Cargando…

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

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 evalu...

Descripción completa

Detalles Bibliográficos
Autores principales: Domingues, Cátia, Jarak, Ivana, Veiga, Francisco, Dourado, Marília, Figueiras, Ana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10610377/
https://www.ncbi.nlm.nih.gov/pubmed/37896191
http://dx.doi.org/10.3390/pharmaceutics15102431
_version_ 1785128240156770304
author Domingues, Cátia
Jarak, Ivana
Veiga, Francisco
Dourado, Marília
Figueiras, Ana
author_facet Domingues, Cátia
Jarak, Ivana
Veiga, Francisco
Dourado, Marília
Figueiras, Ana
author_sort Domingues, Cátia
collection PubMed
description 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.
format Online
Article
Text
id pubmed-10610377
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-106103772023-10-28 Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies Domingues, Cátia Jarak, Ivana Veiga, Francisco Dourado, Marília Figueiras, Ana Pharmaceutics Review 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. MDPI 2023-10-06 /pmc/articles/PMC10610377/ /pubmed/37896191 http://dx.doi.org/10.3390/pharmaceutics15102431 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Domingues, Cátia
Jarak, Ivana
Veiga, Francisco
Dourado, Marília
Figueiras, Ana
Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title_full Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title_fullStr Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title_full_unstemmed Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title_short Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies
title_sort pediatric drug development: reviewing challenges and opportunities by tracking innovative therapies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10610377/
https://www.ncbi.nlm.nih.gov/pubmed/37896191
http://dx.doi.org/10.3390/pharmaceutics15102431
work_keys_str_mv AT dominguescatia pediatricdrugdevelopmentreviewingchallengesandopportunitiesbytrackinginnovativetherapies
AT jarakivana pediatricdrugdevelopmentreviewingchallengesandopportunitiesbytrackinginnovativetherapies
AT veigafrancisco pediatricdrugdevelopmentreviewingchallengesandopportunitiesbytrackinginnovativetherapies
AT douradomarilia pediatricdrugdevelopmentreviewingchallengesandopportunitiesbytrackinginnovativetherapies
AT figueirasana pediatricdrugdevelopmentreviewingchallengesandopportunitiesbytrackinginnovativetherapies