A focus on building a nationwide system of real-world data could provide the opportunity for more randomized clinical trials to generate needed evidence to inform health care decisions.
System-wide changes in the ecosystem of randomized clinical trials are needed to conduct trials more efficiently and at a lower cost, according to a recent paper in JACC written by current and former representatives of the DCRI.
The assertions in the paper build on and respond to findings published in JAMA in March 2019 by a writing group that included the JACC paper’s co-authors, DCRI’s Renato Lopes, MD, PhD, former DCRI faculty member Robert Califf, MD, and former DCRI fellow Alexander Fanaroff, MD, MHS (now of the University of Pennsylvania). The March paper found that 90 percent of guidelines used to manage care for heart patients are not based on randomized clinical trial data, the gold standard for determining the efficacy of medical practices. In their more recent paper, Lopes, Califf, and Fanaroff explore barriers to conducting randomized clinical trials, and suggest solutions to overcome them.
A major challenge is that trials are typically funded by a company seeking data to support approval for a new medication or device. This structure creates “chasms in the evidence base” because it does not enable certain types of trials to be conducted, “including trials that compare treatment strategies, health services interventions, or the relative efficacy of approved medications and devices, evaluations of combinations of treatments, or trials intended to reduce the use, duration, or dose of a therapy,” the authors write.
The paper points to innovations that have emerged in the past decade in an effort to reduce costs, from adaptive designs that require smaller sample sizes, to virtual trials that can alleviate both cost and complexity. However, these advances have not moved the needle on the proportion of guideline recommendations that draw from randomized clinical trial data. “To move toward a world in which most clinical decisions are supported by high-quality evidence will require transformative, simultaneous structural changes in clinical trials and clinical care ecosystems,” the authors write.
The authors suggest that building randomized clinical trials within a robust real-world data system would create an environment in which multiple trials could use the renewable resource of data collected during routine patient care. Currently, limitations hinder the creation of such a system, including data inoperability challenges among health care systems and regulatory constraints.
In the U.S., the authors write, a culture change is also needed: Healthcare systems, hospitals, and healthcare providers must place more value on research participation and encourage their patients to participate in randomized clinical trials.
These changes will require collaboration and both public and private investment. The authors note that pharmaceutical companies would benefit from investing in building a system that will ultimately lead to reduced costs. The government should also participate in the creation of such a system, the authors write, because “the lack of high-quality evidence from RCTs to support most patient care decisions is an urgent public health issue that deserves an appropriately strong response.”