TY - JOUR
T1 - Creating a unique, multi-stakeholder Paediatric Oncology Platform to improve drug development for children and adolescents with cancer
AU - Vassal, Gilles
AU - Rousseau, Raphaël
AU - Blanc, Patricia
AU - Moreno, Lucas
AU - Bode, Gerlind
AU - Schwoch, Stefan
AU - Schrappe, Martin
AU - Skolnik, Jeffrey
AU - Bergman, Lothar
AU - Bradley-Garelik, Mary Brigid
AU - Saha, Vaskar
AU - Pearson, Andy
AU - Zwierzina, Heinz
N1 - Publisher Copyright:
© 2014 The Authors. Published by Elsevier Ltd.
PY - 2015/1/1
Y1 - 2015/1/1
N2 - 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.
AB - 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.
KW - Drug development
KW - Long-term follow up
KW - Paediatric Investigation
KW - Paediatric oncology
KW - Plan
KW - Precompetitive development
KW - Regulatory framework
UR - http://www.scopus.com/inward/record.url?scp=84925449413&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2014.10.029
DO - 10.1016/j.ejca.2014.10.029
M3 - Article
C2 - 25434924
AN - SCOPUS:84925449413
SN - 0959-8049
VL - 51
SP - 218
EP - 224
JO - European Journal of Cancer
JF - European Journal of Cancer
IS - 2
ER -