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Oct 25 CAPHC Breakfast Symposium - Sponsored by Alexion - CORD


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Alexion Sponsored Breakfast Symposium
Canada's Rare Disease Strategy: Why Now and What Next?

Published in: Healthcare
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Oct 25 CAPHC Breakfast Symposium - Sponsored by Alexion - CORD

  1. 1. 1 Canadian Organization for Rare Disorders October 2016 Canada’s Rare Disease Strategy: Why Now and What Next?
  2. 2. 2 Overview 1. Who is CORD? 2. What are Rare Diseases? 3. Why Does Canada Need a Strategy? 4. What Does the Strategy Call For? 5. Support Rising Across Canada 6. Implementing the Strategy:  Rare Alliance Canada  Acting now on regulatory framework & orphan drug plan 2
  3. 3. 3 1. Who is CORD?
  4. 4. About CORD  Canadian Organization for Rare Disorders (CORD) —Network of 102 Patient Groups  Mission: Improve lives of all those affected by rare diseases  Mandate: Advance rare disease policy; improve screening, diagnose and access to clinical trials and treatment; develop patient group capacity; support research; collaborate  Durhane Wong-Rieger, PhD, President and CEODurhane Wong-Rieger 4
  5. 5. 5 What Does CORD Do?  Support Rare Disease Patient Groups: organize, develop, connect to researchers, healthcare providers, policy makers, drug developers  Advance rare disease programs and policy, including screening, diagnosis, research, access to therapies  Promote public awareness and support for rare diseases  Produce and train/support patient organizations to produce submissions to HTA and regulatory reviews  Advocate with or on behalf of patient groups and individual patients for access diagnosis, treatment and services 5
  6. 6. 6 2. What Are Rare Diseases?
  7. 7. 7 Key Stats on Rare Diseases 2/3 of children with a rare disease will not reach their 5th birthday 30% 1 in 12 Canadians has a rare disease of Canadians with Rare Diseases are Children That’s MORE than 2.8 MILLION! 7
  8. 8. 8 # Canadians with Common vs. Rare Diseases 0 0.5 1 1.5 2 2.5 3 3.5 Milions Affected Cancer Diabetes Heart disease Rare Diseases1.4 M 2.8 M 2.4 M 1.8 M (millions) 8
  9. 9. 9 3.Why Does Canada Need a Strategy
  10. 10. 10 Now is the Time for a Rare Disease Strategy  Rare disease is a Major Public Health Issue  RD Patients rarely access effective therapies  Health systems waste resources, achieve limited benefits  Rare disease strategies work in other countries  Canadian strategies work in other areas: Mental health, cancer, diabetes, cardiovascular disease  Leverage & coordinate expertise and resources across disciplines and sectors and internationally 10
  11. 11. 11 Canada: Catching up With Rest of the World… 1980 2020 USA 1983 JAPAN 1993 AUSTRALIA 1997 EUROPEAN UNION 1999 TAIWAN 2000 SOUTH KOREA 2003 USA & EU Harmonization 2007 Canada? 11 Adoption of Orphan Drug Legislation
  12. 12. 12 4. What Does the Strategy Call For?
  13. 13. 13 Launch of Canada’s Rare Disease Strategy  Extensive consultations with rare disease stakeholders since 2012  Strategy Launched on Parliament Hill in May 2015  Now is the Time  Shared across Canada:  Political parties, policy makers  Healthcare providers & admin  Researchers, patients, public May 25, 2015 13
  14. 14. 14 5 Key Goals of Canada’s Rare Disease Strategy 1. Improving early detection and prevention 2. Providing timely, equitable and evidence-informed care 3. Enhancing community support 4. Providing sustainable access to promising therapies 5. Promoting innovative research 14
  15. 15. 15 3 Guiding Principles Fundamental Consensus Principles for Effective, Cost-Effective, and Sustainable RD Programs I. Equity of Access II. Patient-Centered III. Collaboration and Coordination 15
  16. 16. 16 GOAL 1 Improving Early Detection and Prevention  Newborn screening in all provinces  Next-generation diagnostic testing; state-of-the art international labs  Standards for pre-conception, pre- natal genetic screening and counseling  Consistent, comprehensive, up-to- date genetic testing guidelines and tests  Genetic testing linked to RD registries, expert centers, healthcare services CORD survey 2/5 have genetic condition but didn’t receive pre-natal counseling or screening On average: misdi agnoses before proper one 2 to 3 1/4 diagnosed within 3 months waited more than 3 years for diagnosis 1/3 1/5 waited more than for diagnosis 6 years 16
  17. 17. 17 GOAL 1 Improving Early Detection and Prevention  Priority actions: a) Adopt national state-of-the art newborn screening b) Implement early detection and preventive services across Canada 17
  18. 18. 18 CORD survey GOAL 2 Timely, Equitable and Evidence-Informed Care Priorities  RD training for GPs, pediatricians, other HCPs  Clinical practice guidelines  Disease registries  Comprehensive care & support  Centres of Excellence and virtual networks  Linkage to social care, education, disability and work supports did not understand the information they received 2/5 2/3 were not referred to a patient organization did not receive adequate information from their doctor did not receive resources or contacts to seek additional information 18
  19. 19. 19 GOAL 3 Enhancing Community Support  RD patient community key in patient & family support  Priorities:  Adequate funding  Accessible information on Canadian resources to HCPs, patients and public  Well-resourced / utilized Canadian Orphanet database  The RD community:  Supports patients & families  Connects patients to resources and one another  Communicates RD information to policy- makers, decision-makers, the media & the public  Ensures patient voices are informed, empowered and heard 19
