A progress report on the development of the European Commission (EC) and European Medicines Agency (EMA) Action Plan on Paediatrics has been published, highlighting in particular, strategies to address the medical needs of children
The ACCELERATE action plan is recognised as a successful example in addressing unmet medical needs in children and adolescents. The progress report provides an update on the achievements in five key areas outlined in the 2018 action plan with the objective to increase efficiency of paediatric regulatory processes in the current legal framework and boost availability of medicines for children.
The EMA/EC action plan contains actions in five key areas: Identifying paediatric medical needs; strengthening cooperation between decision-makers; ensuring timely completion of paediatric investigation plans (PIPs); improving the handling of PIP applications and increasing transparency around paediatric medicines.
The 2020 progress report highlights the contribution of ACCELERATE to the overall strategy of the EMA and the EC. In particular, the report highlights that key area one has achieved important results in defining strategies to address the needs of children with malignancies through the joint ACCELERATE/EMA Paediatric Strategy Forums, with the participation of the FDA, to address paediatric medical needs and provide a basis for strategic decision-making on paediatric medicine development.
Three ACCELERATE/EMA Paediatric Strategy Forums were listed as successful examples in addressing the needs of children with malignancies.
- ‘Paediatric Strategy Forum for medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients’ (September 2018)
- ‘Paediatric Strategy Forum for medicinal product development for acute myeloid leukaemia in children and adolescents’ (April 2019)
- ‘Paediatric Strategy Forum for epigenetic modifiers in paediatric malignancies’ (January 2020)
The first of these was organised and hosted in the U.S. The conclusions of these three multi-stakeholder forums were subsequently published in the European Journal of Cancer in open access
ACCELERATE is also currently running a project to evaluate the impact of Paediatric Strategy Forums on the development of innovative therapies for children and adolescents with cancer.
The progress report furthermore highlighted the uniqueness of ACCELERATE in ensuring participation and engagement of both the EMA and the FDA at the highest level to address the needs of children with malignancies.