Pushing the boundaries of science to beat childhood cancers (Guest blog)

Cancer can strike at any age – including early in life. While childhood cancer is uncommon, its diagnosis is always heart-breaking and has a deep impact on the child and their family in the short- and long-term.
This is the sad reality that affects thousands of families every year: Almost 16,000 children are estimated to have been diagnosed with cancer in Europe in 2020[i]. One in every 300 children born that year is likely to develop cancer by the age of 19. This means that every large school or sports club is likely to be touched by cancer at some point.
Time is of the essence when it comes to patient outcomes. By the time they are diagnosed, 80% of paediatric cancers have already spread to other parts of the body, compared to about 20% of adult cancers. While, thanks to advances in cancer care, 80% of children now survive 5 years or more[ii], we know that more can be done.
To beat childhood cancer, progress is urgently needed to ensure earlier diagnosis, advance multidisciplinary care, foster medical innovation, expand psychological services for patients, families and caregivers, and support survivors.
Increase collaboration across countries
Childhood cancers are relatively rare compared to the most common adult cancers, making research and treatment more challenging. That is why it is even more important to accelerate progress in paediatric oncology by sharing good practices from across Europe to advance diagnosis, therapy and care.
It is also vital to collect and analyse data on paediatric cancer incidence, on the natural history of diseases, and on how patients respond to treatment. For rare cancers in particular, specialists across Europe must have the skills, technological tools and support required to share anonymised information for research purposes and to facilitate clinical care.
Thankfully, several initiatives will move Europe forward in this regard. For example, one of the aims of Europe’s Beating Cancer Plan (EBCP) is to address the significant differences between and within Member States, and between socioeconomic groups, in access to prevention and care. This work will rely on the collection and analysis of data across health systems.
Indeed, the EBCP has the potential to be a game-changer for children’s cancers if it is implemented with vigour. One of the flagships of the Plan is the “Helping Children with Cancer” Initiative. It aims to ensure that children have access to rapid and optimal detection, diagnosis, treatment, and care. It will aid early diagnosis and quality specialist treatment through the network of centres of excellence, which are part of the European Reference Network for paediatric cancers (ERN PaedCan).  
Sound policy, high-impact research and excellent patient care relies on access to quality information. The European ‘childhood incidence data’ section of the European Cancer Information System brings data together from across Europe. This holds real potential for researchers, policymakers, patients, citizens, and stakeholders in Member States to better monitor trends and outcomes for different diagnostic groups of childhood cancer.
Access to trials and therapies
These initiatives are most encouraging, and we believe there is much more we can do together that will positively impact paediatric oncology. Last month, the EU-X-CT Initiative was launched jointly by the EFGCP and EFPIA to improve access to clinical trials and ground-breaking treatments across the EU. Together, we aim to tackle the hurdles that patients, their physicians and investigators face when seeking to join trials in another EU country.
The EFPIA Oncology Platform also commissioned the Innovation for Sustainable Cancer Care report, which was published last month and endorsed by a dozen organisations. It highlights current technologies that can be incorporated to help redesign cancer care to improve outcomes and treatment accessibility in a sustainable manner. These include eHealth tools used to support the well-being of children and teenage cancer survivors.
Up to 30% of children affected by cancer suffer severe long-term consequences. In keeping with our commitment to improve cancer survival, we must continue to expand the focus on the needs of survivors.
That’s where initiatives including the ‘Cancer Survivor Smart-Card’ comes in. It will help childhood cancer survivors, through long-term monitoring of outcomes and potential toxicity of treatments, rehabilitation, psychological support, educational, connectivity with healthcare staff, and information about past clinical history.
In all of this, our focus must be on patients. Children affected by cancers should always be at the centre of muti-stakeholder discussions. We must, at every opportunity, listen to the voice of young patients. This is why we are proud to support the EU Network of Youth Cancer Survivors.
First-in-child innovation
Finally, EFPIA and its members will maintain our commitment to advancing innovation in childhood cancer. In the context of the forthcoming reviews of European pharmaceutical legislation, orphan medicines and the Paediatric Medicines Regulation, we advocate a shift from an ‘adult centric’ approach to become more ‘child-centric’. To this end, we should encourage ‘first-in-child’ innovation where possible.
Embracing this concept requires us to think about how we define diseases. Today, the underlying cause of many conditions is better understood than ever before. This presents opportunities to develop therapies that directly target the cause of the disease.
A medicine’s mechanism of action (MoA) may mean that it can be used for more than one condition, if there is evidence that two or more diseases share an underlying cause. That is why the definition of a disease should include its fundamental cause.
EFPIA proposes a framework for this child-centric approach, where the Paediatric Investigation Plans (PIPs) can be based not on adult indications, but on the paediatric unmet medical need and the MoA of a therapy. This would open up new opportunities to study existing adult therapies in different childhood illnesses if both have the same underlying cause. However, this approach must always be underpinned by unequivocal scientific evidence.
As we mark International Childhood Cancer Day, we acknowledge the distance we have travelled together over several decades, the major collaborative initiatives under way, and commit ourselves to press ahead in the future fight against paediatric cancers. We owe it to children affected by cancer today and in the years ahead.
[i] European Commission. (2021). Europe’s Beating Cancer Plan 2021. Brussels: European Commission.

Marie-Sharmila Blandino

Marie-Sharmila Blandino is vice chair of the EFPIA Oncology Platform and director global access strategy lead oncology...
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