Year: 2012
Type: review
Abstract: Adaptive designs allow planned modifications based on data accumulating within a study. The promise of greater flexibility and efficiency stimulates increasing interest in adaptive designs from clinical, academic, and regulatory parties. When adaptive designs are used properly, efficiencies can include a smaller sample size, a more efficient treatment development process, and an increased chance of correctly answering the clinical question of interest. However, improper adaptations can lead to biased studies. A broad definition of adaptive designs allows for countless variations, which creates confusion as to the statistical validity and practical feasibility of many designs. Determining properties of a particular adaptive design requires careful consideration of the scientific context and statistical assumptions. We first review several adaptive designs that garner the most current interest. We focus on the design principles and research issues that lead to particular designs being appealing or unappealing in particular applications. We separately discuss exploratory and confirmatory stage designs in order to account for the differences in regulatory concerns. We include adaptive seamless designs, which combine stages in a unified approach. We also highlight a number of applied areas, such as comparative effectiveness research, that would benefit from the use of adaptive designs. Finally, we describe a number of current barriers and provide initial suggestions for overcoming them in order to promote wider use of appropriate adaptive designs. Given the breadth of the coverage all mathematical and most implementation details are omitted for the sake of brevity. However, the interested reader will find that we provide current references to focused reviews and original theoretical sources which lead to details of the current state of the art in theory and practice.
Source: Trials
Language: English
Subfield: Statistics and Probability
Field: Mathematics
Domain: Physical Sciences
Open Access status: gold
Estimated APC paid: $2,790
Funders: University of Florida, China Scholarship Council, National Institutes of Health, National Institute on Alcohol Abuse and Alcoholism, National Institute on Aging, National Heart, Lung, and Blood Institute, National Institute on Drug Abuse, National Institute of Nursing Research, National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Neurological Disorders and Stroke, National Institute of Dental and Craniofacial Research, National Center for Research Resources, National Center for Advancing Translational Sciences
Awards: Ancillary Functional Studies for the CCTRN, UF Clinical and Translational Science Award, G2848036350, G3233374885, Substance Use and Immunity in HIV+ Adolescents by Systems Biology, UF Clinical and Translational Science Award, A collaborative model to improve BP control and minimize racial disparities--DCC, Southeast Center for Research to Reduce Disparities in Oral Health, G5321427439, G640430542, Amitriptyline and Topiramate in the Prevention of Childhood Migraine: DCC, Vitamin B6 Effects on One-Carbon Metabolism, SECONDARY PREVENTION OF SMALL SUBCORTICAL STROKES (SPS3), Etiology of Alcohol Use and Consequences Among Multi-Ethnic Urban Youth, Improving Dementia Caregiver Sleep and The Effect on Heart Disease Biomarkers, Multilevel and Longitudinal Study Sample Size Tools for Behavioral Scientists, Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) DCC
OpenAlex ID: W2171364060