On May 16, 2018, the NIH Common Fund sponsored a workshop on embedded pragmatic clinical trials (PCTs) of therapeutic A vs. B interventions. This type of PCT, which compares two or more therapeutic medical interventions in real-world settings, holds exciting potential for bridging the gap between evidence, practice, and policy. The one-day workshop included information on the progress of PCTs and strategies for conducting future research.
The workshop focused on experiences from the NIH Health Care Systems Research Collaboratory, also known as the NIH Collaboratory. Initiated in 2012, the NIH Collaboratory has launched nine large-scale PCTs in partnership with several U.S. health care systems. The trials are being conducted in real-world settings of routine clinical care and have served as case studies for addressing challenges of pragmatic research. Knowledge gained from the trials has allowed the NIH Collaboratory to develop an extensive set of methods and best practices for embedded PCTs. There is now considerable interest to expand the interventions tested within the NIH Collaboratory and specifically to develop additional tools and strategies that test distinct therapeutic medical interventions, such as A vs. B trials.
The workshop included five moderated discussions on opportunities and approaches for partnering with health care systems, and other stakeholders, to conduct embedded A vs. B trials. Panelists from Anthem, Inc., Hospital Corporation of America, and the Centers for Medicare and Medicaid discussed stakeholder perspectives on such trials. Ongoing real-world A vs. B trials in the United States—supported by NIH, the Patient-Centered Outcomes Research Institute (PCORI), and the Department of Veterans Affairs (VA) Health Systems—were presented as potential models for future studies. Also, in view of the diversity of health care systems and delivery in the United States, different design and analytic strategies for capturing real-world evidence were discussed in the context of studies supported by NIH, the Food and Drug Administration (FDA), and the pharmaceutical industry.
As regulatory oversight of the clinical research enterprise is an essential component of PCT planning and implementation, the workshop included two panels that reviewed regulatory aspects, including ethical oversight, of trials that test such interventions. Panelists from the FDA, the Office for Human Research Protections (OHRP), and U.S. institutional review boards guided an informed conversation describing current regulations and perspectives on trials conducted in real-world settings.
NCCIH is excited that emerging information from the NIH Collaboratory will also help with real-world research on complementary and integrative therapies.
You can view the archived workshop sessions on the NIH Videocast Web site now. A report on the May workshop will be published in the near future.