What is a Pediatric Investigation Plan?

A Pediatric Investigation Plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorization of a medicine for pediatric use. PIPs are a crucial aspect of the drug development process, mandated by regulatory authorities such as the European Medicines Agency (EMA) to improve the health of children across the world by ensuring that medicines intended for use in children are of high quality, ethically researched, and appropriately authorized.

What is the Importance of a PIP?

The importance of Pediatric Investigation Plans cannot be overstated. Historically, a significant portion of medicines used in children were not tested for pediatric use, leading to a lack of appropriate dosing information, potential safety concerns, and an overall scarcity of medicines specifically authorized for children. The implementation of PIPs addresses these issues by:

Ensuring Safety and Efficacy: PIPs ensure that pharmaceutical companies conduct the necessary research to verify that medicines are safe and effective for use in pediatric populations.

Bridging Knowledge Gaps: They help bridge the gap in medical knowledge regarding the treatment of various diseases in children, contributing to more evidence-based pediatric care.

Promoting Innovation:By requiring research into pediatric uses of medicines, PIPs encourage innovation in developing treatments that are specifically designed for or can be adapted to children.

Roles and Functions of a PIP

Planning and Assessment The primary role of a PIP is to outline a detailed plan for the development of a medicine for children of all age groups, from birth to adolescence. This includes the timing of studies, the age groups involved, the methods to be used, and how the studies will contribute to understanding the medicine's efficacy and safety in children.

Research and Development Guidance PIPs serve as a guide for pharmaceutical companies during the research and development phases of a medicine, indicating when and how pediatric studies should be integrated into the overall development program. This ensures that pediatric research is considered early in the development process and carried out efficiently.

Regulatory Requirement A PIP is a regulatory requirement for the marketing authorization of a new medicine in many jurisdictions. It must be agreed upon with the relevant regulatory authority before certain applications for marketing authorization can be submitted. This regulatory role underscores the importance of PIPs in the official approval process for new medicines.

Incentives and Obligations Regulatory frameworks surrounding PIPs often include incentives for pharmaceutical companies to conduct pediatric research, such as patent extensions or market exclusivity. However, these incentives come with obligations to actually conduct the research as outlined in the PIP, ensuring that companies follow through on their commitments to pediatric medicine development.

Ethical Assurance PIPs ensure that pediatric studies are conducted ethically, with minimal risk to participants. They require that studies are scientifically necessary and designed in a way that respects the unique considerations of treating children, including informed consent and assent processes.