Publication: pharmaphorum
Rare disease drug development faces enormous hurdles—tiny patient populations, unpredictable disease progression, and the high cost of traditional trials. This article explores how Virtual Twin® models and virtual trials are transforming the landscape, making research faster, more inclusive, and less dependent on large patient groups. With real-world examples like Pompe disease and insights into regulatory evolution, it highlights a future where advanced simulation drives breakthroughs in personalized medicine.
Author: Piet van der Graaf
Year: 2025 年 7 月 17 日
How Biosimulation is Transforming Rare Disease Drug Development
In this expert video, Piet van der Graaf, PharmD, PhD, explains how biosimulation is reshaping rare disease drug development, using a powerful example from our work with Sanofi on Pompe Disease. This innovative approach is accelerating development, reducing risks, and ultimately bringing life-changing therapies to patients faster.
