By Eva Berglund
When writing this, it’s just before Christmas and 14 days are left in 2020. As we all know, Brexit—the withdrawal of the United Kingdom from the European Union— is coming up and thereby new rules for pediatric drug developers come into place for the UK in 2021.
The EU uses pediatric investigation plans (PIPs) to ensure that sponsors collect the necessary data in pediatric studies to support the authorization of a medicine for children. PIPs and modifications agreed to before Jan 1, 2021 will be adopted also by the UK and do not need to be submitted to the UK’s health authority, the Medicines and Healthcare products Regulatory Agency (MHRA). If an EU PIP has received a negative opinion, the PIP will be considered refused in the UK as well. PIPs or modifications for which a valid request was submitted to the EMA before January 1st, will be adopted if the Paediatric Committee (PDCO) gives the PIP/modification a positive opinion, and if supported by the UK. No resubmission to MHRA is needed in these cases.
After January 1st
Submissions: An UK PIP needs to be submitted along the lines of the EU Paediatric regulation, which principles the UK will follow. The MHRA will in principle accept the PDCO opinion but may do a focused assessment related to UK conditions such as medical need, paediatric-only products, disease incidence, applicability of summary report scientific arguments, available medicines, etc. The MHRA will want to have information on any past EU procedures and assessments as well as on any agreed upon pediatric study plans (PSPs), the US equivalent to the PIP. The EU list of class waivers will be adopted by the MHRA. The MHRA will make their own assessment in case no EU PIP exists. However, to avoid having two extensive assessment procedures and to minimize the burden on sponsors, in most cases, it will be suitable to apply for an UK PIP when the EU PIP has been agreed upon.
Compliance checks: If the approved PIPs in the EU and UK are equivalent, the MHRA will adopt PDCO compliance checks (CCs) or interim CCs. The PDCO CC outcome should be submitted before or at the time of the marketing authorization application (MAA). However, the applicant must pay attention to the agreed timelines of those measures which would need to be completed after the PDCO CC to ensure compliance on the date of the UK MA submission. There is a need for a MHRA CC, for example, if there are any differences between the UK and EU PIP, or if there is no EU PIP, applicants are encouraged to request an UK CC ahead of the MA application.
More information is available at:
Communication from the Commission — Guideline on 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 and on criteria for assessing significant studies Text with EEA relevance (europa.eu)
About Eva Berglund
Eva is a pharmacist by training and has a PhD in Clinical Pharmacology, both from Uppsala University, Sweden. She has been a Clinical Pharmacology reviewer at the Swedish Medical Products Agency for over 20 years and a Senior Expert for 12 years, working with all types of molecules in marketing applications, clinical trials and scientific advice procedures in the EMA Network of National agencies. Eva has been working in all therapeutic areas and has extensive knowledge in antivirals, antibiotics, CNS active drugs, oncology, rheumatology, inhalation products etc.
Eva has been Rapporteur and actively involved in drafting of several EU Clinical Pharmacology guidance documents (drug-drug and drug-food interactions, PBPK, pediatrics, pharmacogenetics, etc.), in inter-regional harmonization activities and in the work of EMA working parties Pharmacokinetics Working Party and Paediatrics Working Party. Eva joined Certara in 2019 and provides her Clinical Pharmacology experience and Regulatory strategy knowledge in GAP analyses, regulatory stress tests and moc meetings, regulatory interactions, filing and clin pharm response support, pediatric submissions (PIP, PSP, new indications). Her inspiration is scientific development and its practical application, optimizing drug development, pushing regulatory science forward, and improving patient access to efficacious and safe drugs.