Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and...

Area of Science:

• Pharmaceutical Sciences
• Pediatric Drug Development
• Formulation Science

Background:

• Global access to age-appropriate pediatric drug formulations remains a priority, yet development lags behind adult products due to inherent complexities.
• Regulatory incentives have not fully overcome the challenges in bringing pediatric formulations to market, highlighting a critical unmet need.

Purpose of the Study:

• To advocate for the use of a pediatric Quality Target Product Profile (p-QTPP) as a strategic tool for efficient pediatric formulation development.
• To define essential attributes of pediatric formulations and promote enhanced collaboration among multidisciplinary teams.
• To address hurdles in pediatric drug development, such as lack of early information on indications and dosing.

Main Methods:

• This position paper proposes the utilization of a pediatric Quality Target Product Profile (p-QTPP) framework.
• It outlines key attributes for pediatric formulations and emphasizes collaborative approaches.
• Recommends incorporating acceptability testing as a secondary endpoint in clinical trials.

Main Results:

• The p-QTPP is identified as an efficient tool for early planning and decision-making in pediatric formulation design.
• Essential attributes for pediatric formulations are suggested to guide development.
• Increased collaboration between formulation scientists and clinical professionals is advocated.

Conclusions:

• The p-QTPP can expedite the development of new chemical entities and the repurposing of existing drugs for pediatric populations.
• Enhanced collaboration and defined formulation attributes are key to creating safe, effective, and acceptable pediatric medicines.
• Addressing early knowledge gaps regarding indications and dosing through tools like the p-QTPP is vital for successful pediatric drug development.