Over ten years ago, the Institute of Medicine (IOM) set a goal that by the year 2020, “90 percent of clinical decisions will be supported by accurate, timely, and up-to-date clinical information.”¹ The development of learning health care system was predicated on the notion that evidence to support clinical decision-making could be gathered from data collected during the course of routine care, such as the electronic health record and administrative claims data, and ePCTs are a part of that solution.

Yet it has been challenging to meet the National Academy of Medicine’s 2020 goal for the development of a learning health system. If the goal had been met, 90% of clinical decisions would be based on accurate evidence gathered from health systems that continually learn from data collected as part of routine care.

“Absent such evidence, the type of care provided is determined by the haphazard influences of financial incentives, clinicians’ anecdotal experiences, and patients’ or clinicians’ exposure to marketing messages.” — Simon et al., New England Journal of Medicine, April 16, 2020

Most pragmatic trials, including the NIH Pragmatic Trials Collaboratory Trials and clinical trials conducted through the National Patient-Centered Clinical Research Network (PCORnet), have compared standard care with proposed improvements to standard care, or “A vs A-plus trials.” Trials that compare 2 alternative treatments that are in current use, or “A vs B trials,” are rarely done.

“Achievement of the NAM’s goal of basing 90% of clinical decisions on accurate evidence remains distant, and meaningful progress will involve engagement by many parties,” write Simon and colleagues. “It will require health system leaders to consider rigorous evidence generation a core function of ordinary health care, research funders to prioritize practical questions relevant to population health and to support infrastructure for embedded research, research regulators to align consent procedures with incremental risks of research, and researchers to ask real-world questions that patients, caregivers, and clinicians want answered. But the first step is for everyone involved to honestly acknowledge the lack of evidence supporting the majority of common medical decisions and the urgent need for more relevant and efficient clinical research.” (Simon et al. 2020)

To encourage health system participation, a robust national ePCT capability could help generate evidence to guide decisions by patients, clinicians, health systems, and regulators. (Platt et al. 2021.)

Researchers and funders could consider a 5-pronged strategy to increase healthcare system participation in ePCTs:

• Engage with health system leaders and clinicians to identify priority research questions
• Reimburse for the additional costs of trial participation
• In some highly engaged systems, support permanent, reusable infrastructure
• Offload research-specific tasks to minimize burden on sites (such as IRB oversight, obtaining informed consent, and mailing medications to participants)
• Assign and promote reputational benefit for these activities

For more information, see the NIH Pragmatic Trials Collaboratory workshop, Embedded Pragmatic Clinical Trials of Therapeutic A vs B Interventions. The workshop explored challenges and strategies for planning and implementing ePCTs that compare 2 or more therapeutic medical regimens.

• Workshop Summary
• Videocast
• Individual Presentation Slides