Recent Trends in Drug Safety: The Growing and Evolving Role of Real-World Evidence

SUMMER 2022, THE EVIDENCE FORUM, WHITE PAPER

Summary

Historically, major drug safety issues have contributed to an evolution of the regulatory framework for drug safety, particularly in the post-approval period. In recent years, real-world evidence (RWE), derived from real-world data (RWD), has been playing an increasingly fundamental role in the post-authorization supervision of medicines. In this new era, changes are not only driven by emerging drug safety issues, but rather by a wider regulatory context on evidence needs throughout the development and lifecycle management of drugs.

In this paper, we outline the shifting landscape of safety studies over the years and explore the recent regulatory guidelines issued by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) from the perspective of post-approval and post-marketing safety evaluation of drugs and biologicals to bring a clearer global picture of:

• Shifts in the regulatory environment
• Evolving approaches with real-world data sources
• Consequences in terms of needs related to the use of real-world evidence for post-approval/authorization research
• Expectations on the future of post-marketing safety studies

Authors

Delphine Saragoussi, MD, MScPH, Executive Director, Strategic and Scientific Affairs, Real-World Evidence
Evidera, a business of PPD, part of Thermo Fisher Scientific

Sara Angleman, PhD, MPhil
Senior Research Associate, Strategic and Scientific Affairs, Real-World Evidence
Evidera, a business of PPD, part of Thermo Fisher Scientific

Katheryne Downes, PhD, MPH, Research Scientist, Strategic and Scientific Affairs, Real-World Evidence
Evidera, a business of PPD, part of Thermo Fisher Scientific

Debra A. Schaumberg, ScD, OD, MPH, Vice President and Head, Strategic and Scientific Affairs, Real-World Evidence
Evidera, a business of PPD, part of Thermo Fisher Scientific