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Executive Summary:
The European Network for Cancer research in Children and Adolescents’ (ENCCA - funded by the European Union's FP7 program) aimed to improve the quality of life of children, teenagers and young adults with cancer by efficiently structuring collaboration within the paediatric oncology community.
The project was driven by 34 organizations in 11 countries and together with the SIOPE community the following areas of interest were developed: biology, bio-banking, drug development, policy actions to improve the clinical trial framework, population-based cancer registries, special needs of teenager and young adult patients (TYA), innovative statistical methodology, and close collaboration with European Parent and Patient Groups, including ethical aspects in clinical research.
ENCCA established important platforms for Paediatric Oncology: the SIOPE Clinical Research Council as a community voice, the CDDF – ITCC – ENCCA - SIOPE platform to facilitate collaborative partnership with Pharma (re-enforcing mode-of-action driven drug developments), a European Parents and Patients Forum, an Ethical Advisory Committee and a TYA network. The new platforms engaged successfully in European policy initiatives including new Clinical Trial Regulation and forthcoming EU Data Protection Regulation. ENCCA designed an “Advanced Biomedical Collaboration Domain”, a cloud-based solution for the “European Virtual Institute” and the ENCCA Unified Patient Identity (EUPID), a context–based identity management concept. A new Syllabus for Paediatric Haemati-Oncology education was agreed and collaborative training courses (including e-learning) were organised.
ENCCA developed a survivorship passport summarising the patients’ journey through cancer treatments and providing long-term recommendations based on individual cancer treatment related risks. In Austria it became integral part of the Austrian National Cancer Plan in 2014. EU member states are encouraged to follow and to integrate this secondary prevention and quality of life supporting tool. ENCCA also supported an on-line neuro-oncology health e-tracker tool in eight languages for patients after brain tumour treatment to report the outcomes in a standardised way.
To ensure long-term sustainability, ENCCA partners and SIOPE have defined the strategic European Cancer Plan for Children and Adolescents ( - strongly supported by parents, patients and survivors. It was first presented during the European Cancer Congress (25-29 September 2015, Vienna, Austria) and was further discussed at the EU-policy level. This Plan will give directions to the community in the years to come to increase cure rates, improve quality of survivorship, and foster the multi-stakeholder dialogue and cooperation to achieve best results.

Project Context and Objectives:
Around 15,000 young people aged 18 and under, are diagnosed with cancer in Europe each year and with present cure rates about 3000 will die. This makes cancer the biggest cause of death for this age group beyond infancy. The strengthening of research and care is still an urgent need in the field of paediatric oncology in Europe, i.e. the integration of basic, epidemiological, preclinical and clinical research with the implementation and evaluation of interventions in prevention, diagnosis, prognosis, treatment and care.

The objectives of the ENCCA project were:
Creation of a sustainable "European Virtual Institute" for clinical and translational research in childhood and adolescent cancers facilitating the investigator-driven clinical trials to introduce new generations of biologically targeted drugs into standard of care.
Definition of the European Strategy to increase both cure and quality of cure by networking and integrating the different leukaemia and tumour groups for the development of new drugs and biology-driven therapeutic strategies and for the implementation of clinical and translational research in all paediatric malignancies.
Integration all relevant stakeholders, enhance collaboration, reduce knowledge fragmentation and improve communication to increase the critical mass of expertise and capacity to run trials to ultimately spread ‘know -how’ also to countries who are not currently major participants in clinical research.
Improved therapeutic strategies by enabling better access to innovative therapies, knowledge sharing and innovative technology that will be exploited to prioritise drug development and to develop a biology-based therapeutic strategy for each of the different paediatric malignancies.
Substantially improved quality of life of children and adolescents with cancer with particular emphasis on long term treatments side effects to cancer survivors “survivorship passport” to facilitate adequate risk-based advice, follow up treatment and care.
Improve access to care and research for teenagers and young adults through the creation of a European Steering Groups of leading professionals and stakeholders in TYA oncology, to share practice and promote service development, interdisciplinary support and develop specific practice guidance.
Organization of a comprehensive education and training programme for specialists in paediatric oncology on new protocols and standards for clinical trials, for parents/patients to improve their awareness of clinical trials and oncological research.
Fostering substantial discussions on ethical aspects of clinical research engaging all stakeholders involved to elaborate sound proposals and improved adopted modes of actions.
The ENCCA project addressed the scientific and technological objectives in 18 work packages, grouped into three types of activities:
➢ Networking/Integrating Activities
• European sustainable strategy for clinical trial paediatric oncology
• Clinical trial facilitation
• Establishment of the Virtual Institute information portal
• Biology to guide innovative targeted therapy development
• Standardised and innovative methodology for clinical trial design and analysis
• Integrating clinical trials and tumours biology research in bone sarcoma
• Management
➢ Joint Research Activities
• Early evaluation and prioritisation of new anticancer drugs
• Improved therapeutic strategies using predictive biomarkers in leukaemia
• Risk adaptation of therapeutic strategies using prognostic biomarkers in malignant solid tumours
• Clinical epidemiology and prospective registries for patients on standardized protocols
• Clinical research in very rare tumours
• Quality of survivorship
➢ Spread of Excellence Activities
• Dissemination activities
• Facilitation of Collaboration with Pharmaceutical Companies and SME
• Improving Outcomes for Teenagers and Young Adults with Cancer
• Education and training
• Ethical aspects of clinical trials

