| Title: |
ACCELERATE and European Medicines Agency Paediatric Strategy Forum for medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients |
| Authors: |
Pearso, Andrew D. J.; Rossig, Claudia; Lesa, Giovanni; Diede, Scott J.; Weiner, Susan; Anderson, John; Gray, Juliet; Geoerger, Birgit; Minard-Colin, Veronique; Marshall, Lynley V.; Smith, Malcolm; Sondel, Paul; Bajars, Marcis; Baldazzi, Claudia; Barry, Elly; Blackman, Sam; Blanc, Patricia; Capdeville, Renaud; Caron, Hubert; Cole, Peter D.; Jimenez, Jorge Camarero; Demolis, Pierre; Donoghue, Martha; Elgadi, Mabrouck; Gajewski, Thomas; Galluzzo, Sara; Ilaria, Robert, Jr.; Jenkner, Alessandro; Karres, Dominik; Kieran, Mark; Ligas, Franca; Lowy, Israel; Meyers, Michael; Oprea, Corina; Peddareddigari, Vijay G. R.; Sterba, Jaroslav; Stockman, Paul K.; Suenaert, Peter; Tabori, Uri; van Tilburg, Cornelis; Yancey, Todd; Weigel, Brenda; Norga, Koenraad; Reaman, Gregory; Vassal, Gilles |
| Source: |
0959-8049 ; European journal of cancer |
| Publication Year: |
2020 |
| Collection: |
IRUA - Institutional Repository van de Universiteit Antwerpen |
| Subject Terms: |
Human medicine |
| Description: |
The third multistakeholder Paediatric Strategy Forum organised by ACCELERATE and the European Medicines Agency focused on immune checkpoint inhibitors for use in combination therapy in children and adolescents. As immune checkpoint inhibitors, both as monotherapy and in combinations have shown impressive success in some adult malignancies and early phase trials in children of single agent checkpoint inhibitors have now been completed, it seemed an appropriate time to consider opportunities for paediatric studies of checkpoint inhibitors used in combination. Among paediatric patients, early clinical studies of checkpoint inhibitors used as monotherapy have demonstrated a high rate of activity, including complete responses, in Hodgkin lymphoma and hypermutant paediatric tumours. Activity has been very limited, however, in more common malignancies of childhood and adolescence. Furthermore, apart from tumour mutational burden, no other predictive biomarker for monotherapy activity in paediatric tumours has been identified. Based on these observations, there is collective agreement that there is no scientific rationale for children to be enrolled in new monotherapy trials of additional checkpoint inhibitors with the same mechanism of action of agents already studied (e.g. anti-PD1, anti-PDL1 anti-CTLA-4) unless additional scientific knowledge supporting a different approach becomes available. This shared perspective, based on scientific evidence and supported by paediatric oncology cooperative groups, should inform companies on whether a paediatric development plan is justified. This could then be proposed to regulators through the available regulatory tools. Generally, an academic-industry consensus on the scientific merits of a proposal before submission of a paediatric investigational plan would be of great benefit to determine which studies have the highest probability of generating new insights. There is already a rationale for the evaluation of combinations of checkpoint inhibitors with other agents ... |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/isi/000514572100006 |
| Availability: |
https://hdl.handle.net/10067/1677210151162165141; https://repository.uantwerpen.be/docstore/d:irua:882 |
| Rights: |
info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.C19ABB35 |
| Database: |
BASE |