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 discussed how pediatric considerations fit into the overall drug development program. They also made recommendations for addressing issues from both a practical and scientific perspective. Anonymized case studies were presented with a review of historical issues with a focus on solutions for today (ie, licensed comparators differences in US and EU, importation issues, and endpoint selection).