Drug development in rare diseases requires the most efficient use of limited data, across both clinical trials and real-world settings. In this webinar, we will explore how combining predictive disease and care modeling with real-world data (RWD) can help translate efficacy observed in clinical trials into real-world effectiveness.
Using practical examples from rare disease settings, we’ll demonstrate how bridging modeling studies can augment trial data to support regulatory and access decisions, while also addressing key data requirements and limitations.
What You’ll Learn
- The difference between efficacy and effectiveness, and why it matters for health authorities
- What a bridging modeling study is and how it works
- How RWD can extend clinical trial insights to real-world populations
- Key use cases and impact on regulatory and strategic decision-making
Speaker:
Billy Amzal, Ph.D., Head of Strategic Consulting, Phastar
