Recommendations for the design of small population clinical trials

Orphanet Journal of Rare Diseases

Volumn 13, Issue 1, 2018, Pages

  Day, Simon ^a   Jonker, Anneliene Hechtelt ^b   Lau, Lilian Pek Lian ^b   Hilgers, Ralf Dieter ^c   Irony, Ilan ^d   Larsson, Kristina ^e   Roes, Kit Cb ^f   Stallard, Nigel ^g  

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

^b INSERM   (France)

^c RWTH AACHEN UNIVERSITY   (Germany)

^d CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^e EUROPEAN MEDICINES AGENCY   (United Kingdom)

^f UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^g UNIVERSITY OF WARWICK   (United Kingdom)

Author keywords

Clinical trials; IRDiRC; Patient engagement; Rare diseases; Recommendations; Small population studies; Small populations; Statistical methods

Indexed keywords

ANALYTIC METHOD; ARTICLE; CLINICAL PROTOCOL; CLINICAL TRIAL (TOPIC); DECISION MAKING; METHODOLOGY; PATIENT PARTICIPATION; PRACTICE GUIDELINE; RARE DISEASE; SAFETY; STUDY DESIGN; HUMAN; MEDICAL RESEARCH; PROCEDURES;

BIOMEDICAL RESEARCH; CLINICAL TRIALS AS TOPIC; HUMANS; RARE DISEASES; RESEARCH DESIGN;

EID: 85056121395     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-018-0931-2     Document Type: Article

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