1. Childhood cancers across the rare cancers ERN
JARC General Assembly
26 October 2018, Milan (IT)

2. ERN PaedCan: Vision
Equal and possible best paediatric cancer care and outcomes no matter where in Europe children live
EC Vision on ERNs: A sustainable European Structure

3. ERN PaedCan: Mission
Reduce inequalities of childhood cancer outcomes across Member States
By supporting access to up to date diagnostics and treatments by facilitating the exchange of expertise and knowledge
Implement Virtual Tumour Boards
Medical expertise and knowledge travels rather than patients
Proportionate & appropriate Cross-Border Healthcare
To unite the best specialists across Europe to tackle complex or rare paediatric cancer conditions that require highly specialized interventions and a concentration of knowledge and resources.

4. ERN PaedCan Members 2017 57 Full Members 18 Members States
28% members with gross national income <90% of EU average

5. ERN PaedCan Governance

6. The Strength of Collaboration & Shared Knowledge
16 European SIOPE Clinical Trial Groups
Composition of the Network:
Management Team (NMT): ERN-PAEDCAN Coordinator, Network Manager (NM), SIOPE (European Society for Paediatric oncology) Officer and president, SIOPE dissemination manager (DM)
Quality Assurance Board (QAB): CCI- E (Childhood Cancer International )– Europe , Internal ERN-PAEDCAN evaluators (Richard Sullivan and Pam Kearns tbc)
Advisory Board (AB): 2 representatives from: CCI-E, Ethics Advisory Committee, European Clinical Research Council, -Innovative Therapies for Children with Cancer, TYA ( Teenager and Young Adults with Cancer European Group Leaders, Non-EU expert collaborators;
Ethics Advisory Committee: The EAC will be composed of 2 EAC members who will provide advice to the ERN-PAEDCAN consortium. The role of the EAC is to keep participants well informed about ethical regulations relevant to the clinical trials. It also may also study broader ethical and social issues, like the protection of patients in research etc.
Hubs of Coordination:
International coordinating centre of paediatric oncology trials in Europe (European Clinical Trial Group [ECTG] coordinating centres)
HoCs fulfilling the criteria above identified by the respective representative of European National Paediatric Haemato-Oncology Societies (NAPHOS)
HoCs with tumour entity specific special expertise/techniques as identified by the respective ERN-PAEDCAN subnetwork (examples: Retinoblastoma, Hepatoblastoma, Wilms tumour, Very Rare Tumours, etc.)
HoCs from countries with low health economy rate (LHEAR countries HoCs, mostly former Eastern European countries): WP6 of the ERN-PAEDCAN pilot project ExPO-r-Net has developed and distributed a self-assessment questionnaire for LHEAR countries to check their ability to provide standard baseline care for patients and are going to be supported by virtual advice in tumour boards with twinning ECTG coordinating HoC. This survey identified a number of eligible centres with sufficient quality and patient numbers, which were invited to participate in the ERN-PAEDCAN. LHEAR country HoCs which meet specified criteria to fulfil the European Standards of Care as assessed in survey during the ExPO-r-Net project
Affiliated centers: Centers of expertise outside the ERN-PAEDCAN, which provide significant expertise to the ERN. i.e.: Vienna is a HoC but for special treatment of Proton Therapy patients are sent to Wiener Neustadt)
National Parent´s and Survivors` Organisation (PaSOs): As identified in the roadmap present an important point of contact to support patients and parents in the respective cross-border healthcare situation. They are an integral part of the ERN-PAEDCAN structure.
Strategic Management (Between NMT, Advisory Board und QAB)
Implementation management (implementation of activities by the strategic management)

7. ERN PaedCan Implementation Plan
01: Roadmap development
02: Patient and Survivor Organisation (PASO) integration
04: Twinning Programs
05: Survivorship Passport Roll-Out
06: Knowledge sharing and dissemination
08: Best Practice Sharing
07: Very Rare Tumours integration
09: e-Training and capacity building
2017
2018
2019
2020
2021
03: Virtual Tumour Board M1
M10 M9 M8 M6 M7 M5 M3 M2 M4

8. JARC WP9 Childhood cancers
Task 9.1: Mapping accessibility of essential medicines and high quality radiotherapy used to treat paediatric malignancies across Europe Overview of resources across Europe to further foster coordinated movement of patients (ERN PaedCan Implementation plan area 01: Roadmap development)
Task 9.2: Making recommendations on access to innovative therapies through referrals to early clinical trials across Europe
Mapping ERN PaedCan against the ITCC network of expertise and identifying barriers to cross- border referrals (ERN PaedCan Implementation plan area 01: Roadmap development)
Task 9.3: Identifying solutions for optimal care and research of very rare tumours (VRTs) in the paediatric population
ERN PaedCan implementation plan area 07: Integration of VRT
Task 9.4: Making recommendations on models of healthcare for childhood cancer survivors (CCS) based on prior projects
ERN PaedCan implementation plan area 05: Survivorship Passport rollout

9. ERN PaedCan Implementation Plan
01: Roadmap development
02: Patient and Survivor Organisation (PASO) integration
04: Twinning Programs
05: Survivorship Passport Roll-Out
06: Knowledge sharing and dissemination
08: Best Practice Sharing
07: Very Rare Tumours integration
09: e-Training and capacity building
2017
2018
2019
2020
2021
03: Virtual Tumour Board M1
M10 M9 M8 M6 M7 M5 M3 M2 M4
JARC Task 9.1
JARC Task 9.2
JARC Task 9.4
JARC Task 9.3

10. ACCESS TO ESSENTIAL TREATMENTS – ESSENTIAL MEDICINES AND RADIOTHERAPY
JARC Task 9.1
ACCESS TO ESSENTIAL TREATMENTS – ESSENTIAL MEDICINES AND RADIOTHERAPY

11. Task 9.1 Access to essential therapies
Strategic Partners
In the scope
Standard anticancer agents and supportive, palliative and pain control medicines
Radiotherapy equipment and personnel
(Surgery addressed in ERN PaedCan directly)
Relevance for ERN PaedCan
Overall vision of medicines supply/access across Europe
Roadmap identifying centres for high quality RT interventions

12. JARC Task 9.2
ACCESS TO INNOVATION IN EARLY CLINICAL TRIALS FOR PATIENTS IN RELAPSE OR TREATMENT FAILURE

13. Task 9.2 Access to innovation in CTs
Strategic Partner: Innovative Therapies for Children with Cancer (ITCC)
54 European Paediatric Oncology Departments with expertise in conducting early phase trials in children and adolescents and 22 European research laboratories
Common ERN PaedCan and ITCC membership: incl. CCRI
Draft JARC recommendations:
Make information on early phase clinical trials across Europe publicly available, easily accessible, and understandable with particular consideration for the needs of parents;
Set up Virtual Tumour Boards to discuss all relapsed patients and define referral paths;
Strengthen cooperation between centres to ensure continuity of care when patients are referred to participate in an early trial;
Streamline policies for reimbursement of cross-border healthcare (CBHC) in relation to early clinical trial participation (currently do not cover treatments in early development and hence prohibit access of patients to innovation);
Explore potential solutions to facilitate travel and accommodation of patients and their family.
Key for ERN PaedCan:
Adopt current rules for CBHC reimbursement - Negotiation between MS needed

14. JARC Task 9.3
SOLUTIONS FOR OPTIMAL CARE AND RESEARCH OF VERY RARE TUMOURS (VRTS) IN THE PAEDIATRIC POPULATION

15. Task 9.3 Very rare tumours
Strategic Partner: European Cooperative Study Group on Paediatric Rare Tumours
Draft JARC recommendations on:
Definition of VRTs in relation to incidence and availability of established structures
Aetiology considerations
Guidelines availability: therapeutic and diagnostic
Access to novel therapies
Research and funding considerations
Key for ERN PaedCan:
Synergies with Implementation Plan area 07: Integration of VRTs
One should consider that a whole group of cancers, pediatric cancers, are rare. This leads pediatric oncologists to make further distinctions between hematological malignancies, brain tumors and solid cancers that are all rare but commonly diagnosed in children and adolescents. In addition, some cancers occurring in children and adolescents are very rare with an incidence of less than 2 cases/million/year [14] such as pancreatoblastoma and pleuropulmonary blastoma, or adult malignancies diagnosed in young people such as colon adenocarcinoma in children.

16. Work Package 9 – Childhood Cancers
Since 2008
the website
harmonized recommendations/guidelines
the virtual tumor board and advisory desk
Work Package 9 – Childhood Cancers
Task 3: “Identifying solutions for delivering optimal care and research for young people with extremely rare cancers”
Implementation partner

17. Defining and listing very rare cancers of pediatric age: consensus of the Joint Action on Rare Cancers (JARC) in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT)
Andrea Ferrari, MD1, Ines B. Brecht, MD2, Gemma Gatta, MD3, Dominik T. Schneider, MD4, Daniel Orbach, MD5, Giovanni Cecchetto, MD6, Jan Godzinski, MD7, Yves Reguerre, MD8, Ewa Bien, MD9, Teresa Stachowicz-Stencel, MD9, Michael Ost, MD2, Chiara Magni, MD1, Pamela Kearns, MD10, Gilles Vassal, MD11, Maura Massimino, MD1, Andrea Biondi, MD, Gianni Bisogno, MD12, Annalisa Trama, MD3
Introduction
Tumors are rare in pediatric age, and some particularly rare childhood cancers have not benefited from advances made by the international pediatric oncology network. While various initiatives have focused on the challenge posed by such very rare pediatric cancers, these entities have yet to be accurately defined and listed. The EU Joint Action on Rare Cancers promoted a consensus effort to produce a definition and a list of very rare pediatric cancers based on epidemiological data.
Methods
Incidence rates were estimated for all the cancers listed by the RARECAREnet (an information network on rare cancers). This list groups cancers by combining ICD-O3 morphology and topography codes, thereby properly capturing the peculiarities of tumors in childhood and adolescence. These incidence rates estimated from the RARECAREnet database (which pools details from 94 population-based cancer registries, and 27 countries, covering 46% of the EU population) were discussed by a panel of specialists to ascertain whether a cancer considered very rare on the basis of its incidence was actually “very rare” from the clinical standpoint too.
Results
Very rare pediatric cancers were identified as those with an annual incidence <2/1,000,000 and corresponded to 11% of all cancers in patients aged 0-14 years. Applying this definition revealed two subgroups: tumor types typical of childhood (i.e. hepatoblastoma, pleuropulmonary blastoma, pancreatoblastoma); and those typical of adult age (i.e. carcinomas, melanoma). The threshold of 2/1,000,000 could also be adopted in populations aged 0-19 years, but in this case there were three (additional?) tumor types (i.e. thyroid and testicular cancers, and skin melanoma) considered “very rare” by the experts (with an incidence rate >2/1,000,000).
Conclusions
The present definition and list of very rare pediatric cancers represents a starting point for prioritizing research based on data and patients’ clinical needs, and for promoting innovative solutions for conducting clinical studies on these cancers.
submitted

18. JARC Task 9.4
Models of healthcare for childhood cancer survivors (CCS) based on prior projects

19. Task 9.4 Childhood cancer survivorship
JARC recommendations incl.:
Systematic inclusion of CCS in cancer policies and programmes (incl. NCCPs)
Meaningful involvement of CCS and professionals across policy cycle
Support to collaborative approaches to define or/and implement:
Standardised surveillance guidelines for late-occurring side effects
Organised health care transition (paediatric to adult) as a standard of care;
Survivorship follow up care plans & interoperable tools to apply them cross- border;
Foster research on CCS: long-term effects, outcomes of novel approaches
Key for ERN PaedCan:
European Long Term Quality Care Models for Cancer Survivors across Member States - need non-competitive European Funding for high-quality guideline development
Secure life-long relevant treatment information about treatment burden and late effects, capitalizing on European eHealth developments - need EU and national support for implementation of Survivor Care Plans & Survivorship Passport

20. Synergies with other JARC WPs
WP6 – Clinical Practice Guidelines
European Standard of Care
Quality care parameters of hospital settings, staffing including psychosocial care [Kowalczyk et al., Eur J Cancer 2016]
Standard of Treatments – European Clinical Trial Groups
Standard reflected in the ECTG - SIOPE - ERN PaedCan Road Map
Tumour entity specific guidelines
Standard for Follow Up Care
PanCare guidelines
Guideline development is a work-intensive, lengthy process currently “in-kind” experts time
To establish European Quality Care Models : Non- competitive European Funding for high- quality guideline development is a need

21. Synergies with other JARC WPs
WP7 – Innovation
EU Clinical Patient Management System (CPMS)
Information travels rather than the patients – concept is brilliant!
The system for free – excellent !
Modern and GDPR compliant consulting system
But
Work intensive processes and major time investments of experts
No reimbursement scheme Europe for advisory functions within health care pathways!
Needs a good balance of HCP engaged – number of partners in ERN PaedCan to increase, but that needs support and management
Need for a smart compensation scheme between individual HCPs across boarders for virtual advise
CPMS needs to be secured as a sustainable European structure

22. Synergies with other JARC WPs
WP8 – Education
Twinning of HCP across Europe for enhanced education, research and better quality in care & treatments
Aspects of Twinning - SIOPE perspectives
Support exchange of Health Care Personnel
Day to day knowledge exchange using “Twinning” within the CPMS platform
Continued medical education WEBinars /CME points
1 funded major educational event to embrace the whole community in need of upgrading knowledge of treatment standards
Enhancement of twinning by non- competitive initiatives to foster research & education
To overcome inequalities across Europe “Twinning” is key and requires non-competitive funding

23. LHEAR country HoCs: Targets of CBHC Cooperation
Country
Institute, Location 1
Croatia
Zagreb UHC 2
Zagreb KDBZ 3
Czech Republic
Praha 4
Brno 5
Hungary
Budapest SEMMELWEIS 6
Latvia
Riga 7
Lithuania
Vilnius 8
Poland
Wroclaw 9
Warsaw 10
Cracow 11
Romania
Bucharest ICF 12
Bucharest JOB 13
Jasi 14
Slovakia
Bratislava 15
Slovenia
Ljubljana

24. ERN PaedCan: Vision
Equal and possible best paediatric cancer care and outcomes no matter where in Europe children live
EC Vision on ERNs: A sustainable European Structure
Enhanced communication of EC & relevant ministry representation of MS-NHS to achieve enhanced MS commitment for the multiple actions to improve patient centric care models on the national level and CBHC commitments!

25. ERN PaedCan and other ERNs
Common ERN topics

26. 24 PaedCan-ERN Paediatric Cancer
ERNS – Common Topics Current Interactions
& Cooperations planned 24
PaedCan-ERN
Paediatric Cancer
BOND ERN
Bone Diseases
CRANIO
Craniofacial anomalies and ENT disorders
Endo-ERN
Endocrine Conditions
EpiCARE
Rare and Complex Epilepsies
ERKNet
Kidney Diseases
ERN GENTURIS
Genetic Tumour Risk Syndromes
ERN-EYE
Eye Diseases
ERNICA
inherited and congenital anomalies
ERN-LUNG
Respiratory Diseases
ERN-RND
Neurological Diseases
ERN-Skin
Skin Disorders
EURACAN
Solid Adult Cancers
EuroBloodNet
Onco-Hematological Diseases
EUROGEN
Urogenital Diseases
EURO-NMD
Neuromuscular Diseases
GUARD-HEART
Diseases of the Heart
ITHACA
Congenital Malformations and Intellectual Disability
MetabERN
Hereditary metabolic diseases
RARE-LIVER
Hepatological Diseases
ReCONNET
Connective Tissue and Musculoskeletal Diseases
RITA
Immunodeficiency, AutoInflammatory and Auto Immune Diseases
TRANSPLANT-CHILD
Transplantation in Children
VASCERN
Multisystemic Vascular Diseases

27. Continuous cross-talk & knowledge sharing
ERN PaedCan started to established closer contacts to investigate synergies and future common activities serving the population needs:
Invitation to ERN Coordinators or representatives to join the ERN PaedCan General Assembly
ERN EUROCAN, ERN GENTURIS, MetabERN,
More joined planning to come!

28. Continuous cross-talk & knowledge sharing
First Result:
ESMO-ERNPaedCan-EURACAN: Clinical Practice Guidelines for diagnosis, treatment and follow-up of bone sarcomas (2018 online!)
Future aspects: Overarching ERN policy actions and distinct “commons” , i.e:
EURACAN: Cross-cutting projects on “common diagnostics entities” with potentially different prognosis related to age and associated inherent genomic profiles & correlation to treatments offered
ERN EuroBloodNet: topics of benign haematology in young people and children
ERN GENTURIS: Cooperation with the ERN PaedCan Familial Leukaemia Network started ( board member), synergies in the Li Fraumeni Syndrom aspects, …
ERN RARE-LIVER: Synergies with the Childhood Liver Tumours Strategy Group (SIOPEL) community of ERN PaedCan (related to specialised surgery including liver transplantation and very rare liver tumour entities apart from hepatoblastoma)
ERKNet Kidney Diseases: Synergies with the SIOPE – Umbrella Study regarding skilled surgery and diagnostics
TRANSPLANT-CHILD: Existing know – how within ERN PaedCan on allo / auto SCT procedures and supportive care for children ( best practice sharing).
………

29. Towards a brighter future for children and adolescents with cancer
THANK YOU!