TY  - JOUR
A1  - Vassal, Gilles
A1  - Rousseau, Raphaël
A1  - Blanc, Patricia
A1  - Moreno, Lucas
A1  - Bode, Gerlind
A1  - Schwoch, Stefan
A1  - Schrappe, Martin
A1  - Skolnik, Jeffrey
A1  - Bergmann, Lothar
A1  - Bradley-Garelik, Mary Brigid
A1  - Saha, Vaskar
A1  - Pearson, Andy
A1  - Zwierzina, Heinz
T1  - Creating a unique, multi-stakeholder paediatric oncology platform to improve drug development for children and adolescents with cancer
T2  - European journal of cancer
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.
KW  - Drug development
KW  - Paediatric oncology
KW  - Regulatory framework
KW  - Paediatric Investigation Plan
KW  - Precompetitive development
KW  - Long-term follow up
Y1  - 2014
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/37184
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-371840
SN  - 0959-8049
N1  - (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/).
VL  - 51
IS  - 2
SP  - 218
EP  - 224
PB  - Elsevier
CY  - Amsterdam
ER  -