  20. 20. 20 GOAL 4 Sustainable Access to Promising Therapies  Challenges for drug access: small patient populations; lack causes, natural history, and long-term benefits of therapy; high individual cost  Priorities:  Canadian Orphan Drug Regulatory Framework  HTA process for common disease drugs inappropriately used for orphan drugs  Consistent pan-Canadian access  Immediate access through risk sharing/managed access programs CORD survey couldn’t access appropriate drug treatments 1 in 3 20
  21. 21. 21 GOAL 5 Promoting Innovative Research  Leverage pre-clinical research strengths  Priorities:  Collaborative research programs (SPOR PARTNERS)  Patient registries to enable Canadians in clinical trials  Patient-reported outcome measures and input on acceptable harm / benefit trade-offs  Studies on disease etiology and natural history of disease  Small clinical trial designs, adaptive designs  Applied research; pilot projects toward best practices 21
  22. 22. 22 GOAL 5 Promoting Innovative Research  Priority actions: a) Provide increased and dedicated funding for RD research and Centres of Excellence on RDs b) Establish new Canadian Partnership for RDs to coordinate national research agenda and Centres of Excellence 22
  23. 23. 23 5. Support Rising Across Canada
  24. 24. 24 Rare Disease Strategy Tour 24
  25. 25. 25 Stakeholders Discussing the Need for Canada’s Rare Disease Strategy 25
  26. 26. 26 Media Coverage of Cross-Country Tour 26
  27. 27. 27 CORD Advocacy Efforts Ottawa, ON (Mar. 10, 2016) Advocacy Day on Parliament Hill Toronto, ON (Feb. 29, 2016) International Rare Diseases Day Vancouver, BC (Jan. 19, 2016) Patient Rally 27
  28. 28. 28 6. Implementing the Strategy
  29. 29. 29 Early Wins  Orphan drugs recognized as priority at Jan. 2016 health ministers meeting  “The ministers affirm that strong, long-term solutions are needed to address the ongoing challenges.”  Agreed to work toward more consistent assessments of drugs and coverage decisions and a fair pricing strategy  ON Health Minister Feb. 2016 commits to establish :  EDS clinics that could expand to other rare diseases  Working group of patients and clinicians care for rare disease patients  1st Meeting WG on 25 Oct:  CORD Rare Disease Strategy as platform 29
  30. 30. 30 Rare Alliance Canada  Launched by CORD in winter 2016  Multi-stakeholder alliance comprised of patients, researchers, clinicians and industry representatives  Will oversee the implementation of the 5 goals and actions of Canada’s Rare Disease Strategy  Next steps:  Formalize membership  Establish steering committee  Develop plan for implementing priority actions 30
  31. 31. 31 Urgent Action # 1 Implement Orphan Drug Framework Background  2012 commitment by federal government to adopt a framework  Draft framework now ready but implementation timelines unclear Why is this Issue Urgent?  Will incentivize the development of new therapies to Canada  Will facilitate more timely patient access to new therapies  Will allow more Canadians to participate in clinical trials  Canada is the only developed country without an Orphan Drug Regulatory Framework  Need to harmonize Canadian regulations with those of Europe and USA Action Needed: Rally stakeholders under Rare Alliance Canada to call on Federal Government to implement framework now
  32. 32. 32 Urgent Action # 2 Adopt a Rare Disease Drug Program Background  Access challenges:  Many drugs don’t get listed or take many years to be reimbursed, often under very limited conditions  Access varies across provinces and among public/private insurance plans  2006 health ministers’ commitment to propose a funding model for rare disease drugs – but no action taken yet  Interprovincial working group established in 2014 on drugs for rare diseases  Expected to propose recommendations to health ministers in fall 2016 Why is this Issue Urgent?  Lack of access to the right therapies results in increased morbidity, loss of life or poorer quality of life and increased costs to the family, the healthcare system and the Canadian economy • Action Needed: Rally stakeholders under Rare Alliance Canada to call on governments to adopt a rare disease drug program
  33. 33. 33 Developing a Rare Disease Drug Program Objective  Provide timely, sustainable and affordable access to rare disease treatments for all Canadians Key Elements  Provide early access to patients and adjust price later to avoid delays (e.g., Germany and France)  Single set of eligibility criteria across the country so that no patient is left behind  Flexibility and exceptional adjudication measures  National pooled funding across all public drug plans to ensure equitable and affordable access across the country  Use managed access programs as a tool to provide sustainable access 33
  34. 34. 34 Managed Access Programs What is a Managed Access Program? • Arrangement between manufacturer and payer that enables payment for a drug under specified conditions Key Features • Accommodate high uncertainty in safety and effectiveness with on- going monitoring and data collection through patient registries • Address budget impact of uncertainty of patient numbers (diagnosis, eligibility) through risk-sharing plans • Collect cost-effectiveness data to address uncertainty of long-term benefit vs. harms and health outcomes (QoL, survival) • Assure high cost of individual use and total budget impact are justifiable in terms of appropriate patient use, adherence, documented patient outcomes, and new knowledge about disease 34
  35. 35. 35 CORD ARCTIC QUEST — August 2011 35
  36. 36. 36 Thank You! Durhane Wong-Rieger, PhD President Canadian Organization for Rare Disorders 416-969-7435 36