EMA/EC progress report on the action plan on paediatrics highlights the work of ACCELERATE.

The European Commission (EC) and European Medicines Agency (EMA) have published a progress report of the 2018 EMA/EC joint action plan on paediatrics to support the development of medicines for children in Europe.

This 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 and namely:

1. Identifying paediatric medical needs 

2. Strengthening cooperation between decision-makers

3. Ensuring timely completion of paediatric investigation plans (PIPs)

4. Improving the handling of PIP applications and

5. Increasing transparency around paediatric medicines.

The 2020 progress report clearly highlights the contribution of ACCELERATE to the overall strategy of the EMA and the EC.

In particular, Key Area 1 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, namely: 

1. ‘Paediatric Strategy Forum for medicinal product  development of checkpoint inhibitors for use in combination therapy in paediatric patients’ (September 2018)

2. ‘Paediatric Strategy Forum for medicinal product development for acute myeloid leukaemia in  children and adolescents’ (April 2019); and

3. ‘Paediatric Strategy Forum for epigenetic modifiers in paediatric malignancies (Jan 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.

The manuscripts are available here.

ACCELERATE is currently also running a project to evaluate the impact of Paediatric Strategy Forums on the development of innovative therapies for children and adolescents with cancer.

More information will become available soon.

The progress report furthermore highlighted the uniqueness of the platform in ensuring participation and engagement of both the EMA and the FDA at the highest level to address the needs of children with malignancies.

As a multi-stakeholder transparent forum, bringing together worldwide representatives from academia, industry, patient advocates and regulators, overarching issues are discussed and addressed in a timely fashion to drive forward the development of innovative medicines for children and adolescents with cancer.

All members and diverse participants are committed to continue and expand the work, which was initiated more than five years ago by engaging all relevant stakeholders to speed up innovation in drug development for children and adolescents with cancer. Within this context, new initiatives in the pipeline for 2021 include:

4-5 February 2021: ACCELERATE will hold its annual Paediatric Oncology Conference virtually. More information available here.

25-27 May 2021: The (virtual) 6th Paediatric Strategy Forum for Medicinal Product Development of CAR T-cells in Children and Adolescents, in collaboration with the European Medicines Agency and with participation of the Food and Drug Administration is scheduled to take place. More information available here.

Together with our international multi-stakeholder members and partners, ACCELERATE is committed to improving global development of new paediatric oncology drugs.

Source: ACCELERATE