Today I’m writing a short blog to introduce Paediatric Investigation Plan (PIP) submissions to the European Medicines Agency (EMA).
What is a Paediatric Investigation Plan (PIP)?
• A PIP is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorisation of a medicine for children.
• All applications for marketing authorisations for new medicines have to include the results of studies, as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver.
• This requirement also applies when a marketing authorisation holder (MAH) wants to add a new indication, pharmaceutical form or route of administration for a medicine that is already authorised.
PIP deferrals and waivers
The Paediatric Committee (PDCO) is the EMA’s scientific committee responsible for activities on medicines for children, and may grant PIP deferrals for some medicines (for example until there is enough information to demonstrate its effectiveness and safety in adults). This will mean an applicant can delay development of the medicine in children. However the PIP will still need to include details of the paediatric studies and their timelines. For more detailed information please see here: PIP deferrals.
The PDCO may also grant waivers when development of a medicine in children is not needed or is not appropriate (for example for diseases that only affect the adult population). The EMA maintains a list of class waivers for medicines not requiring a PIP submission and you can find more information here: Class waivers for PIPs.
If you need to apply for a PIP deferral or waiver, see PIPs: questions and answers.
When should I submit the application for a PIP or waiver?
According to Article 16 of the Paediatric Regulation, applications should be submitted, unless duly justified, ‘not later than upon completion of the human pharmaco-kinetic (PK) studies’, as specified in Section 5.2.3 of Part 1 of Annex 1 of Directive 2001/83/EC. Recital 10 of the Regulation states that ‘paediatric investigation plans should be submitted early during product development, in time for studies to be conducted in the paediatric population, where appropriate, before marketing authorisation applications are submitted’.
The timing of submission should not be later than the end of healthy subject or patient PK, which can coincide with the initial tolerability studies, or the initiation of the adult phase-II studies (proof-of-concept studies); it cannot be after initiation of pivotal trials or confirmatory (phase-III) trials.
Applicants are welcome to submit their PIP applications during or even before initial PK studies in adults.
The EMA published submission deadlines, together with forms and templates, can be found here.
See our next blog, Paediatric Investigation Plans (PIPs) in the UK, for detail on how UK PIPs are handled by the Medicines and Healthcare Products Regulatory Agency (MHRA) following Brexit.
While the information in this article is considered to be true and correct at the date of publication, changes in guidance and/or experience may impact on the accuracy of the information. Please refer to the relevant guidance for the latest information.