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Oct 25 CAPHC Breakfast Symposium - Sponsored by Alexion - CORD
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Transcript of Oct 25 CAPHC Breakfast Symposium - Sponsored by Alexion - CORD
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Canadian Organization for Rare Disorders
October 2016
Canada’s Rare Disease Strategy:
Why Now and What Next?
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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 Canada6. Implementing the Strategy:
Rare Alliance Canada Acting now on regulatory framework & orphan drug
plan
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1. Who is CORD?
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 CEO Durhane Wong-Rieger
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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
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2. What Are Rare Diseases?
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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 than2.8 MILLION!
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# Canadians with Common vs. Rare Diseases
0
0.5
1
1.5
2
2.5
3Milions Affected
Series3
1.4 M
2.8 M
2.4 M
1.8 M
(mill
ions
)8
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3.Why Does Canada Need a Strategy
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Now is the Time for a Rare Disease Strategy
Rare disease is a Major Public Health IssueRD Patients rarely access effective therapiesHealth systems waste resources, achieve limited
benefitsRare disease strategies work in other countries Canadian strategies work in other areas: Mental
health, cancer, diabetes, cardiovascular diseaseLeverage & coordinate expertise and resources
across disciplines and sectors and internationally
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Canada: Catching up With Rest of the World…
1980 2020
USA1983
JAPAN1993
AUSTRALIA1997
EUROPEANUNION1999
TAIWAN2000
SOUTHKOREA2003
USA & EUHarmonizatio
n2007 Canada
?
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Adoption of Orphan Drug Legislation
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4. What Does the Strategy Call For?
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Launch of Canada’s Rare Disease Strategy
Extensive consultations with rare disease stakeholders since 2012
Strategy Launched on Parliament Hill in May 2015Now is the Time
Shared across Canada: Political parties, policy
makers Healthcare providers &
admin Researchers, patients,
public
May 25, 2015
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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
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3 Guiding Principles
Fundamental Consensus Principles for Effective, Cost-Effective, and Sustainable RD Programs
I. Equity of AccessII. Patient-CenteredIII. Collaboration and
Coordination
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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
2/5have genetic
condition but didn’t receive pre-natal
counseling or screening
On average:
misdiagnoses before proper
one
2 to 3
1/4diagnosed
within 3 months
waited more than 3 years for diagnosis
1/3
1/5waited more than
for diagnosis6 years
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GOAL 1 Improving Early Detection and Prevention
Priority actions:a) Adopt national state-of-the art newborn screeningb) Implement early detection and preventive
services across Canada
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CORD survey
GOAL 2Timely, Equitable and Evidence-Informed
Care
PrioritiesRD training for GPs,
pediatricians, other HCPs Clinical practice guidelines Disease registriesComprehensive care &
support Centres of Excellence and
virtual networksLinkage 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
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GOAL 3Enhancing 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
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GOAL 4Sustainable 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
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GOAL 5Promoting Innovative Research
Leverage pre-clinical research strengthsPriorities:
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
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GOAL 5Promoting Innovative Research
Priority actions:a) Provide increased and dedicated funding for RD
research and Centres of Excellence on RDsb) Establish new Canadian Partnership for RDs to
coordinate national research agenda and Centres of Excellence
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5. Support Rising Across Canada
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Rare Disease Strategy Tour24
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Stakeholders Discussing the Need for Canada’s Rare Disease Strategy
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Media Coverage of Cross-Country Tour
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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
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6. Implementing the Strategy
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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
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Rare Alliance Canada
Launched by CORD in winter 2016Multi-stakeholder alliance comprised of patients,
researchers, clinicians and industry representativesWill oversee the implementation of the 5 goals and
actions of Canada’s Rare Disease StrategyNext steps:
Formalize membership
Establish steering committee
Develop plan for implementing priority actions
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Urgent Action # 1Implement 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
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Urgent Action # 2Adopt a Rare Disease Drug
ProgramBackground 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
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Developing a Rare Disease Drug Program
ObjectiveProvide timely, sustainable and affordable
access to rare disease treatments for all Canadians
Key ElementsProvide 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 behindFlexibility and exceptional adjudication measuresNational pooled funding across all public drug plans to ensure
equitable and affordable access across the countryUse managed access programs as a tool to provide sustainable
access
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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
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CORD ARCTIC QUEST — August 201135
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Thank You!
Durhane Wong-Rieger, PhDPresident
Canadian Organization for Rare Disorders
www.raredisorders.ca416-969-7435
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