Since the beginning of NCCAM, the starting point for us has been the “real-world” use of complementary health approaches. Over time, we have supported a number of surveys and other observational studies, and have learned a good deal about the choices Americans are making in complementary approaches, the reasons they cite, and the associated costs. There is more to learn, of course, but we have pretty good descriptive data about real-world practices. And we have learned that the focus of most use is on pain management. But observational data, by itself, does not answer some important questions. In fact, it does not really test what works and what doesn’t work. So now we are beginning to ask: How can we bring the rigor of the randomized controlled trial to learn more about what is actually helping people manage pain in the real world?
The “real world” question is an active topic across the National Institutes of Health (NIH), reflected in growing interest in what are being called effectiveness studies or pragmatic trials. Of course, NIH clinical studies happen in the real world, but usually under conditions that are tightly specified and controlled. In the typical interventional study—whether it is of a new drug, a procedure, or a new behavioral approach—simultaneous use of other therapies is limited, patient eligibility criteria are closely specified, only highly experienced practitioners are engaged, nurse coordinators closely monitor compliance, and so on. Careful control of all aspects helps ensure study results can be replicated. It helps to create “internal validity.” And, by reducing the sources of variability, we reduce the need for large numbers of participants. But, there is a tradeoff in this approach to trial design. At least sometimes, when the results are implemented in real-world conditions, the intervention does not work as expected. Hence, the concept is gaining acceptance that we need both explanatory studies to test the efficacy of therapies and pragmatic studies to examine real-world effectiveness.
Investigators interested in improving the translation of research findings to the real world have developed an approach to assessing trial design—called PRECIS criteria, a pragmatic-explanatory continuum indicator summary. This tool categorizes clinical trials on a number of criteria, aiming to explore whether the trial design fits the overall goals of the research. Trials are considered to be pragmatic if they have broad participant eligibility, typical practitioners, interventions implemented flexibly, and an analysis that captures objective evidence for all patients. I urge our investigators to look at the literature on pragmatic study designs and to think with us about their potential applications to NCCAM’s research mission.
There are many practical questions emerging from our pain portfolio that seem ready for a more pragmatic approach. We have growing evidence, reflected in systematic reviews and practice guidelines, that a number of the mind-body therapies can have real benefit in pain management. But these results raise many new questions. Do these approaches improve patient well-being when they are integrated into primary care settings? What works well in pain clinics that see referral patients? Do the mind-body approaches reduce opioid abuse? What patient populations are best targeted? How can providers effectively encourage models of self-management?
The list is long. And the problem—chronic pain—is a very tough one.
We welcome your thoughts.