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

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Canadian Organizat ion for Rare Disorders

N o v e m b e r 2 0 1 6

Canada’s Rare Disease Strategy:

Brief Overview

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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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!

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# Canadians with Common vs. Rare Diseases

0

0.5

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1.5

2

2.5

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3.5 Milions Affected

Cancer Diabetes Heart disease Rare Diseases 1.4 M

2.8 M

2.4 M

1.8 M

(mill

ions

)

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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

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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?

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Adoption of Orphan Drug Legislation

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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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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

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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

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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

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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

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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

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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

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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 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

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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

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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

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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

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

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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 �  Dravet.ca �  Dystonia Medical Research

Foundation Canada �  Ehlers-Danlos Syndrome Canada �  Encephalitis Global Inc

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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

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

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Thank You!

Durhane Wong-Rieger, PhD President

Canadian Organization for Rare Disorders

www.raredisorders.ca 416-969-7435

[email protected]

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