A Paediatric Investigation Plan (PIP) is a development programme reviewed by the European Medicines Agency that describes how the efficacy, safety and appropriate use of a medicinal product in children and adolescents are to be investigated.
The PIP is a key element of the EU Paediatric Regulation and is intended to ensure that paediatric data are collected systematically—rather than deriving paediatric treatment solely from adult data.
What is a Paediatric Investigation Plan used for?
The PIP defines which studies, formulation development and other measures are required to assess a medicinal product for paediatric age groups.
Typical objectives include:
- Definition of appropriate age cohorts (e.g. newborns, infants, children, adolescents)
- Specification of clinical studies and, where applicable, non-clinical investigations
- Development of a child-appropriate dosage form (e.g. syrup, orally disintegrating tablet, dosing aids)
- Planning of pharmacokinetic/pharmacodynamic investigations and safety monitoring
When must a PIP be submitted?
In the EU, a PIP is generally required when marketing authorisation is sought for a new medicinal product or a new indication and the disease may also be relevant in childhood.
The PIP is typically submitted early in development (often before Phase I/II planning is finalised), as many requirements are time-critical.
Who assesses the PIP?
The scientific assessment is carried out by the EMA’s Paediatric Committee (PDCO). The PDCO may:
- approve the PIP (where applicable, with conditions)
- request changes (modification of the plan)
- reject it if the rationale or methodology is not convincing
Deferral and waiver: important exceptions
In many development programmes, exemptions are used:
- Deferral: certain paediatric studies may be postponed (e.g. until more adult data are available).
- Waiver: a (partial or full) exemption from the obligation if the medicinal product is not suitable for children or the indication is not relevant in childhood.
Why is the PIP also economically relevant?
An approved PIP can structure regulatory obligations, but can also trigger incentive mechanisms (e.g. extension of the supplementary protection certificate or additional market exclusivity in certain constellations). At the same time, it affects the cost, timeline and feasibility of the development programme.
FAQ
Is a PIP the same as a paediatric study protocol?
No. The PIP is an overarching development programme and may include multiple studies and measures. Individual protocols are more detailed and are developed later.
Does the PIP also apply to generics?
Generics are often subject to exemptions; the specific obligation depends on the authorisation pathway and the indication applied for.
Can a PIP be amended retrospectively?
Yes. PIPs are often adjusted during development, for example when new data become available or studies are not feasible in practice.