Researchers & Healthcare Professionals

In paediatric cancer research, the overall objective of ENCCA with respect to research matters centers on several points:

Harmonisation of clinical trials for children and adolescents with cancer in Europe

The majority of clinical trials for children and adolescents with cancer are investigator-driven and sponsored by academic institutions. However, in every EU Member States there are different organisational and sponsorship models, resulting in a lack of co-ordination and in a significant duplication of efforts and costs. The EU Clinical Trials Directive (2001/20/EC) tried to address this problem, but it has been differently implemented at the national level. Moreover, 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) across Europe. Although the Directive is currently under revision to find better solutions (see the proposal for a new Clinical Trials' Regulation), there is still a strong need to work with the European clinical trial groups to define standard approaches to increase the efficiency of investigator-driven clinical trials and risk-based assessment of trials in children.


Share data and tools

While there is an increasing amount of high-quality genomic and biological data available on childhood cancers, it is challenging to share data at the European level and, thereby, reach a higher level of understanding of the cancer biology in this particular age group. Often, it is difficult to obtain data on licensed drugs from drug manufacturers. However, sharing data is becoming increasingly mandatory to integrate knowledge from the different trials in Europe and effectively monitor outcome and long-term sequel. ENCCA is to encourage data-sharing through a common knowledge management platform that will improve access to datasets and support data-sharing management with the relevant infrastructure settings.


Enhance innovative methodology and designs for clinical trials

The challenge in paediatric oncology is to run trials in rare diseases.
The current trial classification does not make the distinction between studies where the risk is equivalent to standard care and those that are aimed at innovation (e.g. testing a new drug). Having similar requirements for all trials on medicinal products is not a ‘one-size-fits-all’ solution, and as such, a clinical trial should follow a development path that places the proposed product within the therapeutic arm of the disease.
It is obviously important to understand the disease (e.g. its incidence, prevalence, etiology, etc.), its manifestations, and its standard of care. ENCCA will address the issue of methodology and designs for clinical trials for drug development in rare and very rare paediatric malignancies by developing a more sophisticated patient stratification through its network of biostatisticians.


Financial sustainability and co-funding of the research agenda

Innovative therapies transformed into standard of care should be a priority in Europe to improve child health in the forthcoming decade. This research needs to be supported by funding from EU Member States and charities. ENCCA will address this issue by creating a model to streamline existing funding opportunities in EU Member States to stimulate collaborations between academia and industry, so as to improve visibility of the research agenda and incite public and private funders.


Improve access to new anti-cancer drugs for children in Europe through biology-driven drug development

To date, paediatric anti-cancer drugs developed in Europe remain scarce when compared to the average of 600 new anticancer compounds developed every year to meet adult cancer needs. Hence, there is an urgent need to select some of the most promising compounds to introduce them into standard of care for children. ENCCA is to reinforce early drug development to prioritise and streamline the development of innovative therapies in a comprehensive programme. A biology-based drug development programme will be run, addressing each main malignancy type within the European Paediatric Medicines regulatory framework. ENCCA aims to establish a strong partnership incorporating all stakeholders: the paediatric oncology community, the pharmaceutical industry and regulatory bodies.


Biology-driven treatments encouraging tailored-made medicines for leukaemias and malignant solid tumours

ENCCA endeavours to facilitate the integration of biological prognostic factors into the main Phase III clinical trials in Europe, aiming to improve therapeutic strategies by combining complex biological and genetic data with novel molecular and imaging treatment measures response to risk groups. These personalised biology-driven treatments shall limit recourse to unnecessary adjuvant therapies.


Patient-oriented research as an attractive career for clinicians

The attractiveness of career in clinical research has declined in recent years, resulting in a shortage of qualified researchers. This is mostly due to the current constraints generated by the implementation of the clinical trials' regulations, indirectly affecting creative and innovative research. ENCCA is keen to encourage clinical research careers through accredited training schemes and improved conditions for clinicians. To this end, it will create a fully comprehensive e-mobility centre where high-quality information, training and optimal facilities will be available for young clinicians.


Training and Education

Paediatric oncology is currently witnessing a lack of coordination resulting in training gaps in certain fields. To remedy this problem, ENCCA is to set up a series of multidisciplinary paediatric specific oncology training sessions to harmonise knowledge and standard of care.