Robert L. Ternik, Ph.D., R.Ph., is senior research advisor, Small Molecule Design and Development, at Eli Lilly and Company. He is responsible for creating and implementing human-centered design capability for Lilly’s small molecule drug product portfolio. He is a member of the Pediatric Working Group of the IQ Consortium.
Julia Gao, Ph.D., is an associate director at Bristol-Myers Squibb Company, where she is responsible for commercial oral product development for BMS’s small molecule portfolio. He is a member of the Pediatric Working Group of the IQ Consortium.
Robert Ju, Ph.D., is an associate director at Abbvie, where his group is responsible for formulation development of AbbVie’s small molecule portfolio. He chairs the Pediatric Working Group of the IQ Consortium.
Globally, the design and development of pediatric drug products has significantly lagged behind the development of drug products for adult populations. The lag is largely attributable to a combination of economic factors, therapeutic and bioethical factors, and the challenges associated with conducting robust pediatric clinical trials with the innate diversity of the pediatric population. This lag has greatly affected pediatric patients by limiting the availability of beneficial and potentially life-saving medicines and requiring health care professionals to use medicines studied and designed for adults in children.
Consequently, regulatory bodies and industry have acted to address this issue. Since 1997, a number of regulatory approaches have been implemented to incentivize the development of pediatric products. These efforts have increased in their intensity and effectiveness over the past decade, most notably with the 2006 passing of the pediatric regulation in the European Union and the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) legislation in the United States. As a direct consequence of these pieces of legislation, more attention to the challenges of pediatric drug development has been generated across the medical and pharmaceutical industries. In response, a number of focused groups and initiatives have been established across the globe to provide guidance, clarity, and support to improve the design, development, and manufacture of pediatric products for clinical evaluation and commercial use.
While progress has been made, significant challenges remain in all areas of pediatric development. Creating synergy across the relevant scientific disciplines through collaboration among industry, academia, and regulatory bodies across the globe is necessary to overcome these challenges. This collaboration could harmonize regulatory approaches, promote basic research, share best practices, and solidify the economic incentives available. These efforts are directed toward the goal of expediting the availability of high-quality pediatric products for global use.
The March issue of the AAPS Newsmagazine highlights the impact of recent regulation and provides an overview of existing challenges and opportunities to accelerate the development of pediatric drug products. Read No Small Challenge: Can the Barriers to Pediatric Drug Development Be Overcome? from the Formulation Design and Development section of AAPS and then participate in the discussion questions below.
1. What is the biggest barrier or limitation in developing pediatric pharmaceutical products and suggestions for overcoming it?
2. Have the legislative approaches in the EU and USA been effective in advancing pediatric research? What could be done to improve legislation?
3. What is the biggest opportunity to rapidly advance pediatric product development?