Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders

Journal of Comparative Effectiveness Research

Volumn 3, Issue 4, 2014, Pages 399-422

  Schlander, Michael ^a,b   Garattini, Silvio ^c   Holm, Søren ^d   Kolominsky Rabas, Peter ^e   Nord, Erik ^f   Persson, Ulf ^g   Postma, Maarten ^h   Richardson, Jeff ⁱ   Simoens, Steven ^j   De Solà Morales, Oriol ^k   Tolley, Keith ^l   Toumi, Mondher ^m  

^a MEDICAL FACULTY MANNHEIM HEIDELBERG UNIVERSITY   (Germany)

^b Institute for Innovation and Valuation in Health Care (InnoValHC)   (Germany)

^c MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH   (Italy)

^d UNIVERSITY OF MANCHESTER   (United Kingdom)

^e UNIVERSITY OF ERLANGEN NUREMBERG   (Germany)

^f UNIVERSITY OF OSLO   (Norway)

^g SWEDISH INSTITUTE FOR HEALTH ECONOMICS   (Sweden)

^h UNIVERSITY OF GRONINGEN   (Netherlands)

ⁱ MONASH UNIVERSITY   (Australia)

^j UNIVERSITY OF LEUVEN   (Belgium)

^k INSTITUT D INVESTIGACIÓ SANITÀRIA PERE VIRGILI IISPV   (Spain)

^l Tolley Health Economics Ltd   (United Kingdom)

^m UNIVERSITÉ LYON 1   (France)

more..

Author keywords

consensus statement; cost effectiveness; economic evaluation; fairness; health technology assessment; medical ethics; medical need; orphan medicines; quality adjusted life year; social preferences

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALGLUCERASE; ARSENIC TRIOXIDE; BOSENTAN; BUSULFAN; CARGLUMIC ACID; GALSULFASE; IMATINIB; LARONIDASE; MIGLUSTAT; PEGVISOMANT; RECOMBINANT BLOOD CLOTTING FACTOR 9;

CLINICAL EFFECTIVENESS; COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG COST; ECONOMIC EVALUATION; EVIDENCE BASED MEDICINE; FABRY DISEASE; GAUCHER DISEASE; HEALTH CARE COST; HEALTH STATUS; HEMOPHILIA B; HUMAN; HURLER SYNDROME; MEDICAL ETHICS; QUALITY ADJUSTED LIFE YEAR; RARE DISEASE; REIMBURSEMENT; RESOURCE ALLOCATION; REVIEW; BIOMEDICAL TECHNOLOGY ASSESSMENT; CONSENSUS; COST BENEFIT ANALYSIS; ECONOMICS; OUTCOME ASSESSMENT; PROCEDURES; RARE DISEASES; STATISTICAL MODEL;

CONSENSUS; COST-BENEFIT ANALYSIS; EVIDENCE-BASED MEDICINE; HEALTH CARE COSTS; HUMANS; MODELS, ECONOMIC; OUTCOME ASSESSMENT (HEALTH CARE); QUALITY-ADJUSTED LIFE YEARS; RARE DISEASES; TECHNOLOGY ASSESSMENT, BIOMEDICAL;

EID: 84907859265     PISSN: 20426305     EISSN: 20426313     Source Type: Journal    
DOI: 10.2217/cer.14.34     Document Type: Review

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