To mark Rare Disease Day 2026, a new article authored by Billy Amzal, Head of Strategic Consulting at Phastar, explores how external control arms, Bayesian borrowing, and AI-enabled digital twins can strengthen evidence generation and accelerate rare disease drug development, while meeting evolving expectations from regulators such as the FDA and MHRA.
”Innovative trial designs that harness data augmentation, RWD, and the power of AI are enabling a new era of rare disease research. However, seizing the opportunities on offer requires careful planning and execution. By working with expert statisticians and data scientists, pharma and biotech companies can de-risk development, improve evidence generation, and unlock a new era of research that accelerates rare disease drug development.”
Read the article to see how these model-informed approaches can help de-risk development and bring treatments to rare disease patients faster.