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EMA Guidance: Procedural Advice on Paediatric Applications

The European Medicines Agency (EMA) yesterday(04 June 2024) updated and released the guidance document "Procedural Advice on Paediatric Applications" to ensure that medicinal products developed for children are safe and effective.

The legislative framework for paediatric applications is based on the Paediatric Regulation, which outlines the requirements for submitting paediatric investigation plans (PIPs), modifications, waivers, and compliance checks.

The primary objective of the guidance is to ensure that paediatric medicinal products undergo rigorous evaluation, ensuring their safety, efficacy, and quality. It aims to streamline the process of submitting and managing paediatric applications, providing clear instructions for applicants.

The general principles outlined in the guidance emphasize the importance of early and continuous dialogue between applicants and the EMA. It also highlights the need for thorough documentation and adherence to timelines to facilitate the efficient review and approval of paediatric applications.

Applicants are advised to carefully plan their submission timelines, taking into consideration the time required for the EMA's review and any potential need for additional data or clarification. Applicants are encouraged to engage with the EMA to address any general questions related to their paediatric application.

Pre-submission meetings with the EMA can provide valuable feedback and guidance, helping to ensure that the application meets all regulatory requirements. During the clock-stop period, applicants can seek clarification from the EMA to address any issues raised during the initial review. This helps streamline the re-submission process and ensures that all concerns are adequately addressed.

The initial PIP outlines the proposed studies and measures to ensure that the medicinal product can be safely and effectively used in the paediatric population. It is a critical component of the paediatric application.

Applicants may need to modify an agreed PIP based on new data or scientific advancements. The guidance provides instructions on how to submit modifications and the necessary documentation. Before submitting the final application, applicants must request a compliance check to ensure that all PIP requirements have been met.

The EMA will validate the application to ensure all necessary documents and information are included. Once validated, the formal review process begins. The assessment phase involves a detailed review of the submitted data and documentation to ensure the medicinal product's safety, efficacy, and quality for paediatric use.

PDCO (Paediatric Committee)will provide feedback and may request further information before adopting a final opinion on the paediatric application. If necessary, applicants can request a re-examination of the PDCO opinion to address any disagreements or concerns. Based on the PDCO opinion, the EMA will make a final decision on the paediatric application, determining whether the product can be approved for the paediatric population.

This comprehensive EMA guidance on procedural advice for paediatric applications is essential for ensuring the safe and effective development of medicines for children. It provides clear instructions and timelines for applicants, facilitating efficient submission and review processes. For detailed information, refer to the full document available here.


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