Creating a unique, multi-stakeholder paediatric oncology platform to improve drug development for children and adolescents with cancer

  • Seven years after the launch of the European Paediatric Medicine Regulation, limited progress in paediatric oncology drug development remains a major concern amongst stakeholders – academics, industry, regulatory authorities, parents, patients and caregivers. Restricted increases in early phase paediatric oncology trials, legal requirements and regulatory pressure to propose early Paediatric Investigation Plans (PIPs), missed opportunities to explore new drugs potentially relevant for paediatric malignancies, lack of innovative trial designs and no new incentives to develop drugs against specific paediatric targets are some unmet needs. Better access to new anti-cancer drugs for paediatric clinical studies and improved collaboration between stakeholders are essential. The Cancer Drug Development Forum (CDDF), previously Biotherapy Development Association (BDA), with Innovative Therapy for Children with Cancer Consortium (ITCC), European Society for Paediatric Oncology (SIOPE) and European Network for Cancer Research in Children and Adolescents (ENCCA) has created a unique Paediatric Oncology Platform, involving multiple stakeholders and the European Union (EU) Commission, with an urgent remit to improve paediatric oncology drug development. The Paediatric Oncology Platform proposes to recommend immediate changes in the implementation of the Regulation and set the framework for its 2017 revision; initiatives to incentivise drug development against specific paediatric oncology targets, and repositioning of drugs not developed in adults. Underpinning these changes is a strategy for mechanism of action and biology driven selection and prioritisation of potential paediatric indications rather than the current process based on adult cancer indications. Pre-competitive research and drug prioritisation, early portfolio evaluation, cross-industry cooperation and multi-compound/sponsor trials are being explored, from which guidance for innovative trial designs will be provided.

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Author:Gilles Vassal, Raphaël Rousseau, Patricia Blanc, Lucas Moreno, Gerlind Bode, Stefan Schwoch, Martin Schrappe, Jeffrey Skolnik, Lothar BergmannORCiDGND, Mary Brigid Bradley-Garelik, Vaskar Saha, Andy Pearson, Heinz Zwierzina
URN:urn:nbn:de:hebis:30:3-371840
DOI:https://doi.org/10.1016/j.ejca.2014.10.029
ISSN:0959-8049
Pubmed Id:https://pubmed.ncbi.nlm.nih.gov/25434924
Parent Title (English):European journal of cancer
Publisher:Elsevier
Place of publication:Amsterdam
Document Type:Article
Language:English
Date of Publication (online):2014/11/27
Date of first Publication:2014/11/27
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2015/04/29
Tag:Drug development; Long-term follow up; Paediatric Investigation Plan; Paediatric oncology; Precompetitive development; Regulatory framework
Volume:51
Issue:2
Page Number:7
First Page:218
Last Page:224
Note:
(c) 2014 The Authors. Published by Elsevier Ltd.
This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/3.0/).
HeBIS-PPN:368975312
Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Licence (German):License LogoCreative Commons - Namensnennung-Nicht kommerziell-Keine Bearbeitung 3.0