Less than 10% of young patients with a relapsed terminal cancer have access to innovative therapies in Europe, and very few new medicines reach children and adolescents with cancer in Europe. Better access to these therapies – increasingly available in the case of adult cancers – and an improved referral of patients to expert investigating centres are urgently needed.
Despite the EU Paediatric Medicine and the Orphan Drug Regulations, innovative drug development in paediatrics is far from being satisfactory, as:
- The legal requirements for paediatric drug development are often waived, because the treatment being researched is for a disease which does not occur in children. This is despite the fact that many cancers in children do not occur in adults. Furthermore, the way the drug works (its “mechanism of action”) in an adult type of cancer may be relevant to a cancer type that only occurs in children;
- There are major delays in starting clinical trials of oncology drugs for children, as the process often requires to wait for the drug to show promise in adult cancer patients without regard to whether they could already benefit children. Financial incentives to develop drugs for a cancer which only occurs in children often come too late for pharmaceutical companies to consider investing in paediatric oncology research;
- When eventually mandated, Paediatric Investigation Plans (PIPs) are approved following lengthy negotiations with health authorities and too often prove unfeasible because they focus on the rare occurrence of an adult cancer in a child rather than the potentially wider use of the new drug in other relevant paediatric cancers.
SIOPE, CDDF (Cancer Drug Development Forum) and ITCC (Innovative Therapies for Children with Cancer) joined forces and successfully built a Multistakeholder Platform to improve new oncology drug development for children and adolescents. Created in 2013 within the ENCCA project by representatives of parent/patient advocates, academia, industry and regulators, the Platform works on proposals to address the identified bottlenecks in this field (a high-level
article on the Paediatric Platform has been published in press).
Building on the constructive discussions of this active platform, SIOPE shared some important recommendations on the subject during two recent events: the
CDDF-ITCC-SIOPE Paediatric Conference (20-21 January 2016, Brussels) and the
International Childhood Cancer Awareness Day (27 January 2016, European Parliament, Brussels).
Open discussions characterised both events, where the need to accelerate drug development for paediatric cancers and to amend the Paediatric Medicines Regulation was emphasized. In particular, participants in the SIOPE event to mark International Childhood Cancer Day
‘Speeding up Innovation, Saving Lives’ – hosted by Member of the European Parliament Glenis Willmott (S&D, UK), a strenuous supporter of our community over the past years – suggested an increase in public-private partnerships with the pharmaceutical industry and observed the successful reward system of the American “Creating Hope Act” for companies developing innovative paediatric drugs. Members of the
Unite2Cure network of parents, parent organisations and patient advocates from across Europe had the opportunity share their vision for a better access to quality treatment for children and young people with cancer.
On these occasions, different categories of stakeholders in the field made a call for concrete action to improve the availability of effective innovative cancer medicines for children and adolescents. Their requests were channelled into a
stakeholders’ joint proposals, focusing on the following proposed amendments to the Regulation:
- Mandatory paediatric investigation of medicines based on ‘mechanism of action’ rather than adult disease;
- Prioritisation of drugs in order to preserve and match rare and frail children with cancer to the best available therapies;
- More effective and flexible rewards to drive early clinical development of drugs for childhood cancers and specifically for those cancers which only occur in children.
SIOPE representatives encouraged all stakeholders to increase their cooperation in order to suggest solutions for safer and more effective innovative treatments, one of the objectives addressed by the widely endorsed
SIOPE Strategic Plan (for more information, please read the
SIOPE press release).
More information:
CDDF-SIOPE-ITCC Paediatric Conference
International Childhood Cancer Awareness Day