This document summarizes a panel discussion on bringing rare disease drugs to Canada. The panel discussed opportunities and challenges, including having the National Pharmacare Council focus on rare disorders, implementing an orphan drug framework, and adopting managed access programs. Panelists represented patients, industry, and government perspectives on issues like national pharmacare and harmonizing provincial formularies. The discussion focused on improving access to treatments for rare diseases in Canada's universal healthcare system.

1. Bringing Rare Disease Drugs to Canada:
Opportunities, Challenges, and More Challenges
National Pharmacare and Rare Disorders
March 21, 2017

2. Federal context
2

3. Provinces / Federal-Provincial-Territorial Context
3

4. CORD on the Hill yesterday
4
1. Mandate the National Pharmacare
Council to pay special attention to the
2.8 million Canadians with a rare
disorder as it develops national
pharmacare – an opportunity to to fix the
gaps in accessing treatments for rare
disorders and remedy this problem in our
universal healthcare system
2. Implement the federal orphan drug
regulatory framework
3. Adopt a specific drug program or
funding approach – managed access
programs can help bring products through
the system in a sustainable way

5. Panelists
5
 John Oliver: Member of Parliament for Oakville;
Member of the House of Commons Standing
Committee on Health (HESA)
 Neil Palmer: Founder and Principal at PCDI
Market Access
 Sherry O’Quinn: Managing Principal at MORSE
Consulting and formerly Senior Pharmacist with
the Ontario Ministry of Health and Long-Term Care
 Durhane Wong-Rieger: President and CEO of
CORD

6. 6

7. National Pharmacare:
Where should the focus be?
W. Neil Palmer
Ottawa, March 2018

8. Why National Pharmacare?
1. Universal / comprehensive pharmacare coverage
– Providing coverage for those with no or inadequate coverage
– Ability to pay should not be a barrier
2. Equity / equitable access to prescription drugs across Canada
– National formulary of clinically and cost effective drugs
3. Reducing overall drug expenditures
– Increased bargaining power should result in lower prices
4. Public Only – eliminating private drug insurance
– Consistent with Canada Health Act principles for medically necessary services
March 2018 8

9. But…
1. Universal coverage – we still don’t know the extent of the lack of coverage
– Estimates are based on limited surveys with poor response rate
– True rate of inadequate coverage is unknown
– All jurisdictions have catastrophic coverage (but is it adequate?)
2. Equity / equitable access – pan-Canadian process already in place and evolving
– Provincial formularies are > 90% harmonized already
– CADTH (CDR, pCODR) and pCPA (all public jurisdictions) continue to improve harmonization
– National Formulary may further restrict / limit access to effective drugs
3. Reducing drug expenditures – mechanisms already in place and evolving
– National Pharmacare program is not a prerequisite for lower prices or lowering drug
expenditures
– PMPRB, pCPA , private insurers are actively reforming pricing and funding policies to limit
drug prices and expenditures – how would national pharmacare be any different
4. Public Administration – what will be gained by eliminating private drug insurance?
– Reduced coverage (fewer drugs covered)
– Private drug plans are an employee benefit (like dental, eye care)
– Opportunity Cost (of using scarce health care dollars to cover patients already covered by
private plans)
March 2018 9

10. Recommendations
• Universal Coverage – Yes
– Public plans should expand to cover those with no or inadequate private
coverage but not replace private coverage
– Funding from Feds?
• National Formulary – No
– Provincial Formularies must continue to reflect underlying provincial health
care systems and priorities
– CADTH, pCPA already harmonize >90%
• Physician prescribing
– Physicians (particularly specialists) should have greater latitude to decide
which medications are best for specific patients
March 2018 10

11. Biography
W. Neil Palmer
Founder & Principal Consultant
PDCI Market Access Inc
Neil.Palmer@pdci.ca
www.pdci.ca
Neil Palmer President and Principal Consultant of PDCI Market Access Inc. (PDCI) a leading pricing and reimbursement consultancy
founded in 1996.
Prior to PDCI, Neil worked with the Canadian Patented Medicine Prices Review Board (PMPRB) where his responsibilities included
policy development, overseeing the price review of patented medicines and conducting economic research. Prior to the PMPRB,
he worked with the Health Division of Statistics Canada where he was responsible for economic and statistical analysis of health
care costs and utilization. Neil also worked with RTI Health Solutions (Research Triangle Park, North Carolina) where he served as
global vice president for pricing and reimbursement. After completing his studies at the University of Western Ontario, Neil began
his career in Montreal with the research group of the Kellogg Centre for Advanced Studies in Primary Care. He has written
extensively on pharmaceutical pricing and reimbursement issues and is a frequent speaker at conferences in North America and
Europe.
Since 2015, Neil is Adjunct Assistant Professor at the University of Southern California School of Pharmacy graduate program in
Health Care Decision Analysis where he is an occasional lecturer on health technology assessment, pricing and market access
from a global perspective.
March 2018 11

