WP 7: Innovation and Access to Innovation Marc Van den Bulcke Cancer Centre Scientific Institute of Publlic health Brussels – Belgium JARC, Milano, 16 November 2017 Rue Juliette Wytsmanstraat 14 | 1050 Brussels | Belgium T +32 2 642 57 20 | F +32 2 642 50 01 | email: belgianCancerCentre@wiv-isp.be | www.e-cancer.be

WP 7: Innovation and Access to Innovation The goal of WP7 is to establish optimal ways to bring the best treatment and care to patients by advancing translational and clinical research on rare cancers trough ERNs 1. to stimulate translational research on RCs by exploiting networking, namely through the new ERNs 2. to promote exploitation of “big data”, namely those generated through the new ERNs, as a mean to advance our knowledge on rare cancers to provide recommendations on how to optimize long-term surveillance of rare cancer patients in an effort to address the many survivorship’s issues 3. to make proposals on how to exploit available regulations across the EU, and/or how to improve them, on collaborative prospective interventional clinical research, especially academic clinical research joining national collaborative groups (inter-group research, etc.)

WP 7: Innovation and Access to Innovation Deliverables linked to this work package D7.1 Report on standardization of clinical data and patient-centered clinical outcomes for big data optimal exploitation and connection to virtual biobanks and quality assurance / quality control (QA/QC) in regard to biological samples, data collection and molecular screening (with special reference to SPECTARare) (M24) D7.2 Recommendations on requirements for long-term surveillance of rare cancer patients (for each family of rare cancers) (M30) D7.3 Proposals to improve collaborative international clinical trials in the EU, particularly inter-group clinical trials (M18) D7.4 Roadmap on precision medicine in rare cancer care within ERNs (M30)

WP 7: Innovation and Access to Innovation Partners Instituion Referent Univerity of Athens (Greece) Maria Gazouli Istituto Nazionale Tumori (Italy) Paolo Casali, Lisa Licitra, Annalisa Trama Ministry of Health of the Czech Republic   Petr Čermák Vilniaus universiteto ligoninės santariškių klinikos (Lithuania) Ramunas Janavicius University of Szeged, Albert Szent-Györgyi Health-Center, Department of Oncotherapy (Hungary) Fábián Gabriella University of Thessaloniki - pneumonological clinic (Greece) Efimia Boutsikou Cancer Society of Finland Sakari Karjalainen Heikki Joensuu EURORDIS Ariane Weinman Ministry of Health, Malta Domenic Agius ECPC Kalliopi Christoforidi UCL Institute of Child Health Kathy Pritchard-Jones Belgian Cancer Registry Elisabeth Vaneycken EORTC Stephane Lejeune Anticancerfund Lydie Meheus ESMO (CAREFOR) to be defined

Task 7.1 RC Translational Research landscape (M24) This Task will be aimed at: standardizing clinical data and patient-centred clinical outcomes defining a robust quality assurance / quality control (QA/QC) framework for SPECTARare with regard to biological samples, data collection and molecular screening in order to address the specific research needs of the 12 different families of rare cancers included in the present JA.   Lead partner: UoA. Applicants: IPH, EORTC, EURORDIS.

Milestones to be reached M7.1 Survey on data collection and handling in Rare Cancers in Europe (UoA) completed (Task 7.1, M12) Version & Date: v1.0 / 3rd September 2017 Deliverable type: Report Milestones to be reached M7.1 Survey on data collection and handling in Rare Cancers in Europe completed (Task 7.1, M12)  

Task 7.2. Big data (M30) Recommendations will be provided with regard to the requirements for long-term surveillance of rare cancer patients This task will address the many regulatory and legal issues posed by clinical big data. It will address the issues concerning the harmonization of these data, especially in terms of their semantics. The goal will be to explore the possibility to set up a clinical epidemiology framework program for outcome research in rare cancers including the use of patient-reported outcomes, including quality of life end-points. This Task will contribute to solve the theoretical and methodological issues posed by the use of these data to advance medical. Lead partner: University College of London-Institute of Child Health (UCL-ICH). Applicants: BCR (Belgian cancer Registry-BCR), EORTC, European School for Medical Oncology (ESMO), INT.

