Paediatric drug optimization for sickle cell disease: meeting report, 8-10 September 2025

Overview

The paediatric drug optimization (PADO) exercise for sickle cell disease (SCD) aimed to identify short- and long-term therapeutic priorities for children with SCD by reviewing currently approved products and emerging products in the research and development pipeline, including gene therapies. The outcome of this process is intended to inform and support the alignment of funders, procurers, market coordination entities, researchers, innovators, generic manufacturers, product development partnerships and regulators on priority products and strategies to improve access to SCD therapeutics.

This meeting report describes the priority-setting process and summarizes the consensus reached by participants on therapeutic priorities for paediatric SCD. It presents the PADO priority list, including the development of a target product profile for age-appropriate hydroxyurea formulations, and identifies a watch list of promising investigational therapies to guide future paediatric research and development. The report also highlights key research priorities and actions to accelerate the availability of safe, effective and child-appropriate treatments for children living with sickle cell disease.