Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

European Journal of Clinical Pharmacology

Volumn 69, Issue 4, 2013, Pages 1009-1024

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

^a MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH   (Italy)

^b Local Health Unit of Verona   (Italy)

Author keywords

Clinical efficacy; Clinical trials; Development; Drug regulation; Orphan drug; Outcome measures; Pharmacological research; Placebo; Preclinical studies; Rare disease

Indexed keywords

3,4 DIAMINOPYRIDINE; AGALSIDASE ALFA; AGALSIDASE BETA; AMBRISENTAN; AMINOLEVULINIC ACID; ANAGRELIDE; ARSENIC TRIOXIDE; AZACITIDINE; AZTREONAM LYSINE; BOSENTAN; BUSULFAN; CAFFEINE CITRATE; CANAKINUMAB; CARGLUMIC ACID; CELECOXIB; CLADRIBINE; CLOFARABINE; DASATINIB; DEFERASIROX; ECULIZUMAB; HYDROCARBAMIDE; MITOTANE; ORPHAN DRUG; PEGVISOMANT; PHOTOFRIN; PLACEBO; RAZOXANE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; UNCLASSIFIED DRUG; UNINDEXED DRUG; UREA DERIVATIVE;

ACUTE LYMPHOBLASTIC LEUKEMIA; ARTICLE; CHRONIC MYELOID LEUKEMIA; CINCA SYNDROME; CLINICAL RESEARCH; COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG COST; DRUG EFFICACY; DRUG INFORMATION; DRUG LEGISLATION; DRUG WITHDRAWAL; EATON LAMBERT SYNDROME; EUROPEAN UNION; FABRY DISEASE; FAMILIAL COLON POLYPOSIS; FOLLOW UP; FUNDING; GLYCOGEN STORAGE DISEASE TYPE 2; HAIRY CELL LEUKEMIA; HEALTH CARE ORGANIZATION; HUMAN; HYPERAMMONEMIA; IRON OVERLOAD; METHODOLOGY; MYELODYSPLASTIC SYNDROME; NEWBORN APNEA; NONHUMAN; ORPHAN DISEASE; OUTCOME ASSESSMENT; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PRIORITY JOURNAL; PROMYELOCYTIC LEUKEMIA; PSEUDOMONAS INFECTION; PULMONARY HYPERTENSION; RARE DISEASE; THROMBOCYTHEMIA;

ANIMALS; CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; DRUG EVALUATION, PRECLINICAL; DRUGS, INVESTIGATIONAL; EUROPEAN UNION; EVIDENCE-BASED MEDICINE; HEALTH POLICY; HUMANS; LEGISLATION, DRUG; MARKETING; ORPHAN DRUG PRODUCTION; OUTCOME ASSESSMENT (HEALTH CARE); RARE DISEASES;

EID: 84876743099     PISSN: 00316970     EISSN: 14321041     Source Type: Journal    
DOI: 10.1007/s00228-012-1423-2     Document Type: Article

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