Please use this identifier to cite or link to this item: https://hdl.handle.net/10316/111848
Title: Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
Authors: Domingues, Cátia 
Jarak, Ivana 
Veiga, Francisco 
Dourado, Marília 
Figueiras, Ana 
Keywords: pediatrics; nanoparticles; gene therapy; cell- and tissue-based therapy
Issue Date: 6-Oct-2023
Publisher: MDPI
Project: PTDC/NAN-MAT/1431/2021 
Ph.D. grant 2021.08095.BD 
Serial title, monograph or event: Pharmaceutics
Volume: 15
Issue: 10
Abstract: The paradigm of pediatric drug development has been evolving in a "carrot-and-stick"-based tactic to address population-specific issues. However, the off-label prescription of adult medicines to pediatric patients remains a feature of clinical practice, which may compromise the age-appropriate evaluation of treatments. Therefore, the United States and the European Pediatric Formulation Initiative have recommended applying nanotechnology-based delivery systems to tackle some of these challenges, particularly applying inorganic, polymeric, and lipid-based nanoparticles. Connected with these, advanced therapy medicinal products (ATMPs) have also been highlighted, with optimistic perspectives for the pediatric population. Despite the results achieved using these innovative therapies, a workforce that congregates pediatric patients and/or caregivers, healthcare stakeholders, drug developers, and physicians continues to be of utmost relevance to promote standardized guidelines for pediatric drug development, enabling a fast lab-to-clinical translation. Therefore, taking into consideration the significance of this topic, this work aims to compile the current landscape of pediatric drug development by (1) outlining the historic regulatory panorama, (2) summarizing the challenges in the development of pediatric drug formulation, and (3) delineating the advantages/disadvantages of using innovative approaches, such as nanomedicines and ATMPs in pediatrics. Moreover, some attention will be given to the role of pharmaceutical technologists and developers in conceiving pediatric medicines.
URI: https://hdl.handle.net/10316/111848
ISSN: 1999-4923
DOI: 10.3390/pharmaceutics15102431
Rights: openAccess
Appears in Collections:FFUC- Artigos em Revistas Internacionais
I&D ICBR - Artigos em Revistas Internacionais
FMUC Medicina - Artigos em Revistas Internacionais
I&D CEISUC - Artigos em Revistas Internacionais

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