“Pragmatic trials” are the new buzz in clinical research. I want to spend this post talking a bit about why—and the reasons for NCCAM’s particular interest in this topic.
Many complementary approaches are readily available in the marketplace. As a consequence, NCCAM sits at the crossroads between research and real-world consumer use. The general public wants to know what works and what doesn’t, and increasingly health care providers also want reliable information. Complementary health approaches are being integrated into the care offered in many nursing homes, hospices, and hospitals, and these health care organizations want good information to drive decisions about which therapies to provide or recommend. NCCAM wants to take on the challenge of meeting this need, but by and large we do not have the kind of rigorous, high-quality data that would help answer these questions.
This unique situation has made us aware of the importance of better methods to do real-world, or pragmatic, research. Driven by this interest, we volunteered about 2 years ago to take on a major administrative and leadership role in an NIH Common Fund initiative called the Health Care Systems Research Collaboratory. This program is engaging health care delivery organizations as research partners, with the goal of building methods to conduct rigorous large-scale clinical trials in real-world settings. Through the Collaboratory, NIH is pioneering the development of approaches to conduct large-scale, cost-effective clinical research studies in the settings where patients already receive their care.
The focus of this activity is pragmatic trials. What makes a pragmatic trial, well … pragmatic? Pragmatic trials, sometimes called effectiveness trials, test an intervention, or compare several interventions, delivered under conditions as close to the “real world” as possible. In contrast, efficacy studies typically test interventions under ideal conditions, with very close monitoring.
The interventions tested in a pragmatic trial should not require a complex structure for implementation. The study design should incorporate rigorous controls, prospectively identified and preferably randomized. The monitoring and outcome assessment should be as simple as possible and potentially use electronic health records. And finally, the research question to be answered should be important—to patients, payers, and health care delivery systems.
Two of the Collaboratory studies will address pain management. The LIRE study, a partnership with the National Institute of Arthritis and Musculoskeletal and Skin Diseases and a number of Health Maintenance Organizations, looks at the impact of more detailed radiology reports for back pain imaging studies on subsequent use of resources. The second partnership, a study called PPACT, which involves a number of Kaiser health systems, with oversight from the National Institute on Drug Abuse and the National Institute of Neurological Disorders and Stroke, will examine the impact of an integrated pain management strategy implemented in primary care practices.
This type of research represents a paradigm shift in clinical research. These trials are rigorous randomized studies, but performed in real-world settings with the practical goal of testing the effectiveness of practical approaches. We at NCCAM, like our colleagues at many other NIH Institutes and Centers, are eagerly watching the progress of the Collaboratory projects, not only because of our involvement, but because we hope the lessons being learned will help us and our grantees move clinical research forward.