FDA Rare Disease Innovation Hub Future Programming; Request for Comments

UCB, Inc. and a team of academic experts submitted a proposal to include a workshop on Digital Twins and In Silico Trials in the FDA's Rare Disease Innovation Hub Future Programming. They argue that these computational strategies are essential for addressing the evidentiary challenges of developing treatments for rare diseases with small patient populations.

PhRMA, representing the biopharmaceutical research industry, supports the FDA's Rare Disease Innovation Hub and proposes specific topics for future workshops. They advocate for discussing innovative statistical approaches, predictive and mechanistic modeling, and Bayesian analyses to better establish associations between biomarkers and clinical outcomes in rare disease drug development.