Gregory Knipp

Abstract:

The term “Therapeutic Orphans” was first used by Dr. Harry Shirkey in 1962 to describe pediatric populations. Over fifty years later, pediatric drug development is still largely an unmet area of therapeutic need based on the various physiological, clinical, and formulation challenges associated with age-based population dosing. Moreover, access to pediatric dosage forms is limited throughout the world as highlighted by the World Health Organization’s Global Accelerator for Paediatric Formulations business plan. Access to pediatric formulations is most limited in remote areas where resources are limited, and there is a greater utility of off label use of compounded adult formulations where the dose control is not ensured. Here we will highlight our efforts to address the global need for sustainable, verified strategies to develop reproducible, extemporaneous formulations that can easily be transferred to remote areas where resources are limited. Towards this aim, easily generated pediatric formulation strategies developed at Purdue with the intent to transfer the approaches to collaborators in Africa will be discussed. One of the primary causes for this lack of pediatric drug development is the inability to accurately predict clinical safety and efficacy at different pediatric developmental stages early in translation. This lecture will also highlight some of the challenges that currently face preclinical development of pediatric medicines and the potential utility of the juvenile porcine model as a preclinical model for pharmacokinetic testing during formulation development. Our aim is to develop reproducible pediatric formulation approaches that require minimal technological advances, which can be produced at different doses in remote clinics. We will also highlight our efforts to verify performance by ensuring reproducible exposure in preclinical testing utilizing the juvenile swine model to ensure that safety and efficacy can be met. An illustration of a simple formulation strategy that has been transferred from Purdue to Tanzania