Transcript 10 19 2015_Wong-Reiger_CA RD Strategy Clinical Present Oct

Canada’s Rare Disease Strategy:
Blueprint for Action
Durhane Wong-Rieger, PhD
President,
Canadian Organization for Rare
Disorders
October 2015
Canadian Organization for Rare Disorders
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 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
CORD CA Framework
March 2015
What Does CORD Do?
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 Support Rare Disease Patient Groups: organize, develop,
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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
Conduct, train, and support patient organization
submissions to HTA and regulatory reviews
Advocate with or on behalf of patient groups and individual
patients for access diagnosis, treatment and services.
CORD CA Framework
March 2015
Early detection, screening, and diagnosis
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“At 33, I finally got a diagnosis
at about the same time as my
2-year-old daughter who was
diagnosed with the same
genetic condition. But we
started her on therapy right
away so hopefully she’ll never
suffer the way I did.” (Ian, 34year-old man with MuckleWells)
CORD CA Framework
May 2015
Timely, equitable and evidence-informed care
5
“Surgery and radiation did not
successfully remove the tumour causing
my rare disorder. Treating the tumour
resulted in other rare conditions for
which medications are generally not
covered. After a long battle, coverage
was attained for one medication, but a
new drug that could treat the tumour is
not yet available in Canada. So the fight
by our patient community continues.”
(Tracy, 44-year-old mom with Cushing’s
Disease)
CORD CA Framework
May 2015
Community support
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“When you grow up thinking
monthly blood transfusions and
nightly 10-hour drug infusions
are “normal”, almost anything
else is possible. A rare disease
isn’t limiting if you have
treatment but most important
friends and family support.“
(Cassandra, 16-year-old girl
with Thalassemia major)
CORD CA Framework
May 2015
Sustainable appropriate access
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"I have an ultra-rare blood disease that
leads to kidney failure. The only effective
therapy is a drug approved by Health
Canada but denied by the provincial public
drug plans because of cost. This disease
destroyed my own kidneys as well a
transplanted kidney donated by my wife.
The doctors won't allow another
transplant unless I’m on this drug, but my
province refuses to approve this drug for
transplants. So I must remain on
dialysis." (Michael, 50-year old with
aHUS).
CORD CA Framework
May 2015
Why Canadian Strategy for Rare Diseases?
But Canada’s federation makes
national approach a challenge!
# Canadians with Common vs. Rare Diseases
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Milions Affected
3.5
3
2.8 M
2.5
Cancer
2.4 M
Diabetes
2
Heart disease
1.8 M
1.5
1
0.5
0
1.4 M
Rare Diseases
Why A Canadian Strategy for Rare Diseases
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 Rare disease: 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
Next Step:
Canada’s Rare Disease Strategy
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 Extensive consultations with
rare disease stakeholders
since 2012
 Strategy Launched on
Parliament Hill in May 2015
 Now is the Time
 Shared across Canada:
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Political parties, policy makers
Healthcare providers & admin
Researchers, patients, public
May 25, 2015
5 Key Goals of
Canada’s Rare Disease Strategy
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1.
2.
3.
4.
5.
Improving early detection and
prevention
Providing timely, equitable and
evidence-informed care
Enhancing community support
Providing sustainable access to
promising therapies
Promoting innovative research
3 Guiding Principles
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Fundamental Consensus Principles
for Effective, Cost-Effective, and
Sustainable RD Programs
Equity of Access
II. Patient-Centered
III. Collaboration and Coordination
I.
5 Components of
Canada’s Rare Disease Strategy
Timely Access;
Centres of
Expertise
Screening,
Detection &
Prevention
Harmonized Orphan Drugs/Rare Diseases Policy - Latin America
Innovative
Access to
Therapies
Engaging
Patints &
Communities
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Research: Finding
Genes to Finding
Cures
May 2015
GOAL 1
Improving Early Detection and Prevention
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CORD survey
2/5
have genetic
condition but didn’t
receive pre-natal
counseling or
screening
On average:
2 to 3misdi
agnoses before
proper
one
1/4
diagnosed
within 3
months
1/5
waited more than
6 years
1/3
for diagnosis
waited more
than 3 years
for diagnosis
1. Detection & Prevention
2. Access to Care
Newborn screening in all provinces
 Next-generation diagnostic testing;
state-of-the art international labs
 Standards for pre-conception, prenatal genetic screening and
counseling
 Consistent, comprehensive, up-todate genetic testing guidelines and
tests
 Genetic testing linked to RD
registries, expert centres, healthcare
services
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3. Community Support
4. Access to Therapies
5. Research
GOAL 1
Improving Early Detection and Prevention
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 Priority actions:
1.
