Module232025

12/05/2025

Global Regulatory Landscape

The Organisation for Professionals in Regulatory Affairs

5

M Dehlinger-Kremer, 14 May 2025

5

The Regulatory Framework and Paediatric Regulatory Requirements

United States

Europe

EU Paediatric Regulation (No 1901/2006), Jan 2007 − Paediatric Investigation Plan (PIP) and compliance with PIP at time of product Marketing Authorisation Application − PIP submission after PK in adults EU new proposed Pharma Legislation approved parliament April 2024- Council as next step. Adoption outstanding − Mechanism of action PIP

Better Pharmaceuticals for Children Act (BPCA, 2002, Law 2012) − Reward

Paediatric Research Equity Act (PREA, 2003, Law 2012) − Paediatric Study Plan (iPSP) mandatory for NDAs/BLAs − PSP submission after end phase II meeting with FDA Research to Accelerate Cures and Equity (RACE) for Children Act (Aug 18, 2020) − New molecularly targeted drugs and biologics “intended for the treatment of adult cancers and directed at a molecular target substantially relevant to the growth or progression of a paediatric cancer” must be evaluated for paediatrics − ODD exemption from PREA eliminated

UK and CH Paediatric Regulations − Paediatric Investigation Plan required for MAA

Canada

Paediatric Rule policy draft − The policy under piloted since February 2024 and for 2 years − Guidance on submitting paediatric development plans and paediatric studies, published May 2024

Paediatric Rare Disease Priority Review Voucher - Until Sept. 30, 2026 - PRV program sunset started Dec. 20, 2024. Extension requested

Paediatric Plans to be agreed with the respective authorities at early stage of the drug development

The Organisation for Professionals in Regulatory Affairs

6

M Dehlinger-Kremer, 14 May 2025

6