State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases.

ABSTRACT: Pediatric rare diseases present unique challenges for drug development due to small patient populations, ethical constraints on clinical trial design, and limited prospectively defined natural history data. Model-Informed Drug Development (MIDD) has emerged as a powerful paradigm to address these challenges by leveraging quantitative methods to enhance decision-making across all stages of drug development. This paper reviews the state-of-the-art MIDD approaches being applied to pediatric rare disease therapeutics, including the traditional pharmacometrics methodologies of population pharmacokinetic/pharmacodynamic (PK/PD) modeling, physiologically based pharmacokinetic (PBPK) modeling, disease progression modeling, and more future-facing Bayesian trial designs, and real-world data integrat

SUBMITTER: Krishna R 

PROVIDER: S-EPMC12625129 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature