PN002 - Drug Regulatory Sciences and Real-World Evidence

The current drug regulatory landscape is shifting, driven by need to keep up pace with scientific and technological advancements. Using science to inform regulatory decision making throughout the drug life-cycle is essential. This is the domain of regulatory science. Regulatory science is essential to improve regulatory effectiveness and efficiency and to enable timely access to new therapies. As such, regulatory science has become a key focus of regulatory agencies, including the EMA and FDA. Pharmacoepidemiological methods are often applied to regulatory science related questions and use of Real World Evidence in decision making on medicines. Current developments of pharmacoepidemiological methods are often driven or intertwined with new needs and challenges within the drug regulatory field. [1] There is a striking imbalance between the contribution of RWE/observational data to safety compared to efficacy/effectiveness issues to be sorted out. Can we improve? [2] RWE/observational data enable better insight in the landscape of the exposure-outcome association under regulatory review. Do regulators like context? [3] More sophistication of RWE/observational data has not necessarily resulted in better and informed regulatory decisions. Do we understand why?

Learning objectives:
- to understand how pharmacoepidemiological methods can be applied to drug regulatory science
- to understand the importance of drug regulatory science for better decision making on medicines
- to reflect on the future challenges within drug regulation and how the field of pharmacoepidemiology can play a role in analyzing and using real-world evidence for advancing drug regulation.