Date: Wednesday, September 4, 2019 Time: 12:00pm – 1:00 pm EST Why did the Liberal government wait until the dead of summer … when Parliament had left town and most people were on holidays … to put into effect their new regulations on prescription drug pricing? Perhaps they were hoping to avoid close scrutiny of the biased statistics and flawed analyses to justify the draconian regulated price reductions. Perhaps they hoped the patients, who would lose out on medicines NOT coming to Canada, would not notice. Perhaps they hoped the summer media would just “buy” their version of these changes. Which is why CORD’s next Webinar, Debunking the Myths of the New Regulations on Prescription Drug Pricing Controls is a must-attend opportunity to learn more about the federal pricing regime. We have less than a year before these new regulations come into effect. The Patented Medicine Prices Review Board (PMPRB) will soon consult on guidelines for how the new rules will operate. The fast-approaching October 21 federal election is another important opportunity to ensure our voices are heard. Join CORD and other experts to unpack the regulatory changes and how they will impact medicines available today and those yet to come. Presenters: Neil Palmer, Senior Strategic Adviser at PDCI Market Access Inc (PDCI) Durhane Wong-Rieger, President & CEO, CORD Moderator: Bill Dempster, CEO, 3Sixty Public Affairs Biographies Neil Palmeris Senior Strategic Adviser at PDCI Market Access Inc (PDCI), Canada’s leading pricing and reimbursement consultancy that he founded as Palmer D’Angelo Consulting Inc (PDCI) in 1996. Neil’s current role at PDCI is to provide strategic advice and expertise to PDCI’s clients on important pricing and market access matters. Neil has more that 30 years of pharmaceutical pricing and market access experience and formerly held the titles of Founder, President and Principal Consultant at PDCI. Durhane Wong-Rieger,PhD, is the President & CEO of the Canadian Organization for Rare Disorders (CORD) and the Institute for Optimizing Health Outcomes (Canada). She is also Chair of the Consumer Advocare Networkand Chair of Canadian Heart Patient Alliance. Internationall, she isChair of the Council for Rare Disease International, member of the Steering Committee of the UN NGO Committee for Rare Diseases, Board member of Asia Pacific Rare Disease International, member of the Editorial Board of The Patient- Patient Centred Outcomes Research, member of the Global Commission to End the Diagnosis Odyssey for Rare Diseases and member of Health Technology Assessment International Patient /Citizen Involvement Interest Group. Bill Dempster, CEO and co-founder of 3Sixty Public Affairs Inc., is an in-demand health and pharmaceutical policy expert who works with a range of clients to navigate complex political, policy, regulatory and reimbursement challenges in the Canadian life challenges in the Canadian life sciences field.

1. PMPRB Webinar
Debunking the
Myths of the New
Regulations on
Prescription Drug
Pricing Controls
Sep. 4, 2019
12pm – 1pm

2. Today’s speakers:
Neil Palmer
Principal
PDCI Market Access Inc.
Bill Dempster
CEO
3Sixty Public Affairs
2
Durhane Wong-Rieger
President
CORD

3. Recap: what is the PMPRB?
• Quasi-judicial federal agency
• Regulates medication prices while under patent
• Reports to the federal health minister
• Twofold mandate
o Regulatory – Ensures that prices charged by patentees for patented
medicines sold in Canada are not “excessive”
o Reporting – Tracks pricing trends related to all medicines and the research
and development spending of patentees
• Classifies medicines based on level of therapeutic improvement
• Ex-factory price regulated – not consumer price
• PMPRB approval of price not required before sale
3

4. Drug Development
Health Canada
Review
Patented Medicine
Prices Review Board
Patient Access
Late 1980’s:
PMPRB was the
only organization
reviewing
drug prices
4

5. Drug Development
Health Canada
Review
Patented Medicine
Prices Review Board
Health Technology
Assessment
Hospitals
Patient Access
Private Drug PlansPublic Drug Plans
Today:
dozens of
organizations
review prices
5

6. PMPRB imposes a maximum non-excessive
price by applying tests: International
Comparisons
(7 comparator countries)
FRANCE UK
GERMANY ITALY
SWEDEN USA
SWITZERLAND
Level of Clinical
Improvement
Price Test
All Patented
Medicines
Breakthrough Median International Price
Prices of patented
medicines can never
exceed the
International Maximum
Price (i.e., the highest
price among the
PMPRB7 comparator
countries
Substantial
improvement
Higher of the Therapeutic
Class Comparison (TCC) and
the International Median
Moderate
improvement
Midpoint of the Therapeutic
Class Comparison and
International Median (but
not lower than the TCC)
No or little
improvement
Therapeutic Class
Comparison or Reasonable
Relationship Test
6

7. It also engages in active monitoring of the market…
• Price can never be higher than
highest international price
• Maximum allowable price set at
launch
• Annual price increases limited to
inflation
• Regular reporting requirements by
manufacturers
• Four market segments - retail,
distributor, pharmacy and hospital
7

8. … and enforcement for non-compliance
• Investigations
• Non-compliance options
o Board orders
o Voluntary compliance
undertakings (VCUs)
• Federal court
8

9. 9
Finalchangestothe Patented MedicinesRegulations– very few
substantive differencescompared toDecember2017proposals
• Released Friday, August 9, 2019
• Coming-into-force: July 1, 2020

