Canadian Expert Patients in Health Technology Conference Nov 7 – 8, 2016: Day 2

1. Expensive
Drugs
for
Rare
Diseases:
Challenges
and
Opportuni9es
for
Public
Payers
Angie
Wong,
Director
Drug
Programs
Policy
&
Strategy
Branch,
Ontario
Public
Drug
Programs
November
8,
2016

2. Agenda
• Background on Expensive Drugs For Rare
Diseases (EDRD)
• Key Themes & Challenges
• Summary
2

3. BACKGROUND
3

4. Background
• Recognize that rare diseases are complex, multi-
system and usually have a high burden of illness
• Growing gap between patients’ needs, patient and
provider expectations, system sustainability and
industry innovation and profitability
• Patient involvement in funding decisions
4

5. Put Your Screenshot
Here

6. National Approaches to Rare Disease Drugs
2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016
FABRY INITIATIVE
NATIONAL PHARMACEUTICAL
STRATEGY
1989
NATIONAL AGENCY FOR HEALTH TECHNOLOGY ASSESSMENT
HEALTH CARE INNOVATION
WORKING GROUP
PAN-CANADIAN PHARMACEUTICAL ALLIANCE
EDRD Working
Group
Case-by-Case P/T Decisions

7. EDRD WORKING GROUP
OPPORTUNITIES & CHALLENGES
7

8. EDRD Working Group
• January 2015: Provincial & Territorial (P/T) Health
Ministers supported the establishment of a P/T
Expensive Drugs for Rare Diseases (EDRD)
Working Group (WG)
• Objective: to explore the management of DRDs
with evidence-based approaches.
• Alberta, British Columbia and Ontario co-lead this
WG.
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9. Major Themes of Framework
• Communication
• Evidence
• Pricing
• Access
9

10. Communication
Objective:
• Recognize the need to enhanced communications
with patients and clinicians.
Key Opportunity:
• Need to bring forward objective information
• Opportunity to hear from stakeholders on
perspectives
10

11. Evidence
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Appropriate
design and
comparator
Understanding
natural
progression and
drug benefits
Uncertainty in
relative efficacy
and safety
Meaningful
clinical
outcomes
Accountability
for evidence
generation
Align regulatory
and
reimbursement
reviews
Establish robust evidence standards to
demonstrate incremental benefits of drugs for
rare diseases

12. Pricing:
• DRDs typically cost
hundreds of thousands of
dollars per patient per year
• Assessing cost-
effectiveness
12
Fair and affordable pricing to support
reasonable access to effective drugs for
rare diseases
• Opportunity costs
• Affordability and
sustainability

13. Access
Objective:
• Consistent implementation of CADTH
recommendations
Key Challenges:
• Timely access and stakeholder pressures
• Exceptions and compassionate access
• Balancing industry accountability including post
marketing evidence generation
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14. Types of Managed Access Programs
Performance based schemes
Managed Access Programs
Financial Based Outcome Based
market share
price volume
utilization or
price cap
manufacturer
funded
treatment
initiation
conditional coverage performance linked
reimbursement/outcomes
guarantee
patient level
population level
coverage with
evidence
development
conditional
treatment start
or continuation
only in research only for research
Adapted From: Sullivan, S. (2009). ISPOR Plenary.
Available from http://www.slideshare.net/raredisorders/rare-disease-access-issues-2015-v2

15. SUMMARY & QUESTIONS
15

16. Summary
Key Success Factors:
• Evidence-informed
• Accountability by all stakeholders
• Equity
• Patient education/empowerment
• Transparency
• Fair, non-excessive pricing
• Sustainability
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