The Pediatric Research Equity Act (PREA) was signed into law on December 2003 and reauthorized in 2007 and 2012. PREA mandates paediatric studies for new drugs or biologics which are required only on adult indication (s) under review. FDA could thus waive studies in children if the disease for which the drug is being used in adults does not exist in children, such as prostate cancer. In addition, orphan indications were exempt of paediatric studies and paediatric studies could be deferred. As in Europe, the development of new oncology drugs in paediatric malignancies was neither mandated no incentivised by PREA.
On August 18, 2017, the RACE for Children Act, i.e. Research to Accelerate Cures and Equity Act, was signed into law as Title V of the 2017 FDA Reauthorization Act (FDARA) to amend the Pediatric Research Equity Act (PREA). This act aims to promote research into, and development of, new treatments for children with cancer. Paediatric investigation can be required if the drug or biological product is intended for the treatment of an adult cancer and directed at a molecular target that FDA determines to be “substantially relevant to the growth or progression of a paediatric cancer.” FDA will publish a list of molecular targets considered, on the basis of data the Agency determines to be adequate, to be substantially relevant to the growth and progression of a paediatric cancer and that may trigger the new paediatric investigation requirement. In addition, this act amends the PREA exemption for orphan-designated indications.
The RACE for Children Act will come into force on August 18, 2020.
The new regulatory environment is likely to promote the paediatric development of new anticancer drugs through their MoA with respect to the biology of paediatric malignancies instead of their adult indication. It is aimed at accelerating research and starting paediatric investigations earlier in the product development. Considering the number of oncology medicinal products under development in adults and the rarity of paediatric cancers, prioritization will be crucial to meet the needs of children.