Drug product development and case studies for patient centric pediatric protein-based therapeutics

Area of Science:

• Pharmaceutical Sciences
• Pediatric Drug Development
• Biotechnology

Background:

• Pediatric drug development necessitates a holistic approach, recognizing diverse users beyond the patient.
• Protein-based therapies present unique challenges in pediatric formulation and administration.

Purpose of the Study:

• To outline quality target product profile parameters for pediatric protein-based therapies.
• To detail a patient-centric strategy for developing these therapies, considering all stakeholders.
• To address formulation, administration routes, and excipient selection for pediatric use.

Main Methods:

• Defining quality target product profile parameters.
• Describing use environment, formulation design, and stability considerations.
• Presenting an acceptability profile for parenteral administration routes in pediatric age groups.
• Applying a risk assessment approach for excipient selection in pediatric biopharmaceuticals.

Main Results:

• Illustrative case studies demonstrate the selection of drug product parameters (formulation, dose volume, route) tailored for pediatric users.
• A comprehensive framework for patient-centric development of pediatric protein therapies is provided.

Conclusions:

• A patient-centric approach is critical for successful pediatric protein therapy development.
• Consideration of all users and specific pediatric needs is essential for optimizing drug product design and delivery.