A framework for applying unfamiliar trial designs in studies of rare diseases

Journal of Clinical Epidemiology

Volumn 64, Issue 10, 2011, Pages 1085-1094

  Gupta, Samir ^a,b,c   Faughnan, Marie E ^a,b,c   Tomlinson, George A ^a,d,e   Bayoumi, Ahmed M ^a,c,e,f  

^a UNIVERSITY OF TORONTO   (Canada)

^b ST MICHAEL'S HOSPITAL   (Canada)

^c ST MICHAEL'S HOSPITAL   (Canada)

^d UNIVERSITY HEALTH NETWORK   (Canada)

^e UNIVERSITY OF TORONTO   (Canada)

^f ST MICHAEL'S HOSPITAL   (Canada)

Author keywords

Bayes theorem; Crossover studies; Methods; Orphan drug production; Rare diseases

Indexed keywords

AMITRIPTYLINE; DOCETAXEL; IMMUNOGLOBULIN; ITRACONAZOLE; METHOTREXATE; PLACEBO; RECOMBINANT DEOXYRIBONUCLEASE; RECOMBINANT ENZYME; UNCLASSIFIED DRUG; VANDETANIB;

ALLOPATHY; BAYES THEOREM; CHRONIC GRANULOMATOUS DISEASE; CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY; CLINICAL RESEARCH; CLINICAL TRIAL (TOPIC); CONCEPTUAL FRAMEWORK; CROSSOVER PROCEDURE; CYSTIC FIBROSIS; EXTRACORPOREAL OXYGENATION; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; LUNG NON SMALL CELL CANCER; NEWBORN RESPIRATORY FAILURE; PERSISTENT PULMONARY HYPERTENSION; PILOT STUDY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; RESPIRATORY FAILURE; REVIEW; SENSITIVITY ANALYSIS; SEQUENTIAL ANALYSIS; SYSTEMATIC REVIEW; SYSTEMIC SCLEROSIS; TREATMENT OUTCOME;

BAYES THEOREM; CLINICAL TRIALS AS TOPIC; HUMANS; RARE DISEASES; RESEARCH DESIGN;

EID: 80052034189     PISSN: 08954356     EISSN: 18785921     Source Type: Journal    
DOI: 10.1016/j.jclinepi.2010.12.019     Document Type: Review

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