Worldwide collaboration for orphan drug designation

Nature Reviews Drug Discovery

Volumn 15, Issue 6, 2016, Pages 440-

  Mariz, Segundo ^a   Reese, James H ^b   Westermark, Kerstin ^a,c   Greene, Lesley ^a,d   Goto, Takahiro ^e   Hoshino, Tatsuro ^e   Llinares Garcia, Jordi ^b   Sepodes, Bruno ^a,f  

^a EUROPEAN MEDICINES AGENCY   (United Kingdom)

^b CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^c MEDICAL PRODUCTS AGENCY   (Sweden)

^d Europ Org for Rare Dis (EURORDIS)   (France)

^e Japan Ministry of Health Labour and Welfare   (Japan)

^f RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

Author keywords

[No Author keywords available]

Indexed keywords

ORPHAN DRUG;

DRUG INDICATION; DRUG LEGISLATION; DRUG MARKETING; DRUG RESEARCH; DRUG SURVEILLANCE PROGRAM; EUROPEAN UNION; FOOD AND DRUG ADMINISTRATION; HUMAN; JAPAN; LETTER; MEDICAL SOCIETY; PREVALENCE; PRIORITY JOURNAL; PUBLIC-PRIVATE PARTNERSHIP; RARE DISEASE; UNITED STATES; WORKSHOP; COOPERATION; DRUG MANUFACTURE; GLOBAL HEALTH; PROCEDURES; RARE DISEASES;

COOPERATIVE BEHAVIOR; GLOBAL HEALTH; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84973352529     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd.2016.80     Document Type: Letter

References (7)

1. Braun, M. M., et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat. Rev. Drug Discov. 9, 519-522 (2010
2. Westermark, K., et al. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat. Rev. Drug Discov. 10, 341-347 (2011
3. Song, P., Gao, J., Inagaki, Y., Kokudo, N., & Tang, W. Rare diseases, orphan drugs, and their regulation in Asia: current status and future perspectives. Intractable Rare Dis. Res. 1, 3-9 (2012
4. [No authors listed]. Orphan Drug Act. H. R. 5238, Public Law No. 97-414, 97th Congress. National Institutes of Health https://history.nih.gov/research/downloads/PL97-414.pdf (1983
5. European Commission. Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another. European Commission: Health and Consumers Directorate-General http://ec. europa.eu/health/files/orphanmp/2014-03-guideline- rev4-final.pdf (2014
6. Tsigkos, S., et al. Establishing medical plausibility in the context of orphan medicines designation in the European Union. Orphanet J. Rare Dis. 9, 175-178 (2014
7. European Commission. Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products. European Commission: Health and Food Safety http://ec.europa.eu/health/files/orphanmp/doc/orphan-inv-report-20160126.pdf (2015