1. WP 7: Innovation and Access to Innovation
Marc Van den Bulcke
Cancer Centre
Sciensano - Belgium
JARC, Milano, Italy 26 October 2018

2. WP 7: Innovation and Access to Innovation Goals
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 Status of activities
JARC WP7 Deliverables
Start:
9/30/2016
Del 7.1
Report on standardization of clinical data and patient-centered clinical outcomes
UoA
Report
Public OK
Del 7.2
Recommendations on requirements for long-term surveillance of rare cancer patients (for each family of rare cancers)
SLR/UCL/
Sciensano
Del 7.3
Proposals to improve collaborative international clinical trials in the EU, particularly inter-group clinical trials
INT/Sciensano
3/30/2019
Del 7.4
Roadmap on precision medicine in rare cancer care within ERNs

4. WP 7: Innovation and Access to Innovation Key Messages Del 7.1
Task 7.1 RC Translational Research landscape
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
Recommendations:
Standardization of clinical data and patient follow-up data collection, protocols for biological samples collection as well as efficient communication and possible sample exchange between the centers joining the JARC program is essential for the maximization of results production and the lowering of the costs. The aim of D7.1 deliverable is to specify methods for the collection and storage of participant samples that give maximum scientific return.
We propose the collection of those information:
Clinical Data: • Demographics and clinical status, including the results of examinations for specific markers (if existed) for each type of cancer, disease progression and symptoms, patient outcome, treatment and complications

5. WP 7: Innovation and Access to Innovation
Key Messages Del 7.1
data will be updated on a regular basis (for example every 6 months or 1 year, depending on the health condition of the patient)
a unique code to all biological samples taken from same patient and “assembled” following specific instructions
Biobank-Samples Collection :
- Centralized of federated databases
Ethical issues addressed in the same way
Communication between centers
- adopt effective global strategies for sharing cancer-related data.

6. WP 7: Innovation and Access to Innovation Key Messages 7.2
Task 7.2. “Big data”
requirements for long-term surveillance of rare cancer patients; regulatory and legal issues, the harmonization of these data (semantics)
clinical epidemiology framework program for outcome research in rare cancers (theoretical and methodological issues)
Recommendations:
Definition of ‘big data’ in this context
Status of the activities run by:
National Cancer Register (Be)
University College of London (UK)

7. Contributing to WP7 – Feasibility approach
Pilot project – HPV oropharynx carcinoma in population-based cancer registries
Evaluate data availibity in cancer registries
8 national or regional registries contacted by (JARC countries)
Response of 5  telephone survey
Not every registry has detailed information about treatment modalities
HPV/p16 not yet available in a structured format
Manual effort needed to retrieve the data from medical files, pathological reports, …
UCL partnering for UK (difficulties in obtaining permissions)

8. Future steps
Participation of some cancer registries to share the available data
Describe weaknesses and strengths
Make recommendations for the cancer registries to implement HPV/p16 status in a structured way

9. WP 7: Innovation and Access to Innovation Key Messages 7.2
Comments by UCL (UK) on progress in the evaluation of the application of Toronto Staging Guidelines by population based CR
(in collaboration with WP4) :
12 CR provided staging data for neuroblastoma and Wilms tumor
lots of variability in data content and effort needed to extract data
« Good progress is being made »

10. WP 7: Innovation and Access to Innovation Key Messages 7.2
Comments by UCL (UK) on progress in the evaluation of the application of Toronto Staging Guidelines by population based CR
(in collaboration with WP4):
‘From Public Health England’s perspective, we have been able to establish through our participation to date that the level of staging completeness held by PHE’s current records for the calendar year 2014 for Wilms tumour and neuroblastoma was only about 30-50%.  Whilst a much higher level of staging completeness could be obtained by linking to clinical trial data (that I hold) for WT and to other clinical data held at treatment centres (for neuroblastoma), the processes and timescales to do this linkage and obtain the approval through PHE”s Office for Data Release would require resourced manpower time and a timescale that went beyond the data collection period assigned to the task.’

11. WP 7: Innovation and Access to Innovation Key Messages 7.2
Comments by UCL (UK) on progress in the evaluation of the application of Toronto Staging Guidelines by population based CR
(in collaboration with WP4):
Next steps:
write up a report on the principles and resources required for the PHE (and indeed other UK population based registries) to participate in a future joint
hold a workshop in early 2019 with all interested registries (who covers the costs of inviting the registries, including those who expressed interest)

12. WP 7: Innovation and Access to Innovation Key Messages 7.2
« Big data» Belgian ‘Precision’ Project data collection:
Demo website :
You can find PRECISION project in the left panel.

