A recent JAMA Cardiology paper describes several design elements that make ADAPTABLE, a demonstration study of PCORnet®, the National Patient-Centered Clinical Research Network, a novel pragmatic clinical trial. The manuscript’s authors detailed these trial elements with an aim to inspire efficiency and patient-centricity in future trials.
“A strong need has arisen to develop and execute more streamlined, pragmatic trials leveraging real-world data and technologies to generate real-world evidence to answer important clinical questions that have a direct impact on public health,” the paper’s authors write.
Using the broad and vast resources of PCORnet, ADAPTABLE is positioned to deliver answers in a way that is less expensive, more efficient, and less burdensome for both patients and clinicians. A key PCORnet-enabled design element of the trial included large-scale recruitment using EHRs from members of the Network. One of the goals of the trial was to test PCORnet capabilities for patient identification, recruitment, and follow-up. ADAPTABLE successfully recruited over 15,000 participants from among its 40 participating sites.
Patient engagement has been carefully planned and implemented throughout the trial, including constitution of a Patient Review Board for the study comprised of “Adaptors,” the patient leaders engaged to help lead ADAPTABLE. By design, one Adaptor has been identified from each of the seven participating PCORnet Clinical Data Research Networks in order to represent patient viewpoints from the geographic region of the sites. Through their participation, the Adaptors have reviewed various aspects of study policy and a variety of specific patient-facing study materials. They have contributed both reactively as well as proactively, providing many specific suggestions and also new unsolicited ideas about how to approach and engage participants in a way that will help the trial succeed.
In an accompanying commentary on this paper, Patrick T. O’Gara, MD, from the Cardiovascular Division, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical writes that “JAMA Cardiology has not previously published trial design articles. The editors believe the methods incorporated in the ADAPTABLE trial represent important next steps in the more rapid and less expensive generation of reliable real-world evidence to enable better decision-making. The efficacy and safety outcomes of this 15 000-patient study are awaited with interest. In the interim, the design features should spur further efforts to improve the clinical research ecosystem.”