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: July 17, 2025
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.
