I was delighted to be part of the “Ethical and Regulatory Issues of Pragmatic Clinical Trials Workshop” here at the National Institutes of Health (NIH) on May 10. Dr. Wendy Weber and I joined a handful of colleagues from NIH, Johns Hopkins University, Duke University, and the University of Pennsylvania to organize this workshop, now available as an archived videocast. A brief written summary is planned for later this year. Topics included informed consent, vulnerable populations, institutional review boards, data monitoring committees, and privacy issues.
The workshop idea emerged after a special issue of Clinical Trials was published in October 2015 including a series of 12 articles on ethical and regulatory issues of pragmatic trials. That project was planned and carried out by the ethics working group of the NIH Collaboratory: Health Care Systems Research Collaboratory, and sponsored by NIH and The National Patient-Centered Clinical Research Network.
The NIH Collaboratory, an NIH Common Fund project since 2012, aims to “strengthen the national capacity to implement cost-effective large-scale research studies that engage healthcare delivery organizations as research partners.” Ultimately, the goal is to ensure that health care providers and patients will have the best available clinical evidence upon which to make decisions. Demand for quality evidence in the health care arena is very high, and thus new, efficient approaches to conduct large-scale randomized, controlled trials that answer important health care questions are urgently needed.
To help address this need, the NIH Collaboratory supports the design and execution of Pragmatic Clinical Trial Demonstration Projects. Pragmatic trials test interventions in usual or real-world settings, in contrast to explanatory trials, which test interventions in more targeted settings.
Dr. Josephine Briggs, NCCIH Director, has previously blogged about the NIH Collaboratory and NCCIH’s interest in pragmatic trials. The 10 projects awarded to date address questions of major public health importance in a variety of medical conditions, with trials conducted in partnership with health care-delivery systems; and produce data, tools, and resources to be shared with the greater research community. Nine have now been launched as full-scale trials.
I consider this innovative methodology for clinical trials a new phase for clinical research, and even a new era of transformative methods for NIH in the clinical research space. But as with any startup, there are challenges, and unlike explanatory trials, pragmatic trials have some fairly unique ones in terms of regulatory and ethical aspects―including issues of informed consent, participant privacy, and determination of research risks.
The Demonstration Projects and their investigators have already provided much useful information for NIH and the community about these issues, and identified potential problems as well as solutions for future pragmatic trials.
View the videocast (May 10, 2016, 8:00 a.m. – 4:30 p.m. ET).