Review
Open access
Jul 2026
From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development
Clinical pharmacology is expected to continue playing an important role in evaluating mechanistic plausibility, ensuring analytic rigor, and translating small datasets into meaningful evidence to inform development and regulatory decisions in rare diseases.
Yan Xu, Alissa Verone-Boyle, Natalie Schmitz et al.
· Journal of clinical pharmaco... · 0 citations