Work Package 9 Childhood Cancers For a Brighter

Work Package 9: Childhood Cancers For a Brighter Future for Children & Adolescents with Cancer JARC General Assembly 16 th Sept. 2017 Milan, Italy Joint Action ‘ 724161/JARC’ has received funding from the European Union’s Health Programme (2014 -2020) www. siope. eu

WP 9: Childhood Cancers Ø Task 9. 1: Make recommendations on accessibility of essential treatments across Europe Ø Task 9. 2: Make recommendations on access to innovative therapies through referrals to clinical trials across Europe Ø Task 9. 3: Identify solutions for optimal care and research for young people with extremely rare cancers Ø Task 9. 4: Make recommendations on models of healthcare for survivors of childhood cancers based on prior projects. www. siope. eu 2

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

JARC WP 9: Childhood Cancers Task 9. 1: To make recommendations to solve barriers in accessibility of standard treatments across Europe For a Brighter Future for Children & Adolescents with Cancer www. siope. eu SIOPE Strategic Plan Objective 4: Equal Access

Task 9. 1: Essential treatments Eighty percent (80 %) of children and adolescents with cancer can be disease-free at 5 years provided that they receive current multidisciplinary treatments Ø Are the essential medicines to cure, support and palliate children and adolescents with cancer available and accessible across Europe? Ø What improvements can we request to decision makers based on the current situation and needs? www. siope. eu 5

Task 9. 1: Essential treatments Milestones and Deliverables: § M 9. 1: Survey completed & available (Month 21 – July 2018) § M 9. 2: Mapping of issues to solve done (Month 22 – Aug 2018) § M 9. 3: Consensus meeting on recommendations (Month 30 – April 2019*) Ø D 9. 1: Report summarising survey results/issues and recommendations (M 36 M 30: April/May 2019) *or May 2019 www. siope. eu 6

Task 9. 1: Essential treatments SCOPE: o Medicines: Standard anticancer agents and palliative and pain control medicines incl. for administration during procedures o Radiotherapy (RT): Equipment and personnel qualified to work with children STRATEGIC PARTNERS: o Medicines: CCI Europe, ESOP and ESMO o Radiotherapy (RT): CCI Europe, QUARTET (SIOPE committee with EORTC), dialogue ongoing with ESTRO and researchers in the field www. siope. eu 7

Task 9. 1: Essential treatments METHODOLOGY (Medicines): § Core Working Group to 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 www. siope. eu 8

Task 9. 1: Essential treatments PROGRESS YEAR 1* - MEDICINES § Core Working Group of experts constituted § SIOPE – CCI Europe – ESOP – ESMO § 1 st 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) www. siope. eu 9

Task 9. 1: Essential treatments 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 www. siope. eu 10

Task 9. 1: Essential treatments PROGRESS YEAR 1 - RT § Cooperation initiated with key stakeholders/researchers: § 1 st face-to-face exchange: 13 Sept. 2017, Brussels, BE § Scope of Task refined: § 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. www. siope. eu 11

Task 9. 1: Essential treatments 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 www. siope. eu *(focus: Oct. 2017 – April 2018) 12

JARC WP 9: Childhood Cancers Task 9. 2: To make recommendations to increase and facilitate timely access to innovative therapies within clinical trials For a Brighter Future for Children & Adolescents with Cancer www. siope. eu SIOPE Strategic Plan Objective 1: Innovative treatments

Task 9. 2: Innovative therapies 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%)* Ø 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 14

Task 9. 2: Innovative therapies Milestones and Deliverables: § M 9. 4: Survey on referral patterns completed & available (Month 21 – July 2018) § M 9. 5/D 9. 3: List of preclinical models and research teams available (Month 24 – Oct. 2018) § M 9. 6: Consensus meeting on recommendations (Month 28 – Feb 2019) Ø D 9. 2: Report summarising survey results/issues and recommendations (M 36 M 30: April/May 2019) www. siope. eu 15

Task 9. 2: Innovative therapies SCOPE § Innovative therapies in early clinical trials for patients in relapse/failure STRATEGIC PARTNERS § CCI Europe, ITCC METHODOLOGY § First set of recommendations based on experience in ITCC countries on facilitating clinical trial referrals § Recommendations developed and supplemented with input from other countries in Europe § Recommendations consolidated and final consensus meeting www. siope. eu

Task 9. 2: Innovative therapies PROGRESS YEAR 1 § 1 st 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 WP 9 partners with interest in Task § 1 st Draft Recommendations agreed: Ø Make information on early phase clinical trials across Europe publicly available, easily accessible, and understandable with particular consideration for the needs of parents; Ø Set up Virtual Tumour Boards to discuss all relapsed patients and define referral paths; Ø Strengthen cooperation between centres to ensure continuity of care when patients are referred to participate in an early trial; Ø Continue exploring the S 2 certificate policy in relation to early clinical trial participation in view of putting forward concrete proposals; Ø Explore potential solutions to facilitate travel and accommodation of patients and their family. * AT, BE, DK, FI, FR, DE, IL, IT, NL, ES, SE, CH, UK (12 EU countries + Switzerland Israel) www. siope. eu 17

Task 9. 2: Innovative therapies ACTION PLAN YEAR 2* § 2 nd face-to-face meeting: early 2018 § Scope: non-ITCC countries in Europe § Including preparatory and follow up liaison by email/TC § Consolidating 2 nd Draft Recommendations with inputs from non-ITCC countries § Milestone 9. 4 (Month 21 – July 2018): Survey on referral patterns completed & available www. siope. eu *( focus: Oct. 2017 – April 2018) 18

Task 9. 2: Innovative therapies 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 www. siope. eu 19

JARC WP 9: Childhood Cancers Task 9. 3: Identify solutions for optimal care and research for young people with extremely rare cancers For a Brighter Future for Children & Adolescents with Cancer www. siope. eu SIOPE Strategic Plan Objective 4: Equal Access

Task 9. 3: Very Rare Tumours Extremely rare cancers (<2/1, 000) are i) extremely rare pathologies in children, such as pleuropulmonary blastoma ii) 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. Ø What can we formulate for decision makers to ensure that these specific needs are addressed? www. siope. eu 21

Task 9. 3: Very Rare Tumours Milestones and Deliverables: § M 9. 7: Report on issues raised by care and research for young people with extremely rare cancers (Month 12 – Oct 2017) Ø D 9. 2: Report summarising proposals to address VRT in paediatric patients (M 36 M 30: April/May 2019) www. siope. eu 22

Task 9. 3: Very Rare Tumours SCOPE § VRT Guidelines – VRT Expert advice / Centres of excellence – VRT Research STRATEGIC PARTNERS § CCI Europe, EXPe. RT - European Study Group for Paediatric Rare Tumours METHODOLOGY § Development of report on specific issues faced by children and adolescents with VRT § Development of draft recommendations incl. based on prior projects (Ex. PO-r-Net) § Recommendations consolidated and final consensus meeting TBC www. siope. eu

Task 9. 3: Very Rare Tumours PROGRESS YEAR 1 § Cooperation established with key partners (e. g. EXPe. RT) § 1 st face-to-face exchange: 29 -30 March 2017, Padova, IT § § 2 nd face-to-face exchange: 29 -30 June 2017, Brussels BE § § Methodology defined as receiving information from database of RARECAREnet (83 European data bases, 2000 -2007) – Synergies with WP 4 Preparation of online questionnaire for parents with CCI: § § Task presented at Ex. PO-r-Net final conference and ERN Paed. Can Launch event Analysis of incidence of rare paediatric tumours in Europe § § Streamlining methodology and synergies with Ex. PO-r-Net /JARC WP 4 - Epidemiology 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 www. siope. eu 24

Task 9. 3: Very Rare Tumours ACTION PLAN YEAR 2* § Launch of survey on paediatric VRT to parents across Europe § 1 nd face-to-face meeting: 17 Nov. 2017, Frankfurt, DE § Discussion of preliminary survey results § Identifying future orientations § Report on incidence of rare paediatric tumours in Europe data from 2000 -2007, database of RARECAREnet, 83 cancer registries – synergies with WP 4 § Drafting recommendations on optimal care and research for young people with extremely rare cancers www. siope. eu *( focus: Oct. 2017 – April 2018) 25

JARC WP 9: Childhood Cancers Task 9. 4: Make recommendations on models of healthcare for survivors of childhood cancers based on prior projects For a Brighter Future for Children & Adolescents with Cancer www. siope. eu SIOPE Strategic Plan Objective 6: Improve quality of life of Childhood Cancer Survivors (CCS)

Task 9. 4: Survivorship 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. Ø What should we request that policy makers do to effectively help achieve these objectives? www. siope. eu 27

Task 9. 4: Survivorship Milestones and Deliverables: § M 9. 8: Consolidated conclusions from EU projects on CCS (Month 18 – April 2018) § M 9. 9: Consensus meeting on final recommendations (Month 30 – April 2019*) Ø D 9. 5: Report summarising guidelines and recommendations on models of care for CCS (M 36 M 30: April/May 2019) *or May 2019 www. siope. eu 28

Task 9. 4: Survivorship SCOPE § Survivorship Passport § Surveillance guidelines § Long-Term Follow Up delivery guidelines incl. transition to adult care STRATEGIC PARTNERS § Community participating 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 www. siope. eu

Task 9. 4: Survivorship PROGRESS YEAR 1 § 1 st face-to-face meeting: 3 May 2017, Lund, SE § Partners’ roles and interest areas defined § Consolidated information on prior EU projects available www. siope. eu 30

Task 9. 4: Survivorship ACTION PLAN YEAR 2 § First draft recommendations to be developed: § Consensus building and exchange by email and TC § (2 nd face-to-face meeting TBC) § Milestone 9. 8 – Month 18 (April 2018): Consolidated conclusions from EU projects on CCS www. siope. eu * (focus Oct. 2017 – April 2018) 31

ACTION PLAN YEAR 2 § First draft recommendations to be developed: § Consensus building and exchange by email and TC § (2 nd face-to-face meeting TBC) § Milestone 9. 8 – Month 18 (April 2018): Consolidated conclusions from EU projects on CCS www. siope. eu * (focus Oct. 2017 – April 2018) 32

For a Brighter Future for Children & Adolescents with Cancer www. siope. eu THANK YOU