This keynote presentation by Mark McClellan, Director of the Duke-Margolis Institute for Health Policy and former commissioner of the U.S. Food and Drug Administration, provides a thought-provoking analysis of the current state of Real-World Data and Evidence in regulatory use. Major themes include “regulatory grade” RWD/E, audit readiness and data fitness, learning health systems, and fit-for-purpose clinical trial design.
Following the keynote, a panel of experts discussed what’s on the regulatory horizon, from the use of alternative data sources to novel trial designs to AI-generated evidence.
This paper examines the benefits and challenges of using supplemental and external controls in regulatory submissions. Data platforms are an integral part of the ecosystem, enabling trial sponsors to access and use patient data from real-world settings and prior clinical trials. The paper recommends specific steps data platforms can take to support sponsors in meeting health authority expectations.
Innovative trial designs that make use of the totality of available evidence can reduce the burden on patients and meaningfully improve the drug development process. In February 2023, the FDA released for public comment “