Journal Article

Pragmatic clinical trials embedded in healthcare systems: generalizable lessons from the NIH Collaboratory

Heagerty, Patrick J., Staman, Karen L., Larson, Eric B., Sugarman, Jeremy, Vazquez, Miguel, Coronado, Gloria D., Mor, Vincent, Septimus, Edward J., Simon, Gregory E., Rosenthal, Gary E., Curtis, Lesley H., DeBar, Lynn L., Hernandez, Adrian F., Weinfurt, Kevin P., James, Kathryn T., Platt, Richard, Green, Beverly B., Jarvik, Jeffrey G., Dember, Laura M., Huang, Susan S., Zatzick, Douglas

2017

Overview

Background The clinical research enterprise is not producing the evidence decision makers arguably need in a timely and cost effective manner; research currently involves the use of labor-intensive parallel systems that are separate from clinical care. The emergence of pragmatic clinical trials (PCTs) poses a possible solution: these large-scale trials are embedded within routine clinical care and often involve cluster randomization of hospitals, clinics, primary care providers, etc. Interventions can be implemented by health system personnel through usual communication channels and quality improvement infrastructure, and data collected as part of routine clinical care. However, experience with these trials is nascent and best practices regarding design operational, analytic, and reporting methodologies are undeveloped. Methods To strengthen the national capacity to implement cost-effective, large-scale PCTs, the Common Fund of the National Institutes of Health created the Health Care Systems Research Collaboratory (Collaboratory) to support the design, execution, and dissemination of a series of demonstration projects using a pragmatic research design. Results In this article, we will describe the Collaboratory, highlight some of the challenges encountered and solutions developed thus far, and discuss remaining barriers and opportunities for large-scale evidence generation using PCTs. Conclusion A planning phase is critical, and even with careful planning, new challenges arise during execution; comparisons between arms can be complicated by unanticipated changes. Early and ongoing engagement with both health care system leaders and front-line clinicians is critical for success. There is also marked uncertainty when applying existing ethical and regulatory frameworks to PCTS, and using existing electronic health records for data capture adds complexity.

• Publisher: BioMed Central,BioMed Central Ltd,Springer Nature B.V,BMC
• Subject: Clinical trials / Clinics / Cluster randomized trials / Collaboration / Cost-Benefit Analysis / Decision Making / Delivery of Health Care - economics / Delivery of Health Care - methods / Delivery of Health Care - standards / Design / Electronic health records / Electronic Health Records - standards / Embedded clinical trials / Epidemiology / Ethics / Health care industry / Health care policy / Health Sciences / Hemodialysis / Humans / Informatics / Internet / Medical screening / Medicine / Medicine & Public Health / Mortality / Narcotics / National Institutes of Health (U.S.) / Pain / Patients / Planning / Pragmatic clinical research / Pragmatic Clinical Trials as Topic - economics / Pragmatic Clinical Trials as Topic - methods / Pragmatic Clinical Trials as Topic - standards / Pragmatic research / Primary care / Research Article / Research Design - standards / Research Report - standards / Researchers / Stakeholder engagement / Staphylococcus infections / Statistical Theory and Methods / Statistics for Life Sciences / Study design / Theory of Medicine/Bioethics / United States