Day 1: RD Fall 2022 Conference Strengthening Health Canada’s Commitment: Michelle Mujoomdar/ Daniel MacDonald, Health Canada

1. CORD Conference – 21 November 2022
Developing a National Strategy for
Drugs for Rare Diseases

2. Summary of key issues and areas of consensus emerging
from 18 months of extensive stakeholders consultation
2
Issues heard in consultations
• Fragmented decision-making across drug plans creates “postal code lottery” for DRD coverage
• Inadequate real-world data on safety and effectiveness poses challenges to drug coverage and
continued listing decisions
• Patients, families, and care providers feel powerless and disenfranchised
• High cost of drugs and absence of risk-sharing leaves drug plans financially vulnerable
Areas of consensus for national strategy
• Common governance framework and tools to move towards national consistency in coverage
• Better data to support decision-making and management of DRD formulary decisions
• Patients and families need to be at the centre of the strategy and any future government actions
• Long-term, predictable federal funding to support existing drug plans and improve access in a
sustainable way, for the benefit of patients

3. Framework of a national strategy for drugs for rare diseases
3
VISION Patients with rare diseases have improved access to effective drugs and better health
outcomes
PILLARS
Seek national
consistency
Invest in
innovation
Collect and
use evidence
Support patient
outcomes and
sustainability
PRINCIPLES
• Patient-centered
• Transparent and
accountable
• System alignment
and sustainability
• Efficient and effective
• Evidence-informed
• Collaborative and
Inclusive
• Adaptive

4. Potential Activities for the National Strategy
4
Ensure national
consistency
• Adopt a common vision and commitment for the national
strategy
• Enhance coordination and shared decision-making around
reimbursement decisions
Support patient
outcomes and
sustainability
• Promote risk-sharing and equitable sharing of costs among payers
• Explore innovative drug reimbursement models
Collect and use
evidence
• Develop data standards to enable the collection of data and
building of knowledge of rare disease and effective treatments
• Leverage and analyze health data, including real-world evidence
generation, to improve ongoing decision-making
Invest in innovation
• Support research by building on Canada’s RD research capacity
• Support access to data to spur clinical trials

5. Current Work & Next Steps
• Internal policy development steps
• Ad-hoc engagement with stakeholders
• Engagement with health system partners
• Engaging with PTs (FPT Pharmaceutical Executives Group)
• Launch strategy by the end of FY 22/23 and work with
partners and stakeholders to move forward
5