Orphan drug development is progressing too slowly

British Journal of Clinical Pharmacology

Volumn 61, Issue 3, 2006, Pages 355-360

  Joppi, Roberta ^a,b   Bertele, Vittorio ^a   Garattini, Silvio ^a  

^a MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH   (Italy)

^b Local Health Unit   (Italy)

Author keywords

Development; Orphan drugs; Rare diseases

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALEMTUZUMAB; ALITRETINOIN; ANAGRELIDE; ARSENIC TRIOXIDE; ARYLBUTYRIC ACID DERIVATIVE; BLOOD CLOTTING FACTOR 7A; BLOOD CLOTTING FACTOR 9; BOSENTAN; BUSILVEX; BUSULFAN; CARGLUMIC ACID; CELECOXIB; CLADRIBINE; DEFERIPRONE; IBUPROFEN; ILOPROST; IMATINIB; IMIGLUCERASE; LARONIDASE; LITAK; MERCAPTAMINE; MIGLUSTAT; MITOTANE; ORPHAN DRUG; PEGVISOMANT; PHOTOFRIN; PROTEIN C; RILUZOLE; UNINDEXED DRUG; WILZIN; ZINC ACETATE;

ACROMEGALY; ADENOMATOUS POLYP; ADRENAL CORTEX CARCINOMA; ARTICLE; CARCINOGENICITY; CHRONIC MYELOID LEUKEMIA; CLINICAL TRIAL; DRUG APPROVAL; DRUG DESIGN; DRUG INDUSTRY; DRUG INFORMATION; DRUG MANUFACTURE; DRUG USE; DYSPLASIA; FABRY DISEASE; GAUCHER DISEASE; GENOTOXICITY; HAIRY CELL LEUKEMIA; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INTERNET; LICENSING; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PRIORITY JOURNAL; PROMYELOCYTIC LEUKEMIA; PULMONARY HYPERTENSION; THROMBOCYTHEMIA; WILSON DISEASE;

ANIMALS; DOCUMENTATION; DRUG INDUSTRY; EUROPEAN UNION; HEALTH SYSTEMS AGENCIES; PHARMACEUTICAL PREPARATIONS; RESEARCH; RETROSPECTIVE STUDIES;

EID: 33644905861     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/j.1365-2125.2006.02579.x     Document Type: Article

References (4)

1. Regulation (EC) 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products Official Journal L 18 22/1/2000 1 5
2. Federal Drug Administration. Orphan drug act Available on the URL (last accessed 17 March 2005).
3. Committee for Proprietary Medicinal Products Note for Guidance on Repeated Dose Toxicity July 2000 Available on the URL (last accessed 17 March 2005).
4. Stolk P Willemen M Leufkens HG Rare essentials? Drugs for rare diseases on the essential medicines list Utrecht (NL) WHO Expert Committee on the Selection and Use of Essential Medicines, UISP 2005