Pediatric Formulations and Dosage Forms and Future Opportunities: Impact of Regulations in the USA and Implementation of Quality by Design

The advances in pharmaceutical sciences and technology have been so significant that Peter Drucker’s quote that “the future has already happened” applies readily for medicines developed for adults. For pediatric patients, the future is about to happen. The continuation of focused partnerships and knowledge sharing and leveraging are critical to ensure that pediatric patients have timely access to high quality drug products that were developed with pediatric patients in mind. An overview of regulatory efforts, regulations and legislation to address the challenges for pediatric drug development are discussed. The implementation of regulatory incentives, the Pediatric Rule, Best Pharmaceuticals for Children Act, and Pediatric Research Equity Act, is having an impact and has led to incorporation of information for dosing of approximately 500 drug products since 1998 starting with implementation of the Pediatric Rule. There are significant accomplishments and a lot more work ahead for the pediatric community. The labeling information is usually for older pediatric patients and the need for information for safe and effective dosing of patients of 6 years old or younger remains. Some of the study outcomes are inconclusive with respect to safety and efficacy, and support the hypotheses that better understanding of drug delivery to pediatric patients is needed for determining and delivering the right dose to the pediatric patients. The 2012 Food and Drug Administration Safety and Innovation Act and implementation of Quality by Design paradigm focusing on drug product design and manufacturing process are expected to have a synergistic effect for continuing to advance development of pediatric dosage forms and formulations for the benefit of the pediatric patients.