title: Pragmatic clinical trials: Emerging challenges and new roles for statisticians.
abstract: Patients, clinicians, and policymakers alike need access to high-quality scientific evidence in order to make informed choices about health and healthcare, but the current national clinical trials enterprise is not yet optimally configured for the efficient creation and dissemination of such evidence. However, new technologies and methods hold significant potential for accelerating the rate at which we are able to translate raw findings gathered from both patient care and clinical research into actionable knowledge. We are now entering a period in which the quantitative sciences are emerging as the critical disciplines for advancing knowledge about health and healthcare, and statisticians will increasingly serve as critical mediators in transforming data into evidence. In this new, data-centric era, biostatisticians not only need to be expert at analyzing data but should also be involved directly in diverse efforts, including the review and analysis of research portfolios in order to optimize the relevance of research questions, the use of "quality by design" principles to improve reliability and validity of each individual trial, and the mining of aggregate knowledge derived from the clinical research enterprise as a whole. In order to meet these challenges, it is imperative that we (1) nurture and build the biostatistical workforce, (2) develop a deeper understanding of the biological and clinical context among statisticians, (3) facilitate collaboration among biostatisticians and other members of the clinical trials enterprise, (4) focus on communication skills in training and education programs, and (5) enhance the quantitative capacity of the research and clinical practice worlds.
authors.affiliation: US Food and Drug Administration, Silver Spring, MD, USA Robert.Califf@fda.hhs.gov.
authors.forename: Robert M
authors.initials: RM
authors.lastname: Califf
mesh_terms: D035843:Biomedical Research / Q000379:methods*; D002986:Clinical Trials as Topic / Q000379:methods*; D003625:Data Collection / Q000379:methods*; D057286:Electronic Health Records; D006801:Humans; D015203:Reproducibility of Results; D013223:Statistics as Topic / Q000379:methods*
keywords: Biostatistics; clinical trial; electronic health record; evidence; pragmatic clinical trial; quality by design; randomization; statistics