13. WP 7: Innovation and Access to Innovation Key Messages 7.3
Task 7.3. Collaborative clinical trials
Recommendations:
The solutions in the new Clinical Trials Regulation (CTR)
Will support harmonisation across countries because a EU regulation comes into force automatically in all EU countries and overtakes national rules
The provision in the CTR of a risk-based approach should greatly benefit investigator-driven trials. Several academic studies in rare cancers may be expected to be low-risk studies
- National health care systems could actively promote and support research for rare cancers, at the very least with a view to trials which could decrease health costs (and
which companies may not be interested to sponsor). In any case, possible extra-costs for health systems implied by such trials should be limited in trials implemented within ERNs

14. WP 7: Innovation and Access to Innovation Key Messages 7.3
Task 7.3. Collaborative clinical trials
Recommendations:
The establishment of an ERN-specific research infrastructure
1) A dedicated CRO for ERNs or a consortium of CROs sharing open infrastructures could serve collaborative trials set up within ERNs. Minimum requirements of any research infrastructure should:
- serve all the HCPs of a specific ERN;
- be interoperable with the electronic health records of the HCPs and available bio-banks;
- be flexible to accommodate the requirements pertaining to different rare cancers and possible study objectives;
- be compliant with privacy requirements, as foreseen by the CTR and the EU General Data Protection Regulation;
- be interoperable with administrative data bases in the EU countries and cancer registries
2) An entity serving as the trial sponsor for this kind of studies would be needed. Possible solutions in principle may be:
1. establishing a consortium of all Health Care Providers (HCPs) of a ERN operating as a legal entity;
2. exploiting a scientific/professional society or any multi-stakeholder initiative being a legal entity.
Funding
It would be highly recommended that the EU research funding infrastructures could launch dedicated calls for clinical trials on rare cancers.

15. Towards a European health research and innovation cloud (HRIC)
DG RTD initiative (9th framework)
Key message:
Health research data in Europe are collected by a multitude of organisations and stored across different ICT systems and physical locations. The EU initiative on the Digital Transformation of Health and Care (i.e., 'Digicare'), which was announced in the mid-term review of the Digital Single Market Strategy, aims at providing conditions for the building of a secure, flexible and decentralized digital health infrastructure to enable data sharing and analysis for health research across the EU in compliance with data protection legislation and preserving the full trust of patients and research participants. We envision that this digital health infrastructure could be implemented through a European Health Research and Innovation Cloud (HRIC).

16. Roadmap : Precision medicine in rare cancer care
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. Belgian Cancer Centre, Sciensano
The IPAAC « Roadmap on Implementation and Sustainability of Cancer Control Actions”
Belgian Cancer Centre, Sciensano
Marc van den Bulcke
Régine Kiasuwa Mbengi
Laure Bakker

18. WP4: Main objectives MAIN OBJECTIVE
To ensure the (timely) development of the (high quality) key deliverable: « the
Roadmap on Implementation andSustainability of Cancer Control Actions”
(support MS in implementing iPAAC and CANCON recommendations)
MAIN CONTENT
The Roadmap will mainly consist in the combination of 2 types of outputs:
1. The experience of EU MS in implementing CanCon recommendations
2. Results from iPAAC WPs 5 – 10 (recommendations and/or advices for implementation)
TARGET GROUPS:
The primary target group of the iPAAC Joint Action will be EU-level policymakers and decision makers at national, regional and local level
The secondary target group will consist of professional and scientific organisations
iPAAC WP4 Background, July 2018

19. Sources for the Roadmap
10 topics and 2 types of outputs:
1. experience of EU MS in implementing CanCon recommendations
Cancer screening
Integrated cancer care
Community-level cancer care
Survivorship and rehabilitation
2. Results from iPAAC WPs 5 – 10
- Cancer prevention -> CanCon policy paper
- Genomics in cancer control and care -> CanCon policy paper
- Information system -> adressed throughout the whole guide (+JA IS)
- (economic) Challenges in cancer care -> CanCon policy paper
- Innovative therapies (immunotherapies) -> very briefly in chapter 5 (CCCNs)
- Integrated and comprehensive cancer care -> EU ERNs
iPAAC WP4 Background, July 2018

20. INTERACTIVE PROCES GB RCC WP SG IPAAC Partners IPAAC WP leaders
Governmental Board SG
IPAAC WP leaders
RCC
IPAAC
Partners WP
Stakeholder forum
Stakeholders
iPAAC WP4 Background, July 2018

21. IPAAC Work Progres WP10 WP9 WP8 WP7 WP6 WP5 2018 2019 2020 2021 Yearly
ROADMAP
RCC GB
WP4
RCC GB
WP4
Stakeholder Forum
WP3
WP2
WP1
6 monthly
iPAAC WP4 Background, July 2018

