Statistical Considerations for an Adaptive Design for a Serious Rare Disease

Therapeutic Innovation and Regulatory Science

Volumn 50, Issue 3, 2016, Pages 375-384

  Patra, Kaushik ^a   Cree, Bruce A C ^b   Katz, Eliezer ^a   Pulkstenis, Erik ^a   Dmitrienko, Alex ^c   Cutter, Gary ^d  

^a MEDIMMUNE   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c IQVIA   (United States)

^d UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

Author keywords

adaptive design; adjudication process; chain procedure; futility; inter and intrarater reliability; masked sample size re estimation; neuromyelitis optica; rare disease

Indexed keywords

AQUAPORIN 4 ANTIBODY; AZATHIOPRINE; IMMUNOGLOBULIN; IMMUNOGLOBULIN G ANTIBODY; INEBILIZUMAB; PLACEBO; RITUXIMAB; STEROID;

ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG MEGADOSE; HUMAN; INTERRATER RELIABILITY; INTRARATER RELIABILITY; MYELOOPTIC NEUROPATHY; NEUROMYELITIS OPTICA SPECTRUM DISORDER; NUCLEAR MAGNETIC RESONANCE IMAGING; OVERALL SURVIVAL; PHYSICAL DISABILITY; PLASMAPHERESIS; PRIORITY JOURNAL; RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL; RARE DISEASE; RESEARCH ETHICS; SAMPLE SIZE; SEROLOGY; STATISTICAL CONCEPTS; STRATIFICATION; STUDY DESIGN; SURVIVAL RATE; TREATMENT DURATION; VISUAL IMPAIRMENT;

EID: 84964284206     PISSN: 21684790     EISSN: 21684804     Source Type: Journal    
DOI: 10.1177/2168479015619203     Document Type: Article

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