FDA Support For Pragmatic Clinical Trials for Post-Market Studies
(Thursday, June 6, 2024) While the gold standard for pre-approval clinical trials is the randomized clinical trials (RCTs) design, post-market studies should follow the pragmatic clinical trial (PCT) design for better information about the drugs in the real world setting. FDA’s top management published a perspective this week that asserts that this would help better understand how well the new drug works compared to other treatments and the best ways to use it. After a treatment is approved, real-world evidence becomes important. This evidence comes from data collected in everyday healthcare settings, like electronic health records and wearable technology. These data help us understand how treatments work in real life. The major limitation of real world data is the high variability of the quality of the information available from medical records and biases. Pragmatic clinical research offers the middle ground between traditional RCTs and real-world evidence. PCTs use real-world data combined with data collected specifically for clinical studies. PCTs include randomization but are more flexible and cost-effective than traditional RCTs. Pragmatic trials are more reflective of real-life practice and more inclusive of diverse populations since they are embedded in health care systems. The FDA strongly recommends PCT design for postmarket studies where the trials are integrated into routine care and conducted in diverse settings to ensure broad applicability. For chronic diseases, pragmatic trials can answer questions that arise after initial approval, like the best order of treatments or which patients will benefit most. These trials can be designed to be more efficient and cost-effective, making them accessible to underresourced settings. This is crucial for conditions like diabetes and obesity, which are more common in minority and low-income populations. By using electronic health records, wearable sensors, and virtual methods, these trials can enroll diverse patients at a lower cost. FDA believes that PCTs offers a balanced approach between traditional RCTs and observational studies. It uses advances in technology and methodology to evaluate medical treatments in real-world settings, providing valuable insights at a lower cost. This research can inform regulatory decisions and help patients, doctors, and health systems make better treatments. AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: [email protected] Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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