The document outlines the context and developments surrounding Canada's rare drug program and the adoption of a UN resolution addressing the challenges faced by individuals with rare diseases. It highlights the need for a national rare disease drug strategy that promotes patient access, research, equity, and various support measures, as well as the establishment of a Canadian Rare Drug Agency to facilitate timely access to treatments. Additionally, it discusses the social and economic challenges faced by families affected by rare diseases, exacerbated by the COVID-19 pandemic.

1. DEC 1 & 2, 2021 | 11 AM- 2 PM
VIRTUAL CONFERENCE
BUILDING CANADA’S
RARE DRUG PROGRAM:
A BOLDACIOUS
BILLION$ INVESTMENT
FALL 2021 CONFERENCE

2. 2015: CORD releases Canada’s Rare Disease Strategy
2019: Federal government announces $1 billion to set up national Expensive Drugs for
Rare Diseases Strategy
2020: CORD “Jump Starts” consultations on Rare Drug Strategy with series of webinars
and conference
2021: Health Canada hosts consultations and releases report reinforcing “patient-
centred” access; drops reference to “expensive drugs”
2021-22: CORD continued public consultations on implementation
2022: April target date for launch of national rare disease drug program
2022: Implementation and assessment of “learning” projects
Building Canada’s Rare Drug Program:
How We Got Here and Where Do We Go From Here?

3. UN Resolution on Addressing Challenges of Persons Living
With a Rare Disease and their families
UN Resolution has been
adopted by consensus
by the Third Committee of the
UNGA under Agenda item 28
“Social Development”
on 15 Nov 2021
Qatar
Brazil
Spain
3 Member States: leading the Negotiations with 54
Countries Co-sponsoring

4. A draft proposal at the Third Committee of the UNGA
(Social, humanitarian, human rights, and cultural issues)
December: official adoption by UN General Assembly

5. Snapshot of a Life with a Rare Disease
For a child with a rare disease, discrimination strikes very early in life with accessing education,
and in adulthood it is difficult to find, maintain or return to work. The same for lifelong leaning.
Women are disproportionately discriminated and excluded from society, either as persons with
a rare disease or as mothers.
Families with a member living with a rare disease are at greater risk of impoverishment as they
have more expenses and less income.
With the COVID pandemic, all the pre-existing inequalities and vulnerabilities of this population
have been severely aggravated.
Heterogeneous but rare: with rarity comes lack of knowledge and expertise, lack of research
and innovation, lack of appropriate health and social care. Rarity means lack of hope.

6. Five overarching asks in UN Resolution

7. HUMAN RIGHTS &
INCLUSION
• Address root causes of discrimination including through awareness-raising and dissemination of information
• Collect disaggregated data on PLWRD to identify discrimination and assess progress
• Right and access to educating and lifelong learning
• Promote access to decent work along with appropriate measures for financial inclusion
APPROPRIATE CARE
• Strengthen health systems and accelerate achievement of UHC by 2030 for PLWRD and realize their right to
physical & mental health and enhance health equity
• Encourage creation of networks of experts and multidisciplinary specialized centres
• Support research by strengthening international collaboration, coordination of research efforts and sharing of data
• Encourage promotion of mental health & psychological support for PLWRD, well-being of families & caregivers
NATIONAL ACTION &
MEASURES
• Encourage UN Member States to adopt national strategies, action plans and legislations to ensure PLWRD are not
left behind and promote full participation in society of PLWRD, families and caregivers
• Encourages Member States to provide affordable, accessible and good quality care facilities for children and
measures promoting equal sharing of household responsibilities
INCLUSION IN THE
UN SYSTEM
• Member States, UN Agencies and other stakeholders - in consultation with PLWRD and their representatives
organizations - to design and implement policies and share experience to ensure that AGENDA 2030 is inclusive and
accessible to PLWRD
• Encourages elimination of barriers in accessing water, sanitation and hygiene in both rural and urban areas
MONITOR
PROGRESS &
IMPLEMENTATION
• Given the multifaceted nature of these challenges, decides to consider the issue of challenges faced by PLWRD and
their families at its 78th session (Sep 2023) under the item “Social Development”

