Model-informed drug development is revolutionizing the way we approach rare diseases, where traditional large-scale clinical trials may be impractical or even unethical. By integrating nonclinical and clinical data, these quantitative approaches provide a comprehensive understanding of a drug’s risk–benefit profile, helping to reduce uncertainty in technical and regulatory outcomes.
In this publication, you’ll discover how innovative modeling techniques—such as quantitative systems pharmacology, disease progression modeling, and artificial intelligence—are transforming the landscape of drug development. Real-world case studies will showcase how these tools can optimize trial design, reduce the need for additional clinical data, and accelerate the path to regulatory approval.
Key Takeaways:
- Learn how model-informed approaches maximize the use of all available data to support rare disease drug development.
- Explore novel strategies like patient-reported outcomes, external control arms, and clinical trial simulations.
- Read real-world examples and case studies demonstrating the impact of these techniques on regulatory review.
- Gain insights into the future of rare disease drug development and the role of advanced modeling in shaping that future.