A RESEARCH ON REGULATORY REQUIREMENTS FOR PEDIATRIC MEDICINES: INDIA, EU, AND USA

Sanjyot Sanjay Jagtap*, Shoaib Ali

Children are not simply small adults; their pharmacokinetic and pharmacodynamic profiles differ substantially across neonatal, infant, toddler, child and adolescent sub-populations, and this physiological heterogeneity has historically left the paediatric population as “therapeutic orphans”, treated largely with off-label and unlicensed medicines lacking dedicated safety and efficacy data. This dissertation undertakes a comparative regulatory analysis of the legal, procedural and administrative requirements governing the development, evaluation and marketing authorisation of pediatric medicines in three major jurisdictions — India, the European Union and the United States of America. The study traces the evolution of pediatric medicine regulation from the International Conference on Harmonisation guideline ICH E11 through region-specific legislative instruments: the Drugs and Cosmetics Act, 1940 and the New Drugs and Clinical Trials Rules, 2019 together with the National List of Essential Medicines for children in India; Regulation (EC) No 1901/2006 (the “Paediatric Regulation”), the Paediatric Committee (PDCO), the Paediatric Investigation Plan (PIP) and the Paediatric-Use Marketing Authorisation (PUMA) mechanism in the European Union; and the Pediatric Research Equity Act (PREA), the Best Pharmaceuticals for Children Act (BPCA) and the FDA Safety and Innovation Act (FDASIA) in the United States. A structured review of literature (2011–2026), a comparative evaluation of ethics committee composition, informed consent/assent requirements, waiver and deferral mechanisms, incentive structures and post-marketing pharmacovigilance obligations was carried out. Three comparative case studies — the diethylene glycol/ethylene glycol-contaminated pediatric cough syrup episodes (2022–2025), the first European Paediatric-Use Marketing Authorisation for midazolam oromucosal solution (Buccolam®), and the persistence of off-label prescribing in pediatric practice — were analysed to illustrate how the three regulatory systems respond in practice. The findings indicate that while the EU and the USA operate mandatory, incentive-linked pediatric development frameworks with dedicated committees, waiver/deferral systems and exclusivity-based incentives, India continues to rely on general clinical trial provisions supplemented by ethics-committee-level pediatric expertise, without a dedicated, mandatory pediatric investigation requirement equivalent to the PIP or iPSP. The dissertation concludes with a comparative regulatory pathway for market entry of pediatric medicines in the three jurisdictions and offers recommendations — including the introduction of a mandatory Pediatric Study Plan requirement, harmonised age-appropriate formulation guidance, and strengthened post-market surveillance — to strengthen India's pediatric regulatory framework, together with directions for future research.