Project Results:
ENCCA has founded several steering structures:
• European Clinical Research Council (ECRC – as of 12.2015 SIOPE Clinical Research Council):
• Parent-Patient Advocacy Committee (PPAC)
• Scientific and Ethics Advisory Committees
• European Steering Group for Teenager and Young Adults (ENTYAC)
• International Steering Group for Bone Sarcoma
Other major achievements:
• Major policy activities resulting in contributions to the new European Clinical Trial Regulation satisfying major common needs of rare diseases and in particular Paediatric Haemato-Oncology (PHO) (WP2, 4)
• Framework of master contracts allowing delegation of sponsor duties within multinational clinical trials (WP2, 4)
• Advanced Biomedical Collaboration Domain 4 ENCCA (ABCD-4-E), as cloud-based solution for the “European Virtual Institute” building on Integrating the Healthcare Enterprise (IHE) interoperability with context–based Patient Identity Management concept respecting data protection requirements. The prototype and the EUPID (the ENCCA Unified Patient Identity) code were demonstrated at the ENCCA General Assembly in 01.2015 the Beta Version of was presented at the Final ENCCA General Assembly on 18 December 2015.
• Development of a roadmap towards the federation of ENCCA biobanking resources.
• High level statisticians engaged in publishing new methodological approaches, innovative trial designs and analysis of competing events and long term outcome in high profile publications based on ENCCA inherent activities.
• SIOPEL web-based platform holding special features of communications and advice as well as e-learning sessions satisfying the needs of very rare tumour settings (WP11)
• The Children’s International Hepatoma Cooperation (CHIC) database initiative for liver tumours culminated in the establishment of an international consensus classification system for all paediatric liver tumours leading to the new CHIC International Hepatoblastoma Risk Grouping.
• The Cancer Drug Development Forum (CDDF) Paediatric Platform was created in partnership with ITCC, ENCCA, SIOPE to meet this urgent need and promote progress in the field of paediatric oncology drug development (WP8, 16).
• Development of an exemplar prospective clinical biomarker renal tumours study (open in the UK as IMPORT (Improving Population Outcomes for Renal Tumours of Childhood) (WP11).
• The final implementation of a local biobank solution as proof-of-concept for the feasibility of interconnection of existing biobanks towards a pan-European biobanking solution via interfacing of key resources representing the most developed solutions currently in use
• Boosting ground-breaking findings and several landmark translational research papers in the fields of neuroblastoma, brain tumors and laeukemia based on ENCCA-supported biomaterial and bioinformatics (R2) resources in addition to expert networking with high potential impact for future clinical trial design and novel therapeutic options
• Establishment of a consortium of institutions to sponsor phase I and II clinical trials
• Several clinical trials embedded in the ENCCA environment (WP5,6,7,8,9,10,12) with introduction of molecularly targeted treatment in clinical trials on leukaemia (WP9)
– Phase III trials: LINES (neuroblastoma), EUROEWING99/EWING 2008 (Ewing tumours), AIEOP BFM ALL2009 (ALL), EUROEWING2012 (Ewing tumours), rEECur (Ewing tumours),
– Phase II/III trials: PNET 5 MB (Medulloblastoma)
– Phase II trials: Beacon (neuroblastoma), Herby trial (high grade glioma), Bortezomib (relapsed ALL)
– Phase I / II: NECTAR (relapsed T-cell ALL or T-LL), Blinatumomab ( relapsed/refractory ALL), VIDAZA (MDS and JMML)
– PHITT (SIOPEL6 trial, liver tumours) – in planning phase (Phase III)
– Other trials: IMPORT (Wilms tumours and other renal tumours)
• SIOP-E PNET5-MB study has been approved by EC and CA in Germany, France, Finland, Sweden, Italy, Switzerland and Belgium. Ethics Committee approval has been also obtained for Austria. For the first time molecular diagnostics (beta-cathenin mutations and MYC-gene status) is going to be applied prospectively in an international multi-centric study, in order to propose an adaptation of treatment intensity according to biological risk profiling in clinically standard risk group medulloblastoma
• The sponsors consortium was made sustainable beyond ENCCA through the creation of the Sponsor Institutions Committee within the ITCC consortium
• Videoconference based European Tumour Board performed by the bone sarcoma consortium
• European Relapsed Osteosarcoma registry (EURELOS) as part of ENCCA activities (WP7)
• Proposal for standard dataset for population –based cancer registries (WP11) allowing ultimately outcome research.
• 2.0 prototype of the Survivorship Passport providing long term guidance based on informative data sets related to the patients journey of cancer treatment
• Inclusion of the Survivorship Passport project in the Austrian Cancer care plan for the next 5 years
• Commitment of Childhood Cancer Switzerland to introducing the Survivorship Passport in all paediatric hospitals in Switzerland (expected by early 2017)
• Inclusion of the Survivorship Passport in the SIOPE strategic plan for the next decade presented at the EU parliament.
• Online module for assessing quality of survivorship in medulloblastoma (Health tracker).
• New Syllabus for PHO education and several collaborative training courses (including e-learning)
• Proposal for an online educational platform “medworks” for the Paediatric Haemato-Oncology community developed
• PPAC Guidelines - “A Guide to Clinical Trials – For young people with cancer and their parents”:
• A total of 74 research and advocacy papers published
• Establishment of strong relationships with other FP7 and /or EU projects
• The IPR committee created a consensus on the principles and recommendations for contracting with Pharmaceutical Companies
• TYA webpage was developed and is now fully functioning. The Beta Version of the TYA Forum has been set up.
• An agreement in place that all UK NIHR Cancer Trials entering the portfolio will have a lower age of 16 years, following extensive interactions with the National Cancer Research Institute Clinical Studies Groups and the chairs forum
• The Strategic strategic European Cancer Plan for Children and Adolescents
The 7 objectives of the Plan are:
1. Introduce safe and effective innovative therapies into standard care;
2. Install precision cancer medicine to help guide therapeutic decisions;
3. Increase knowledge of tumour biology and speed up translation;
4. Bring about equal access across Europe to standard care, expertise and clinical research;
5. Address the specific needs of teenagers and young adults in cooperation with adult oncology;
6. Improve the quality of survivorship;
7. Understand the causes of paediatric cancers.

