Learning From Failure, Leveraging Modeling and Simulation for Pediatric Drug Development Success

Historically, clinical trials have not examined most pediatric medications. This is due to the ethical and practical challenges of conducting pediatric drug trials. Children are 40 percent of the world’s population. Yet, regulatory agencies have approved only 10 percent of the drugs on the market for pediatrics.1 Without a proper clinical process, pediatricians are stuck with inaccurate dosing and therapeutic approaches. The result is a continuation of off-label, experiential drug prescribing.

To address this urgent need, both the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) now require pediatric trial plans for new drugs. These trials plans are known as the Pediatric Study Plan (PSP) and the Pediatric Investigation Plan (PIP), respectively. The combination of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) and these new regulatory requirements are moving the pendulum towards safer, more effective medicines for children. Between 2007 and 2013, 469 pediatric studies were completed under BPCA and PREA. And by August 2014, 526 labeling changes were made.2 Since 2007, around 300 products have had label changes approved for safety, efficacy or dosing for pediatrics in the European Union.2