Clinical trials for paediatric cancers under new legislation in the USA.

In their Health Policy, Gilles Vassal and colleagues 1 Vassal G de Rojas T Pearson ADJ Impact of the EU Paediatric Medicine Regulation on new anti-cancer medicines for the treatment of children and adolescents. Lancet Child Adolesc Health. 2023; 7: 214-222 Summary Full Text Full Text PDF PubMed Scopus (2) Google Scholar surveyed anticancer medicine development following the European Paediatric Regulation of 2007. In 2017, the US Congress passed the Research to Accelerate Cures and Equity (RACE) For Children Act to accelerate the development of targeted therapies for paediatric cancers. From August, 2020, this law authorised the US Food and Drug Administration (FDA) to require paediatric studies for new drugs and biologics for the treatment of adult cancers if the molecular targets of these therapies were relevant to the growth or progression of a paediatric cancer. 2 Hwang TJ Bourgeois FT New legislation to promote paediatric studies for new cancer medicines. Lancet Oncol. 2022; 23: e368-e369 Summary Full Text Full Text PDF PubMed Scopus (2) Google Scholar Impact of the EU Paediatric Medicine Regulation on new anti-cancer medicines for the treatment of children and adolescentsThe European Paediatric Medicine Regulation was launched in 2007, aiming to provide better medicines for children. However, its benefit for paediatric patients with cancer has been questioned and the European Paediatric and Orphan Regulations have been under review since November, 2020. To ascertain the effect of the European Paediatric Medicine Regulation, all paediatric anti-cancer medicines assessed by the European Medicines Agency from 1995 to 2022 were identified and reviewed using the agency's public assessment reports, and all Paediatric Investigation Plans granted since 2007 were analysed. Full-Text PDF