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Detailed guide: Procedures for UK Paediatric Investigation Plan (PIPs) from 1 January 2021

Medicines Healthcare Products Regulatory Agency

September 1
14:01 2020

The guidance also covers information for applicants or companies with an agreed EU-PIP opinion conferred prior to 1 January 2021.

General approach to UK Paediatric Investigation Plans

We will simplify the PIP application process for applicants by offering an expedited assessment where possible, and by mirroring the submission format and terminology of the EU-PIP system. Therefore, the scientific content and assessment required will be kept in line with European Medicines Agency (EMA) guidance documents.

Northern Ireland (will continue to be part of the EUs system for paediatric medicines development including agreement of EU paediatric investigation plans (PIPs) or waivers.

We will take decisions on PIP and waiver opinions, modifications and compliance statements to support paediatric market authorisation decisions, while acknowledging that EU PIPs remain applicable for Northern Ireland.

Read the European Commissions principles that the UK will follow.

The format and submission procedure for UK-PIP applications will be published separately. Applicants should include information relevant specifically to the UK, particularly with respect to any areas of unmet therapeutic need that this product intends to cover in the UK [Section 6 of this document].

This guidance addresses the common scenarios that may occur when a UK paediatric procedure is submitted to the Medicines and Healthcare products Regulatory Agency (MHRA) after 1 January 2021.

A case by case discussion should always be considered for any UK paediatric submissions that do not fall into any of the prespecified criteria listed below.

Further step by step information on the process of submitting PIPs via the new MHRA submissions portal will be available in a user reference guide which will be published separately.

Section 1: PIP submissions

1.1. EU-PIP or modifications to PIPs submitted before 1 January 2021

EU-PIPs and modifications agreed by the EMA prior to 1 January 2021, will be adopted as UK-PIPs on or after that date:

  • These PIPs will not require re-submission to the MHRA.

Where a valid request for an EU-PIP or modification or waiver has been made to the EMA, but no decision has been given before 1 January 2021, the EU-PIP will be adopted as a UK-PIP if the EMA Paediatric Committee (PDCO) has given a positive opinion with which the UK has concurred.

  • These PIPs will not require re-submission to the MHRA.

Where a valid request for an EU-PIP or modification or waiver has been made to the EMA, but the PDCO has issued a negative opinion:

  • In this case, the MHRA will treat the application as refused. However, applicants can submit an updated PIP to the MHRA which addresses the reasons for refusal.

Where a valid request for an EU-PIP or modification or waiver has been made to the EMA, but the PDCO has not yet given any opinion, or where the UK disagreed with the PDCO opinion:

  • These PIPs should be resubmitted to the MHRA unless the applicant notifies MHRA that they do not wish the application to proceed.

EU-PIPs which become UK-PIPs under these transitional provisions will be referred to as adopted UK-PIPs in this guidance. New UK-PIP submissions after 1 January 2021 that have been assessed and agreed by the MHRA, will be referred to as agreed UK-PIPs.

1.2. UK-PIP submissions after 1 January 2021

When a UK-PIP is submitted, information should be provided on whether there is:

  • an agreed EU-PIP and the opinion and supporting documentation is included
  • an ongoing EU-PIP assessment, its timeline in the PDCO assessment cycle (i.e. day 30, 60, clock stop, day 90 or 120)
  • any scientific divergence between the submitted UK-PIP and the EU-PIP

This information will be used to establish the MHRA assessment process as described in Sections 1.3 to 1.4.

The assessment pathways for UK-PIP submissions from 1 January 2021 are Annexes I and II (PDF, 90.9KB, 2 pages).

1.3. UK-PIP with agreed EMA opinion from 1 January 2021 or ongoing assessment at EMA from 1 January 2021

In principle, the MHRA will aim to accept a positive PDCO opinion. A focused assessment may be needed considering (but not restricted to) the following:

  • unmet UK paediatric needs [Section 6 of this guidance];
  • paediatric only development particularly for an innovative product (such as a new drug class, mechanism of action)
  • the incidence of the disease in the UK population
  • the relevance of the scientific arguments by EMA / PDCO in the summary report (SR) to the UK paediatric population
  • any additional safety or efficacy concerns for the UK population
  • the nature and number of licensed products already available for the intended paediatric indication
  • the feasibility of performing the proposed paediatric studies in the UK

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