Enhancing Representative Enrollment through Point-of-Care Trials

Clinical trials are an important part of the drug development process and help to ensure new treatments are safe and effective. Known for their rigor, traditional randomized controlled trials have strict inclusion and exclusion criteria, multitude of required assessments, and carefully crafted outcome measures. Data collection occurs in controlled environments, often requiring patients who do not reside near trial sites to travel long distances to access innovative treatments. Data from clinical trials are then reviewed by regulatory agencies such as the U.S. Food and Drug Administration (FDA) to determine whether a medical product is safe and effective. However, despite the merits and successes of clinical trials, the current clinical trial enterprise faces structural challenges including a notable lack of representation and a fragmented post-market evidence generation system. In 2023, FDA Commissioner Dr. Robert Califf emphasized that the current post-market evidence system does not collect sufficient data after a product is approved to address the evidentiary needs of all Americans.

Clinical trial modernization is needed to create a system that eliminates barriers to efficient evidence generation and increases the representativeness of enrolled patients. Recent efforts to innovate the traditional clinical trial design include steps to reduce data collection burdens on patients and staff, support and incentivize involvement from frontline staff and patients in data collection and increase representativeness while maintaining analytical rigor.

To research these issues more in-depth, the Duke-Margolis Institute for Health Policy conducted a multiphase research study between Fall 2022 and Spring 2024 specifically on the topic of increasing representativeness in clinical trials. This research included multiple convenings, a landscape review of existent literature and published materials as well as structured stakeholder interviews with key experts across the clinical trial enterprise including patient advocacy organizations, researchers, and pharmaceutical representatives. Stakeholder interviews were conducted primarily in Spring 2023 and public workshops were hosted in July 2023 and March 2024.

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