Paediatric investigation plans
Table of contents
- Rapid procedure for COVID-19 treatments and vaccines
- Joint EMA / FDA procedural information for COVID-19 medicines
- Joint EMA / FDA guidance on cancer medicines for use in children
- Stepwise PIP pilot supporting authorisation of innovative medicines (new)
- Deferrals and waivers
- Post-assessment guidance
- Scientific advice
- Checking compliance
A paediatric investigation plan (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 authorisation 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 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.
For treatments and vaccines for COVID-19, EMA reviews applications in an expedited manner for agreement of a PIP, deferrals or waivers and accelerates compliance checks, to speed up these products' development and approval.
There are no pre-specified submission deadlines and EMA's review of a PIP may take only twenty days, depending on its complexity and the applicant's preparedness to respond to questions, followed by two days to issue an EMA decision instead of the usual ten.
Developers may provide focused scientific documentation. Compliance checks, if required, may take only four days.
EMA encourages applicants to use the template below to submit a letter of intent ahead of their PIP application, providing EMA with key information about the product, its intended use in preventing or treating COVID-19 and timelines. This helps ensure that EMA is duly prepared to assess the application when it arrives:
Applicants may use the letter to request a pre-submission meeting with EMA to obtain procedural guidance in the format of a virtual meeting.
For more information, see:
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 a COVID-19 vaccine or treatment:
The 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.
EMA and the FDA are encouraging medicine developers to submit PIPs and iPSPs early.
EMA and the FDA published the joint document in June 2020.
Joint guidance 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 on the use of a cancer medicine in children:
The purpose is to speed up the development and authorisation of cancer medicines for children, given the rarity of childhood cancers.
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 'cluster activities' on paediatric medicines;
- international collaboration in the design and conduct of clinical trials.
EMA and the FDA published the joint document in April 2021.
New: EMA runs a pilot to test 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:
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.
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.
- For information on how to apply for a PIP deferral or waiver, see PIPs: questions and answers.
- For information on the classes of diseases subject to waivers, see Class waivers for PIPs.
- Approved deferrals and waivers are included in the searchable list of opinions and decisions on PIPs.
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.
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.
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: