Let’s make it right: A fit-for-purpose EU Paediatric Regulation can deliver for all children and adolescents with cancer

A political commitment to unleash the life-saving potential of the EU Pharmaceutical Strategy is needed say Gilles Vassal, Board Member of SIOP Europe, and Delphine Heenen, Committee Member of CCI Europe.
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01 Jul 2022

Each year in Europe, over 35,000 new cases of paediatric cancer are diagnosed, with more than 6,000 children and young people dying from cancer. The majority of the estimated 500,000 survivors in Europe experience a wide array of adverse long-term side-effects caused by the disease and treatment, and face barriers accessing appropriate follow-up care and full participation in society.

Against this backdrop, the EU Paediatric Regulation was launched in 2007 with the aim of meeting the highly specific therapeutic needs of children by committing to developing better medicines. Unfortunately, 15 years into its implementation, only 12 anticancer medicines in the EU have been approved for use in children, in contrast to over 150 for adult cancer patients. This insufficient pace of innovation cannot serve paediatric cancer patients appropriately across the full range of specific malignancies that affect this population.



In the upcoming Paediatric Regulation’s revision, we need to put the needs of children first. Placing the focus on First-In-Child innovation and finding the right incentives can lead to more and better medicines for this vulnerable population. Curing more children with cancer, curing them better and reducing inequalities is SIOP Europe’s mission which we cannot attain without the continued support of committed EU decision-makers. The European childhood cancer community stands ready to engage in fruitful dialogue to define a forward-looking Pharmaceutical Strategy for Europe’s youngest ones.” Gilles Vassal, SIOP Europe Board Member

In 2021, the European Society for Paediatric Oncology (SIOP Europe), representing all clinicians and researchers working in the field of childhood cancers, and Childhood Cancer International – Europe (CCI Europe), being the voice of patients, survivors and their families, contributed to the Open Public Consultation on the revision of EU rules on medicines for children and rare diseases. Both organisations united around the persisting lack of sufficient investment and stalled innovation, concluding that the current Paediatric Regulation cannot address the urgent needs of young cancer patients. In light of the pronounced lack of new anticancer medicines for children and adolescents across Europe, the shortcomings of the legislation need to be met with a strong political will for change.

Urgent action is required to revise the regulatory environment in line with the European childhood cancer community’s evidence-based proposals for the development of innovative medicines for children and adolescents with cancer, and improving access to new and essential medicines for these patients. Among others, these recommendations call for paediatric medicines development driven by the mechanism of drug action, disease biology and patient needs, which would require the deletion of Article 11b of the Paediatric Regulation. In addition, there is a strong need for better-tailored incentives to ensure both an early start of paediatric development as well as ‘First-in-Child’ studies and marketed authorisation against specific paediatric biological targets. The revised Paediatric Regulation should also provide a framework for the continuous identification and evaluation of patients’ needs while allowing for a multi-stakeholder discussion on the prioritisation of novel medicinal products. Multi-stakeholder Paediatric Strategy Forums are a hallmark of the ACCELERATE Platform – a successful model bringing substantial added value to medicines development in paediatric oncology by uniting all interested parties with the goal of finding common solutions.

“When my 12-year-old son relapsed from cancer in 2015, he was offered old toxic off-label drugs to fight off his cancer. 7 years down the road the treatment options for his disease have not improved. Young patients need political backing and hands-on expertise to transform the EU Paediatric Regulation: only then will they have access to more on-label targeted drugs that are more efficient and less toxic for their growing bodies. We count on the European Parliament and all political groups to identify strong MEP Champions to lead on the Paediatric Regulation file and help all children and adolescents with cancer in Europe.” Delphine Heenen, CCI Europe Committee Member

To further elucidate the existing issues and bring forward the need for increased political commitment by all EU institutions in revising the Paediatric Regulation, SIOP Europe and CCI Europe are co-organising a virtual event with the MEPs Against Cancer (MAC) Interest Group and the Association of European Cancer Leagues (ECL).

On the occasion of Gold September – Childhood Cancer Awareness Month, EU stakeholders are invited to tune in on Thursday 29 September 2022 at 12:00 – 13:30 CEST to the policy exchange on championing the Paediatric Regulation in the future revision of EU pharmaceutical legislation. 

The European childhood cancer community looks forward to the upcoming revision of the EU pharmaceutical legislation and will continue to advocate for committed and concerted action towards a fit-for-purpose Paediatric Regulation and a brighter future for children and adolescents with cancer all across Europe.



Championing the Paediatric Regulation in the future revision of EU pharmaceutical legislation

Gold September – Childhood Cancer Awareness Month 2022

SIOP Europe and CCI Europe invite you to register for the Gold September – Childhood Cancer Awareness Month 2022 – EU Policy Webinar on 29 September 2022 (12:00 – 13:30 CEST).

This year’s event will be dedicated to championing the Paediatric Regulation in the future revision of EU pharmaceutical legislation.

Find out more on the event webpage

Read the most recent articles written by SIOPE - Shining a light on Childhood Cancer Awareness Month