Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group

Value in Health

Volumn 21, Issue 5, 2018, Pages 493-500

  Nestler Parr, Sandra ^a   Korchagina, Daria ^b   Toumi, Mondher ^c   Pashos, Chris L ^d   Blanchette, Christopher ^e   Molsen, Elizabeth ^f   Morel, Thomas ^g   Simoens, Steven ^g   Kaló, Zoltán ^h,i   Gatermann, Ruediger ^j   Redekop, William ^k  

^a Rare Access Ltd   (United Kingdom)

^b UNIVERSITÉ PARIS SACLAY   (France)

^c AIX MARSEILLE UNIVERSITY   (France)

^d TAKEDA PHARMACEUTICAL COMPANY LIMITED   (Japan)

^e UNIVERSITY OF NORTH CAROLINA AT CHARLOTTE   (United States)

^f ISPOR   (United States)

^g UNIVERSITY OF LEUVEN   (Belgium)

^h EÖTVÖS LORÁND UNIVERSITY   (Hungary)

ⁱ Syreon Research Institute   (Hungary)

^j CSL BEHRING GMBH   (Germany)

^k ERASMUS UNIVERSITY ROTTERDAM   (Netherlands)

more..

Author keywords

cost effectiveness; health policy; health technology assessment; orphan designation; rare diseases

Indexed keywords

ALPHA 1 ANTITRYPSIN; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR;

ARTICLE; BIOMEDICAL TECHNOLOGY ASSESSMENT; CLINICAL EFFECTIVENESS; CLINICAL EVALUATION; CLINICAL RESEARCH; CLINICAL STUDY; CLINICAL TRIAL (TOPIC); DIAGNOSTIC ERROR; DIAGNOSTIC TEST; HEALTH CARE; HEALTH CARE ACCESS; HUMAN; LEGAL ASPECT; MEDICAL ETHICS; MEDICAL EXPERT; MEDICAL HISTORY; OUTCOME ASSESSMENT; PREVALENCE; PRIORITY JOURNAL; PROGNOSIS; QUALITATIVE ANALYSIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; SELECTION BIAS; THERAPY EFFECT; TREATMENT INDICATION; CONSENSUS DEVELOPMENT; COST BENEFIT ANALYSIS; HEALTH CARE POLICY; MEDICAL RESEARCH; PROCEDURES; STANDARDS;

BIOMEDICAL RESEARCH; CONSENSUS DEVELOPMENT CONFERENCES AS TOPIC; COST-BENEFIT ANALYSIS; HEALTH POLICY; HUMANS; RARE DISEASES; TECHNOLOGY ASSESSMENT, BIOMEDICAL;

EID: 85045187145     PISSN: 10983015     EISSN: 15244733     Source Type: Journal    
DOI: 10.1016/j.jval.2018.03.004     Document Type: Article

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