Availability of and access to orphan drugs: An international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia

PharmacoEconomics

Volumn 29, Issue 1, 2011, Pages 63-82

  Blankart, Carl Rudolf ^a,b   Stargardt, Tom ^a,b   Schreyögg, Jonas ^a,b  

^a INSTITUTE OF EPIDEMIOLOGY   (Germany)

^b UNIVERSITY OF HAMBURG   (Germany)

Author keywords

Chronic myeloid leukaemia, treatment; Cost effectiveness; Decision making; Fabrys disease, treatment; Formularies; Hereditary angioedema, treatment; Orphan drugs; Pricing; Pulmonary hypertension, treatment; Regulatory process; Reimbursement.

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; AMBRISENTAN; BOSENTAN; CETOR; COMPLEMENT COMPONENT C1S INHIBITOR; DASATINIB; ECALLANTIDE; FRESH FROZEN PLASMA; ICATIBANT; ILOPROST; IMATINIB; KALBITOR; NILOTINIB; PLACEBO; SILDENAFIL; SITAXSENTAN; TYVASO; UNCLASSIFIED DRUG; UNIPROST;

ALLOGENEIC STEM CELL TRANSPLANTATION; ANGIONEUROTIC EDEMA; ARTICLE; AUSTRALIA; CANADA; CHRONIC MYELOID LEUKEMIA; CLINICAL TRIAL; DRUG INDICATION; DRUG MARKETING; FABRY DISEASE; FRANCE; GERMANY; HEALTH INSURANCE; HUMAN; HUNGARY; NETHERLANDS; PATIENT RIGHT; POLAND; PREVALENCE; PRIORITY JOURNAL; PULMONARY HYPERTENSION; SLOVAKIA; SWITZERLAND; UNITED KINGDOM; UNITED STATES;

ANGIOEDEMAS, HEREDITARY; FABRY DISEASE; HEALTH SERVICES ACCESSIBILITY; HUMANS; HYPERTENSION, PULMONARY; INTERNATIONALITY; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; ORPHAN DRUG PRODUCTION;

EID: 78650240029     PISSN: 11707690     EISSN: 11792027     Source Type: Journal    
DOI: 10.2165/11539190-000000000-00000     Document Type: Article

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