This requirement also applies when a marketing-authorisation holder wants to add a new indication, pharmaceutical form or route of administration for a medicine that is already authorised and covered by intellectual property rights.
Joint procedural information is available from EMA and the United States Food and Drug Administration (FDA) for medicine developers submitting a PIP to EMA and an initial pediatric study plan (iPSP) to the FDA.
For more information, see:
Cancer medicines for use in children
Given the rarity of childhood cancers, this joint guidance aims to speed up the development and authorisation of cancer medicines for children. It describes the information the two regulators typically require for their evaluations to support:
EMA is developing a feasible and rigorous approach to support early and timely engagement with medicine developers on the development of medicines addressing unmet medical needs in children.
This is one of the main takeaways of a report covering the nearly three years of EMA's 'stepwise paediatric investigation plan (PIP)' pilot.
The pilot proposes an alternative to PIPs that are fully defined at the time of the initial PIP application. This approach introduces the concept of a partial development programme for innovative paediatric medicines.
It applies only to cases where there is a lack of crucial information needed to decide on key elements of a PIP. This includes rare and complex cases with major uncertainty.
Between 2023 and 2025, EMA received 27 eligibility requests and adopted eight stepwise PIPs, mainly in rare diseases and oncology.
A stepwise PIP is conditional on the development of a full PIP once sufficient evidence becomes available. It relies on predefined steps agreed with the EMA's Paediatric Committee (PDCO), such as applicants being able to hold discussions with the PDCO once they obtain more data.
Next steps in the pilot include:
The need for the stepwise PIP approach has been recognised in the proposed reform of the EU pharmaceutical legislation.
For guidance on how to apply for a stepwise PIP, see Paediatric investigation plans: questions and answers: Stepwise paediatric investigation plans (sPIPs).
Read the full pilot stepwise PIP pilot report below.
The PDCO may grant PIP deferrals for some medicines. These allow an applicant to delay development of the medicine in children until, for instance, there is enough information to demonstrate its effectiveness and safety in adults.
Even when studies are deferred, the PIP will include details of the paediatric studies and their timelines.
More information on requirements for companies is available under PIP deferrals.
The PDCO may also grant waivers when development of a medicine in children is not needed or is not appropriate, such as for diseases that only affect the adult population.
After assessing an application for a PIP, deferral, waiver or modification, the PDCO formulates an opinion, which is notified to the applicant. The applicant is then able to request a re-examination of the opinion if it wishes. Once the Committee has issued its final opinion, after re-examination if requested, EMA adopts a decision.
EMA makes all opinions and decisions on PIPs, deferrals and waivers public, after deletion of information of a commercially confidential nature.
Applicants can request scientific advice from EMA in preparation of a PIP, which is free of charge for questions relating to the development of paediatric medicines.
For more information, see:
Applicants must follow agreed PIPs exactly. Once the plan is complete, the European Medicines Agency or the medicines authorities in Member States check that companies comply with the agreed measures listed in each PIP.
These checks are necessary before the applicant can apply for a marketing authorisation or a change to an existing marketing authorisation.
For more information, see:
An update log is available to show the date and summary of changes to this webpage. It does not include updates to linked documents or minor edits like typos or broken link fixes.
The tracking of updates begins in February 2026.
12 February 2026
Update of the 'Stepwise PIP pilot supporting authorisation of innovative medicines' section to publish a report reviewing the outcomes of the stepwise paediatric investigration plan (sPIP) pilot from 2023 to 2025.