Convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University and supported by a cooperative agreement with FDA, the purpose of the public workshop is to bring the stakeholder community together to discuss a variety of topics related to the use of real-world data (RWD) and real-world evidence (RWE) in drug development and regulatory decision making. Topics will include an update on FDA's activities to address the use of RWE in regulatory decisions and the development of a framework for tackling challenges related to RWE's regulatory acceptability. In addition, panelists will discuss opportunities to improve data development activities, study designs, and analytical methods used to create robust RWE.
RWD (data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources) and RWE (clinical evidence regarding the usage and potential benefits or risks of a drug derived from analysis of RWD) are increasingly being used by multiple stakeholders within the health care system. Payers may rely on RWD and RWE to refine formularies or assist in coverage decisions. Physicians and professional societies can utilize RWE to further tailor clinical practice guidelines and decision-support tools. Medical product developers can use RWE to further develop a product's benefit-risk profile, monitor postmarket safety and adverse events, or generate additional hypotheses for continued clinical development.
The 21st Century Cures Act, section 3022 (Pub. L. 114-255), enacted on December 13, 2016, directed FDA to establish a program to evaluate the potential use of RWE. The framework of the program was to include information describing the sources of RWE, the gaps in data collection, standards and methods for collection and analysis, and the priority areas and challenges.
To date, RWD and RWE have been used in very specific regulatory contexts. Some treatments for rare diseases, for example, have utilized RWE as part of the historical controls used for clinical study and, ultimately, regulatory submission. Postmarket safety surveillance has also relied heavily on RWD-generating networks. As part of exploring the opportunities for enhanced use of these types of data and evidence in additional regulatory decision-making contexts, FDA is seeking input on the opportunities and challenges in using RWE to support the approval of a new indication for an already approved drug, and to help support or satisfy postapproval study requirements.
More information is available via the Federal Register notice.