Pragmatic clinical trials (PCTs) offer a promising alternative to explanatory trials, according to a recent commentary published in the ESMO Real World Data and Digital Oncology. Such trials feature simplified study protocols and data collection methods and enable a wider patient selection pool, which the authors believe can enhance clinical research.
Clinical trials with pragmatic elements can incorporate use of real-world data (RWD) and real-world evidence (RWE) from electronic health records, registries, and patient-reported outcomes, the authors wrote.
The article, written by Mark Stewart, vice president of Science Policy at the Friends for Cancer Research, and colleagues, states that there is “growing interest in using PCTs as alternatives to explanatory clinical trials to support regulatory decision making.”
The authors note that while explanatory clinical trials represent the conventional approach to conducting clinical research, a method driven by familiarity with the methodologies and acceptance by regulatory authorities, there has been a shift toward PCTs, reflecting “growing recognition of the challenges with conventional trials, such as increasing design complexity and highly selected patient populations.”
The authors recommended that drug sponsors consider a hybrid PCT method integrating pragmatic elements into traditional randomized controlled trials to streamline research and expand access to diverse patient populations.
Officials at the Us Food and Drug Administration support the use of PCTs, including FDA Commissioner Robert Califf, who endorsed this research approach in a June article he co-wrote with other FDA officials.
Richard Pazdur, director of the FDA’s Oncology Center of Excellence (OCE), also advocated for using the pragmatic design at a 2022 Friends of Cancer Research Meeting. (RELATED: This Week at FDA: Califf and Pazdur on return to office, accelerated approval, and more, Regulatory Focus 18 November 2022)
The study cites the Pragmatica-Lung case study as an example of how pragmatic elements can be integrated into a clinical trial. Pragmatica-Lung is a federally funded clinical study examining a combination of therapies to treat patients with advanced non-small cell lung cancer (NSCLC). (RELATED: Pragmatica: Lung cancer trial tests streamlined design, inclusive eligibility, Regulatory Focus 12 April 2023)
The Pragmatica-Lung study features broadened eligibility criteria to encompass diverse patient populations and streamlined data collection methods that prioritizes “critical” safety and efficacy endpoints.
The study was “rapidly” implemented and is now at over 500 sites within the National Clinical Trials Network. The trial is “on pace to complete enrollment in half the estimated timeframe across a more representative set of patients compared with historical rates, demonstrating the efficiency and ability of PCTs to reach more patients.”
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