From the analyst's couch: Rare diseases and orphan drugs

Nature Reviews Drug Discovery

Volumn 11, Issue 4, 2012, Pages 267-268

  Melnikova, Irena ^a  

^a Strategy and External Innovation at Sanofi   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; BOTULINUM TOXIN A; IMATINIB; IMIGLUCERASE; INFLIXIMAB; ORPHAN DRUG; RECOMBINANT ERYTHROPOIETIN; RITUXIMAB; SILDENAFIL; VELAGLUCERASE ALFA;

DRUG APPROVAL; DRUG INDICATION; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG PROGRAM; DRUG RESEARCH; ENZYME REPLACEMENT; ERECTILE DYSFUNCTION; FABRY DISEASE; GAUCHER DISEASE; GENE EXPRESSION REGULATION; GENE MUTATION; GENE THERAPY; GOVERNMENT REGULATION; HUMAN; INTEGRATED HEALTH CARE SYSTEM; PRIORITY JOURNAL; PULMONARY HYPERTENSION; RARE DISEASE; SHORT SURVEY; DRUG DEVELOPMENT; DRUG MANUFACTURE; NOTE;

DRUG APPROVAL; DRUG DISCOVERY; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84859326107     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd3654     Document Type: Short Survey

References (4)

1. Field, M. J & Boat, T. F. (eds). Rare Diseases and Orphan Products: Accelerating Research and Development (The National Academies Press, Washington DC, 2011).
2. Tambuyzer, E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nature Rev. Drug Discov. 9, 921-929 (2010).
3. Philippidis, A. Orphan drugs, big pharma. Hum. Gene Ther. 22, 1037-1040 (2011).
4. Hyde, R. & Dobrovolny, D. Orphan drug pricing and payer management in the United States: are we approaching the tipping point? American Health and Drug Benefits 3, 15-23 (2010).