Understanding Recent Regulatory Changes For Pediatric Oncology Trials

By Kristi Miller

Of the 1.7 million new cases of cancer in the U.S. in 2018, 10,590 involved children aged 14 or younger.[1] While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for innovative therapies for pediatric cancers is becoming increasingly vocal.

Until recently, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act of 2003 (PREA) constituted the primary legislation on pediatric trials in the U.S. The objective of PREA, in conjunction with BPCA, was to increase the availability of evidence for pediatric medication use by giving regulatory agencies the authority to require sponsors to conduct pediatric testing and to produce formulations appropriate for children.[2] These regulations are intended to not only support the need for pediatric safety, efficacy, and dosing information but also to provide increased transparency into the drug approval process.