Pediatric Study Plans (iPSP) and Pediatric Investigation Plans (PIP)

A Pediatric Investigation Plan (PIP) submission in the EU must include several key components to ensure it meets regulatory requirements. These components are: a detailed scientific rationale for including or excluding pediatric studies based on existing data and clinical need; a comprehensive outline of the planned pediatric studies, including objectives, design, methodologies, and timelines; information on formulations suitable for different pediatric age groups; a description of ethical considerations and measures taken to protect pediatric participants; justification for any requested waivers or deferrals for specific pediatric studies, if applicable; and assurance that the submission complies with current regulatory guidelines and requirements. 

In the US, a waiver for an Initial Pediatric Study Plan (iPSP) can be requested under specific circumstances. Such waivers may be granted if the drug or biologic product is intended for a condition that does not affect the pediatric population. Additionally, a waiver may be appropriate if the product is unlikely to be used by a significant number of pediatric patients due to the rarity of the disease or condition in children. If existing data strongly suggest that the product would be ineffective or unsafe for pediatric use, a waiver could also be considered. Other justifications supported by robust evidence may also warrant a waiver.

For timing, the iPSP should be submitted by the end of Phase I clinical trials in the US. This ensures that pediatric considerations are addressed before advancing to later stages of development.

In the EU, a waiver for a Pediatric Investigation Plan (PIP) can be requested under different criteria. A waiver may be granted if the medicinal product is likely to be ineffective or unsafe for part or all of the pediatric population. Another reason for requesting a waiver is if the disease or condition the medicine is intended to treat occurs exclusively in adults. Additionally, if the medicine does not provide a significant therapeutic benefit over existing treatments for pediatric patients, a waiver might be justified.

For PIPs in the EU, the plan must be submitted early in the clinical development process, typically before the start of Phase II/III trials. This early submission allows for timely integration of pediatric considerations into the development strategy.

The requirements for pediatric study plans primarily apply to pharmaceutical drugs and biological products. However, for medical devices, there are distinct regulations and requirements both in the EU and the US, focusing on ensuring the safety and effectiveness of medical devices for pediatric use. In the EU, medical devices are regulated under the Medical Device Regulation (MDR) (EU) 2017/745, which came into full application on May 26, 2021. The regulation emphasizes the need to consider pediatric use where applicable.