Emerging Issues for Pharma R&D: A Practical Approach for Pediatric Drug Programs

Pediatric patients are not simply small adults. Children differ from adults in both disease pathophysiology and pharmacokinetics/pharmacodynamics (PK/PD). Maturation of metabolic capacity and renal function can impact drug clearance. In addition, changes in body composition can alter volumes of distribution, and changes in GI function can affect drug absorption. Yet historically, 80 percent of medicines used in children had little to no data guiding prescribers on proper use.

 The pharmaceutical industry has numerous reasons not to develop pediatric drugs. Often, pediatric indications are not particularly profitable, and pediatrics in the US market represents only about seven percent of drug sales. From a practical perspective, pediatric development tends to be a low priority for product development teams who are focused on gaining regulatory approval for adults.

 To address this market failure, regulatory legislation for drug development in pediatric patients was passed worldwide over the past decade. The number of drugs tested in and labeled for children has increased dramatically as a result. Despite this progress, pediatric drug developers still face numerous hurdles: heterogeneous patient populations, limited numbers of patients, and practical/ethical challenges with performing clinical studies in this vulnerable population.

 In this webinar, Dr. Barry Mangum from Paidion Research and Dr. JF Marier from Certara will discuss how pediatric considerations fit into the overall drug development program. They will also make recommendations for addressing issues from both a practical and scientific perspective. Anonymized case studies will be presented with a review of historical issues with a focus on solutions for today (i.e. licensed comparators differences in US and EU, importation issues, and endpoint selection).

 By attending this webinar, you will learn

• What trends are emerging in the global pediatric drug development landscape with a focus on the US perspective
• • How to optimize the interactions with regulatory agencies, before or after the pediatric investigation plan (PIP) and managing the FDA-EMA Committee for Medicinal Products for Human Use (CHMP) parallel advice procedure

• What to look for when engaging external expertise in pediatric drug development

Speakers

Jean-Francois Marier, Senior Vice President Canda, Consulting Services, Certara

Dr. Marier has directed pharmacokinetic/pharmacodynamic (PK/PD) analyses, reporting, and modeling and simulation projects for hundreds of studies in early and late clinical research. His drug development experience spans non-compartmental and population PK/PD modeling and simulation across all phases of development and in specific populations (pediatric/geriatric, renal/liver impaired). His expertise includes scientific and regulatory consultation on drug development programs for small and large molecules, particularly in the therapeutics areas of oncology, inflammation, immunosuppressants, cardiovascular, infectious diseases and metabolic diseases. Marier has authored or co-authored more than 100 abstracts/manuscripts and book chapters in the field of PK/PD, and has leveraged modeling and simulation to build and optimize drug development programs for dozens of industry sponsors. Marier earned his PhD in pharmaceutical sciences from the University of Montreal, Faculty of Pharmacy and is a fellow of the American College of Clinical Pharmacology.

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Barry Mangum, Founder and CEO, Paidion Research

As Paidion’s Founder and CEO, Dr. Mangum is responsible for their vision of profound and meaningful change in global pediatric clinical research. Paidion is catalyzing change in an old paradigm and laying the groundwork for developing safe and effective medicines for children. Responsible for partnering with clients to implement clinical trials for all phases of pediatric drug development, Dr. Mangum brings decades of pediatric clinical research expertise to Paidion. His extensive academic experience includes serving as faculty and researcher in pediatrics and clinical pharmacology/pharmacotherapy at Duke’s and UNC’s medical schools. His corporate experience includes serving as Senior Director of Pediatric Clinical Development Services at Quintiles. He has numerous publications, presentations and memberships, including co-authorship of NeoFax: A Manual of Drugs Used in Neonatal Care, the leading guide for dosing neonatal medications in the US, as well as the NeoFax software package and mobile device app.

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