Adopt national state-of-the art newborn screening
2.
Implement early detection and preventive services
across Canada
1. Detection & Prevention
2. Access to Care
3. Community Support
4. Access to Therapies
5. Research
GOAL 2
Timely, Equitable and Evidence-Informed Care
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CORD survey
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 receive adequate
information from their doctor
2/5
did not understand the information
they received
did not receive resources
or contacts to seek
additional information
2/3
were not referred to a
patient organization
1. Detection & Prevention
2. Access to Care
3. Community Support
4. Access to Therapies
5. Research
GOAL 3
Enhancing Community Support
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 RD patient community key in
patient & family support
 Priorities:
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 The RD community:
Adequate funding
Accessible information on
Canadian resources to HCPs,
patients and public
Well-resourced / utilized
Canadian Orphanet database
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1. Detection & Prevention
2. Access to Care
3. Community Support
Supports patients &
families
Connects patients to
resources and one another
Communicates RD
information to policymakers, decision-makers,
the media & the public
Ensures patient voices are
informed, empowered and
heard
4. Access to Therapies
5. Research
GOAL 4
Sustainable Access to Promising Therapies
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CORD survey
1 in 3
couldn’t access
appropriate drug
treatments
1. Detection & Prevention
 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 panCanadian access
 Immediate access thru risk
sharing/managed access programs
2. Access to Care
3. Community Support
4. Access to Therapies
5. Research
GOAL 5
Promoting Innovative Research
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 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
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1. Detection & Prevention
2. Access to Care
3. Community Support
4. Access to Therapies
5. Research
GOAL 5
Promoting Innovative Research
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 Priority actions:
19. Provide increased and dedicated funding for RD
research and Centres of Excellence on RDs
20. Establish new Canadian Partnership for RDs to
coordinate national research agenda and Centres of
Excellence
1. Detection & Prevention
2. Access to Care
3. Community Support
4. Access to Therapies
5. Research
Canada: Catching up with rest of the world…
 1983: USA incentivize development of “unprofitable” drugs
 Definition Rare Disease: <200,000 Americans (1:1,570)
 Incentives: Protocol assistance, subsidies, 7-yr exclusivity, tax credits, fee waivers, fast track
 1993: Japan develop drugs for “intractable” diseases
 Rare disease < 50,000 (1:2,550) no effective treatment, major financial/psychological burden
 Incentives: protocol assist, subsidies, 10-yr exclusivity, tax credits, priority review, fast track
 1999: European Union attract R&D to Europe ($ incentives by country)
 Rare disease: <1:2,000; life-threatening/chronically debilitating; no treatment/significant better
 Incentives: protocol assistance, 10-yr market exclusivity, fee waivers, fast track approval
 2000: Taiwan attract drug development
 Rare disease: <1:10,000; genetic origin, difficult to diagnose and treat
 Incentives: protocol assistance, 10-yr market exclusivity, subsidies, fast track approval
 2003: South Korea support local drug development
 Rare disease: 1:20,000 with no appropriate treatment or alternative
 Incentives: protocol assistance, 10-yr market exclusivity, subsideis, fast track approval
 2007: USA and EU Harmonization
 Single orphan designation application; separate regulatory approval filing; single 5-year update
CORD CA Framework
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March 2015
Orphan Drugs: Key Success Factor
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 Innovative drugs for rare diseases, aka orphan drugs, are a key to
detection, prevention, treatment, and understanding of rare diseases.
Hope of an effective treatment galvanizes patients, clinicians,
researchers, drug companies, and investors.
 Canada’s Rare Disease Strategy provides a framework for research on
rare diseases and discovery of targeted therapies, capitalizing on incountry expertise and capacity.
 Canada is still the only developed country without orphan drug
legislation. Canadian patients miss out on clinical trials and access,
still receiving only about 60% of what is available elsewhere.
 Canada’s Orphan Drug Regulatory Framework, announced in 2012, will
harmonize Canadian regulations with those of Europe and USA,
providing support and incentives for R&D on rare disease drugs. It is
not yet implemented.
