance: good practice in prescribing medicines. 2008. www.gmc-uk.org/static/documents/content/Good- Practice-in-Prescribing-Medicines-0911.pdf.
6. Medicines and Healthcare Products Regulatory Agency. Guidance note 14. The supply of unlicensed relevant medicinal products for individual patients. MRHA, 2008.
7. Royal Pharmaceutical Society. Dealing with specials. Pharmacy Professional June 2010; 27-32. www.rpharms.com/support-pdfs/ppjune2010-specials. pdf.
8. Folk JC, Stone EM. Clinical therapeutics: ranibizumab therapy for neovascular age-related macular degeneration. N Engl J Med 2010;363:1648-55.
9. McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the National Health Service: should we value rarity? BMJ 2005;331:1016-9.
10. 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.
11. Hughes D. Rationing of drugs for rare diseases. Pharmacoeconomics 2006;24:315-6.
12. Ferner RE, Hughes DA, Aronson JK. NICE and new: appraising innovation. BMJ 2010;340:b5493.