A healthy holiday topic—pragmatic trials

Pragmatic trials address real-world issues. In his monthly newsletter, Dr. Eric B. Larson suggests asking your guests what's important to them as physicians, patients, and caregivers.

What will you be talking about during your holiday dinners? Presidential debates, football, who gets the drumsticks? Today, I'm suggesting a topic that anyone who has interacted with the health care system might find intriguing: pragmatic trials.

We've all heard of randomized clinical trials. They're conducted under controlled conditions with extensively screened patients. As a first step for testing new drugs, these trials are great. But they aren't right for many clinical issues because in the real world, patients, their illnesses, and their treatments are diverse.

Pragmatic clinical trials take place wherever everyday care happens: in community clinics, hospitals, even online in homes. These studies include as many patients as possible for a widely representative population. In pragmatic trials, researchers don’t create idealized situations. Instead, they work with patients, physicians, and health system leaders to develop the study, often starting with a pilot phase, and continuing the collaboration as the full trial is conducted, analyzed, and communicated. Compared to efficacy trials (performed in idealized circumstances), and explanatory trials (that collect extensive information to understand why an idea did or did not work as expected), pragmatic trials aim for results that apply to real-world care. If an intervention improves health and care in a pragmatic trial, it's likely to work when implemented more generally since it was tested in typical care settings.

Pragmatic trials can also reduce research waste. This aligns with The Lancet REWARD campaign that I wrote about last month. Pragmatic trials start with an issue of high priority to people who will use the results—a REWARD recommendation. The studies are designed so that promising results move quickly into practice, which is the goal of translational science. In the language of this field, pragmatic trials break down silos, eliminating physical and cultural barriers between research and clinical practice. They require researchers to work closely with health system operational, management, and clinical personnel.

Pragmatic examples from the NIH Collaboratory

As anyone who has worked on a diverse team can imagine, pragmatic clinical trials aren't easy. That's why the National Institutes of Health Common Fund, which supports health innovations, created the Health Care System Collaboratory. The Collaboratory funds pragmatic studies and is learning how best to conduct them. Group Health is involved in several Collaboratory trials underway now:

• Psychiatrist and GHRI Senior Investigator Gregory Simon, MD, MPH, is testing programs, including at Group Health sites, to reduce suicide risk.
• Group Health Physician and GHRI Associate Investigator Beverly B. Green, MD, MPH, with Kaiser Permanente's Gloria Coronado, PhD, is studying how to increase colorectal cancer screening in minority and low-income populations.
• Group Health is a lead site in a trial led by Jeffrey Jarvik, MD, MPH, University of Washington, with GHRI Senior Investigator, Dan Cherkin, PhD, to improve interpretation and communication of medical imaging for low back pain. 

Watch for results from these trials in a few years and for pragmatic trials in your health system. I predict they'll become more popular since they provide valid, actionable information that health systems can apply to support evidence-based medicine.

To be honest, your guests will probably talk about food, family, and the local football team. But if anyone asks what's new in your line of work, tell them about pragmatic trials. Explain that they answer practical clinical questions that are important to doctors, nurses, and patients. Ask what health care questions your guests would like answered and tell me what you find out. Have a happy, healthy holiday season.

Read more about what we're learning about planning pragmatic trials in our recent publications:

• Trials without tribulations: Minimizing the burden of pragmatic research on healthcare systems
• A guide to research partnerships for pragmatic clinical trials

Eric B. Larson, MD, MPH
Vice President for Research, Group Health
Executive Director, Group Health Research Institute

post updated December 2, 2015