An Exciting New Year for Pragmatic Clinical Trials

Jan. 8, 2018
It’s an exciting time in the world of pragmatic clinical trials, as the big data from EHRs and claims data starts to bring back answers to interesting research questions.

It’s an exciting time in the world of pragmatic clinical trials, as the big data from EHRs and claims data starts to bring back answers to interesting research questions. Last week I saw a great presentation about the design and launch of an effort to improve treatment with oral anticoagulants in patients with atrial fibrillation (AFib) and at risk of stroke. It involves a randomized controlled trial of direct mail to thousands of health plan members with AFib and to their providers to encourage consideration of oral anticoagulation.

I wrote about pragmatic clinical trials a few times last year. They are designed to reflect “real-world” medical care by recruiting broad populations of patients, embedding the trial into the usual healthcare setting, and leveraging data from health systems to produce results that can be readily used to improve patient care.

For instance, I wrote about a presentation at Duke University by Russell Rothman, M.D., the vice president for population health research at Vanderbilt University Medical Center, in which he described some of the informatics infrastructure of one PCORI-funded study, ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness).

ADAPTABLE is a $14 million, three-year pragmatic clinical trial that is comparing the effectiveness of two different daily doses of aspirin widely used to prevent heart attacks and strokes in individuals living with heart disease. Its goal is to enroll 20,000 patients. For that project, the Mid-South Clinical Data Research Network (CDRN) takes EHR data from health systems and transforms it into a common data model to run queries against. 

In another talk last year, Eric Larson, M.D., M.P.H., executive director and senior investigator in the Kaiser Permanente Washington Health Research Institute and co-principal investigator of the NIH Collaboratory Coordinating Center, explained that pragmatic is the key word because these trials are practical. The researchers are embedded in healthcare systems and they seek to engage everyday patients.  “They have chosen questions relevant to decision making by patients and providers,” he said. This is the genesis of the learning health system, he argued. As researchers bring their hypotheses to the health system, providers can ask questions that influence the research and it creates a virtuous cycle that ends ultimately with dissemination of evidence-based improvements. Evidence informs practice and practice informs evidence.

Yet another example of a pragmatic clinical trial is LIRE (Lumbar Imaging with Reporting of Epidemiology) run out of the University of Washington. The LIRE study is testing the effectiveness of a simple and inexpensive intervention: inserting epidemiologic benchmarks into lumbar spine imaging reports. The goal of the trial is to reduce subsequent tests and treatments, opioid prescriptions, spinal injections, or surgery. The LIRE team is doing a presentation in a few weeks. I will try to report on that project as well.)

Back to the presentation I saw last week at the NIH Collaboratory: The study is called Impact AFib, with the word Impact standing for “IMplementation of a randomized controlled trial to imProve treatment with oral AntiCoagulanTs in patients with Atrial Fibrillation.”

The aim is to increase the use of oral anticoagulation (OAC) among patients with AFib and at risk of stroke. Sean Pokorney, M.D., M.B.A., of the Duke Clinical Research Institute, said that preliminary research of claims data suggested that among the population with AFib and stroke risk factors, only 52 percent were being treated with anticoagulants.

“The underuse of anticoagulation is a major public health priority and a priority for health plans as well. They want to deliver optimal care,” he said.

The study designed educational mailings about OAC for patients and their providers, with the help of patient representatives. The trial uses the FDA’s Sentinel database and claims data from five large commercial insurers. Noelle Cocoros, DSc, MPH, an epidemiologist and research associate in the Department of Population Medicine at Harvard Medical School and Harvard Pilgrim Health Care Institute, described how the study uses the Sentinel system, one of the FDA’s tools for post-market safety surveillance of medical products. Sentinel is a distributed data network with the data transformed into a common data model at the coordinating center at Harvard Pilgrim. “We can write queries against the data and what gets returned is aggregated data from each site. It translates into an incredibly robust and efficient system with rapid analytic capability,” she said.

The IMPACT team randomized 80,000 patients with AFib diagnoses and stroke risk into an early intervention group and a delayed intervention group, who will get the same notices a year later. A commercial IRB waived the need to consent patients into the study. The mailings started going out to the first group of more than 40,000 patients in September. The trial will also assess outcomes associated with the treatment, including stroke.

One of the project leaders is Christopher Granger, M.D., of the Duke Clinical Research Institute, whose research background includes conducting large randomized clinical trials in heart disease. “This has been the most innovative clinical trial I have ever been involved with,” he said, adding it would soon be able to do analyses involving 80,000 patients on one of the most important issues in public health — preventing stroke. “It is being done so efficiently. It is truly a pragmatic clinical trial. It is not selective in terms of population. Developing this incredibly efficient design at modest cost across these collaborators has been a remarkable experience.”

Stay tuned over the next few years as they publish their results and determine whether their interventions are easily replicable by other health systems.

By the way, here is a year in review blog post from the folks at PCORnet, the national patient-centered clinical research network making these pragmatic trials possible. In 2017, among many other milestones, PCORnet opened its Front Door, offering potential investigators (including patient groups, healthcare organizations, clinicians, government, industry scientists, and sponsors) a central gateway to access and leverage PCORnet’s unique infrastructure and collaborate on patient-centered clinical research.

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