The art and science of choosing efficacy endpoints for rare disease clinical trials

American Journal of Medical Genetics, Part A

Volumn 176, Issue 4, 2018, Pages 759-772

  Cox, Gerald F ^a,b,c  

^a EDITAS MEDICINE   (United States)

^b BOSTON CHILDREN S HOSPITAL   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

Author keywords

biomarker; efficacy; endpoint; minimal important difference; proof of concept; rare disease; standardized response mean

Indexed keywords

BIOLOGICAL MARKER;

BIOASSAY; DISEASE EXACERBATION; EFFECT SIZE; GOOD CLINICAL PRACTICE; HEALTH ASSESSMENT QUESTIONNAIRE; HUMAN; MEDICAL RECORD REVIEW; MINIMAL CLINICALLY IMPORTANT DIFFERENCE; PREDICTIVE VALUE; PRIORITY JOURNAL; PROCESS DEVELOPMENT; PROOF OF CONCEPT; PSYCHOLOGICAL WELL-BEING; QUALITY OF LIFE; RARE DISEASE; REVIEW; SAMPLE SIZE; SHORT FORM 36; STUDY DESIGN; CANCER STAGING; CLINICAL TRIAL (TOPIC); CONTROLLED CLINICAL TRIAL (TOPIC); TREATMENT OUTCOME;

BIOMARKERS; CLINICAL TRIALS AS TOPIC; CONTROLLED CLINICAL TRIALS AS TOPIC; DISEASE PROGRESSION; ENDPOINT DETERMINATION; HUMANS; NEOPLASM STAGING; RARE DISEASES; TREATMENT OUTCOME;

EID: 85041705349     PISSN: 15524825     EISSN: 15524833     Source Type: Journal    
DOI: 10.1002/ajmg.a.38629     Document Type: Review

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