title: Innovative designs of point-of-care comparative effectiveness trials.
abstract: One of the provisions of the health care reform legislation in 2010 was for funding pragmatic clinical trials or large observational studies for comparing the effectiveness of different approved medical treatments, involving broadly representative patient populations. After reviewing pragmatic clinical trials and the issues and challenges that have made them just a small fraction of comparative effectiveness research (CER), we focus on a recent development that uses point-of-care (POC) clinical trials to address the issue of "knowledge-action gap" in pragmatic CER trials. We give illustrative examples of POC-CER trials and describe a trial that we are currently planning to compare the effectiveness of newly approved oral anticoagulants. We also develop novel stage-wise designs of information-rich POC-CER trials under competitive budget constraints, by using recent advances in adaptive designs and other statistical methodologies.
authors.affiliation: VA Palo Alto Cooperative Studies Program Coordinating Center, Mountain View, CA, USA. Electronic address: mei-chiung.shih@va.gov.
authors.forename: Mei-Chiung
authors.initials: MC
authors.lastname: Shih
authors.affiliation: Department of Medicine, Stanford University, Stanford, CA, USA; VA Palo Alto Health Care System, Palo Alto, CA.
authors.forename: Mintu
authors.initials: M
authors.lastname: Turakhia
authors.affiliation: Department of Statistics, Stanford University, Stanford, CA, USA; Department of Health Research and Policy, Stanford University, Stanford, CA, USA.
authors.forename: Tze Leung
authors.initials: TL
authors.lastname: Lai
mesh_terms: D000925:Anticoagulants / Q000627:therapeutic use; D001281:Atrial Fibrillation / Q000188:drug therapy; D057186:Comparative Effectiveness Research / Q000191:economics / Q000379:methods*; D006801:Humans; D011897:Random Allocation; D016032:Randomized Controlled Trials as Topic / Q000191:economics / Q000379:methods; D012107:Research Design
chemical_list: D000925:Anticoagulants
keywords: Adaptive design; Anticoagulants; Comparative effectiveness; Point-of-care trials; Pragmatic trials; Shared decision making