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Lancet Oncol. 2017 Jul;18(7):e394-e404. doi: 10.1016/S1470-2045(17)30442-4.

From class waivers to precision medicine in paediatric oncology.

Author information

1
Paediatric Drug Development, Children and Young People's Unit, The Royal Marsden NHS Foundation Trust, Sutton, UK; Division of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, Sutton, UK. Electronic address: andy1pearson@btinternet.com.
2
Division of Pediatric Neuro-oncology, German Cancer Research Center, Heidelberg, Germany; German Cancer Consortium, Heidelberg, Germany; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany.
3
Pediatric Hematology-Immunology Department, University Hospital Robert Debré and Paris Diderot University, Paris, France.
4
Division of Oncology and Hematology, University Children's Hospital Zurich, Children's Research Center, Zurich, Switzerland.
5
Paediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
6
Paediatric Drug Development, Children and Young People's Unit, The Royal Marsden NHS Foundation Trust, Sutton, UK; Division of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, Sutton, UK.
7
Department of Paediatric, Adolescents and Young Adults Oncology and INSERM U830, Institut Curie, Paris, France.
8
Department of Pediatric Oncology and Hematology, Charité University Hospital, Berlin, Germany; German Cancer Consortium, Berlin, Germany; Berlin Institute of Health, Berlin, Germany.
9
Department of Pediatric and Adolescent Oncology, CNRS UMR 8203 Vectorology and Anticancer Treatments, Gustave Roussy, Université Paris-Sud, Villejuif, France.
10
Division of Pediatric Neuro-oncology, German Cancer Research Center, Heidelberg, Germany; German Cancer Consortium, Heidelberg, Germany.
11
Cancer Research UK Clinical Trials Unit Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
12
Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlands.
13
Department of Paediatric Oncology, Birmingham Women's and Children's Hospital, Birmingham, UK.
14
Wolfson Childhood Cancer Research Centre, Northern Institute for Cancer Research, Newcastle University and Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
15
Department of Pediatric Oncology/Hematology, Erasmus MC/Sophia Children's Hospital, Rotterdam, Netherlands; Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlands.
16
Department of Clinical Research, Gustave Roussy, Paris-Sud University, Paris, France.

Abstract

New drugs are crucially needed for children with cancer. The European Paediatric Regulation facilitates paediatric class waivers for drugs developed for diseases only occurring in adults. In this Review, we retrospectively searched oncology drugs that were class waivered between June, 2012, and June, 2015. 147 oncology class waivers were confirmed for 89 drugs. Mechanisms of action were then assessed as potential paediatric therapeutic targets by both a literature search and an expert review. 48 (54%) of the 89 class-waivered drugs had a mechanisms of action warranting paediatric development. Two (2%) class-waivered drugs were considered not relevant and 16 (18%) required further data. In light of these results, we propose five initiatives: an aggregated database of paediatric biological tumour drug targets; molecular profiling of all paediatric tumours at diagnosis and relapse; a joint academic-pharmaceutical industry preclinical platform to help analyse the activity of new drugs (Innovative Therapy for Children with Cancer Paediatric Preclinical Proof-of-Concept Platform); paediatric strategy forums; and the suppression of article 11b of the European Paediatric Regulation, which allows product-specific waivers on the grounds that the associated condition does not occur in children. These initiatives and a mechanism of action-based approach to drug development will accelerate the delivery of new therapeutic drugs for front-line therapy for those children who have unmet medical needs.

PMID:
28677575
DOI:
10.1016/S1470-2045(17)30442-4
[Indexed for MEDLINE]

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