ReviewCPT: pharmacometrics & systems pharmacology2025
State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases.
Review in CPT: pharmacometrics & systems pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
11 citing papers in PubMed.
- Integration of biological avatars and digital twins for "ex vivo clinical trials".EBioMedicine · 2026Review
- Translational Paradox of Triplet Repeat Expansion Disorders: Synthesizing Clinical Trial Failures to Guide Future Therapeutics.Clinical pharmacology and therapeutics · 2026Review
- Model-Informed Decision Making from Drug Discovery to Reimbursement: A Roadmap for Clinical Pharmacology in Value-Based Healthcare.Journal of clinical pharmacology · 2026Review
- Empowering Clinical Development With Disease Progression Modeling: Recommendations From the Clinical Trials Transformation Initiative.Clinical and translational science · 2026Article
- Innovative Clinical Pharmacology, Modeling, and Simulation Strategies for Accelerating Rare Disease Drug Development.Journal of clinical pharmacology · 2026Review
- From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026Review
- Global Trends in Integrating Machine Learning (ML) with Model-Informed Drug Development (MIDD): A Bibliometric and Systematic Review (2015-2025).Pharmaceutics · 2026Review
- Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals.Clinical and translational science · 2026Article
- Consensus for the most suitable trial design to assess therapy for rare vascular malformations: a Delphi study.Scientific reports · 2026Article
- Practical Pharmacokinetic-Pharmacodynamic Models in Oncology.Pharmaceutics · 2025Review
- The Advance of In Silico Evidence to Transform Pediatric Drug Development for Rare Diseases.CPT: pharmacometrics & systems pharmacology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
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 integration. We highlight how these methods facilitate dose optimization, support extrapolation from adult or other pediatric data, and enable more efficient and ethical clinical trial strategies. Case studies from recent regulatory submissions illustrate the growing acceptance of MIDD in pediatric rare disease contexts. Finally, we discuss the technological and regulatory advances driving this field forward, as well as current limitations and future opportunities for expanding the impact of MIDD on accelerating safe and effective treatments for children with rare diseases.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.