The role of collaboration in tackling paediatric cancer

paediatric cancer

Prof. Martin Schrappe, President of the European Society for Paediatric Oncology details the vital role of collaboration in tackling paediatric cancer across Europe

Paediatric cancer is a rare disease that comes in multiple subtypes, represents both a life-threatening disease and a major public health issue. Each year, more than 6,000 children and young people die of cancer and 35,000 new cases are diagnosed. Despite improvements in the cure rates over the last decades, childhood cancer remains the leading cause of death in children above one year of age in Europe.

Moreover, childhood cancer survivors may face long-term side effects that negatively affect their quality of life and participation in society. Among the factors that define the policy and regulatory priorities for the paediatric cancer sector, are the rarity of individual paediatric cancer types, their dissimilarity from adult cancers and their high collective health burden across Europe.

Cross-border collaborative research is, therefore, vital to overcome small sample sizes at the national level and to foster scientific advancement towards more and better cure. The development of innovative therapies for children with cancer should require urgency since the medicines currently available are sometimes decades old, leading to stagnating cure rates and long-term sequelae.

Another important orientation is cross-border access to the right expertise to ensure optimal treatment and follow up – and to address inequalities in survival rates across Europe.

Since 1998, the European Society for Paediatric Oncology (SIOP Europe or SIOPE) has been active on the European landscape, working closely with parents, patients and survivors to influence legislation and taking part in European-funded projects. In the process, the community has formulated and is currently implementing the SIOPE Strategic Plan with the goal of increasing the cure rate and the quality of cure for children and adolescents with cancer by the year 2025.

Coordinated funding for clinical trial activities across borders is a key challenge. An important past EU project – ENCCA (European Network for Cancer Research in Children and Adolescents) – enabled the creation of the SIOPE Clinical Research Council, bringing together all clinical trial groups and national societies for paediatric haemato-oncology in Europe, Continuity is instrumental to build on the achieved results and SIOPE has been advocating for the next EU Framework Programme for Research and Innovation to further support stable and sustainable clinical trial platforms and international collaborations for paediatric haematology and oncology. In addition, SIOPE is closely monitoring the process leading up to the implementation of the EU Clinical Trials Regulation and has contributed to the legislative process.

Boosting access to innovative therapies for childhood cancer is vital. In 2007, the EU Paediatric Medicines Regulation was launched with the aim of meeting the specific therapeutic needs of children with better evaluated and authorised medicines. However, whereas the adult cancer sector has seen a flurry of innovation over the past years, only very few new medicines have been authorised for cancer affecting children since 2007.

SIOPE Europe together with partners from the parents and patients’ community are working with all stakeholders, including industry and regulatory agencies in the ACCELERATE platform, to improve the implementation of the EU paediatric medicines regulation and continue to advocate a series of targeted changes to accelerate the development of medicines for paediatric cancers.

Specific proposals include running the paediatric strategy forums, jointly coordinated by ACCELERATE and the European Medicines Agency (EMA), to share information and advance learning in a pre-competitive setting, breaking the 18-years dogma for participation in clinical trials, FDA and EMA alignment, earlier submission of paediatric investigation plans and implementing development plans based on paediatric tumour biology and drugs’ mechanism of action.

The poor diagnosis and treatment of childhood cancer is still a reality in certain cancer types and countries, with large disparities in access to standards of care and specialised interventions across Europe. The EU Directive on Cross-Border Healthcare has engendered the development of the European Reference Networks (ERNs), one of which is fully dedicated to childhood cancers (ERN PaedCan). This network has been instrumental in furthering the paediatric cancer agenda, making it possible to share expertise across Europe, thereby facilitating the lives of both healthcare providers and patients whose conditions require specialist expertise and tools not widely available due to low case volumes or lack of resources.

As the EU Commissioner for Health and Food Safety, Vytenis Andriukaitis has said: “no country alone has the knowledge and capacity to treat all rare and complex conditions”. ERN PaedCan aims to increase childhood cancer survival and quality of life in the long term, by fostering cooperation and training, with the ultimate goal of reducing current inequalities in childhood cancer survival and healthcare capabilities across the EU Member States.

Virtual tumour boards provide quality assurance and are a cost-effective way to deliver guidance in paediatric cancer treatments. Sustainability of this new mechanism is vital to ensuring equal access to the best knowledge for all children and young people with cancer, no matter where they live in Europe.

Another area that calls for policymakers’ attention is childhood cancer survivorship. The population of childhood cancer survivors in Europe is growing. These survivors are often faced with distinct and severe chronic health challenges throughout their lifespan. It will become increasingly important to put in place mutually agreed guidelines on the surveillance of childhood cancer survivors and the appropriate models of care.

Other orientations include the exploitation of e-health to underpin research, outcomes data collection and long-term health surveillance. Successful approaches in this field include multi-stakeholder dialogue and collaboration, as well as balancing data privacy and the need for research progress.

As it has become common knowledge that multidisciplinary collaboration in therapeutic research would make improvements in outcomes be possible, SIOP Europe continues furthering dialogue and cooperation with various EU actors to address the urgent needs of children and adolescents with cancer. Progress is only possible through joint efforts and an enabling legislative and regulatory environment that facilitates cross-border approaches and targets the priority areas identified jointly by professionals, parents, patients and survivors. The importance of collaboration between all relevant stakeholders in developing paediatric cancer therapies cannot be stated enough.


Prof. Martin Schrappe


The European Society for Paediatric Oncology (SIOP Europe)

Tel: +32 2 775 02 12


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