Access to orphan drugs despite poor quality of clinical evidence

British Journal of Clinical Pharmacology

Volumn 71, Issue 4, 2011, Pages 488-496

  Dupont, Alain G ^a,b   Van Wilder, Philippe B ^b  

^a Commission for Reimbursement of Medicines ^*  (Belgium)

^b VRIJE UNIVERSITEIT BRUSSEL   (Belgium)

Author keywords

Clinical evidence; Orphan drug; Orphan medicinal product; Rare diseases

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ANAGRELIDE; ARSENIC TRIOXIDE; BORTEZOMIB; BOSENTAN; BUSULFAN; CARGLUMIC ACID; DASATINIB; DEXAMETHASONE; IBUPROFEN; IDURONATE 2 SULFATASE; INDOMETACIN; LARONIDASE; LENALIDOMIDE; MIGLUSTAT; MITOTANE; NELARABINE; NITISINONE; ORPHAN DRUG; PEGVISOMANT; PENICILLAMINE; PROSTACYCLIN; RAZOXANE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; SILDENAFIL; SORAFENIB; SUNITINIB; UNINDEXED DRUG; ZINC ACETATE;

ACROMEGALY; ACUTE LYMPHOBLASTIC LEUKEMIA; ADRENAL CORTEX CARCINOMA; ADVANCED CANCER; ARTICLE; BELGIUM; CHRONIC MYELOID LEUKEMIA; DISEASE SEVERITY; DRUG COST; DRUG EFFICACY; DRUG EXPOSURE; DRUG LEGISLATION; ENZYME REPLACEMENT; EVIDENCE BASED MEDICINE; FABRY DISEASE; GASTROINTESTINAL STROMAL TUMOR; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HEALTH CARE QUALITY; HUMAN; HYPERAMMONEMIA; KIDNEY CARCINOMA; LYMPHOMA; MUCOPOLYSACCHARIDOSIS; MULTIPLE MYELOMA; NATIONAL HEALTH INSURANCE; PRIORITY JOURNAL; PROMYELOCYTIC LEUKEMIA; PULMONARY HYPERTENSION; REIMBURSEMENT; THROMBOCYTOSIS; TYROSINEMIA; WILSON DISEASE;

BELGIUM; DRUG INDUSTRY; EUROPEAN UNION; HEALTH POLICY; HEALTH SERVICES ACCESSIBILITY; ORPHAN DRUG PRODUCTION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REIMBURSEMENT MECHANISMS;

EID: 79952581846     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/j.1365-2125.2010.03877.x     Document Type: Article

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