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Publication: pharmaphorum

The article “Digital Twins and Virtual Trials: A New Era in Rare Disease Drug Development” discusses how these advanced simulation technologies are transforming rare disease drug development. Digital twins refer to virtual representations of patients. They enable precise modeling of real-world responses to medicines, reducing reliance on traditional control groups and large patient populations. Virtual trials further enhance this by simulating the impacts of drugs across diverse scenarios. Leveraging these technologies not only expedites therapy development, but also drives personalized medicine, addressing underserved populations more effectively. The integration of AI-based modeling promises to revolutionize clinical trials, saving time and costs, while accelerating life-changing discoveries.

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.

Watch the videoContact us