12. Man i & O ’Qu in n Reimb u rsement S trateg y E xp erts
( MO RS E )
Public Reimbursement of Drugs for Rare Diseases
(DRDs) in Canada
Canadian Organization for Rare Disorders (CORD)
Rare Disease Day 2018 Conference: March 21, 2018
Presented by: Sherry O’Quinn
Managing Principal
Note: Analysis results are preliminary and require additional internal validation

13. Objective
13
To provide an analysis of drugs for rare diseases
(DRDs) which have made their way through the
Canadian reimbursement pathway in order to
better understand the specific challenges for
DRDs versus other drugs.

14. Health Canada
CADTH: CDR
CADTH:
pCODR
INESSS
pCPA
Public Payer Private Payer
Regulatory
HTA HTAHTA
Reimbursement
Reimbursement Reimbursement
How do drugs become a part of the
pharmaceutical market and achieve
funding in Canada?
PMPRB
Pricing
THE REIMBURSEMENT PATHWAY
LEGEND
PMPRB – Patented
Medicine Prices Review
Board
HTA – Health Technology
Assessment
INESSS – Institut National
d’Excellence en Santé et en
Services Sociaux
CADTH – Canadian Agengy
for Drugs & Technologies in
Health
pCODR- pan-Canadian
Oncology Drug Review
CDR – Common Drug
Review
pCPA – pan-Canadian
Pharmaceutical Alliance
The reimbursement process is complex with little customization for DRDs specifically.

15. MORSEAnalysis:
Objectives &
Assumptions
15
MORSE conducted a preliminary analysis* of DRDs for this presentation
Objective: to develop an understanding of how DRDs are considered in the
Canadian reimbursement landscape with a focus on CADTH recommendations
and pCPA negotiations
Methodology & Assumptions:
• Drugs for rare diseases were based on a list provided by CORD.
• All drugs reviewed by CADTH are based on MORSE’s internal database
• The relevant pCPA files for the list of DRDs were identified as were the
applicable HTA reviews in order to assess the reimbursement
recommendation, pCPA status and timelines associated with market access.
• Data from INESSS was not included
*Preliminary analysis with results still to be validated

16. CDRRecommendations:
AlldrugscomparedtoDRDs
16
3%
33%
2%
62%
Reimburse
Do Not Reimburse
Do not list at submitted
price
Reimburse with
conditions
14 (3%) recommend to reimburse
274 (62%) recommend to reimburse with criteria
and/or conditions
144 (33%) do not recommend to reimburse
11 (2%) do not reimburse at submitted price
CDR Recommendations for
all drugs*
n=443
CDR Recommendations for
Drugs for Rare Diseases**
n=52
Key Take-Away:
**only includes drugs for rare diseases which have been referred to the pCPA as identified by CORD
* Approximation based on MORSE data (as of March 2018)
2%
38%
2%
58%
1 (2%) recommend to reimburse
30 (58%) recommend to reimburse with criteria
and/or conditions
20 (38%) do not recommend to reimburse
1 (2%) do not reimburse at submitted price
Within the non-oncology space, there is a relatively small difference between the “positive”
reimbursement recommendations for DRDs when compared to all CDR recommendations as a whole.

17. pCODRRecommendations:
AlldrugscomparedtoDRDs
• Since its initiation in 2010 through to June 2017, pCODR has released 88 “Notifications to
Implement”
• Source: CADTH Drug Portfolio Slides – October 3, 2017
10%
68%
22%
Positive
Recommendation
Conditional
Recommmendation
Negative
Recommendation
9 (10%) recommend to reimburse
59 (68%) recommend to reimburse with clinical criteria
and/or conditions
19 (22%) do not recommend to reimburse
All pCODR Recommendations
As of June 2017
pCODR Recommendations for
Drugs for Rare Diseases*
68 (78%) received a recommendation recognizing clinical
benefit, with the majority noting a need for a price reduction to
improve cost-effectiveness
*only includes drugs for rare diseases which have been referred to the pCPA as identified by CORD
7 (10%) recommend to reimburse
47 (69%) recommend to reimburse with clinical criteria
and/or conditions
14 (21%) do not recommend to reimburse
10%
21%
69%
For oncology drugs, the recommendations for DRDs are very consistent with all drugs.

