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 EMA / FDA guidance

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.

COVID-19 medicines:

This joint document aims to make it easier for developers to submit paediatric development plans simultaneously to the regulators, to help speed up the development and approval of COVID-19 treatments and vaccines:

FDA / EMA common commentary on submitting an initial paediatric study plan (iPSP) and paediatric investigation plan (PIP) for the prevention and treatment of COVID-19

The guidance in this section was intended for use during the COVID-19 public health emergency.

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:

  • simultaneous submission to both regulators;
  • coordinated discussions between the regulators in the context of the Paediatric Cluster;
  • international collaboration in the design and conduct of clinical trials.

Common Commentary - EMA/FDA: Common issues requested for discussion by the respective agency (EMA/PDCO and FDA) concerning paediatric oncology development plans (Paediatric Investigation Plans [PIPs] and initial Pediatric Study Plans [iPSPs])

Stepwise PIP pilot supporting authorisation of innovative medicines

EMA is running a pilot for a 'stepwise PIP' agreement which would introduce a partial development programme supporting the authorisation of innovative medicines for children.

This programme would be conditional on the development of a full PIP once sufficient evidence becomes available. It would rely 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. 

Guidance on the stepwise PIP pilot is available below:

Guidance for Stepwise PIP pilot

A stepwise PIP would apply to cases where there is a lack of crucial information needed to decide on certain parts of the paediatric investigation plan, such as whether a clinical study for a whole age group is necessary.

EMA launched this pilot in February 2023. It is due for review upon the adoption of eight initial opinions on stepwise PIPs.

Deferrals and waivers

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.

Post-assessment guidance

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, the Agency adopts a decision.

The Agency makes all opinions and decisions on PIPs, deferrals and waivers public, after deletion of information of a commercially confidential nature.

Scientific advice

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. They can also follow up a PIP with scientific advice, for example on combined adult and paediatric development in light of the PIP requirements.

EMA discourages applicants to submit scientific advice and an PIP applications in parallel.

Checking compliance

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:

Related documents

Contact point

How useful do you find this page?

Average: