National registries dedicated to collect epidemiological, clinical and treatment data of children and adolescents with very rare tumours (VRT) exist in four European countries: France, Germany (including Austrian patients), Italy and Poland. Aim of this project is the creation of a Paediatric rare tumour European Registry (PARTNER) linking the existing national registries.
PARTNER will be also linked to a virtual consultation system, a dedicated website and the elaboration of diagnostic/treatment recommendations.
The value of this project is based on the European wide gathering of information on treatment of VRT and the provision of this information to experts generating new guidance recommendations for daily practice. The platform that will be created using innovative IT tools to link the existing databases will enhance European collaboration and facilitate cross border access to dedicated expertise.
Methods and means
The first step will be a process of harmonization of the national registries, (identification of the VRT of interest, selection of variables, use of common definitions and procedures for data management) and discussions of the regulatory issues relevant for the different countries. PARTNER will be then created and linked to the existing registries through the adoption of EUPID, a privacy-preserving, secure and versatile system for pseudonymised patient registration and record linkage. Standard of care recommendations will be also elaborated by a dedicated Working Group. LHEAR countries will be involved in the whole of process creating the basis for a larger EU registry and facilitating recommendations dissemination.
This project will lead to the creation of a comprehensive EU platform that can be easily accessed by EU Health care providers and will ultimately result in improved patients’ care and reduction of the existing inequalities in cancer outcome across EU member states.
SUMMARY OF CONTEXT: Malignant tumours are rare disease in children with an annual incidence rate of 12,7 new cases per 100.000 children per year in Europe. They are constituted by a wide spectrum of different histologies with various clinical and biological characteristics. Leukaemia and cerebral tumours are the most frequent diagnosis followed by less common entities. There is a group of paediatric very rare tumours (VRT), which is extremely heterogeneous. VRT may show variable epidemiological patterns, may arise at virtually all anatomic sites, with variable histology and of note, VRT may often be associated with underlying genetic predisposition. Some VRT characteristically arise at the paediatric age, such as pleuropulmonary blastoma or pancreatoblastoma. Other tumours more commonly develop during adulthood or may even be frequent in adult patients (i.e. colon cancer or malignant melanoma) but they only rarely develop in children and adolescents. There is growing evidence that at least some of them are biologically and clinically distinct from their adult counterpart. Last, a substantial group of “borderline” tumours with uncertain clinical behaviour and no standard treatments are usually also included in the group of VRT. During the last years the European paediatric oncology community has increasingly recognized the necessity to develop projects dedicated specifically to rare paediatric tumours to overcome all these obstacles. In the first decade of the new millennium national initiatives have been launched in different European countries. Albeit with rather different organizations and activity levels, these national groups share the idea that multinational cooperation is needed to improve our knowledge and the outcome for children with VRT. The EU funded (until June 2017) ExPO-r-Net (European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment, www.expornet.eu) project has paved the way to integrate VRT in a European network by promoting the establishment of an international VRT tumour board, the formulation of recommendations for the diagnosis and treatment of an initial group of VRT, the creation of an international advisory desk with a virtual consultation system (VCS) able to give international expert advice on difficult cases. In addition a website dedicated to inform families and the public. (http://www.raretumors-children.eu) was launched in 2016. Within the project a model has been elaborated to create an effective reference subnetwork, part of the ERN PaedCan, dedicated to children and adolescents with VRT.
OVERALL OBJECTIVES: PARTNER aims to create a European Registry dedicated to collect diagnostic, treatment and outcome data of children and adolescents with VRT linking existing national registries and to provide a registry for those countries not already having a registry for VRT in place. The European Registry will be an essential part of the activity of the VRT subnetwork part of the ERN PaedCan. The possibility to link the registry with a virtual consultation system and the elaboration of diagnostic/treatment recommendations will create a platform that can be easily accessed by EU Health care providers. The increasing expertise in VRT based on the data collected in the European registry will increase the capacity to provide international consultation and define standard of treatment recommendations. This will ultimately result in improved patients’ care and reduce currently existing inequalities in cancer outcome across EU member states.
STRATEGIC RELEVANCE and CONTRIBUTION OF THE ACTION TO THE HEALTH PROGRAMME: The strategic value of this project is based on the European wide gathering of information on treatment of VRT and the provision of this information to experts generating new guidance recommendations for daily practice for use by ERN and not ERN Institutions. The proposed tasks and milestones will increase the collaboration among member states in paediatric oncology a
Even though national Groups share the same objectives and in general have a similar organizational model, they still present with many differences mainly due to the national different organizations and regulations. For instance, the different working groups include different VRT in their activities (see table III). Therefore, the existing national VRT registries differ in many aspects: data are collected for different tumours, in different ways, using different IT technologies, and are supported with varying and limited economic resources. In addition, the items included in the respective pre-existing national registries are registered and reported in different languages.Therefore, the important first step within this project will be a process of data harmonization before a European paediatric VRT registry can be established. This processes will includes:
• the identification of the VRT entities that should be included in the EU registry according to international WHO classification,
• the selection of a core of variables of common interest
• the use of common definitions
• the implementation of harmonized procedures for data collection, data quality control, central review, etc.
Moreover this preliminary work will also include the discussion of the regulatory issues relevant for the different countries. The EXPeRT group already started work on a harmonized core data sheet in the context of previous, retrospective analyses (22-25), but this needs further to satisfy the needs of a future internationally harmonized and interoperable registry.
As a second step, a common European registry will be created through the linkage of the existing national registries: the Paediatric Rare Tumours – European Registry (PARTNER). A “collaborative” structure that links national registries with the European Registry, in which the national data are transferred, is more achievable in a short time frame time than a de novo European registry without any link to the national data bases. In addition, the proposed solution fully integrates previous efforts undertaken for VRT on the institutional and national level. Our proposed approach will have the following advantages:
a) No need for new national structures, because the national registries will continue their activities using the already established structures
b) Optimization of economic resources because national registries are using national resources and EU resources will be reserved to the international component of the registry.
c) Reduction of the administrative burden because each national registry will be compliant with the national regulations
d) Easier and better quality of data collection from hospitals because the national language can be used for primary source data documentation and verification
e) Possibility to link data with national biobanks
f) Possibility to link with the future EU proposed IT ERN platform once it becomes available
g) The PARTN- ER project clearly may serve as an interoperable model concept for integrated IT solution for ERNS to connect ERNS whenever topic appropriate.
Implementation action of the following WorkPackages:
- WP1 - Coordination of the project
- WP2 - Dissemination of the project
- WP3 - Evaluation of the project
- WP4 - Analysis and harmonization of data acquisition of the existing national VRT registries
- WP5 - Creation of a European registry for paediatric patients with very rare tumours
- WP6 - Standard of care recommendations for children with VRT
- WP7 - Integration of LHEAR countries in a EU platform dedicated to VRT in paediatric age
- Scheduled meetings
- Creation of the project kit including logo, dedicated brochure, templates
- Integration of PARNTER in the EXPeRT website & SIOPE Intranet
- Surveillance of project management and internal processes – on target evaluation of Deliverables and Milestones.
- Solution-based monitoring and evaluation processes via written feedback to project partners for corrective action, (monitor-review-remedy model). Indicators will include frequency of deadlines being met to deliver reports; participation in meetings and telephone conferences; and internal audit of project targets and resolution of problems.
- Preparation of harmonized recommendations and discussion with international external partners (e.g. ERN EUROCAN in Europe)
The registry activities and its link with the virtual consultation system and the elaboration of diagnostic-treatment recommendations is benefiting
- Patients and their families, patients association are benefiting from an international network that can give advice that may reduce or even avoid in principle patients/families travelling in search for expert care.
- Paediatric oncology centres with low expertise in VRT, because clinician may benefit form a dedicated platform including data registration, expert clinical advice and clinical recommendations
- National Health care systems will benefit from this European effort because patients will be treated according international standards.
- Consortium meetings plan
- PARTNER newsletter disseminated via email and integrated in SIOPE newsletter
- Preparation of the Quality Assurance Plan
EXPECTE OUTCOMES: This project will lead to the creation of a comprehensive EU platform dedicated to the improvement of cares for children and adolescents with VRT.
The registry will be a fundamental part of this platform and its value will be enhanced by its link with the national registry, the VCS and standard of care recommendations.
This platform will increase the EU collaboration and will foster access to expert diagnosis, and treatment ultimately improving the chance of cure of children with VRT across Europe.
National groups interested in VRT will be invited to join the existing EXPeRT creating a much larger Group able establish standard of care applicable in all EU members states. This group will provide a framework to support clinical and biological research on VRT and will be able to increase scientific knowledge. Communication to families will be enhanced and scientific based.
CCRI is developing in collaboration with SIOPE a communication strategy plan to ensure continuity with other
initiatives in the field of paediatric oncology and innovative therapies. Communication target groups for this project will
include all health professionals involved in paediatric haemato-oncology (paediatric oncology researchers, healthcare
professionals, academic experts, nurses, and adult oncologists), parents, patients and survivors’ representatives, but also
project partners, EU policy-makers, industry, regulators and the broader European cancer stakeholders’ community.
Following communication tools should be created:
• PARTNER logo;
• PARTNER brochure;
• PARTNER banner (roll-up);
• PARTNER PowerPoint template for project partners;
• PARTNER 1-page factsheet and presentation for project partners (to disseminate the project at external meetings);
• PARTNER news disseminated via emails and integrated in the SIOPE newsletter;
• PARTNER updates integrated in the SIOPE social media tools.