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1. Overview

This guidance explains the scope and legal basis for the initial and full submission requirements, as well as the processing and assessment of all completed paediatric studies sponsored by marketing authorisation holders (MAHs).

2. Legal background and scope

Regulation 78A(13) and (14) of the Human Medicines Regulations 2012, as amended by the Human Medicines (Amendment etc.) (EU Exit) Regulations 2019 and the Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (HMRs), require that holders of a UK marketing authorisation who sponsor a study which involves use in the paediatric population in respect of the medicinal product to which that authorisation relates must submit to the Medicines and Healthcare products Regulatory Agency (MHRA) results of the study within the period of 6 months beginning with the day on which the trial ended. The paediatric population is defined in the HMRs as that part of the population consisting of persons under the age of 18 years.

This applies whether or not:

• the studies are conducted in accordance with an agreed paediatric investigation plan (PIP)

• the?MAH?intends to apply for a marketing authorisation or a variation/extension for a paediatric indication in relation to the product

From 1 January 2025, the MHRA is making decisions on the licensing of all medicines across the UK, and medicinal products will be designated Category 1 or Category 2. See UK-wide licensing for human medicines. The UK paediatric submission requirements apply irrespective of product categorisation.

This guidance addresses the common scenarios for paediatric submissions to the MHRA, regardless of whether the product is Category 1 or Category 2. A case-by-case discussion should always be considered for paediatric submissions that do not meet the criteria listed in this guidance.

3. Submitting information

In line with the regulation, the?MAH must submit a cover letter. Suggested?cover letter template Suggested?cover letter template (MS Word Document, 41 KB) to the?MHRA?mailbox at?paediatricstudies@mhra.gov.uk within 6 months of completion of the concerned paediatric studies (that is, the date of the last visit of the last subject undergoing the trial, unless otherwise justified in the protocol).

In the cover letter, the MAH should state:

• whether there is a need to update the product information as a result of the paediatric study and if there are any urgent safety or efficacy updates to the product information (PI)

• whether the study(ies) have been submitted for assessment by a non-UK regulator such as the EU procedure under Article 46 of Regulation (EC) No 1901/2006

  • if so, provide a final assessment report from the non-UK regulator as soon as available

  • if the non-UK regulator assessment is ongoing, inform the MHRA of any urgent safety or efficacy updates identified during the review

• whether the study(ies) are linked to other paediatric studies which have been or will be the subject of other submissions under Regulation 78A of the HMRs, if so:

  • provide the study title(s) with approximate date of completion

  • if the study(ies) relate to a UK?PIP, provide the?PIP?number

• whether the study(ies) have been or will be submitted in the UK as part of a marketing authorisation or variation/extension, if so:

  • specify the UK product licence (PL) number and the type of application this will be submitted under, planned timings and the route of submission

  • confirm that the application will be submitted within the next 6 months, if not, provide justification for the delay including an estimated date of submission

  • confirm that, based on the results of the study, no urgent safety update of the product information is required

4.Initial appraisal

On receipt of the cover letter,?the MHRA?will carry out an initial appraisal of whether an assessment procedure is required at this stage. One of the following may apply:

4.1 Assessment of study data is not required

Assessment of the study data is not required at this stage. The MHRA?will maintain records including justification for the decision, for example:

• a regulatory submission is planned in the next 6 months to the MHRA where the study data will be included for review

• the MAH plans to submit the data directly as a variation to the MHRA

• any other agreed reason(s) to defer the procedure

If the study has been submitted or will be submitted to the EU then the MAH must provide the final Article 46 EU assessment report as soon as possible when it becomes available.

If the assessment is ongoing, the MAH must inform the MHRA of any urgent safety or efficacy updates identified during the assessment. The final EU Article 46 outcome (together with cover letter information) will determine further MHRA action, for example, no further action, proceed to variation.

The initial appraisal step does not lead to change(s) to the product information (PI). If changes are required to the PI, the company must submit an appropriate MHRA regulatory submission (refer to section on Legal background and scope).

4.2 Assessment of study data is required

Assessment of the study data is required if, for example:

• there are urgent safety or urgent efficacy updates to PI

• the MAH prefers a full UK assessment

• there is UK-specific concern for public health issues or UK-specific conditions such as pollen immunotherapy for pollen specific to UK only

4.2.1 Limited evaluation

If assessment of the study data is required, limited evaluation of the study data may be undertaken if the?MAH?provides robust justification that the study data is unlikely to warrant PI changes. The?MAH?will need to state in the cover letter that one or more of the following criteria are met:

• the same data has been reviewed in another procedure by?the MHRA?or another regulatory authority and the review has not led to?PI?changes

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