The challenges of orphan drugs and orphan diseases: Real and imagined

Clinical Pharmacology and Therapeutics

Volumn 92, Issue 2, 2012, Pages 151-153

  Hudson, I ^a   Breckenridge, A ^a  

^a MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ORPHAN DRUG;

DRUG APPROVAL; DRUG COST; DRUG EFFICACY; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG SAFETY; ENZYME REPLACEMENT; HUMAN; LICENSING; ORPHAN DISEASE; PRIORITY JOURNAL; RARE DISEASE; REVIEW;

EUROPE; HUMANS; LEGISLATION, DRUG; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES;

EID: 84864132879     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2012.72     Document Type: Review

References (6)

1. Regulation (EC) 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicine Products 〈http://ec.europa.eu/health/ files/eudralex/vol-1/reg-2000-141/reg-2000-141-en.pdf〉 (1999).
2. Garattini, S. Time to revisit the orphan drug law. Eur. J. Clin. Pharmacol. 68, 113 (2012).
3. European Commission. Registry of designated orphan medical products by number 〈http://ec.europa.eu/health/documents/communityregister/html/ orphreg.htm〉 (2000-2012).
4. European Medicines Agency. Medicines for rare diseases 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/special-topics/general/ general-content-000034.jsp&murl=menus/special-topics/special-topics. jsp&mid=WC0b01 ac058002d4eb&jsenabled=true〉.
5. European Commission. Commission Implementing Decision of XXX refusing the designation of "Tecovirimat" as an orphan medicinal product under Regulation (EC) No 141/2000 of the European Parliament and of the Council section pertaining to the indication of monkeypox infection 〈https://docs. google. com/viewer?a=v&q=cache:YUlQ4HSkBooJ :www.europarl.europa.eu/RegData/ docs-autres-institutions/commission-europeenne/comitologie/ros/2011/D012933-02/ COMAC-DR(2011)D012933-02-EN.doc+tecovirim at+monkeypox+orphan&hl=en&gl= uk&pid= bl&srcid=ADGEESiv-7klSEc3OhI9QEOlA8D8 ajM2t1slqva- XU6fEJnWvSvQJKNhATH71tp-MXfEEKVYdFdcJEo-2b3uxkNB1Pll1M-GGR 3bVTJS1DwPr0Ic8HwzPjnza1daFRJ8 DJP8EkvG7zr&sig= AHIEtbQD90upJUF3u6jVz3g5LqB47EkRA& safe=images〉 (2010).
6. European Commission. Commission Implementing Decision of XXX refusing the designation of "Tecovirimat" as an orphan medicinal product under Regulation (EC) No 141/2000 of the European Parliament and of the Council section pertaining to the indication of variola infection 〈https://docs. google.com/viewer?a=v&q=cache:-gSgcl-5bOQJ:www. europarl.europa.eu/RegData/ docs-autres-institutions/commission-europeenne/comitologie/ros/2011/D012932-02/ COM-AC-DR(2011)D012932-02-EN.doc+tecovirimat+vari ola+orphan&hl=en&gl= uk&pid=bl&srcid=ADG EESjGQJf8SfqpfjzzZ-JX5dKIdn7XRDMpVXYA GpysE9fwSHvTruIru8R1vcNreHdX2yuBXQt17Fm cjypTQZ4aMmGeC7m6BNYIk65BLOAlw6thJYD B8voEAyjeBMDblDmW-RVNC7IG&sig=AHIEtb SgjmNf8kUdPvvvST2YIq2pBcLgGg&safe= imag es〉 (2010).