ABSTRACT

Paediatric drug development presents a distinct set of challenges, ranging from economic constraints and formulation complexities to ethical considerations and regulatory hurdles. Economic constraints establishing from smaller market sizes and prolonged development timelines discourage investment in this specialized field. This review charts the evolution of paediatric drug regulations in the United States and Europe, focusing on landmark legislative measures like the paediatric Labelling Rule, the Best Pharmaceuticals for Children Act (BPCA), and the Paediatric Research Equity Act (PREA). These regulatory frameworks have incentivized pharmaceutical companies to undertake paediatric trials, thereby enhancing the safety and labelling standards of medications prescribed to children. The selection of excipients in paediatric formulations demands challenges consideration to ensure both safety and palatability, given the heightened sensitivity of young patients. Selection of dosage forms to suit age-specific requirements further complicates drug formulation and design, posing additional challenges. Ethical considerations play a pivotal role, necessitating stringent protocols for informed consent and ensuring protections for vulnerable paediatric populations. Scientifically, the unique pharmacokinetic and pharmacodynamics profiles in children mandate precise dosing strategies and formulation adjustments, increasingly supported by advanced Modelling and Simulation (M&S) techniques. As the field progresses, collaborative efforts and innovative methodologies are crucial to advancing paediatric therapeutics, ensuring safe andefficacious treatments for young patients worldwide. Efforts to streamline regulatory processes, coupled with advances in telemedicine and biomarker utilization, promise to bolster paediatric medication development, thereby enhancing healthcare outcomes for paediatric populations worldwide. Keywords: Paediatric drug development, formulation complexities, Paediatric Labelling Rule, Best Pharmaceuticals for Children Act (BPCA), Paediatric Research Equity Act (PREA), telemedicine
Publication date: 01/12/2025

https://ijbpas.com/pdf/2025/December/MS_IJBPAS_2025_9646.pdf

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https://doi.org/10.31032/IJBPAS/2025/14.12.9646