10-year report on the European Paediatric Regulation and its impact on new drugs for children's cancers

Two (2%) drugs were considered not to be relevant, and 16 (18%) required further data.5 Supported by these analyses, we proposed a MOA-based approach with five initiatives: an aggregated database of paediatric biological tumour drug targets; a joint academic-pharmaceutical industry preclinical platform to analyse the activity of new drugs (Innovative Therapy for Children with Cancer Paediatric Preclinical Proof-of-Concept Platform [ITCC-P4]); paediatric strategy forums; molecular profiling of paediatric tumours at diagnosis and relapse; and the suppression of article 11b of the European Paediatric Regulation, which allows product-specific waivers on the grounds that the associated condition does not occur in children.5 The first three of these approaches have been implemented: a database of targets has been created, and the ITCC-P4 has been funded as a project of Innovative Medicines Initiative 2 and is operational.Globally, there has been substantial policy change, after the passing of the FDA Reauthorization of 2017 (Research to Accelerate Cures and Equity [RACE] for Children Act) by the US Congress on Aug 3, 2017.7 This Act states that “drugs and biological products should be developed for paediatric cancers, if the drug or biological product is: (i) intended for the treatment of an adult cancer; and (ii) directed at a molecular target that the Secretary determines to be substantially relevant to the growth or progression of a paediatric cancer.”[...]in the USA, there will soon be an operational MOA-based model for the development of oncology drugs for children.[...]the hopes and expectations of parents of children with cancer and the paediatric oncology community have not been met by the report findings.4 AD Pearson, R Herold, R Rousseau, Implementation of mechanism of action biology-driven early drug development for children with cancer, Eur J Cancer, Vol. 62, 2016, 124-131 5 ADJ Pearson, SM Pfister, A Baruchel, From class waivers to precision medicine in paediatric oncology, Lancet Oncol, Vol. 18, 2017, e394-e404 6 European Medicines Agency, Report: paediatric strategy forum for anaplastic lymphoma kinase (ALK) inhibition in paediatric malignancies, May 30, 2017, (accessed Jan 8, 2018).