All Times EDT
Keywords: Regulatory Guidance, RWD, RWE, RCT
The 21st Century Cures Act (Cures Act) encouraged the sponsor to explore and investigate the use of Real-World Data (RWD) and Real-World Evidence (RWE) in facilitating regulatory decision-making, including efficacy and safety demonstration of a drug. Since then, many databases with higher RWD quality and often built-in data extraction and analysis tools have been initiated. In 2021 the FDA published a series of guidance documents on RWD utilization standards and strategies in fulfilment of this mandate. Each guidance focusing on a specific topic, ranging from electronic health records (EHRs) and medical claims (“Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products”), to registries (“Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry”) including specific standards of RWD (“Data Standards for Drug and Biological Product Submissions Containing Real-World Data”) and other considerations (“Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products”) in regulatory decision-making. The open period for these draft guidance documents resulted in several comments on the public docket and discussions in the public domain. This panel brings together experts and stakeholders from industry, RWD vendors and FDA to discuss different aspects of the guidance and its implications. The panelists will address concerns and difficulties, and the impacts on implementing all or portions of these guidance while also speak to how RWD expedites the FDA review process and the rationale behind the requirements. Points of concurrence and potential enhancement in the guidance will be identified. Possible next steps for furthering the use of RWD in clinical research will also be explored.