Paediatric requirements for marketing-authorisation applications
Applications for marketing authorisation submitted to the European Medicines Agency (EMA) that concern medicines not authorised in the European Union (EU) on 26 July 2008 must include the results of studies carried out as part of an agreed paediatric investigation plan (PIP) or information on a PIP deferral or waiver.
Applications must include one of the following elements to be considered valid:
- the results of all studies performed and details of all information collected in compliance with an agreed PIP: this means that the application will have to include the PIP decision and the results in accordance with the agreed PIP;
- an EMA decision on a PIP including the granting of a deferral: this means that the application will have to include the PIP decision including the deferral granted and if applicable, any completed studies;
- an EMA decision granting a product-specific waiver;
- an EMA decision granting a class waiver, together with the EMA confirmation letter of applicability if requested by the marketing-authorisation holder.
Where results of paediatric studies are submitted, applicants should include the rationale for the proposed changes to the product information in the clinical overview. In particular, if the PIP is completed and the results of all studies are available, the applicant should explicitly discuss why the generated data do or do not support the intended paediatric indications stated in the PIP.
Inclusion of the results of all studies performed in compliance with an agreed PIP is necessary to receive the paediatric reward. See Incentives for the development of paediatric medicines.
Applicants should ensure that a PIP compliance check is carried out before submitting a marketing-authorisation application.
This check verifies that the fulfilments of the measures as mentioned in the PIP decision including the timelines for the conduct of the studies or collection of the data are fulfilled.
Full details are available in:
- Questions and answers on the procedure of paediatric-investigation-plan compliance verification at the European Medicines Agency
- Commission guideline
- validation of new MAA, Variation/Extension applications and compliance check with an agreed PIP
- The format and content of applications for agreement or modification of a paediatric investigation plan and request for waivers or deferrals and concerning the operation of the compliance check and on criteria for assessing significant studies