Multinational clinical trials
Multinational clinical trials are vital to ensure optimal treatment for young people diagnosed with cancer as well as to further improve the outcomes after treatment. Clinical research is necessary to combat the burden of cancer: over the past 40 years paediatric oncology in Europe made considerable progresses in increasing patient survival rates of up to 80% from previously 10%. This was only achievable through close collaboration in multinational clinical trials.
The majority of clinical trials for children and adolescents with cancer are multinational, investigator-driven and sponsored by academic institutions. However, paediatric oncology researchers cannot always afford the costs linked to trials in children. This is mainly due to the current lack of commercial sponsorship for rare diseases’ drugs and to the huge bureaucratic burden to initiate a trial. As each EU Member States have implemented differently the Clinical Trials Directive (2001/20/EC), and the high proportion of anti-cancer ‘off label’ drugs used in the paediatric age group (due to the lack of appropriately licensed drugs) led to variable definitions of Investigational Medicinal Products (IMPs), there is a current lack of co-ordination and a significant duplication of efforts and costs in multinational trials across Europe.
Clinical Trials Regulation
SIOPE and all European clinical trial groups made their best efforts to define standard approaches and increase the efficiency of investigator-driven clinical trials in children and adolescents. On 2 April 2014, the Clinical Trials Regulation (CTR was adopted by the European Parliament (594 votes in favour, 17 against and 13 abstentions).
Although still not perfect from our perspective, the new Regulation is a good step towards reversing the negative impact of the former Clinical Trials Directive on the conduct of paediatric clinical trials in Europe. Throughout the whole revision process of the former Directive, SIOPE successfully approached several EU policy makers, including SIOPE’s long-lasting supporter MEP Ms Glenis Willmott, rallying their support to our suggested amendments to the Regulation. This demonstrates SIOPE’s capacity to fruitfully contribute to the amelioration of children’s cancer treatment both in practice and through effective advocacy.