1. 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 | F | |

2. 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.)

3. 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)

4. 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

5. 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.

6. 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)

7. 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.

8. 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

9. 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

10. 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

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

12. 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)

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

14. 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

15. 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).

16. 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)

17. 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