@article{547b146a7beb4aacad9382805d2e2d98,
title = "Adaptive Designs for Clinical Trials: Application to Healthcare Epidemiology Research",
abstract = "Clinical trials with adaptive designs use data that accumulate during the course of the study to modify study elements in a prespecified manner. The goal is to provide flexibility such that a trial can serve as a definitive test of its primary hypothesis, preferably in a shorter time period, involving fewer human subjects, and at lower cost. Elements that may be modified include the sample size, end points, eligible population, randomization ratio, and interventions. Accumulating data used to drive these modifications include the outcomes, subject enrollment (including factors associated with the outcomes), and information about the application of the interventions. This review discusses the types of adaptive designs for clinical trials, emphasizing their advantages and limitations in comparison with conventional designs, and opportunities for applying these designs to healthcare epidemiology research, including studies of interventions to prevent healthcare-associated infections, combat antimicrobial resistance, and improve antimicrobial stewardship.",
keywords = "Clinical trials, Drug resistance, Infection control, Microbial, Randomized controlled trials, Research design",
author = "Huskins, {W. Charles} and Fowler, {Vance G.} and Scott Evans",
note = "Funding Information: Financial support. This study was supported by the National Institute of Allergy and Infectious Diseases (NIAID), NIH (grant UM1 AI104681). Funding Information: Potential conflicts of interest. W. C. H. reports grants from NIH/ NIAID. V. G. F. reports grants from NIH, the Centers for Disease Control and Prevention, MedImmune, Cerexa/Forest/Actavis/Allergan, Pfizer, Advanced Liquid Logics, Theravance, Novartis, Cubist/Merck, Medical Biosurfaces, Locus, Affinergy, Contrafect, Karius, and Genentech; consultancies with Pfizer, Novartis, Galderma, Novadigm, Durata, Debiopharm, Genentech, Achaogen, Affinium, Medicines Co, Cerexa, Tetraphase, Trius, MedImmune, Bayer, Theravance, Cubist, Basilea, Affinergy, Janssen, xBi-otech, and Contrafect; personal fees from Green Cross, Cubist, Cerexa, Durata, Theravance, and Debiopharm; royalties from UpToDate; and a patent pending with Sepsis Diagnostics. S. E. reports grants from NIH/ NIAID, during the conduct of the study; and personal fees from Takeda/ Millennium; Pfizer; Roche; Novartis; Achaogen; the Huntington{\textquoteright}s Study Group; Auspex; Alcon; Merck; Chelsea; Mannkind; QRx Pharma; Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities and Networks (ACTTION); Genentech; Affymax; FzioMed; Amgen; GlaxoSmithKline; Boehringer-Ingelheim; American Statistical Association; the Food and Drug Administration; Osaka University; City of Hope; the National Cerebral and Cardiovascular Center of Japan; the NIH; the Muscle Study Group; the Society for Clinical Trials; Drug Information Association; the University of Rhode Island; New Jersey Medical School (NJMS)/Rutgers; Preclinical Pain Research Consortium for Investigating Safety and Efficacy (PPRECISE); Statistical Communications in Infectious Diseases; Cubist; AstraZeneca; Teva; Repros; Austrian Breast & Colorectal Cancer Study Group/Breast International Group; the Alliance Foundation Trials; Zeiss; Dexcom; the American Society for Microbiology; Taylor and Francis; Claret Medical; Vir; Arrevus; Five Prime; Shire; Alexion; Gilead; and Spark, outside the submitted work. All authors have submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Conflicts that the editors consider relevant to the content of the manuscript have been disclosed. Publisher Copyright: {\textcopyright} The Author(s) 2017.",
year = "2018",
month = mar,
day = "19",
doi = "10.1093/cid/cix907",
language = "English (US)",
volume = "66",
pages = "1140--1146",
journal = "Clinical Infectious Diseases",
issn = "1058-4838",
publisher = "Oxford University Press",
number = "7",
}