Alternative designs for clinical trials in rare diseases

American Journal of Medical Genetics, Part C: Seminars in Medical Genetics

Volumn 172, Issue 4, 2016, Pages 313-331

  Abrahamyan, Lusine ^a   Feldman, Brian M ^a,b   Tomlinson, George ^a,c   Faughnan, Marie E ^d   Johnson, Sindhu R ^b   Diamond, Ivan R ^e   Gupta, Samir ^b,f  

^a UNIVERSITY OF TORONTO   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c MOUNT SINAI HOSPITAL   (Canada)

^d ST MICHAEL'S HOSPITAL   (Canada)

^e NONE

^f ST MICHAEL'S HOSPITAL   (Canada)

Author keywords

adaptive trial design; Bayes theorem; clinical trials; cross over studies; rare diseases

Indexed keywords

BIOLOGICAL MARKER;

ARTICLE; BAYES THEOREM; CLINICAL TRIAL (TOPIC); FACTORIAL DESIGN; GENETIC DISORDER; HUMAN; PILOT STUDY; PRIORITY JOURNAL; PROBABILITY; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; SEQUENTIAL ANALYSIS; STUDY DESIGN; TREATMENT WITHDRAWAL; EVIDENCE BASED MEDICINE; METHODOLOGY; STANDARDS; TRENDS;

EVIDENCE-BASED MEDICINE; HUMANS; RARE DISEASES; RESEARCH DESIGN;

EID: 84997096197     PISSN: 15524868     EISSN: 15524876     Source Type: Journal    
DOI: 10.1002/ajmg.c.31533     Document Type: Article

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