Drug Product Development and Case Studies for Patient Centric Pediatric Protein-based Therapeutics.

The user of a pediatric drug includes not only the patient, but also their caregiver and healthcare provider, including nurses, doctors, and pharmacists. Therefore, adopting a patient-centric approach that focuses on all users is critical for the development of pediatric drug products. This article outlines the quality target product profile parameters and a patient-centric approach for the development of pediatric protein-based therapies. The use environment, formulation design, and preparation and in use stability considerations are described. An acceptability profile for the various routes of parenteral administration is described with a focus on pediatric age groups. Furthermore, a risk assessment approach is presented for the selection of excipients to be utilized in pediatric protein-based biopharmaceuticals. Several case studies are included which illustrate the selection of drug product parameters such as formulation, dose volume, and route of administration with the pediatric user in mind.