siope.eu

1. Work Package 9: Childhood Cancers JARC General Assembly16th Sept. 2017 Milan, Italy Joint Action ‘724161/JARC’ has received funding from the European Union’s Health Programme (2014-2020) www.siope.eu

2. WP 9: Childhood Cancers www.siope.eu • Task 9.1:Make recommendations on accessibility of essential treatments across Europe • Task 9.2: Make recommendations on access to innovative therapies through referrals to clinical trials across Europe • Task 9.3: Identify solutions for optimal care and research for young people with extremely rare cancers • Task 9.4:Make recommendations on models of healthcare for survivors of childhood cancers based on prior projects.

3. WP 9: Childhood Cancers www.siope.eu 24 Partners: SIOPE (EU) • INT (IT) • EASP (ES) • UP (HU) • FFIS (ES) • INCa (FR) • UVEG (ES) • VHIR (ES) • FISABIO (ES) • GPOH (DE) • MoH Republic of Cyprus (CY) • PanCare (EU) • OUS-KSSB (NO) • HSE (IE) • ECL (EU) • BCR (BE) • CCI Europe (EU) • CCRI (AT) • UCL-ICH (UK) • Bambino Gesu (IT) • Uni Padova (IT) •Institut Curie (FR) •ECPC (EU) • Greek Working Group for Rare Cancers and for Childhood Cancer (EL)

4. JARCWP9: Childhood Cancers Task 9.1: To makerecommendationsto solvebarriersin accessibility of standard treatmentsacross Europe SIOPE Strategic Plan Objective 4: Equal Access www.siope.eu

5. Task 9.1: Essential treatments • Are the essential medicines to cure, support and palliate children and adolescents with cancer available and accessible across Europe? • What improvements can we request to decision makers based on the current situation and needs? www.siope.eu Eighty percent (80 %) of children and adolescents with cancer can be disease-free at 5 years provided that they receive current multidisciplinary treatments

6. Task 9.1: Essential treatments www.siope.eu Milestones and Deliverables: • M9.1: Survey completed & available (Month 21 – July 2018) • M9.2: Mapping of issues to solve done (Month 22 – Aug 2018) • M9.3: Consensus meeting on recommendations (Month 30 – April 2019*) • D9.1: Report summarisingsurvey results/issues and recommendations (M36  M30: April/May 2019) *or May 2019

7. Task 9.1: Essential treatments www.siope.eu SCOPE: • Medicines: Standard anticancer agents and palliative and pain control medicines incl. for administration during procedures • Radiotherapy (RT): Equipment and personnel qualified to workwithchildren STRATEGIC PARTNERS: • Medicines: CCI Europe, ESOP and ESMO • Radiotherapy (RT): CCI Europe, QUARTET (SIOPE committee with EORTC), dialogue ongoing with ESTRO and researchers in the field

8. Task 9.1: Essential treatments www.siope.eu METHODOLOGY (Medicines): • CoreWorking Groupto steer the project • Electronic survey focusing on children’s needs across Europe • Drafting of policy recommendations and final consensus meeting METHODOLOGY (RT): • Mapping of initiatives and resources • Stakeholder engagement/Core Working Group TBC • Survey of paediatric haemato-oncology RT across Europe TBC • Drafting of policy recommendations and final consensus meeting

9. Task 9.1: Essential treatments www.siope.eu PROGRESS YEAR 1* - MEDICINES • CoreWorking Group of experts constituted • SIOPE – CCI Europe – ESOP – ESMO • 1st face-to-face meeting: 14 Sept. 2017, Brussels, BE • Agreement on survey content and methodology (preliminary): • List of medicines • Regional coverage • Survey questions • Target respondents • Methods of data collection * (Oct. 2016 – Sept. 2017)

10. Task 9.1: Essential treatments www.siope.eu ACTION PLAN YEAR 2* - MEDICINES • Electronic survey launch in early 2018, in the meantime: • List of medicines: Validation by experts in different disease areas • List of countries and questions: 1-2 TC exchanges to finalise • Fostering participation at annual meetings of respondents: • ESOP: Jan./March 2018 - SIOPE: Jan./Feb. 2018 - CCI Europe: April 2018 • Preliminary survey results: approx. 6 months from launch • Milestone 9.1 (Month 21 – July 2018): Survey questionnaire completed & available • Milestone 9.2 (Month 22 – August 2018): Mapping of accessibility and issues done

11. Task 9.1: Essential treatments www.siope.eu PROGRESS YEAR 1 - RT • Cooperationinitiatedwith key stakeholders/researchers: • 1st face-to-face exchange: 13 Sept. 2017, Brussels, BE • Scope of Taskrefined: • Consolidating information on availability of equipment incl. proton therapy and qualified radiation oncology personnel in each European country; • Putting forward guidelines on requirements for radiation oncology personnel working with children.

12. Task 9.1: Essential treatments www.siope.eu ACTION PLAN YEAR 2* - RT • Constitution of Core Working Group to steer the project • TBC • Defining methodologies to: • Consolidate updated information on equipment incl. proton therapy • Put forward guidelines on RT personnel working with children • Implementation of methodologies • Milestone 9.1 (Month 21 – July 2018): Survey questionnaire completed & available • Milestone 9.2 (Month 22 – August 2018): Mapping of accessibility and issues done *(focus: Oct. 2017 – April 2018)

13. JARCWP9: Childhood Cancers Task9.2: To makerecommendations to increase and facilitate timely access to innovative therapies within clinical trials SIOPE Strategic Plan Objective 1: Innovative treatments www.siope.eu

14. Task 9.2: Innovativetherapies • What improvements can we request to decision makers to increase and faciliate children’s access to innovation through clinical trials? *Information provided by ITCC www.siope.eu Less than twenty percent (<20%) of children with relapsed cancer in ITCC centres are estimated to have access to an innovative therapy through clinical trials. Overall in Europe, this figure is halved to just ten percent (10%)*

15. Task 9.2: Innovativetherapies www.siope.eu Milestones and Deliverables: • M9.4: Survey on referral patterns completed & available (Month 21 – July 2018) • M9.5/D9.3: List of preclinical models and research teams available (Month 24 – Oct. 2018) • M9.6: Consensus meeting on recommendations (Month 28 – Feb 2019) • D9.2: Report summarisingsurvey results/issues and recommendations (M36  M30: April/May 2019)

16. Task 9.2: Innovativetherapies www.siope.eu SCOPE • Innovativetherapies in earlyclinical trials for patients in relapse/failure STRATEGIC PARTNERS • CCI Europe, ITCC METHODOLOGY • First set of recommendations based on experience in ITCC countrieson facilitating clinical trial referrals • Recommendations developed and supplemented with input from other countries in Europe • Recommendations consolidated and final consensus meeting

17. Task 9.2: Innovativetherapies www.siope.eu PROGRESS YEAR 1 • 1st face-to face meeting: 7 July 2017, Brussels, BE • 23 physicians and 11 parents (identified with CCI) from each of the 14 ITCC countries* • (Including preparatory and follow up liaison by email/TC) • JARC WP9 partners with interest in Task • 1st DraftRecommendationsagreed: • Make information on early phase clinical trials across Europe publicly available, easily accessible, and understandablewith 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; • Continue exploring the S2 certificate policy in relation to early clinical trial participation in view of putting forward concrete proposals; • Explore potential solutions to facilitate travel and accommodation of patients and their family. * AT, BE, DK, FI, FR, DE, IE, IL, IT, NL, ES, SE, CH, UK (12 EU countries + Switzerland and Israel)

18. Task 9.2: Innovativetherapies www.siope.eu ACTION PLAN YEAR 2* • 2nd face-to-face meeting: early 2018 • Scope: non-ITCC countries in Europe • Including preparatory and follow up liaison by email/TC • Consolidating 2nd Draft Recommendations with inputs from non-ITCC countries • Milestone 9.4 (Month 21 – July 2018): Survey on referral patterns completed & available *( focus: Oct. 2017 – April 2018)

19. Task 9.2: Innovativetherapies www.siope.eu Sub-Task: To deliver list of existing preclinical paediatric cancer models &skilled research teams involved in preclinical research • Year 1: • Methodology defined (cell lines, patient-derived xenografts and engineered mouse models will be listed). • Year 2: • Review of paediatric preclinical models: • Commercial collections • Consortiums • Literature review • Milestone 9.5/Deliverable 9.3 (Month 24 – Oct 18): List of paediatric preclinical models and research teams

20. JARCWP9: Childhood Cancers Task9.3: Identify solutions for optimal care and research for young people with extremely rare cancers SIOPE Strategic Plan Objective 4: Equal Access www.siope.eu

21. Task 9.3: Very Rare Tumours • What can we formulate for decision makers to ensure that these specific needs are addressed? www.siope.eu Extremely rare cancers (<2/1,000,000) are i) extremely rare pathologies in children, such as pleuropulmonaryblastomaii) adult cancers occurring in children, iii) childhood cancers occurring in adults. There is an urgent need to facilitate access to expertise for diagnosis and treatment and to strengthen cooperation between paediatric and adult oncology.

22. Task 9.3: Very Rare Tumours www.siope.eu Milestones and Deliverables: • M9.7: Report on issues raised by care and research for young people with extremely rare cancers (Month 12 – Oct 2017) • D9.2: Report summarising proposals to address VRT in paediatric patients (M36  M30: April/May 2019)

23. Task 9.3: Very Rare Tumours www.siope.eu SCOPE • VRT Guidelines – VRT Expert advice / Centres of excellence – VRT Research STRATEGIC PARTNERS • CCI Europe, EXPeRT- European Study Group for Paediatric Rare Tumours METHODOLOGY • Development of report on specific issues faced by children and adolescents with VRT • Development of draft recommendationsincl. based on prior projects (ExPO-r-Net) • Recommendations consolidated and final consensus meeting TBC

24. Task 9.3: Very Rare Tumours www.siope.eu PROGRESS YEAR 1 • Cooperation established with key partners (e.g. EXPeRT) • 1st face-to-face exchange: 29-30 March 2017, Padova, IT • Streamlining methodology and synergies with ExPO-r-Net /JARC WP4 - Epidemiology • 2nd face-to-face exchange: 29-30 June 2017, Brussels BE • Task presented at ExPO-r-Net final conference and ERN PaedCan Launch event • Analysis of incidence of rare paediatric tumours in Europe • Methodology defined as receiving information from database of RARECAREnet (83 European data bases, 2000-2007) – Synergies with WP4 • Preparation of online questionnaire for parents with CCI: • Awareness of paediatric VRT structures at national level • Perceived need for action to improve paediatric VRT treatment and care • Awareness of patient initiatives (paediatric) on VRT at national level • Milestone 60 submitted (Month 12 – Oct 17): Report on issues raised by care and research for young people with extremely rare cancers

25. Task 9.3: Very Rare Tumours www.siope.eu ACTION PLAN YEAR 2* • Launch of survey on paediatric VRT to parents across Europe • 1nd face-to-face meeting: 17 Nov. 2017, Frankfurt, DE • Discussion of preliminary survey results • Identifying future orientations • Report on incidence of rare paediatrictumours in Europe data from 2000-2007, database of RARECAREnet, 83 cancer registries – synergies with WP4 • Drafting recommendations on optimal care and research for young people with extremely rare cancers *( focus: Oct. 2017 – April 2018)

26. JARCWP9: Childhood Cancers Task9.4:Make recommendations on models of healthcare for survivors of childhood cancers based on prior projects SIOPE Strategic Plan Objective 6: Improve quality of life of Childhood Cancer Survivors (CCS) www.siope.eu

27. Task 9.4: Survivorship • What should we request that policy makers do to effectively help achieve these objectives? www.siope.eu Two-thirds (2/3) of survivors have late-occurring complications due to their treatments. In one-fourth (1/4) complications are severe, and have a strong impact on the daily lives of survivors. Improving quality of life of survivors of childhood cancer is a major goal.

28. Task 9.4: Survivorship www.siope.eu Milestones and Deliverables: • M9.8: Consolidated conclusions from EU projects on CCS (Month 18 – April 2018) • M9.9: Consensus meeting on final recommendations (Month 30 – April 2019*) • D9.5: Report summarising guidelines and recommendations on models of care for CCS (M36  M30: April/May 2019) *or May 2019

29. Task 9.4:Survivorship www.siope.eu SCOPE • Survivorship Passport • Surveillance guidelines • Long-Term Follow Up delivery guidelines incl. transition to adult care STRATEGIC PARTNERS • Communityparticipating to prior EU projects on CCS METHODOLOGY • Define key policy objectives • Consolidate key messages incl. based on prior project conclusions • Consolidate recommendations and final consensus meeting

30. Task 9.4: Survivorship www.siope.eu PROGRESS YEAR 1 • 1st face-to-face meeting: 3 May 2017, Lund, SE • Partners’ roles and interest areas defined • Consolidatedinformation on prior EU projectsavailable

31. Task 9.4: Survivorship www.siope.eu ACTION PLAN YEAR 2 • First draftrecommendationsto bedeveloped: • Consensus building and exchange by email and TC • (2nd face-to-face meeting TBC) • Milestone9.8 – Month 18 (April 2018): Consolidated conclusions from EU projects on CCS * (focus Oct. 2017 – April 2018)

32. www.siope.eu ACTION PLAN YEAR 2 • First draftrecommendationsto bedeveloped: • Consensus building and exchange by email and TC • (2nd face-to-face meeting TBC) • Milestone9.8 – Month 18 (April 2018): Consolidated conclusions from EU projects on CCS * (focus Oct. 2017 – April 2018)

33. THANK YOU