Legal requirements for children's medicines
An overview of the legal requirements for children’s medicines that Marketing Authorisation Holders (MAHs) need to abide by.
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The Human Medicines Regulations 2012, as inserted by the Human Medicines (Amendment etc.) (EU Exit) Regulations 2019 (HMRs) contain provisions to ensure that where appropriate, medicines are adequately studied in children and developed to meet their needs, including the need for age appropriate formulations.
The HMRs ensure that the necessary data are obtained through studies to support efficacy and safety of medicines and ultimately their authorisation in the paediatric population and /or increase of available paediatric information about these medicines.
Under the HMRs, you need to submit Paediatric Investigation Plans (PIPs) and completed paediatric studies sponsored by Marketing Authorisation Holders (MAHs) to the Medicines and Healthcare products Regulatory Agency (MHRA).
Paediatric Investigation Plans (PIPs)
Paediatric studies are part of the normal lifecycle and development of a medicine. You need to agree and carry out a PIP if you are applying for a marketing authorisation for a new medicine or in certain cases if you are developing a currently licensed medicine, unless you have a waiver for all or a subset of the paediatric population. A PIP includes all the measures that would generate the necessary data from studies in children as well as measures with regards to formulation development.
You must submit your PIP or waiver application via MHRA Submissions.
The MHRA has published guidance on the submission and assessment of UK Paediatric procedures. These are:
- procedures for UK Paediatric Investigation Plans
- the format and content of applications for agreement or modification of a Paediatric Investigation Plan and requests for waivers or deferrals and concerning the operation of the compliance check
For general enquiries about paediatric submissions including PIP and waiver applications, modification procedures, and compliance checks, contact the MHRA Paediatric Unit at ukpip@mhra.gov.uk
Paediatric studies
You need to submit the results of MAH-sponsored studies in children within a period of six months from the completion of the studies. This is in addition to any requirements for registration of clinical trials and publishing of summary trial results. In cases where an initial MHRA appraisal indicates that an assessment is required, you will be asked to submit the paediatric data as a type II variation (see below).
If the results of a paediatric study have been submitted to EMA or CMDh under Article 46 of Regulation (EC) 1901/2006 prior to 1January 2021, the process will remain within the EU assessment framework and no UK equivalent procedure will be initiated unless the MAH indicates that an urgent safety update of the product information (PI) is required.
Upon finalisation of the EU procedure, MAHs should submit the final assessment report to paediatricstudies@mhra.gov.uk.
The MHRA will check the applicability of the outcome of the EU procedure for UK products. If there are proposed changes to the PI which can be directly implemented to relevant UK products, MHRA will request MAHs to submit a Type IB variation to update the PI within 60 days if not already submitted.
MAHs are not required to submit paediatric studies completed by 26 January 2007 which fall under the remit of EU Article 45 of Reg.1901/2006/EC. MHRA will monitor the published public assessment reports and applicability of any recommended changes to the PI for UK licensed products with the same active substance.
If required, you will be asked to submit a type IB variation (or in selected cases, a type II variation, if the MAH should provide supplementary data and further assessment is needed) to update the PI.
Within 30 days from the end of the assessment procedure of completed paediatric studies, the MHRA will publish the Public Paediatric study Assessment Reports (PdPAR).
There is additional guidance on the submission, processing and assessment of completed paediatric studies.
Variation codes
Where paediatric study assessment is required, you should submit a type II variation using change code C.I.13. Where MHRA has requested implementation of changes recommended as the result of an EU procedure, use code C.1.3(z) as under a type IB. In the event where additional data that require assessment are submitted, a type II variation should be submitted with code C.I.3(b).
Email specialpopulationsunit@mhra.gov.uk if you have any queries on variations to children’s medicines or on medicines for children in general.
New medicinal products and variations to existing medicines, including extensions
Your paediatric data will be assessed by the chemical or biological product life-cycle assessment team in the licensing division if you have:
- provided the data to support an application for a new product
- applied for a variation e.g. adding a new indication to an existing product
- applied for an extension e.g. a new pharmaceutical dosage form
Paediatric data for variations affecting the safety of the product will be reviewed by the relevant team in the VRMM division.
You are encouraged to request a compliance check (CC) where one is required for validation of your MA application. PDCO compliance outcome documents, if available, should be submitted ahead of, or at the time of your UK application. These documents will be considered in the MHRA assessment as outlined in Procedures for UK-PIPs.
We do not need to check compliance if:
- a full product-specific waiver (no paediatric studies needed in any age group) for the condition(s) covering the indication(s) in the application has been issued by the MHRA
- there is a class waiver or a letter of confirmation from the MHRA that a class waiver applies for the condition(s) covering the indication(s) in the application
- studies have been deferred and neither the date of initiation or completion of studies relevant to the condition(s) covering the indication(s) in the application fall before the date of submission
Additional information about compliance can be found in Procedures for UK-PIPs.
Email the regulatory information service ris.na@mhra.gov.uk for more information on new marketing authorisations and variations.
Last updated 31 December 2020 + show all updates
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Updated to reflect changes in regulations following Brexit transition.
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Link to new guidance on procedures for UK-PIPs in the event the UK leaves the EU without a deal has been added to the page.
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Added information and link to new guidance page about Completed Paediatric Studies - submission, processing and assessment in the event of a no deal scenario.
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First published.