Health technology assessment of drugs for rare diseases: Insights, trends, and reasons for negative recommendations from the CADTH common drug review

Orphanet Journal of Rare Diseases

Volumn 11, Issue 1, 2016, Pages

  Janoudi, Ghayath ^a   Amegatse, William ^a   McIntosh, Brendan ^a   Sehgal, Chander ^a   Richter, Trevor ^a  

^a CANADIAN AGENCY FOR DRUGS AND TECHNOLOGIES IN HEALTH   (Canada)

Author keywords

Canada; Orphan drugs; Rare diseases; Technology assessment, health

Indexed keywords

ABATACEPT; AGALSIDASE ALFA; AGALSIDASE BETA; AMBRISENTAN; ANGIOPEPTIN; AZTREONAM LYSINE; BOTULINUM TOXIN A; CANAKINUMAB; ECULIZUMAB; ELTROMBOPAG; HUMAN GROWTH HORMONE; IDURONATE 2 SULFATASE; LARONIDASE; MIGLUSTAT; ORPHAN DRUG; OXYBATE SODIUM; PEGVISOMANT; PLERIXAFOR; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RITUXIMAB; ROMIPLOSTIM; RUFINAMIDE; SAPROPTERIN; SITAXSENTAN; SUNITINIB; TADALAFIL; TOCILIZUMAB; TREPROSTINIL; UNINDEXED DRUG; VELAGLUCERASE ALFA;

ACROMEGALY; ANGIONEUROTIC EDEMA; BLEPHAROSPASM; CERVICAL DYSTONIA; CINCA SYNDROME; CLINICAL EFFECTIVENESS; COST EFFECTIVENESS ANALYSIS; CUSHING DISEASE; CYSTIC FIBROSIS; DRUG COST; FABRY DISEASE; FAMILIAL HYPERCHOLESTEROLEMIA; FIBROSING ALVEOLITIS; GASTROINTESTINAL STROMAL TUMOR; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; GROWTH HORMONE DEFICIENCY; HEALTH CARE QUALITY; HEALTH TECHNOLOGY ASSESSMENT; HEMOLYTIC UREMIC SYNDROME; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; IDIOPATHIC THROMBOCYTOPENIC PURPURA; JUVENILE RHEUMATOID ARTHRITIS; LENNOX GASTAUT SYNDROME; MACULAR DEGENERATION; MAROTEAUX LAMY SYNDROME; MORQUIO SYNDROME; MULTIPLE MYELOMA; NARCOLEPSY; NONHODGKIN LYMPHOMA; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PHENYLKETONURIA; PULMONARY HYPERTENSION; QUALITY ADJUSTED LIFE YEAR; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; REIMBURSEMENT; REVIEW; SEVERE MYOCLONIC EPILEPSY IN INFANCY; STATISTICS; TUBEROUS SCLEROSIS; TURNER SYNDROME; WEGENER GRANULOMATOSIS; BIOMEDICAL TECHNOLOGY ASSESSMENT; CANADA; COST BENEFIT ANALYSIS; ECONOMICS; PROCEDURES;

CANADA; COST-BENEFIT ANALYSIS; HUMANS; RARE DISEASES; TECHNOLOGY ASSESSMENT, BIOMEDICAL;

EID: 85000420286     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-016-0539-3     Document Type: Review

References (12)

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