The health care provider's needs in the pediatric drug development process

Abstract

The lack of available medicines with an appropriate label for use in neonates, infants, children, or adolescents presents clinicians with a dilemma: either depriving their patients from the assumed clinical benefits of a medicine or prescribing the medicine in an off-label or unlicensed manner, which may result in uncertainty on efficacy as well as an increased risk of side effects. Even medicines that are labeled for use in the pediatric population are sometimes not suitable to be administered to children or essential pediatric medicines are not available in healthcare facilities, due to inadequate supply, shortage, lack of storage facilities, or inequalities in affordability or reimbursement. This chapter will provide an overview of what health care providers need in order to offer children safe and effective medicine therapy. This chapter will specifically focus on lessons learned as well as the remaining challenges and needs for implementing the health care providers' requirements within pediatric medicine research and development. © 2024 Elsevier Inc. All rights reserved.