ReviewJournal of pharmacokinetics and pharmacodynamics2023
Achieving big with small: quantitative clinical pharmacology tools for drug development in pediatric rare diseases.
Review in Journal of pharmacokinetics and pharmacodynamics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed.
- Phase 2, open-label, noncomparative clinical trial evaluating safety and efficacy of posaconazole in pediatric patients with proven/probable invasive aspergillosis or possible invasive fungal disease.Antimicrobial agents and chemotherapy · 2026Trial
- Safety, outcomes, and pharmacokinetics of isavuconazole as a treatment for invasive fungal diseases in pediatric patients: a non-comparative phase 2 trial.Antimicrobial agents and chemotherapy · 2024Trial
- From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026Review
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases.CPT: pharmacometrics & systems pharmacology · 2025Review
- Clinical pharmacology-how it shapes the drug development journey.European journal of clinical pharmacology · 2025Review
- Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists.Clinical and translational science · 2025Review
- Training the Next Generation of Pediatric Clinical Pharmacologists: Insights and Trainee Perspectives Over 10 Years.Journal of clinical pharmacology · 2025Article
- The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.Frontiers in 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
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pediatric populations represent a major fraction of rare diseases and compound the intrinsic challenges of pediatric drug development and drug development for rare diseases. The intertwined complexities of pediatric and rare disease populations impose unique challenges to clinical pharmacologists and require integration of novel clinical pharmacology and quantitative tools to overcome multiple hurdles during the discovery and development of new therapies. Drug development strategies for pediatric rare diseases continue to evolve to meet the inherent challenges and produce new medicines. Advances in quantitative clinical pharmacology research have been a key component in advancing pediatric rare disease research to accelerate drug development and inform regulatory decisions. This article will discuss the evolution of the regulatory landscape in pediatric rare diseases, the challenges encountered during the design of rare disease drug development programs and will highlight the use of innovative tools and potential solutions for future development programs.
Indexed as
Identifiers
37140724What 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.