Harmonized Orphan Drugs/Rare Diseases Policy - Latin America
May 2015
Canada Orphan Drug Regulatory Framework:
Breakthrough Step for Canadians
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 Definition of and orphan drug designation harmonized to allow joint
application with USA FDA and European Union EMA
 Life-cycle approach to ensure ongoing oversight and collection of a
wide body of evidence before and after a drug is marketed
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Expedited approval based on life-threatening or severely debilitating
condition with no other effective treatments
Regulatory approval for ODs require on-going data collection and
resubmission of outcomes data (5 years)
Patient registries to collect post-market safety and effectiveness
On-going studies with expanded patient population
 Bill C-17 Enhances Post-Market Monitoring
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Opportunities for Managed Access & Coverage with evidence development
Allows for early engagement of HTA (and payers)
Mandates post-market “real world” data collection & evaluation
CORD CA Framework
March 2015
But Rare Disease Drugs: Challenges for Reimbursement
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Incremental Value Added (effectiveness, side effects, tolerability,
improved quality of life) may not equal incremental costs
Pricing criteria may not be established, and willingness to pay
may have little impact on pricing
Medicines for rare and unmet needs tend to have high R&D, high
uncertainty, high cost
Reimbursement strategies may be directed toward reducing
uncertainty in safety, effectiveness, appropriate use, and budget
impact.
Managed access schemes include registries, CED, prior
authorization, limited use, $ capitation.
How Patients Access Drugs in Canada
Manufacturer submits
Up to 2 years
Non-transparent
Health Canada
Issues NOC & DIN
PMPRB Reference-Based
Ceiling Price
Manufacturer submits
pCODR Products
(NCE / new combination product /
new indication)
pERC recommendation to
drug plans
specific to oncology drugs
Common Drug Review products
(NCE / new combination product /
new indication)
CDEC recommendation to drug plans
NOC: Notice of Compliance –
DIN: Drug Identification Number
PMPRB: Patented Med Pricing Review Board
CDR: Common Drug Review
CDEC: Canadian Drug Expert Committee
pCODR: pan-CA Oncology Drug Review
PERC = pCODR Expert Review Committee
INESSS: Institut national d'excellence en
santé et en services sociaux
PCPA = panCanadian Pricing Alliance
Private Drug Plans
INESSS for
Quebec drug plan
Provincial Exceptional Access Programs
Ontario Drugs for Rare Diseases
Alberta Special Access/Off-Label Drugs
BC Rare Disease Drugs Committee
New Brunswick Rare Disease Drugs Fund
panCanadian Pharmaceutical Alliance
(Pricing Negotiations)
Up to 1 year
Transparent
~ varied; not
transparent
Provincial Drug Committee Reviews Health Canada status, CDR/pCODR
recommendation, Province-specific assessment
Provincial process recommends to reimburse (or not) through publicly funded program
Up to 2 mths
Transparent
Negotiations with Provincial Decision Maker on Managed Access/Risk
Sharing
Open ended
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Final Decision by Executive Officer/DOH/Minister
March 2015
Reimbursement Process
Federal
• Health Canada provides NOC
• “Does it work?”
National HTA
• CDR conducts HTA on non-cancer drugs
• pCODR conducts HTA on cancer drugs
• “How does it compare with what we already have?”
Provincial Plans
• Collectively negotiate pricing (pCPA)
• And/or individually negotiate access on the basis of provincial HTA, priorities,
capacity
• “Can we afford it?”
Patients
• Decide which patients covered by public plans (age, income, etc.)
• Decide how to access (open, expert treaters, individual approval)
• Decide conditions for “start-stop” (managed access, reassessment)
• Can we treat (only) patients who benefit?
CORD CA Framework Courtesy of: © 2014, Wyatt Management 27
Consulting Inc. All Rights Reserved
March 2015
Specialized Programs for Rare Disease Drugs
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 2010: Ontario Drugs for Rare Diseases Program evaluation framework
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using Markov Model to set access criteria and outcome benchmarks
2010-11: BC Rare Disease Committee review case-by-case applications
based on evidence and physician submission
2011: Alberta Special Authorization Process for some rare disease
drugs; Start-Stop criteria based on submitted evidence; additional $3
million fund for “off-label” or not Special Authorization drugs
2011-2015: Interprovincial joint action from panCanadian Purchasing
Alliance to panCanadian Pricing Alliance to pC Pharmaceutical Alliance
Quebec: Individual approval through “mesure du patient d’exception”;
now joining pCPA
2012: Private drug plans (CLHIA) implements “pooled” insurance fund
(full coverage plans only) for all drugs > $25k/year
CORD CA Framework
March 2015
CORD ARCTIC QUEST—August 2011
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What can people with rare disorders do?
Anything!
Harmonized Orphan Drugs/Rare Diseases Policy - Latin America
May 2015
Thank You!
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Durhane Wong-Rieger, PhD
President
Canadian Organization for Rare Disorders
www.raredisorders.ca
416-969-7435
[email protected]
Harmonized Orphan Drugs/Rare Diseases Policy - Latin America
May 2015