1

Canadian Organization for Rare Disorders

October 2016

Canada’s Rare Disease Strategy:

Why Now and What Next?

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 Canada6. Implementing the Strategy:

Rare Alliance Canada Acting now on regulatory framework & orphan drug

plan

2

3

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

4

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

2. What Are Rare Diseases?

7

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!

7

8

# 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

9

3.Why Does Canada Need a Strategy

10

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

10

11

Canada: Catching up With Rest of the World…

1980 2020

USA1983

JAPAN1993

AUSTRALIA1997

EUROPEANUNION1999

TAIWAN2000

SOUTHKOREA2003

USA & EUHarmonizatio

n2007 Canada

?

11

Adoption of Orphan Drug Legislation

12

4. What Does the Strategy Call For?

13

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

13

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

3 Guiding Principles

Fundamental Consensus Principles for Effective, Cost-Effective, and Sustainable RD Programs

I. Equity of AccessII. Patient-CenteredIII. Collaboration and

Coordination

15

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

2/5have 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

16

17

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

17

18

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

18

19

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

19

20

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

20

21

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

21

22

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

22

23

5. Support Rising Across Canada

24

Rare Disease Strategy Tour24

25

Stakeholders Discussing the Need for Canada’s Rare Disease Strategy

25

26

Media Coverage of Cross-Country Tour

26

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

6. Implementing the Strategy

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

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

30

31

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

32

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

33

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

33

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

CORD ARCTIC QUEST — August 201135

36

Thank You!

Durhane Wong-Rieger, PhDPresident

Canadian Organization for Rare Disorders

www.raredisorders.ca416-969-7435

durhane@sympatico.ca

36