Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov

Orphanet Journal of Rare Diseases

Volumn 12, Issue 1, 2017, Pages

  Hee, Siew Wan ^a   Willis, Adrian ^a   Tudur Smith, Catrin ^b   Day, Simon ^c   Miller, Frank ^d   Madan, Jason ^a   Posch, Martin ^e   Zohar, Sarah ^f   Stallard, Nigel ^a  

^a UNIVERSITY OF WARWICK   (United Kingdom)

^b Belfast and North West Hub for Trials Methodology Research   (United Kingdom)

^c Clinical Trials Consulting and Training Limited   (United Kingdom)

^d STOCKHOLM UNIVERSITY   (Sweden)

^e MEDICAL UNIVERSITY OF VIENNA   (Austria)

^f INSERM   (France)

Author keywords

Aggregate analysis of clinialtrials.gov; Orphadata; Orphan disease; Orphanet; Prevalence; Rare disease; Sample size

Indexed keywords

ALLERGIC BRONCHOPULMONARY ASPERGILLOSIS; AMYLOIDOSIS; ANALYSIS OF VARIANCE; ARACHNOIDITIS; ARTICLE; ASPERGILLOSIS; CHILD; CLINICAL TRIAL (TOPIC); CONJUNCTIVITIS; CONTROLLED STUDY; DATA BASE; ERDHEIM CHESTER DISEASE; EUROPEAN UNION; FEMALE; FIBROUS DYSPLASIA; GERM CELL TUMOR; HEPARIN INDUCED THROMBOCYTOPENIA; HUMAN; INTRAHEPATIC CHOLESTASIS; LIGNEOUS CONJUNCTIVITIS; LOIASIS; LUNG ALVEOLUS PROTEINOSIS; MAJOR CLINICAL STUDY; MALE; MUCOCUTANEOUS LYMPH NODE SYNDROME; NEPHROTIC SYNDROME; ORPHADATA DATABASE; POEMS SYNDROME; PRECOCIOUS PUBERTY; PREVALENCE; RARE DISEASE; SAMPLE SIZE; SENSITIVITY ANALYSIS; SOLITARY FIBROUS TUMOR; STATISTICAL ANALYSIS; SYNOVIAL SARCOMA; UNITED STATES; ZYGOMYCOSIS; INTERNET;

CLINICAL TRIALS AS TOPIC; HUMANS; INTERNET; PREVALENCE; RARE DISEASES; SAMPLE SIZE;

EID: 85014383015     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-017-0597-1     Document Type: Article

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