1. Access to Personalised Medicine for PDAC patients STSM of the application of an EU-index for barriers Denis Horgan (EAPM) & Angela Brand (IPHG) on behalf of Marleen Jansen COST Action - Working Group 4 CSCDA Meeting, Liege, 24-26 November 2014

2. Department 2 Content STSM: PM for PCAD Barriers to PM –Development of an index –Levels of barriers to PM –Index of barriers PM for PCAD patients –Current state of PM in PCAD –Application of EU Index to PCAD Institute for Public Health Genomics

3. Department 3 STSM: PM for PCAD Working on PM in the EU –Based on and individual’s specific profile PreventionDiagnosis Treatment PM Diagnosis Treatment Patient BARRIERS Institute for Public Health Genomics

4. Department 4 Development of an Index Literature reviews Systematic literature review from scientific articles Gaps & Needs assessment and SWOT analysis Review strategic reports Literature reviews Systematic literature review from scientific articles Gaps & Needs assessment and SWOT analysis Review strategic reports Stakeholder analyses Survey research Gaps & Needs assessment and SWOT analysis Structured interviews Stakeholder analyses Survey research Gaps & Needs assessment and SWOT analysis Structured interviews Prioritizing major areas within access to PM Roundtable discussions Prioritizing major areas within access to PM Roundtable discussions Index for barriers to PM in the EU Institute for Public Health Genomics

5. Department 5 Levels of barriers to PM SectionSubsection ScienceSystem biology and data Research Clinical study design OperationalInformation delivery Education and training Inform, educate, empower patients Regulations EconomicsReimbursement Costs of testing EU-levelBio-banks Translation Reimbursement Legal and ethical Stakeholders involved on several levels: -Patients -Health care professionals -Insurance companies -Policy makers -Industry Institute for Public Health Genomics

6. Department 6 Literature reviews Systematic literature review from scientific articles Gaps & Needs assessment and SWOT analysis Review strategic reports Literature reviews Systematic literature review from scientific articles Gaps & Needs assessment and SWOT analysis Review strategic reports Stakeholder analyses Survey research Gaps & Needs assessment and SWOT analysis Structured interviews Stakeholder analyses Survey research Gaps & Needs assessment and SWOT analysis Structured interviews Prioritizing major areas within access to PM Roundtable discussions Prioritizing major areas within access to PM Roundtable discussions Index for barriers to PM in the EU Development of an Index Institute for Public Health Genomics

7. Department 7 Index of barriers Resulted in an index with eight subgroups including different barriers 1.EU-level policy making 2.Funding 3.Data and research 4.Health care system 5.Patient level 6.Stakeholder involvement 7.Standardization 8.Interoperable infrastructure Institute for Public Health Genomics

8. Department 8 Content STSM: PM for PCAD Barriers to PM –Development of an index –Levels of barriers to PM –Index of barriers PM for PCAD patients –Current state of PM in PCAD –Application of EU Index to PCAD Institute for Public Health Genomics

9. Department 9 Current state of PM in PCAD Literature review to analyze current state of PM (e.g. companion diagnostics) in PCAD. PubMed Web of Science Medline Scientific articles Literature review to analyze current state of PM (e.g. companion diagnostics) in PCAD. –Biomarkers identified –Novel biomarker options: gene profiles, pancreas tissue and juice –Complex disease nature –Detection interval  Translation into routine clinical practice experiences major hurdles Institute for Public Health Genomics

10. Department 10 Application of EU Index to PCAD Institute for Public Health Genomics BarrierRecommendations Involved stakeholders Stakeholder involvement Mismatch in needs and provided information between clinical practice and research Facilitate systematic stakeholder involvement in policy making, research, and implementation by: - Early dialogue and agenda setting - EU-platforms From all fields Standardization Lack of comparable data and information in databases Standardization of methods across EU for: - Evaluation research grants (translational research) - Data collection, interpretation, quality assurance - Information on reimbursement decisions EU-catalogue of (promising) biomarkers From all fields Interoperable infrastructure Lack of ICT-support tools for data- and information- sharing Develop ICT-tools for data- and information sharing Should include information on e.g.: - Biobanks, data-accessibility, guidelines From all fields

11. Department 11 Application of EU Index to PCAD BarrierRecommendationsInvolved stakeholders Policy-making Scattered policies and legislation across EU on research, reimbursement decisions etc. Update and/or develop regulations and legislation on EU-level: - Focus on guidelines to incorporate relevant outcomes for clinical practice Basic science e.g. from industry Translation e.g. HTA-experts Regulatory e.g. member states Healthcare systems e.g. representatives from associations Funding Increased funding, lack of implementation of biomarkers Adjustment of funding models: focus on translation - Best practice guidelines - Conditional reimbursement - Adaptive licensing Basic science e.g. from industry Translation e.g. HTA-experts Regulatory e.g. government bodies Institute for Public Health Genomics

12. Department 12 Application of EU Index to PCAD BarrierRecommendationsInvolved stakeholders Data and research Lack of relevant data: Complexity PCAD (biological processes and interactions) Point-of-care research (CER) to provide relevant biomarkers: - Agreements on type of data, annotation and quality - Adjusted informed consent (EMR) Updated disease models Basic science e.g. from industry Healthcare systems e.g. representatives from associations Patient groups e.g. representatives from associations Lack of information to interpret health data for clinical practice Integrated informational models (communication between clinic and laboratory) - Harmonization of relevant data Turnaround time test Centres of excellence Basic science e.g. from industry Healthcare systems e.g. representatives from associations Lack of information relevant for HTA- evaluation  reimbursement and translation Use HTA-framework in CER: -Best practice guidelines Collaboration between pharma and diagnostic companies Basic science e.g. from industry Translation e.g. HTA-experts Regulatory e.g. government bodies Healthcare systems e.g. representatives from associations Institute for Public Health Genomics

13. Department 13 Application of EU Index to PCAD BarrierRecommendationsInvolved stakeholders Healthcare system Lack of awareness and knowledge of PM for PCAD Training and education - Workshops on e.g. ICT tools, and PM for PCAD - Automated support tools - Adjust curricula healthcare professionals Basic science e.g. from industry Regulatory e.g. government bodies Healthcare systems e.g. representatives from associations Lack of support in clinical decision making Lack of uptake of PM PM to patient Lack of awareness and knowledge of PM for PCAD Develop implementation and dissemination plan for information about PM to PCAD patients and public, e.g.: -Education programs -Communication by healthcare professionals Regulatory e.g. government bodies Healthcare systems e.g. representatives from associations Patient groups e.g. representatives from associations Institute for Public Health Genomics

14. Department 14 Conclusions Challenge to pinpoint what data is relevant for PCAD-care –Barriers exist in effective demonstration of clinical utility of biomarkers –High level of evidence on relevant HTA-aspects needed Barriers are increased due to regulatory issues and budgetary constraints for biomarker research PM-applications need supportive bioinformatics Institute for Public Health Genomics

15. Department 15 Summary Considerable amount of barriers in: – EU-guidelines – Research & funding: focus on translational studies of promising biomarkers – Education of health care professionals and patients Institute for Public Health Genomics

16. Department 16 Acknowledgements Special thanks to all the stakeholders for their input, and the teams from COST Action EUPancreas PerMed EAPM IPHG Institute for Public Health Genomics