Domingues Cátia, Jarak Ivana, Veiga Francisco, Dourado Marília, Figueiras Ana
Univ Coimbra, Laboratory of Drug Development and Technologies, Faculty of Pharmacy, 3000-548 Coimbra, Portugal.
LAQV-REQUIMTE, Laboratory of Drug Development and Technologies, Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.
Pharmaceutics. 2023 Oct 6;15(10):2431. doi: 10.3390/pharmaceutics15102431.
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.
儿科药物研发模式一直在以“胡萝卜加大棒”的策略不断演变，以解决特定人群的问题。然而，给儿科患者超说明书使用成人药物仍是临床实践的一个特点，这可能会影响对适合该年龄段治疗方法的评估。因此，美国和欧洲儿科制剂倡议组织建议应用基于纳米技术的给药系统来应对其中一些挑战，特别是应用无机、聚合物和脂质基纳米颗粒。与此相关的是，先进治疗药物产品（ATMPs）也受到了关注，对儿科人群前景乐观。尽管使用这些创新疗法取得了一些成果，但汇集儿科患者和/或护理人员、医疗保健利益相关者、药物研发人员和医生的工作队伍对于推动儿科药物研发的标准化指南、实现从实验室到临床的快速转化仍然至关重要。因此，考虑到这一主题的重要性，本研究旨在通过以下方式梳理儿科药物研发的现状：（1）概述历史监管概况；（2）总结儿科药物制剂研发中的挑战；（3）阐述在儿科使用纳米药物和ATMPs等创新方法的优缺点。此外，还将关注制药技术专家和研发人员在儿科药物研发中的作用。
