Washington, DC—The Duke-Margolis Center for Health Policy released a new white paper today that explores when and how studies producing real-world evidence (RWE) using data already collected as part of routine clinical care can inform Food and Drug Administration (FDA) regulatory decisions related to a product’s effectiveness.
The paper, “Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility,” reflects the input of the nearly 40 members and observers of the Duke-Margolis RWE Collaborative.
“Real-world evidence has the potential to improve many aspects of health care decision making related to the effectiveness of marketed medical products, but only with a clear understanding of the quality and credibility of the evidence,” said Mark McClellan, MD, PhD, director of Duke-Margolis. “This work focuses on the potential regulatory use of observational studies of product effectiveness that rely on secondary data— data collected for other purposes such as billing, clinical care, and patient reports and monitoring.”
The authors described how observational studies using secondary data can better reflect the broader patient populations, care settings, and medical product uses that are typical of clinical practice and, in some cases, can be conducted more efficiently than traditional randomized clinical trials. The paper both explores ethical, operational, and resource barriers to conducting these studies as well as discusses various methods for conducting observational studies using secondary data, the biases they intend to address, and how they can be mapped to the characteristics of an adequate and well-controlled study as outlined in the Code of Federal Regulations to demonstrate their credibility.
Duke-Margolis will be hosting a webinar to further explore this work on January 15, 2020 (details to follow). The webinar will feature a discussion with expert stakeholders moderated by Dr. McClellan.
The Duke-Margolis RWE Collaborative engages stakeholders to guide high-priority efforts aimed at improving the development and use of RWE for regulatory decision-making. The Collaborative also strives to drive progress in the use of real-world data and evidence to improve patient treatment options and outcomes more broadly. Guided by an advisory group consisting of leaders representing medical product developers, payers, research groups, providers, patient networks, and regulators, the RWE Collaborative is interested in the following 2019 priorities:
- Determining Real-World Data’s Fitness for Use and the Role of Reliability
- Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility
- Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness
- Establishing Guideposts for Developing Real-World Endpoints