18. pCPANegotiationStatus:
DRDscomparedtoalldrugs
18
Drugs for Rare Diseases All Other Files
Individual
P/T Level
3
Referred to pCPA
115 Files
No
Negotiation
25
Negotiate
87
Currently
Underway
20
No Deal
6
Signed Letter
of Intent
61
70%
3%22%
7%
Referred to pCPA
190 Files
No
Negotiation
32
Individual
P/T Level
10
Negotiate
148
Currently
Underway
15
No Deal
15
Signed Letter
of Intent
118
17% 5%
80%
10%
DRDs have had a slightly lower percentage of files negotiated and completed, however, they have a
lower rate of failed negotiations. It is also noteworthy that more DRDs are currently being negotiated.
23%
75% 78%
10%

19. pCPANegotiationStatus:
ByCADTHReviewProcess
19
0
20
40
60
80
100
120
140
Declined Negotiation Completed Negotiation
(LOI)
Closed Negotiation (No LOI) Individual Negotiation Active
NumberofFiles
pCPA Status
pCPA Status of Drugs for Rare Diseases
By CADTH Review Process
CDR pCODR No HTA Review
The pCPA has successfully completed negotiations for more oncology than non-oncology DRDs. In
addition, there are a large number of DRDs under active negotiation right now.

20. pCPAProcessTimelinesfor
CompletedNegotiations:
DRDscomparedtoalldrugs
• It is assumed that pCPA Initiation/closure dates occur mid-month
20
The median calendar days for initiation and a successful negotiation for DRDs were similar compared to
all drugs, however, there were certain non-oncology DRD files that skewed the total duration.
0
50
100
150
200
250
300
350
400
450
CDR
n=67
pCODR
n=37
Median Calendar Days in pCPA
process for All Completed Files (LOI)
from 2015 – 2017*
Time to Initiation
Duration of Negotiation
Total Time from Recommendation to pCPA Completion
* Excludes pCPA initiated files
• n includes all files, however if initiation date was unavailable then that file was not included in the median
time to initiation or duration calculation
0
50
100
150
200
250
300
350
400
450
CDR
n=13
pCODR
n=43
No HTA Review
n=3
MedianCalendarDays
Median Calendar Days in pCPA Process
Completed (LOI) Negotiations
from 2010 – Jan. 2018*
Drugs for Rare Diseases
Time to Initiation
Duration of Negotiation
Total Time from Recommendation to pCPA Completion

21. pCPAProcessTimelinesfor
ClosedNegotiations:
DRDscomparedtoalldrugs
• It is assumed that pCPA Initiation/closure dates occur mid-month
21
For negotiations that are eventually closed with no LOI, DRDs from CDR have negotiations that are
started sooner but are negotiated over a longer time period than all drugs.
0
100
200
300
400
500
600
CDR
n=6
pCODR
n=1
Median Calendar Days in pCPA Process
Closed (No LOI) Negotiations from 2010 to Jan. 2018
for Drugs for Rare Diseases
Time to Initiation
Duration of Negotiation
Total Time from Recommendation to pCPA Closure of Negotiation
0
100
200
300
400
500
600
CDR
n=16
pCODR
n=1
Median Calendar Days in pCPA process for All Closed
Files (LOI) from 2015 – 2017*
Time to Initiation
Duration of Negotiation
Total Time from Recommendation to pCPA Closure of Negotiation
* Excludes pCPA initiated files
• n includes all files, however if initiation date was unavailable then that file was not included in the median
time to initiation or duration calculation

22. Considerations
• This analysis does not show:
o Time to listing across public plans
o Challenges with implementation or restrictive criteria
o Impact of stakeholder input
o Changes within process
o Potential changes in metrics over time
• Surprising findings with less “difference” between HTA review
outcomes and pCPA metrics for DRDs vs. all drugs
• A large number of DRDs are within the oncology space where
there seems to be almost no difference
• More analysis can help shed further light on these issues.
22

23. Panelists
23
 John Oliver: Member of Parliament for Oakville;
Member of the House of Commons Standing
Committee on Health (HESA)
 Neil Palmer: Founder and Principal at PCDI
Market Access
 Sherry O’Quinn: Managing Principal at MORSE
Consulting and formerly Senior Pharmacist with
the Ontario Ministry of Health and Long-Term Care
 Durhane Wong-Rieger: President and CEO of
CORD

25. 25
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