Big Data concepts Big Data: an introduction __________ Big data as a by-product of technology __________ Big data in medicine: a definition __________ Big data in medicine: a history   Sources of big data in medicine __________ Electronic patient records __________ The -omics __________ Radiomics __________ Administrative data bases __________ Disease registries __________ Patient reported outcomes

Big Data concepts How do we innovate state of the art in medicine? __________ The determinants of a clinical decision __________ The paradigm of the clinical trial __________ The paradigm of machine learning __________ This house believes that big clinical data can change the state of the art __________ This house believes that big clinical data cannot change the state of the art __________ This house believes that big clinical data can complement clinical trials   Interoperability as an issue? __________ The technological potential of interoperability today __________ Interoperability will remain an issue __________ Interoperability will be no issue by definition

Big Data concepts The regulatory constraints __________ The European legal framework __________ The ethical questions __________ Technology as a way out?   Perspectives in rare cancers __________ The problem of rare cancers __________ The conceptual framework of precision oncology __________ Clinical networking as an opportunity in rare cancers __________ Big data as an opportunity in rare cancers __________ How to shape clinical networking to make the most of data

Belgian Cancer Registry - Contribution to WP7 Task 7.2 – Big data JARC WP7 Belgian Cancer Registry - Contribution to WP7 Task 7.2 – Big data

HPV oropharynx carcinoma in population-based cancer registries (CR’s) Background CR’s increasing involvement in cancer care due to growing interest in population-based data on diagnosis and treatment CRs well positioned to bring these big data together Aim To explore the possibility to set up a clinical epidemiology framework for outcome research in rare cancers (HPV oropharynx carcinoma)

Work in progress Tender approved Questionnaire Data availability Medical records Pathology reports Clinical registries Administrative Health databases Electronic format? Linkage possibility (unique ID?)  

Under construction Definition of dataset Structured variables in electronic format Diagnostic workup Imaging Treatment modalities HPV status (p16) Invite Member States to pariticapte Collect, analyse and describe the available data  

Task 7.3. Collaborative clinical trials (M18) This Task will try to make an inventory of regulatory constraints for collaborative international clinical trials in the EU, particularly inter-group clinical trials. The Task may also provide proposals to improve national and European regulations and/or their interpretation. Recommendations will be made about the optimal ways to connect the new ERNs to collaborative clinical research, whether investigator-driven or industry-sponsored including quality criteria for reference centers and ERNs based on clinical research performances. Lead partner: INT - IPH. Applicants: UCL-ICH, ESMO, EORTC, EURORDIS, European Cancer Patients Coalition (ECPC).

Task 7.4. Precision medicine in rare cancer care (M30) The aim is to provide recommendations on integrating translational research innovations into rare cancer care, based on conclusions and recommendations developed in the Joint Action and benchmarking with worldwide initiatives in the field. The output will be a Roadmap on the integration of translational research into routine rare cancer care within the evolving scenario of ERNs. Lead partner: IPH Applicants: all WP partners, Anticancer Fund, European Federation of Pharmaceutical Industries and Associations-EuropaBio (EFPIA-EuropaBio)

Next steps JARC Milestones JARC Start: 43 Survey on data collection UoA 1-Oct-17 46 Report on status in European countries on eHealth 48 Report on inventory of regulatory constraints in the field of collaborative clinical trials WIV-ISP (IPH) 44 Draft QA/QC guidance 1-Apr-18 45 Draft SOPs on biological samples and data collection in rare cancers 1-Jul-18 49 Draft index of the recommendations on integrating translational research innovations 47 Report survey on data formats 1-Oct-18 JARC Start: 9/30/2016 WP7 D7.1 D8 Proposals to improve collaborative international clinical trials in the EU, particularly inter-group clinical trials WIV-ISP Report Public 3/31/2018 D7.2 D13 Report on QA/QC guidance for collection of biological samples and data on rare cancers in the EU 9/30/2018 D7.3 D30 Recommendations on requirements for long-term surveillance of rare cancer patients (for each family of rare cancers) 3/30/2019 D7.4 D31 Roadmap on precision medicine in rare cancer care within ERNs