Regulatory Challenges and Strategies for Pediatric Drugs in the EU and US

Abstract

The immense use of unlicensed and off-label medicines in pediatric care points to serious regulatory, ethical, and clinical issues. A subtlety in pediatric drug development and its regulation has been carried out, making it clear the big gap that exists between adult and pediatric pharmacotherapy. In fact, up to 50% of medicines prescribed for children across Europe fall into these categories, for the most part due to a paucity of drugs specifically approved for use in the pediatric population. This only goes to put pediatric patients at unnecessary risks of encountering drug side effects and inefficacies. This underscores the critical need for tailored drug development.

The main changes to the regulatory framework for the European Union (EU) and the United States (US) have been to respond to these new challenges with the implementation of landmark legislation aimed at the safe and effective administration of medicines to children. The Paediatric Regulation of the EU and the Pediatric Research Equity Act (PREA) of the US regulate full Pediatric Investigation Plans (PIPs) and, in fact, even give companies some incentives to make such studies happen. This regulatory approach aims at bridging the gap by ensuring that new and pre-existing medicines are systematically tested for pediatric use, hence expanding the rational arsenal of clinically validated pediatric drugs.

These advancements are not challenge-free, though, and the present article has pointed to some of those challenges that are yet to be cleared. The latter includes complexities in trial design, ethics surrounding enrolling children into clinical trials, and continuing use, in the market, of off-label medications. In this regard, innovative trial designs, like adaptive pathways and model-based approaches, may offer more flexibility and efficiency in collecting pediatric-specific data.

The review goes on further to reveal the ethical dimensions in conducting pediatric drug trials, whereby it highlights the fact that the business interests of the drug trials should be, in every way, subordinated to the health and well-being of the child participants. It discusses the importance of monitoring long-term safety and the role of pharmacovigilance in protecting pediatric patient populations. In conclusion, though, in an attempt at regulation, there has been a significant amelioration of the landscape of pediatric pharmacotherapy, substantial work remains. Some ways are better cooperation between the regulatory bodies, improvement of legislative frameworks, and increasing the quality of the ethical conduct of pediatric trials. This points to the urgency for a joint international effort to ensure that children, a very vulnerable group, have access to the best and safest therapeutic interventions that are most effective.