8. Opportunities for implementation
• Office of the High Commissioner for
Human Rights (OHCHR)
• Continued engagement with WHO
• Continued advocacy for plans and
strategies at national level
• Work with other UN Agencies &
Programmes: UNICEF, UN Women,
World Bank, ILO

9. 12 Steps to a
National
Rare Disease
Framework

10. • Patient empowerment: Empower patient organizations and patient advocates as active
full partners
• Creation of a Canadian Rare Drug Agency: independent, transparent, publicly
accountable agency with responsibility for all aspects of the review of drugs for rare
diseases, in coordination with Health Canada
• Create R&D incentives: Invest in Research and Development to support therapeutic
product accessibility, monitoring, and evaluation. Build capacity for drug discovery,
technological innovation, manufacturing and production, and (global) distribution.
• Speed up access to treatment: Ensure timely availability of new treatments by
establishing a competitive and viable environment, including supportive mechanisms
for clinical trials, early access programs, clinical site development, patient registries,
and patient support programs
12 Steps to RD Framework

11. 12 Steps to RD Framework
• Address regulatory barriers: Ensure PMPRB guidelines do not exceed a “reasonable”
threshold of fairness compared to comparable countries; roll back 2019 PMPRB
regulatory changes by removing use of economic factors
• Improve regulatory approvals process: Ensure Health Canada continues to update its
regulatory process to encourage clinical trial and new drug submissions for rare
disease drugs
• Ensure pathways for special cases: For urgent need, timely access provided through
Special Access Program (SAP) prior to Health Canada approval; create pathway through
Early Access Programs.
• Need for multiple funding options: Multiple separate pathways based on population
size, disease severity, unmet need, evidence uncertainty, potential therapeutic value,
budget impact, annual unit price, and industry

12. 12 Steps to RD Framework
• Leverage Managed Access Programs (MAPs): For drugs receiving an NOC-C where there
is uncertainty about the evidence at the time of approval but where the unmet needs
and benefits outweigh the risks.
• Facilitate concurrent Health Canada and HTA reviews: Joint Health Canada and HTA
application (where appropriate)
• Support real-world evidence generation: Be responsible for real-world monitoring,
data collection, evaluating benefits, risks, and uncertainty
• Enhance centres of clinical expertise: Partner in developing Networked Centres of
Expertise for specific rare diseases related to management of a therapy.

13. 11:00 am - 11:15 am What We Need to Succeed
• Durhane Wong- Rieger, CORD
Canada’s Rare Disease Drug Program
Vision: Integrated, Inclusive, Innovative Rare Drug System
• Single Seamless Pathway from R&D, CT, regulatory approval, access parameters, monitoring, values-based assessment, price negotiations
• Governance board representing all stakeholders: diverse patient community, clinical specialities, public/private drug plan providers, HTA
agencies, pharmaceutical companies, subject matter experts (regulatory, research)
Building for Success: 12 Steps, 4 Platforms
December 1, 2021
Day 1: Getting to Access
Agenda

14. Platform 1: Expedite Timely and Optimal Access to Diagnosis and Care
• Create networks of Specialty Centers for Expert Clinical care and Research and connect to local healthcare providers and social
support programs
• Implement accurate accessible timely diagnosis through national Newborn Screening guidelines and Next-Generation Exome/Genome
Sequencing
• Create R&D incentives including public-private partnerships involving academic and clinical sites, coordinated through institutions such
as CIHR, National Research Council, Genome Canada, Centre for Commercialization of Regenerative Medicine, Regenerative Medicine
Alliance of Canada, and Maternal Infant Child Youth Research Network
Panelists:
• Hugh McMillan, McGill University Health Centre
• Cheryl Rockman-Greenberg, Max Rady College of Medicine
• Thierry Lacaze-Masmonteil, MICYRN
• Frederic Lavoie, Pfizer
• Brent Warner, Novartis
Case Study: Gene Therapy for Inherited Retinal Disease
• In October 2020, Health Canada approved Luxturna as the first therapy for previously untreatable inherited retinal disease. This one-time
therapy replaces dysfunctional RPE65 genes with working copies that restore vision and improve sight.
• In Canada, for children and adolescents with IRD, there is an urgent need for timely access to Luxturna, with several young people moving
beyond the period of efficacy now at high risk of losing their vision. Despite a CADTH positive recommendation for funding, the provinces
have not yet provided access. Private payers have denied access, citing pre-existing condition.
• This situation must be addressed with a national solution in the future Rare Disease Drug Strategy. In the meantime, we urgently need
intervention to save the sight of children who are NOW at risk.
Panelists:
• Elise Heon, The Hospital for Sick Children (Toronto)
• Doug Earle, Fighting Blindness Canada
• Patient Representatives
12:15 pm - 12:45 pm
11:15 am - 12:15 pm

