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Durhane Wong-Rieger (CORD) Canada's Rare Disease Strategy Brief Overview


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Canadian Expert Patients in Health Technology Conference
Nov 7 – 8, 2016: DAY 1

Published in: Healthcare
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Durhane Wong-Rieger (CORD) Canada's Rare Disease Strategy Brief Overview

  1. 1. 1 Canadian Organization for Rare Disorders November 2016 Canada’s Rare Disease Strategy: Brief Overview
  2. 2. 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 2
  3. 3. 3 Key Stats on Rare Diseases 80% Genetic BUT 50% No Family History 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! 3
  4. 4. 4 # 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) 4
  5. 5. 5 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 5
  6. 6. 6 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? 6 Adoption of Orphan Drug Legislation
  7. 7. 7 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 7
  8. 8. 8 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 —  Implement early detection and preventive services across Canada CORD survey 2/5 have genetic condition but didn’t receive pre-natal counseling or screening On average: misdiagnoses 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 8
  9. 9. 9 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 9
  10. 10. 10 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 10
  11. 11. 11 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 11
  12. 12. 12 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 ¡  Studies on disease etiology and natural history of disease ¡  Applied research; pilot projects toward best practices 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 12
  13. 13. 13 Support for Rare Disease Strategy Ottawa, ON (Mar. 10, 2016) Lobby Day on Parliament Hill Toronto, ON (Feb. 29, 2016) International Rare Diseases Day Vancouver, BC (Jan. 19, 2016) Patient Rally 13
  14. 14. 14 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 14
  15. 15. 15 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 15
  16. 16. 16 Priority: 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; adjust price later to avoid delays —  Single set of eligibility criteria across the country —  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 16
  17. 17. 17 Managed Access (Risk- Sharing) Program 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 17
  18. 18. 18 Canadian Rare Disease Centres of Expertise and Community Partners —  Hemophilia Comprehensive Care Clinics & Registry —  Cystic Fibrosis Clinics & Registry —  Neuromuscular Clinics (Muscular Dystrophy) —  Red Blood Cell Clinic (University Health Network)/Sick Kids —  Metabolic Programme –BC Children’s Hospital
  19. 19. CORD Patient Support Members/1 —  Acoustic Neuroma Association of Canada —  aHUS Canada —  AKU Society —  Alpha-1 Canada —  Angioma Alliance Canada —  Answering TTP Foundation —  Aplastic Anemia Myleodysplasia Association of Canada —  Association des Patients Immunodéficients du Québec —  Atlantic Acromegaly Support Society —  Barth Syndrome Foundation of Canada —  Behcet’s Disease Canada —  Bethany’s Hope Foundation —  Canadian Amyloidisis Support Network —  Canadian Association for Alternating Hemiplegia —  Canadian Association for Porphyria/Association Canadienne de Porphyrie —  Canadian Association of Pompe —  Canadian FOP Network —  Canadian Chiari Association —  Canadian Fabry Association —  Canadian Fanconi Anemia Research Fund 19
  20. 20. CORD Patient Support Members/2 —  Canadian Hemophilia Society —  Canadian Immunodeficiencies Patient Organization (CIPO) —  Canadian MPN Network —  Canadian MPS Society —  Canadian Neuropathy Association —  Canadian PBC Society —  Canadian Pemphigus and Pemphigoid Foundation —  Canadian Pulmonary Fibrosis Foundation —  Canadian Skin Patient Alliance —  Carcinoid NeuroEndocrine Tumour Society Canada —  Cassie and Friends Society for Children with Juvenile Arthritis —  CDKL5 Canada —  Choroideremia Research Foundation of Canada —  Chronic Lymphocytic Leukemia Patient Advocacy Group —  CIHR Institute of Genetics / Orphanet-Canada —  Council For Bile Acid Deficiency Diseases —  Cystic Fibrosis Canada —  DEBRA Canada — —  Dystonia Medical Research Foundation Canada —  Ehlers-Danlos Syndrome Canada —  Encephalitis Global Inc 20
  21. 21. CORD Patient Support Members/3 —  Foundation for Prader-Willi Research Canada —  Galactosemia Canada —  GBS/CIDP Foundation of Canada —  GIST Sarcoma Life Raft Group Canada —  GNE Myopathy Support Group —  HAE Canada —  Huntington Society of Canada —  iBellieve Foundation —  Indian Organization for Rare Diseases —  Jesse’s Journey —  Jewish General Hospital, Institute of Community and Family Psychiatry —  Joubert Syndrome & Related Disorders Foundation —  Kidney Cancer Canada —  Medical Cannabis Access Society —  MitoCanada Foundation —  MPN Ontario Patient Support Group —  Muscular Dystrophy Canada —  Myeloma Canada —  National Gaucher Foundation of Canada —  Network of Rare Blood Disorder Organizations (NRBDO) —  Neuroblastoma Canada —  Neurofibromatosis Society of Ontario 21
  22. 22. CORD Patient Support Members/4 —  Ontario Rett Syndrome Association —  PANDAS/PANS Ontartio —  Polycystic Kidney Disease Foundation of Canada —  Pulmonary Hypertension Association of Canada —  PVNH Support & Awareness —  Rare Disease Foundation —  Regroupement québécois des maladies orphelines (RQMO) —  Scleroderma Society of Canada —  Shwachman-Diamond Syndrome Canada —  Sickle Cell Disease Assoc. of Canada —  Soft Bones Canada —  SUDEP Aware —  Tarlov Cyst Society of Canada —  The Anti-NMDA Receptor Encephalitis Foundation —  The Canadian Addison Society —  The Canadian CML Network —  The Foundation Fighting Blindness —  The Hospital for Sick Children —  The Oley Foundation —  Tuberous Sclerosis Canada Sclérose Tubéreuse (TSCST) —  Turner Syndrome Society of Canada —  Vancouver Acromegaly Support Group —  Vasculitis Foundation Canada —  VHL Canada 22
  23. 23. 23 Thank You! Durhane Wong-Rieger, PhD President Canadian Organization for Rare Disorders 416-969-7435 23