1. Josep M. Borras Milano, October 25, 2018
WP10 RARE CANCER POLICY
Josep M. Borras
Milano, October 25, 2018

2. WP10 OBJECTIVES & TASKS
(1) Research and analysis of rare cancer policies, strategies and planning at national level
Task 1.1. Review of NCCPs and rare cancer plans
Task 1.2. Review of rare diseases plans, past and present EU funded initiatives on rare diseases and policy options and priorities for patients’ organisations
(2) Organization, governance and harmonization of rare cancer care and clinical research
Task 2.1. Analysis and case-studies of National reference centers for rare cancers in the context of their inclusion within the ERNs.
Task 2.2. Analysis of the designation of orphan drug medical products and in marketing of medicinal products for rare cancers
(3) Discussion and consensus on the recommendations about key criteria for rare cancer policy and organization at EU and national levels
Task 3.1. Preparation of the workshop meeting between the stakeholders, in cooperation with other WPs, in order to review conclusions and recommendations.
Task 3.2. Report and the consensus recommendations.

3. WP10, global objective 1. Research and analysis of rare cancer policies, strategies and planning at national level
Task 1.1. Review of NCCPs and rare cancer plans across the EU Member States (lead by ICO)
Task 1.2. Review of rare diseases plans, past and present EU funded initiatives on rare diseases and policy options and priorities for patients’ organisations across the EU Member States (lead by EURORDIS)

4. Deadline: M22, July, 2018
Current status: Finalized and ready for circulation and formulation of recommendations
D10.1: Recommendations on policy measures on rare cancers for national plans and strategies on cancer and rare diseases

5. What is the level of information contained in NCCPs on rare and paediatric cancers?
Criteria for appraisal
(1) No reference to rare or paediatric cancers is made
(2) Rare or paediatric cancers should be prioritised but no information or specific planning criteria are given
(3) Some information and planning criteria or recommendations are given.
(4) Specific information and planning criteria or recommendations measures are given
(5) Comprehensive approach

6. RARE CANCERS
PAEDIATRIC CANCERS

7. 25 points for discussion were identified
Process of analysis and development of Rare Cancer Policy recommendations
TASK 1, WP10
Comparative analysis of priorities and recommendations on rare and paediatric cancers in National Cancer Control Programmes (NCCPs)
25 points for discussion were identified
Preliminary review from SIOPE
TASK 2, WP10
Review of rare diseases plans, past and present EU funded initiatives on rare diseases and policy options and priorities for patients’ organisations relevant for rare cancer policy
SINCE NOVEMBER, 2018:
Basis for discussion among JARC stakeholders and trigger a decision-making process for formulation of Recommendations

8. Task 2.1. Analysis and case-studies of National reference centres for rare cancers in the context of their inclusion within the ERNs.
MAIN GOAL:
Providing an in-depth understanding of national experiences and their connection with the developing ERNs based on the perspective of physicians, patients and clinical managers. They may gather relevant perspectives on how different European countries conceive the ERNs.
Specific aims
To describe the processes through which national reference hospitals are embedded, collaborate and are evaluated at National health system level.
To understand the interaction between National and European guidelines and how the knowledge transfer is performed.
To identify the added value of ERNs and the potential opportunities they bring for improving rare cancer care at a national level.

9. Istituto Nazionali di Tumori (Milano)
Multiple case study: fieldwork
(National Reference centres in Sarcoma, involved in EURACAN or not) (February-July, 2018)
FINLAND
Helsinki University Hospital (Helsinki), Turku University Hospital Hospital (Turku)
52 semi-structured interviews were conducted
TXEC REPUBLIC
Masaryk Memorial Cancer Institute (Brno), University Hospital Motol (Prague)
LITHUANIA
Hospital of Lithuanian University of Health Sciences (Kaunas), University Hospital Santaros Klinikos (Vilnius)
FRANCE
Hôpital Ambroise Paré & Institut de Cancérologie Gustave Roussy (Paris), Institut Bergonié (Bordeaux)
ITALY
Istituto Nazionali di Tumori (Milano)
SPAIN
University Hospital San Carlos (Madrid), University Hospital Sant Pau & Catalan Institute of Oncology (Barcelona)
…including Patient Organisations’ representatives, Health Authorities and European Policymakers

10. Current status: Data Analysis
Task 2.1. Analysis and case-studies of nationally based hospitals for rare cancers in the context of their inclusion within the ERNs.
Deadline: M28, January, 2019
Current status: Data Analysis
D10.2: Report on policy and organisational issues affecting the development of ERNs
Special thanks to the National gatekeepers!
Liisa Pylkkanen (Finland), Petr Čermák & Kateřina Kopečková (Txec Republic), Jeanne-Marie Brechot (France), Rasa Jančiauskienė (Lithuania), Annalisa Trama (Italy)

11. Topics for discussion in the meeting:
Task 2.2. Analysis of the designation of orphan drug medical products and in marketing of medicinal products for rare cancers
April 2018: Meeting at EMA headquarters with EMA experts, patient organizations, experts on rare cancers, scientific societies, JARC.
Topics for discussion in the meeting:
Broad advice
Label extension
Potential role of ERN
Two rare cancers as case studies for discussion
Chordoma
Salivary glands tumour

12. Current status: Writing the report
Task 2.2. Analysis of the designation of orphan drug medical products and in marketing of medicinal products for rare cancers
Deadline: M28, January, 2019
Current status: Writing the report
D10.2: Report on the licensing of drugs for rare cancers and the potential role of ERN in the regulatory scenario of approval of rare cancer drugs.

13. Meeting Early June 2019. Barcelona? Final report WP10: September 2019
(3) Discussion and consensus on the recommendations about key criteria for rare cancer policy and organization at EU and national levels Task 3.1. Preparation of the workshop meeting between the stakeholders, in cooperation with other WPs, in order to review conclusions and recommendations. Task 3.2. Report and the consensus recommendations.
Workshop to review conclusions and recommendations: input from all stakeholders required after having received reports from the rest of WPs (March-April 2019)
Meeting Early June Barcelona?
Final report WP10: September 2019

14. Final comments: Major points emerging from analysis of cancer plans
Cancer plans should have specific activities and targets for rare cancers.
Different approaches for paediatric and adult cancers
Model of care: MDT
Organization of health system for rare cancers: centralization of care
Updated approach to therapy
Personalized treatment with experienced professionals
Access to research therapies and trials
Evaluation of outcomes feasible
Continuity of care should be protected between levels of care: professionals designated to coordinate care
Networks as the preferred way to coordinate hospitals and professionals and to avoid or limit barriers to access
Reimbursement should be adapted to these changes in health care organization

15. Summary: major points emerging from analysis of cancer plans
Pathology diagnosis: cornerstone of high quality cancer care. Pathology and molecular tests available with expert pathologists and double reading
Access to second opinion
ERNs should be the most important source of data for patients about therapeutic strategies
Patient associations should play a key role in identifying patient needs and proposing ways to cope with them
ERNs: potential for quality data able to assess therapeutic strategies
ERNs: Different perspectives and expectatives on ERNs according to the country health care system organization and previous experience in centralization of care for rare cancers