The Importance of Early Collaboration in Developing Paediatric Cancer Medicines



In March 2017, a SIOPE event at the European Parliament delivered a clear message: collaboration between all stakeholders is the only way to ensure an effective development of new drugs for paediatric cancers. The SIOPE International Childhood Cancer Awareness Day ‘Development of paediatric cancer medicines - Milestones and the Road ahead towards more life-saving innovation’ (7th March 2017, Brussels, BE) was the occasion to reflect upon the latest accomplishments and the persisting obstacles in the field of paediatric cancer drug development, and it also coincided with the ongoing preparation of the 10-year report regarding the EU Paediatric Medicines Regulation (EC No 1901/2006) by the European Commission. The event was hosted by MEP Elena Gentile (S&D, IT) – a Member of the European Parliament who led the recent European Parliament Resolution on the Paediatric Regulation with the intervention of another leading MEP on the initiative -  Françoise Grossetête (EPP, FR),  and it was attended by other Members supporting the efforts of the SIOPE community such as , MEP Patrizia Toia (S&D, IT), and MEP Nessa Childers (S&D, IE). Children are left behind in terms of research and innovation: more than 6,000 young people die of cancer every year in Europe, and only 10% of the 35,000 childhood cancer patients has access to innovative treatments. A parent advocate from Unite2Cure exemplified this through her personal story as, when her child relapsed, he was offered drugs that were already 15-20 years’ old. ‘Beyond the numbers’ she continued ‘there are children losing weight and hair, and parents falling in despair; moreover, surviving cancer after 5 years doesn’t mean that the rest of the life is a fairy-tale, as treatment long-term toxicities affect the lives of an impressive number of survivors’. Despite the mandate of the Regulation, however, too little  innovation was brought to paediatric oncology. Although the development of new targeted therapies for this age group has a cost, the cost of failing to address this problem will be paid by children and their families. ‘To introduce safe and effective innovative treatments into standard care’ was therefore selected as one of the main objectives of the widely endorsed SIOPE Strategic Plan for the next decade, and paediatric oncology partners have already reached a few milestones such as the European Parliament Resolution on the Regulation as well as the statement of support by the EU Commissioner for Health Vytenis Andriukaitis at the SIOPE Session at the ECCO Congress (January 2017). Partners in the multi-stakeholder ACCELERATE Platform are also addressing the existing obstacles in this field – including proposals on how to amend the Regulation – and last week they agreed on the 5 next objectives to speed up innovation:

  1. Paediatric drug development should be based on the drug’s mechanism of action (MoA), and not on its specific indication (for adult/paediatric patients): this should allow some flexibility and allow the use compounds that could validly treat other diseases than those they were intended for;
  2. Drug prioritisation based on strong biological, preclinical/clinical data as well as on the MoA to guide the choice of compounds to be evaluated in children;
  3. The existing delays in starting a paediatric investigation plan (PIP) should be reduced;
  4. The “18 years’ dogma” in relation to clinical trials enrolment should be challenged, as it has been proven that trials including both adolescents and adults are feasible;
  5. New incentives and rewards should be proposed to making research in this field more “attractive”.

These positive developments were only possible thanks to a strong cooperation and exchange between all the stakeholders. Therefore, also during yesterday’s event, panellists representing different types of expertise – patients and parents’ organisations, academic research, pharmaceutical companies and EU regulatory networks – engaged in an interactive discussion with EU stakeholders on how to synergise their efforts and ensure that these initiatives will not be isolated. For instance, a representative from the Paediatric Rheumatology International Trials Organisation (PRINTO) explained how they successfully collaborate with pharmaceutical companies early in the drug approval process (starting in phase I, before the PIP) and, thus, proposed that the consultation between academia and regulatory agencies should start earlier in the process. At the end of the event, the different perspectives of participants were not an obstacle anymore: change needs happen, and they will not give up until the needs of childhood cancer patients will be met.



SIOPE, the European Society for Paediatric Oncology (, is the only pan-European organisation representing all professionals working in the field of childhood cancers in close cooperation with parents, patients and survivors. With more than 1,600 members across 34 European countries, today SIOPE is leading the way to ensure the best possible care and outcomes for all children and adolescents with cancer across Europe. The SIOPE Strategic Plan – endorsed by all partners in the field – is based on 7 key objectives and will inspire all future initiatives in this field.