ReviewThe AAPS journal2013
Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants.
Review in The AAPS journal, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 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
18 citing papers in PubMed, 29 citations in OpenAlex.
- More Than a Question of Correlation: Characterization of the Evidentiary Basis for Biomarker Surrogates Used in European Marketing Authorizations.Clinical pharmacology and therapeutics · 2026Review
- Exploring Recent Developments in the Manifestation, Diagnosis, and Treatment of Patients with Smith-Lemli-Opitz Syndrome: From Molecular Pathways to Clinical Innovations.International journal of molecular sciences · 2025Review
- Precision medicine in regulatory decision making: Biomarkers used for patient selection in European Public Assessment Reports from 2018 to 2020.Clinical and translational science · 2023Article
- Comprehensive and deep profiling of the plasma proteome with protein corona on zeolite NaY.Journal of pharmaceutical analysis · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Scientific considerations for global drug development.Science translational medicine · 2020Review
- The Revolution in Pediatric Drug Development and Drug Use: Therapeutic Orphans No More.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2020Article
- Role of Patients and Parents in Pediatric Drug Development.Therapeutic innovation & regulatory science · 2019Article
- Innovative Study Designs Optimizing Clinical Pharmacology Research in Infants and Children.Journal of clinical pharmacology · 2018Review
- Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Orphanet journal of rare diseases · 2018Review
- Useful pharmacodynamic endpoints in children: selection, measurement, and next steps.Pediatric research · 2018Review
- Drugs for rare disorders.British journal of clinical pharmacology · 2017Article
- Collaboration in Regulatory Science to Facilitate Therapeutic Development for Neonates.Current pharmaceutical design · 2017Review
- Clinical Pharmacology Studies in Critically Ill Children.Pharmaceutical research · 2017Review
- Effect of Kidney Function on Drug Kinetics and Dosing in Neonates, Infants, and Children.Clinical pharmacokinetics · 2015Review
- Neonatal drug therapy: The first frontier of therapeutics for children.Clinical pharmacology and therapeutics · 2015Review
- 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands.Neuromuscular disorders : NMD · 2015Article
- Regulatory science needs for neonates: a call for neonatal community collaboration and innovation.Frontiers in pediatrics · 2014Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There are similar challenges in developing a product designed to treat patients with a rare disease and drugs to treat critically ill neonates and infants. Part of the challenge in developing such products as well as identifying the optimal dosing regimen for the treatment of young children arises from the complex interrelationship between developmental changes and changes in biomarkers responsive to drug therapy. These difficulties are further compounded by our lack of understanding of the key physiological factors that cause the differences in clinical responses between adults and neonates and infants. Regulatory efforts have succeeded in overcoming these challenges in many areas of pediatric and orphan drug development. Strategic applications of biomarkers and surrogate endpoints for the development and approval of a product used to treat an orphan disease will be highlighted with examples of approved products. Continued efforts are still needed to fill in our knowledge gap and to strategically link biomarkers and surrogate endpoints to clinical responses for rare diseases and diseases affecting neonates and infants.
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.