Journal ArticleDOI
New drugs for children and adolescents with cancer: the need for novel development pathways
Gilles Vassal,C. Michel Zwaan,David M. Ashley,Marie-Cécile Le Deley,Darren Hargrave,Patricia Blanc,Peter C. Adamson +6 more
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TLDR
In this article, the authors proposed a new drug development pathway for children with cancer based on innovative methods and trial designs, investment in biology and preclinical research, new models of partnership and funding including public-private partnerships and precompetitive research consortia, improved regulatory requirements, initiatives and incentives that better address these needs, and increased collaboration between paediatric oncology cooperative groups worldwide.Abstract:
Despite major progress in the past 40 years, 20% of children with cancer die from the disease, and 40% of survivors have late adverse effects. Innovative, safe, and effective medicines are needed. Although regulatory initiatives in the past 15 years in the USA and Europe have been introduced, new drug development for children with cancer is insufficient. Children and families face major inequity between countries in terms of access to innovative drugs in development. Hurdles and bottlenecks are well known-eg, small numbers of patients, the complexity of developing targeted agents and their biomarkers for selected patients, limitations of US and EU regulations for paediatric medicines, insufficient return on investment, and the global economic crisis facing drug companies. New drug development pathways could efficiently address the challenges with innovative methods and trial designs, investment in biology and preclinical research, new models of partnership and funding including public-private partnerships and precompetitive research consortia, improved regulatory requirements, initiatives and incentives that better address these needs, and increased collaboration between paediatric oncology cooperative groups worldwide. Increased cooperation between all stakeholders-academia, parents' organisations and advocacy groups, regulatory bodies, pharmaceutical companies, philanthropic organisations, and government-will be essential.read more
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Sustaining innovation and improvement in the treatment of childhood cancer: lessons from high-income countries
Kathy Pritchard-Jones,Rob Pieters,Gregory H. Reaman,Lars Hjorth,Peter Downie,Gabriele Calaminus,Marianne C Naafs-Wilstra,Eva Steliarova-Foucher +7 more
TL;DR: The introduction of drugs that are less toxic and more targeted than those currently used necessitates a partnership between clinical and translational researchers, the pharmaceutical industry, drug regulators, and patients and their families to ensure that efforts are focused on the unmet clinical needs of young people with cancer.
Journal ArticleDOI
Improving the outcome for children with cancer: Development of targeted new agents.
TL;DR: The collaborative research infrastructure for children with cancer in the United States is well positioned to advance novel treatments into clinical investigations for a spectrum of rare and ultra‐rare childhood cancers.
Journal ArticleDOI
New policies to address the global burden of childhood cancers
Richard Sullivan,Jerzy Kowalczyk,Bharat Agarwal,Ruth Ladenstein,Edel Fitzgerald,Ronald D. Barr,Eva Steliarova-Foucher,Ian Magrath,Scott C. Howard,Mariana Kruger,Maria Grazia Valsecchi,Andrea Biondi,Paul E. Grundy,Malcolm A. Smith,Peter C. Adamson,Gilles Vassal,Kathy Pritchard-Jones +16 more
TL;DR: In this paper, the authors present a key list of time-limited proposals focusing on change to health systems and research and development, including sector and system reforms, policies to promote growth of civil society around both cancer and Millennium Development Goals, major improvements to public health services (particularly the introduction of national cancer plans), improved career development, and increased remuneration of specialist health-care workers and government support for childhood cancer registries.
Journal ArticleDOI
Creating a unique, multi-stakeholder Paediatric Oncology Platform to improve drug development for children and adolescents with cancer.
Gilles Vassal,Raphael Rousseau,Patricia Blanc,Lucas Moreno,Gerlind Bode,Stefan Schwoch,Martin Schrappe,Jeffrey M. Skolnik,Lothar Bergman,Mary Brigid Bradley-Garelik,Vaskar Saha,Andrew D.J. Pearson,Heinz Zwierzina +12 more
TL;DR: 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.
New policies to address the global burden of childhood cancers
TL;DR: In this paper, the authors review the key policy issues for the delivery of better care, research, and education of professionals and patients for children with cancer, and highlight the importance of early childhood cancer screening.
References
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TL;DR: Treatment of metastatic melanoma with PLX4032 in patients with tumors that carry the V600E BRAF mutation resulted in complete or partial tumor regression in the majority of patients.
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