Accelerating the development of priority formulations in childhood cancer: target product profiles for paediatric formulations of cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine, temozolomide
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World Health Organization
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vi, 54 p.
Global disparities in access to appropriate paediatric cancer medicines continue to hinder progress towards improved survival outcomes, particularly in low- and middle-income countries. This technical report presents the outcomes of a WHO-led consultation, aligned with the paediatric drug optimization (PADO) process and global childhood cancer initiatives, to define target product profiles for priority paediatric formulations of six essential cancer medicines, based on a structured development process and expert consensus. The document examines current limitations in age-appropriate formulations and outlines optimal and minimum characteristics for medicines including cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine and temozolomide. It describes the methodology used to assess pharmaceutical feasibility, dosing requirements, safety, acceptability and supply considerations, with particular attention to the needs of children across age groups and settings. Findings highlight challenges related to dosing flexibility, palatability, safe handling and stability, especially in resource-constrained environments. The report also identifies preferred formulation approaches and underscores the role of target product profiles in guiding research and development, supporting regulatory pathways and informing future inclusion of appropriate formulations in essential medicines frameworks.
Global disparities in access to appropriate paediatric cancer medicines continue to hinder progress towards improved survival outcomes, particularly in low- and middle-income countries. This technical report presents the outcomes of a WHO-led consultation, aligned with the paediatric drug optimization (PADO) process and global childhood cancer initiatives, to define target product profiles for priority paediatric formulations of six essential cancer medicines, based on a structured development process and expert consensus. The document examines current limitations in age-appropriate formulations and outlines optimal and minimum characteristics for medicines including cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine and temozolomide. It describes the methodology used to assess pharmaceutical feasibility, dosing requirements, safety, acceptability and supply considerations, with particular attention to the needs of children across age groups and settings. Findings highlight challenges related to dosing flexibility, palatability, safe handling and stability, especially in resource-constrained environments. The report also identifies preferred formulation approaches and underscores the role of target product profiles in guiding research and development, supporting regulatory pathways and informing future inclusion of appropriate formulations in essential medicines frameworks.