15. Platform 2: Create Competitive Access Environment for Innovation
• Develop competitive environment for collaborative drug development, clinical trials, early access, and patient support.
• Roll back 2019 PMPRB changes to refocus on fair, non-excessive pricing and NOT restrictive pricing that discourages and delays new drug
launches
• Enhance Health Canada collaboration and coordination with FDA, EMA, and other regulators on regulatory reviews and approvals; engage
with patients.
• Implement pathways for special access, including early access pre-NOC and pre-reimbursement agreements.
Panelists:
• Eileen McMahon, Torys
• Wayne Critchley, Global Public Affairs
• Michael May, CCRM
• Declan Hamill, Innovative Medicines Canada
• Angela Genge, The Neuro, McGill University
• Laurene Redding, BeiGene
• Fred Little, Pfizer
• Oxana Illiach, CORD
• Tammy Moore, ALS Canada
12:45 pm - 1:45 pm
1:45 pm - 2:00 pm Preparing for Action
• Durhane Wong- Rieger, CORD

16. 11:00 am - 11:15 am Learnings from Day 1 (Access)
• Durhane Wong- Rieger, CORD
December 2, 2021
Day 2: Realizing the Vision
Agenda
11:15 am - 12:30 pm
Platform 3: Create Innovative Financing Pathways for Efficient, Effective, and Cost-Effective Drug Access
• Establish national agency to develop multiple financing pathways specific to needs of patient population and drug characteristics for
universal access across multiple payers
• Leverage Managed Access Programs to provide timely access of approved therapies with potential benefits to specified patients under
conditions of continued monitoring and collection of real-world data
• Facilitate concurrent Health Canada and health technology assessment reviews to promote shared interpretation of safety and efficacy data
and expedite access
• Implement processes for real-world data collection and utilization of real-world evidence throughout drug cycle from clinical trials to real-
world usage to promote timely, evolving, evidence-informed access
Panelists:
• Rosalie Wyonch, CD Howe
• Chris McMaster, CIHR
• Laurie Lambert, CADTH
• Suzanne McMullen, Medlior
• Christopher Pettengell, Pentavere
• Christina Cunningham, Vertex Canada
• Bob McLay, SOBI Canada

17. Platform 4: Implement meaningful representative patient engagement throughout the drug system in every
area, at every level and in every way possible in the Rare Disease Drug Agency
• Through CORD, provide training, support, and resources to enable effective participation.
• Enforce policies and practices of open, transparent, and accountable communications
• Ensure equity through inclusive diversity with attention to those excluded and neglected because of race, ethnicity, biological sex/gender,
age, language, education, literacy, income, immigrant status, indigenous status, and rural geographic residence
• Ensure equitable access to community support programs
• Establish the Canadian Rare Drug Agency as an independent, transparent, publicly accountable agency with responsibility for all aspects of
the review and assessment of drugs for rare diseases, in coordination with accountable agencies (Health Canada, PMPRB, CADTH/INESSS,
pCPA, public drug programs, private drug programs)
• Guarantee sufficient budget to carry out its mandate, with “hands-off” funding from the PT and federal governments
• Establish representative board including all stakeholders
Panelists:
• David Page, Canadian Hemophilia Society
• Maureen Smith
• Thea Discepola, Novartis
• Christian Hansen, Alexion
• Peter Brenders, BeiGene
• Jessica Brcko, Sun Life
• Glenn Monteith, Global Public Affairs
12:30 pm - 1:45 pm
1:45 pm - 2:00 pm Next Steps for Action
• Durhane Wong- Rieger, CORD

18. The Canadian Organization for Rare Disorders acknowledges the contribution of all our Corporate Partners to improving
the lives of patients and families with rare disorders.
We are especially grateful to the following that have supported this conference.