Author: [AUTHOR] Published on 10/16/2024 12:00:00 AM
CALL FOR PAPERS
Clinical Pharmacology & Therapeutics: Pharmacometrics and Systems Pharmacology (PSP), an official journal of the American Society for Clinical Pharmacology & Therapeutics (ASCPT), is inviting submissions for a special themed issue on Model-Based Innovations in Pediatric Rare Disease scheduled for publication in the 4th quarter of 2025.
This special issue will showcase the application of pharmacometrics and systems pharmacology research innovation in the development of therapeutics and advances in understanding disease progression for pediatric rare diseases.
The Orphan Drug Act of 1983 in the US defined a rare disease as a disease or condition affecting less than 200,000 people. The early onset of many rare diseases poses unique challenges to drug development for pediatric rare diseases. These include the classical challenges for pediatric drug development, such as difficulties with recruitment, study design, and lack of assessment tools. Additionally, rare diseases present further challenges, such as a small patient population and often a lack of appropriate outcome measures or relevant prior experience in the adult populations. Pharmacometrics and system pharmacology are established tools to guide drug development and disease understanding across various therapeutic areas and patient populations. This themed issue seeks to spotlight the application of pharmacometrics and systems pharmacology to support and streamline clinical development programs for pediatric rare diseases (pRD), including trial design, biomarker selection, sample size determination, and dose optimisation.
The “Model-Based Innovation in Pediatric Rare Disease” themed issue welcomes the submission of topics that highlight recent advances in pRD:
- Mechanistic and quantitative systems pharmacology modeling
- Leveraging multiple data sources (e.g., real-world data, natural history, registries) to support pRD drug development
- Complementary modeling application using artificial intelligence/machine learning
- Application of model-informed Drug development (MIDD) through case studies
- New approaches to clinical trial designs and simulation studies supported by MIDD
- Exposure-response modeling
- Modeling for developing novel modalities (e.g. gene therapy, oligonucleotides etc.)
The objective of this issue is to illustrate the role of PSP in understanding disease progression and efficient dose selection in pediatric rare diseases.
Original research articles are encouraged. Submissions may also include reviews/mini-reviews, tutorials, position/white papers, PSP case reports, and perspectives from regulatory agencies, academia, societies, and industry. Please contact alethea@ascpt.org for details about manuscript types and format requirements.
To be considered for publication in this themed issue, manuscripts should be submitted via the online submission and tracking system by May 15, 2025.