An overview and comparison of regulatory pathways and guidelines for pediatric study plans in the US and EU

Abstract

Pediatric research is critical to the development of safe and effective treatments for children. The US Food and Drug Administration (FDA) and the European Union (EU) have produced guidelines to promote and oversee pediatric research. Initial Pediatric Study Plan (iPSP) and Paediatric Investigation Plan (PIP) are regulatory documents aiming to ensure therapeutic product development includes studies in pediatric patients. The US FDA requires an iPSP, in the development of all new medications that are not approved for pediatric use. In the European Union (EU), a PIP is required for all new medicinal products, including those intended for adults, unless a waiver or deferral is granted. An iPSP and PIP are similar documents that outline similar information on the drug's pharmacokinetics, safety, and efficacy in young patients. However, there are minor discrepancies between the two documents' requirements. This article provides an overview of iPSP and PIP requirements and highlights the similarities and differences between the two plans. For instance, the iPSP is only necessary for pharmaceuticals that have not yet been licensed for use, whereas the PIP is required for all new medications. In addition, the PIP includes more detailed information on the proposed studies and timelines for completion. In conclusion, regulatory agencies in both the US and EU have developed guidelines to promote and regulate pediatric studies. While their approaches differ, the overarching goal is to ensure children get safe and effective treatments.