Quantitative Pharmacology and Individualized Therapy Strategies in Development of Therapeutic Proteins for Immune-Mediated Inflammatory Diseases

14Quantitative Pharmacology Assessment Strategy Therapeutic Proteins in Pediatric Subjects – Challenges and Opportunities

Jeremiah D. Momper¹Andrew Mulberg²Nitin Mehrotra³Dan Turner⁴William Faubion⁵Laurie Conklin⁶Karim Azer⁷ and Marla C. Dubinsky⁸

¹University of California, Skaggs School of Pharmacy and Pharmaceutical Sciences, La Jolla, San Diego, CA, 92093, USA

²Amicus Therapeutics, Inc., 08512

³Merck & Co., North Wales, PA, 19454, USA

⁴The Juliet Keidan Institute of Pediatric Gastroenterology and Nutrition Shaare Zedek Medical Center, Jerusalem, Israel

⁵Mayo Clinic, Rochester, MN, USA

⁶Children's Hospital, Washington, DC, USA

⁷Bill & Melinda Gates Medical Research Institute, Boston, MA, USA

⁸Icahn School of Medicine at Mount Sinai, New York, NY, USA

14.1 Introduction

Therapeutic proteins (TPs) are critical in the treatment of a variety of diseases in the pediatric population. As TPs are one of the fastest‐growing segments of the pharmaceutical and biotechnology industries, pediatric legislations intended to stimulate pediatric product development in the United States and Europe ensures that TPs will continue to be developed for children. Yet significant challenges and opportunities exist for studying TPs in neonates, children, and adolescents. This chapter covers issues related to studying TPs in the pediatric population, including extrapolation of efficacy, timing, and initiation of pediatric trials, informative priors, and trial design considerations, using inflammatory bowel ...