These guidelines outline the scenarios and relevant procedures for applicants wishing to submit a UK Paediatric Investigation Plan (PIP), request for modification and/or waiver. There is also information on how to submit completed studies for processing and assessment.
European Union (EU) PIPs, modifications to PIPs and full product specific waivers will be adopted as UK-PIPs with no need to re-submit to the Medicines and Healthcare products Regulatory Agency (MHRA) on the condition that a European Medicines Agency (EMA) decision or an EMA Paediatric Committee (PDCO) positive opinion has been conferred before 01 January 2021. If the PDCO delivers a negative opinion, the MHRA will refuse the application, however applicants can resubmit an updated PIP to the MHRA addressing the issues. In situations where the assessment from the EMA/PDCO is still ongoing from 01 January 2021, the MHRA will aim to maintain alignment with their opinions.
For adopted or agreed UK PIPs, a modification request will be assessed based on the scientific arguments provided and the availability of an agreed EU-PIP modification or an ongoing assessment for one.
If there is no agreed EMA modification opinion, a full assessment of the modification will be conducted by the MHRA.
The current EMA class waivers list has been adopted by the UK as of 01 January 2021. The MHRA strives to accept positive EMA opinions on a class waiver request. In the event that an EMA opinion is not forthcoming, the MHRA will conduct its own assessment. An EU full product specific waiver with a positive PDCO opinion or an EMA decision submitted before 01 January 20201 will be adopted as a UK full waiver.
Further components of the guidance detail the relevant compliance checks that should be present in an application, inclusion of paediatric study plans if one is available, and the definition of unmet needs for the UK paediatric population.
Once a trial of a drug which involves use in the paediatric population has been completed, UK Marketing Authorisation Holders (MAHs) must submit a cover letter to the MHRA for appraisal within 6 months. This cover letter should indicate whether the trial is linked to other paediatric studies which have been or will be the subject of other submissions, and also whether it has been or will be submitted in the UK as part of a variation/extension or any other application.
In cases where the results of a paediatric study have been submitted to the European Medicines Agency (EMA) or Co-ordination Group for Mutual Recognition and Decentralised Procedures – Human (CMDh) before 01 January 2021, the MHRA will check the applicability of the outcome of the EU procedure for UK products. If there are proposed changes to the Product Information (PI), the MHRA will request MAHs to submit a Type IB variation to update the PI within 60 days.
To view the full guidance on PIPs, modifications and waivers, click here.
To view the full guidance on submitting completed studies, click here.
To view the full guidance on the format and submission procedure for PIP applications, click here.