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 Bayesian methods continue to gain momentum as an alternative approach to traditional clinical trial design, yet many organizations still have limited experience applying them, particularly when engaging with regulators. With multiple global agencies releasing further guidance in this area, it is useful to understand the current landscape of this trial area.
In this webinar, we present findings from a systematic review of Bayesian clinical trials identified on ClinicalTrials.gov, exploring how adoption has evolved over time.
We’ll also introduce ClinicalTrials.gov as a practical and accessible resource for conducting systematic reviews outside of academic settings.
Using a recent systemic review , this session will provide clarity on where Bayesian methods are being applied, their growing role in trial design, and how they can support regulatory discussions.
What You’ll Learn
Key considerations when introducing Bayesian approaches into development
Where and how Bayesian methods are being used in clinical trials today
How adoption has evolved over time across therapy areas
The potential for regulatory precedent when using Bayesian designs
How to conduct systematic reviews using ClinicalTrials.gov
- 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
Speakers:
Giles Partington, Consultant Statistician, Phastar
Sarah Crossley, Apprentice Statistician, Phastar