Potential Impact:
ENCCA was strongly contributing to the building of a more effective European research area (clinical and translational) by facilitating and coordinating regional, national and joint European paediatric and adolescent oncology research funding programmes between 11 European Member States.
ENCCA aimed to create a sustainable Paediatric Oncology virtual institute and has defined a strategy, tools, explores sustainable resourcing.
ENCCA was bringing together an exceptional consortium of major European clinical trial and research groups with a common aim and commitment to accelerate the translation of scientific discoveries into clinical applications and to accelerate drug development and care quality of children and adolescents with cancer. It will allow translation of the gathered biological and genetic knowledge into molecularly-targeted treatment in clinical trials.
ENCCA was developing solutions to link existing biobanking at European institutions involved in paediatric oncology to better support cooperative efforts to analyse trial-related data. Linking of resources ensured better transfer of information about available tumours and other patient’s samples for collaborative research efforts.
ENCCA was contributing to the realisation of cross-border synergies of human resources in clinical practise and translational cancer research, by supporting capacity building and training activities of multi-disciplinary research teams. This allowed better exploitation of intellectual potential and of pre-clinical and clinical translational cancer research capacities, increasing the quality of translational cancer research and the efficiency of clinical trial process
ENCCA created suggestions for a sustainable legal structure dedicated to paediatric oncology clinical research to manage and coordinate clinical research in Europe and to create mechanisms for financing the organisational structures needed to enhance research and clinical trial structures.
ENCCA created added value with the construction of European Clinical Research Council (ECRC) by developing strategic synergies also on a higher organisational level, namely by further developing strong links with relevant international or European-funded consortia and European Commission.
The joint research activity of ENCCA promoted not only scientific excellence, through peer review evaluation, but also facilitated the achievement of the critical mass needed for conducting long-term research on new preventive, diagnostic, biology-driven therapeutic tools and interventions, with a comprehensive approach and a subject/patient-oriented attitude.
ENCCA had a positive impact in increasing the attractiveness of Europe for biomedical and biotechnology industry focused on the development of anti-cancer interventions for the benefit of the children and young people.
ENCCA established quality measurement processes in its work plan with tools to evaluate long-term outcomes (quality of life, quality of survival) including clinical epidemiology and ethics to explore reasons of outcome inequalities, such as delays in diagnosis or lack of access to clinical trials and relevant expertise.
ENCCA providesd childhood cancer survivors and their health care providers with the Survivorship Passport, a document (in its final version in electronic format) summarizing their cancer related medical history and providing guidance for follow-up based on guidelines approved at European or international level. Aligned to this effort the on-line QoS assessment module for medulloblastoma was a pilot example to be used as a quality assurance instrument both within and without clinical trials.
ENCCA supported an integrated bone sarcoma trial platform which facilitated multinational clinical trials with integrated translational research components.
ENCCA supported the paediatric oncology statistical platforms to develop innovative methodologies, allowing for a more efficient identification of new treatments.

List of Websites:
The address of the project public website:

Contact Us:
Associate Prof. Ruth Ladenstein
ENCCA Project Coordinator
Tel: +43 1 4470 4750, Fax: +43 1 4470 7430
Zoltán Dobai
ENCCA Grant Manager
Children’s Cancer Research Institute
Zimmermannplatz 10, 1090 Vienna, Austria

ENCCA Dissemination Management Team
SIOP Europe
Avenue E. Mounier 83
B-1200 Brussels, Belgium
Follow us on Twitter @SIOPEurope and LinkedIn: SIOPE European Society for Paediatric Oncology