Paediatric Medicines – Regulatory Drivers, Restraints, Opportunities and Challenges

• Published in: Journal of pharmaceutical sciences Vol. 110; no. 4; pp. 1545 - 1556
• Main Authors: Vieira, Isa, Sousa, João José, Vitorino, Carla
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.04.2021
• Subjects: Age-appropriate formulations; European Medicines Agency (EMA); Food and Drug Administration (FDA); Paediatric drug development; Paediatric regulation; EuPFI; PRAC; FDA; PDCO; QTPP; SPC; SmPC; European Medicines Agency (EMA); Age-appropriate formulations; GRiP; MA; CHMP; EMA; FDASIA; FDAAA; WR; EC; ODT; US; CQAs; WHO; USPFI; PeRC; STEP; Food and Drug Administration (FDA); Paediatric drug development; MAA; NIH; PSP; ICH; RA; Paediatric regulation; EU; R&D; QbD; BPCA; PAC; PD; MAH; PIP; API; PPSR; PK; PREA; PUMA; PmRN
• ISSN: 0022-3549; 1520-6017
• DOI: 10.1016/j.xphs.2020.12.036

The investment in the pharmaceutical development of medicines for paediatric use represents a minority when compared to that one made for adult population. Which reasons lie behind this status quo? Which policies have been implemented to reverse such asymmetry? Is there room to new regulatory initiatives? The creation of regulations establishing the obligation to conduct paediatric trials was deemed necessary as a means of producing products of proven quality, safety and efficacy and, in addition, to set forth financial incentives for the pharmaceutical industry reduce this delay. The first regulatory initiatives were carried out by the Food and Drug Administration (FDA) at the end of the 20th century. Later on, the European Medicines Agency (EMA) issued the Paediatric Regulation, which has boosted a closer collaboration between both regulatory agencies. Along with the implemented legislation, pharmaceutical dosage forms, more adapted to the paediatric population have emerged, increasing the availability of age-appropriate formulations. However, a case-by-case analysis is required to ensure the best therapeutic option for the specific child. This review aims at discussing the development of medicines for paediatric use from a regulatory perspective, comparing the policies adopted by the EMA and FDA, following an overview of the drivers, restraints, opportunities and challenges.