3rd Paediatric Strategy Forum: Checkpoint inhibitors in combination

Paediatric Strategy Forum for Medicinal Product Development of Checkpoint inhibitors for use in combination therapy in paediatric patients

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Date: 5&6 September 2018

Venue: EMA, London, UK

Expression of Interest deadline was on 18th May 2018

Background and objectives

The third multi-stakeholder Paediatric Strategy Forum, jointly organised by ACCELERATE and the European Medicines Agency (EMA) will focus on checkpoint inhibitors used in combination therapy, in children and adolescents. Paediatric Strategy Forums have been created to facilitate dialogue and provide an opportunity for constructive interactions between relevant stakeholders (patients/patient representatives, clinicians, academics, pharmaceutical companies and regulators) on topics requiring open discussion on development of medicines in the best interests of children and adolescents with cancer. The goal of this meeting is to share information, in a pre-competitive setting, to facilitate the developments of innovative medicines and ultimately their introduction into the standard-of-care of children with malignancies.

The summary of the first Paediatric Strategy Forum held in January 2017 on ALK inhibition in paediatric malignancies can be found here; the summary of the second Paediatric Strategy Forum held in November 2017 on Medicinal Product Development for Mature B cell Malignancies in Children can be found here.

Immune checkpoint inhibitors have shown impressive success in some adult malignancies, in particular, monoclonal antibodies that block the interaction between programmed death ligand 1 (PD-L1) on the surface of tumour or antigen-presenting cells, and programmed death 1 (PD-1) on the surface of lymphocytes. Many of these products have now been licensed as first or second line treatments for adult malignancies. Furthermore the combination of antibodies targeting PD-1 with those targeting the immune checkpoint molecule CTLA-4 has shown particularly high response rates especially in adult patients with metastatic melanoma. Early phase trials in children of single agent checkpoint inhibitors are currently being presented. High response rates are being observed in some cancers common to children and adults as for example in Hodgkin lymphoma. However, these results appear not to be reflected in typical paediatric malignancies. Some combination studies are in progress and others are planned.  It therefore seems opportune to review the early results of these early phase trials in children and consider opportunities for paediatric studies in which check-point inhibitors are used in combination including also possible other approaches.

In the Forum the immunological environment of paediatric malignancies, checkpoint inhibitors in early phase clinical studies in paediatrics, strategies combining checkpoint inhibitors with other products and alternative immunotherapy strategies will be reviewed.

The output will be a published summary from all participants addressing the challenges and documenting the conclusions.

Click here to download the draft Programme

Click here to download the short Summary

If you have any questions or comments, please contact us: contact@accelerate-platform.eu or visit the EMA webpage.


For more information, please contact contact@accelerate-platform.org
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