Improvement in child cancer survival rates threatened by lack of new drug development

20 February 2013

Kathy Pritchard-Jones

Some of the biggest challenges to the improvement of cancer care for children and young people are set out in a series of studies published in today's Lancet Oncology.

The Series, led by Professor Kathy Pritchard-Jones of the UCL Institute of Child Health, presents the latest results and ideas from some of the world’s most eminent cancer experts from 19 countries, spanning six continents. It highlights the educational, research, and care needs of children and young people with cancer in all countries, and proposes a broad range of solutions for the next decade.

In particular, it looks at how increasingly strict regulatory environments for clinical research are damaging the development of new cancer medicines for children and threatening recent improvements in survival, and outlines how the growing threat of childhood cancer in developing nations could be easily managed.

The Series is published to coincide with a special event hosted by Glenis Willmott (MEP), which will debate these issues at the EU parliament today.

New regulatory approval and research strategies are urgently needed to speed the development of new, effective, and safer treatments for children with cancer if we are to continue to improve the cure rate and reduce toxicity compared to existing treatments.

Professor Kathy Pritchard-Jones, UCL Institute of Child Health

“In high-income countries, we have nearly reached optimisation of present anticancer treatments,” says Professor Kathy Pritchard-Jones.

“New regulatory approval and research strategies are urgently needed to speed the development of new, effective, and safer treatments for children with cancer if we are to continue to improve the cure rate, reduce toxicity compared to existing treatments, and minimise side effects in later life,” she continues.

Although more children and young people in high-income countries are surviving cancer than ever before, cancer remains the leading cause of death from disease in children aged 1 to 15 years. More than 5,000 children still lose their lives to cancer every year in these regions.

Increased participation in international, collaborative clinical trials has successfully raised survival from 30% to 80% over the last half century. “But an increasingly complex and strict regulatory environment for clinical research and data sharing is limiting children’s access to early-phase clinical trials and delaying the development of new drugs”, explains co-leader Professor Richard Sullivan of King’s College London.

Other factors leading to longer clinical development include: the complex nature of the biology underlying childhood cancers; the difficulty of identifying targets suitable for drug treatment; a lack of long-term sustainable funding for research and development, particularly outside the USA; and little economic incentive for pharmaceutical companies to develop anticancer drugs adapted for children.To fast track the most relevant and new medicines for childhood cancers will require a renewed focus on the potential role of adult cancer drugs in children as well as newer methods and clinical trial design that aim to more rapidly predict the optimal (ie, effective and safe) dose.

According to Professor Pritchard-Jones: “New biology-driven approaches are needed, but this will only be achieved through increased international cooperation in clinical trials and sharing of research tissue samples and data. This is necessary as these diseases are already rare, and dividing patients into increasingly smaller biological subgroups might amount to just a handful of trial participants a year in each country.

“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. This therapeutic alliance will ensure that efforts are focused on the unmet clinical needs of young people with cancer.

As well as drug development challenges, more needs to be done to address the long-term consequences of cancer treatment. Estimates suggest that one in 1000 adults in high-income countries are survivors of childhood cancer, and 40% of these survivors experience adverse effects throughout life.

The authors conclude by calling on every country to develop a national cancer plan that recognises the unique demographic and care needs of young people with cancer. They state: “If policy makers continue to fail to pay attention to this issue then in ten years…the infrastructure will not be in place to deal with what will have become the most common disease-related cause of death in childhood."

-Ends-


Media contact: David Weston

Image caption: Professor Kathy Pritchard-Jones, UCL Institute of Child Health


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