22. Roadmap: Methodology Methods: visits into EU countries
Main objectives:
to develop a framework (format) for the Roadmap
to map cancer control initiatives in EU Member States (related to 4/5 domains)
to translate WP5-10 results into implementation advices/guidance
Methods:
visits into EU countries
the Governmental Board (GB)
the Roadmap Coordination Committee (RCC)
2 external experts (Mark Dobrow & Ri De Ridder)
-> the Roadmap will result from the merge of different exercises: MS visits, comparative literature studies (e.g. EC health at glance, HITs,…), surveys and pilots
iPAAC WP4 Background, July 2018

23. Govermental board input
Final deliverable = IT TOOL (comment: new development, need for concrete decision on the process of making such tool) who devolps what & how by when for whom (=why) and where will it be hosted
Some questions: Who will lead this development (content, technical)? Who will partner?
What will be the process?
What is realistic to reach by end of JA?
What will be the needs? Is there budget?
Characteritics of the IT Tool: Practical, documentation (eg guidelines, good practices) and tools, contatcperson inventory, IP issues, benchmarking opportunities (pilots, case studies,…), different entries of access to the data, tiered organisation (in layers for different users), dynamic, governance body (what goes in, what not (Wikipedia entry & control philosophy, gateway clearance, …), ….
iPAAC WP4 Background, July 2018

24. Govermental board input
Sustainability of the final deliverable, the IT tool:
General agreement on the need for maintaining the outputs up-to-date after the JA
Question: do we develop the tool uberhaupt if no committment’ for maintaining the IT too
Preferred solution: DG - Joint Research Centre of EC
- Question: need for a transparant governance body (consortium agreement?)
Issues on GDPR if person names are made available
iPAAC WP4 Background, July 2018

25. Input from RCC Topics identification: Link to Cancon recommendations
Input from MS visits
Input from iPAAC WPs
Include inequalities
Topics selection process:
Inputs to WP4 (different sources, timing) – summary of topics by WP4 – selection agreement at GB/RCC – topic development – topic content presentation at GB/RCC
Topics development:
Examples (pos or neg)
Cost calculation/estimation
Comparative decisions by MS on topics (eg EU level)
Identify contact person/expert per topics
Content management (for: IPAAC topics: WP leader; for Cancon topics: former WP leaders) - during time of JA
Take some examples to demonstrate the feasability of IT tool approach
iPAAC WP4 Background, July 2018

26. Roadmap : the (preliminary) booklet guidance structure
Style: MANUAL to the Roadmap?
iPAAC WP4 Background, July 2018

27. Roadmap : the virtual basics
HPV screening
a) What, where, when, who, why and how
b) Pilots
c) Refs
Tangible example
MammoMSIEUEU CodeeHealthPatientDis
HPV
NGS
Fairness
ERN
Patient
GDPR
Training
Work
CReg
BCS
BMI AI
Nutrition
Mammo
TMB
CCCN
MSI
eHealth
Drones
EU Code
Pancreas
Disinvestment
Screening
Citizen
Education
Inequalities
Economics
HTA
Who is the policymaker?
Target population:
Belgium ‘Cervical cancer screening’
Aim: ‘Cytology to HPV test’
mixed responsability Federal and regional level
- Interkabinet decision (ministers)
Interkabinet working group (governmental administrations)
Technical working group (coordinator)
Experts and stakeholders
ROADBOOK/actionplan
WHAT? & WHY
WHO?
HOW?
Where, when….
iPAAC WP4 Background, July 2018

28. Roadmap : the network expert map
MammoMSIEUEU CodeeHealthPatientDis
HPV
NGS
Fairness
ERN
Patient
GDPR
Training
Work
CReg
BCS
BMI AI
Nutrition
Mammo
TMB
CCCN
MSI
eHealth
Drones
EU Code
Pancreas
Disinvestment
Screening
Citizen
Education
Inequalities
Economics
HTA
iPAAC WP4 Background, July 2018

29. WP4 Visits to member states
27 countries
Visits of one day
Period: July 2018 to March/April 2019
Aim: insight in actions and intentions of MS on cancer control and care within the scope of IPAAC topics
Means: ‘interview survey’
iPAAC WP4 Background, July 2018

30. WP4 Visits to member states
Process:
Invitation sent by WP4 to local iPAAC partner organisations whom send it to the MS responsibles (ASAP)
Note: explain overall framework and purpose of the visits
IPAAC Partner organises meeting with local policymaker(s) (Sept 2018 – March 2019)
Visit and issue discussion with WP4 representatives (topics, gaps, needs, …) (RK, LB and MVDB)
Report / MS by WP4 – sent for validation to each MS (via IPAAC partner)
Summary Report (June/luly 2019)
Presentation and discussion of report at 3th GB (about M18)
Integration of conclusions into the roadmap (e.g. Heatmap presentation)
iPAAC WP4 Background, July 2018

31. THANK YOU
iPAAC WP4 Background, July 2018