10. 10
Modified listofcomparator countries
• All medicines, including those currently marketed in Canada, will be subject
to the new basket (the PMPRB11) as of July 1, 2020
• The December 2017 draft regulations proposed the addition of South Korea
• The new basket – if PMPRB applied the Median International Price
Comparison test – would still bring Canada’s public list price to below the
OECD median (~20% reduction)
Previous list (PMPRB7): New list (PMPRB11)
FRANCE UK
GERMANY ITALY
SWEDEN
USA
SWITZERLAND
FRANCE UK
GERMANY ITALY
SWEDEN AUSTRALIA
BELGIUM
JAPAN
NORWAY
NETHERLANDS
SPAIN

11. 11
New economic criteria
• Mandatory application of new
economic criteria in determining
maximum non-excessive prices for
new medicines, specifically:
o Pharmacoeconomic value (cost per QALY
/ affordability thresholds)
o Market size in Canada
o Gross domestic product & GDP/capita
• Will only apply to medicines that are
assigned a drug identification
number (DIN) after August 21, 2019

12. 12
New reporting obligations
• Net prices (after rebates under product listing agreements,
free goods, etc.)
• Forecasts of “estimated maximum use” of medicines within
30 days of first sale (for all meds sold in Canada after July 1,
2017)
• Additional reporting requirements for “high-cost” medicines:
o “High cost” = medicines that cost 50 percent of Canada’s annual
GDP per capita (approx. $24K/yr)
o Requirement to report all published cost-utility analyses
prepared by publicly-funded organization (i.e., cost-per-QALY)

13. 13
Estimated savings for Canadians over 10 years
• $8.8 billion net present value or $13.2 billion over 10 years
on patented drug costs
• Nearly identical to the December 2017 budget impact
analysis of $8.6 billion over 10 years
o FACT: the model used by Health Canada to estimate the impact
grossly understated the levels of price reduction being considered for
implementation by PMPRB

14. 14
Fact sheet and Regulatory Impact Analysis Statement
• Claims that the amendments will not reduce or delay access to
new innovative medicines or reduce R&D investment; Offers
correlative evidence that ”peer” countries have faster access and
greater levels of investment despite lower prices
o FACT: there is a demonstrated relationship between lower
regulated prices for pharmaceuticals and lower levels of R&D
and access

15. 15
Fact sheet and Regulatory Impact Analysis Statement
• Claims that these changes are similar to or drawn directly from
those in place in other countries, including those with large
pharmaceutical industries
o FACT: no other jurisdiction uses economic factors to regulate
the sale price for all patented medicines in the entire market
o It is not appropriate for a quasi-judicial federal regulatory body
like the PMPRB, with no connection to reimbursement of
medicines, to apply new criteria to regulate affordability,
which falls within the purview of provincial governments, other
payers and individuals

16. Potential Impact of PMPRB Price Reforms
on Product Launches in Canada
Neil Palmer
September 2019
©2019 PDCI Market Access 16

17. How Manufacturers Set Prices
• Global pricing teams oversee price setting and launch sequencing
– Establish pricing corridor – international prices expected to fit within corridor
• Expected prices are estimated for each market
– Applying pricing rules that are expected to be in place at time of regulatory
approval
– Input from local affiliates (using Canada as an example)
• Pricing allowed by PMPRB
• Likely recommendations of CADTH, INESSS
• Likely reimbursement status with public and private plans
• Likely net effective price to be negotiated with pCPA
• Timing of all of the above
• Based on the inputs from each county, the global pricing team establishes
the international launch sequence driven primarily, but not exclusively, by
price achievable in each market
• Uncertainty can be major constraint in authorizing a launch
17©2019 PDCI Market Access

18. Manufacturers optimize launch sequencing primarily based on price
18©2019 PDCI Market Access

19. Canada is currently a 1st or 2nd wave launch country
19©2019 PDCI Market Access
Source: Cutting Edge Information, 2016

20. Overview of new Guidelines framework
Complex and continues to evolve
20
11
Reformed PMPRB Guidelines
Median
This framework (likely) applies to “new” medicines only
Existing medicines will be reviewed against the PMPRB-11.
©2019 PDCI Market Access

21. 21
3.21
1.06
1.00 0.97 0.94 0.94 0.92 0.89 0.88 0.86 0.85 0.84 0.83 0.83 0.81 0.81 0.80 0.80 0.79 0.79 0.78 0.76 0.74 0.72 0.71 0.70 0.69 0.69 0.67 0.67
0.54
0.34
USA
Sw
itzerlandCanadaGerm
any
New
ZealandM
exico
Japan
ChileAustriaSw
eden
ItalyFinland
UKIreland
OECD
M
EDIAN*Hungary
Spain
NetherlandsBelgium
Luxem
bourgNorw
ayFranceAustraliaPortugalSlovakia
Czech
RepublicPolandGreeceEstoniaSlovenia
South
KoreaTurkey
Average Foreign-to-Canadian Price Ratios, Patented Medicines, OECD, 2017 Illustrative only - impact will vary by individual medicine. The
estimated average impact is based on assumptions in the RIAS
and previously proposed PMPRB methodologies – analysis &
illustration will need to be updated once draft Guidelines are
available from PMPRB.
Current comparator
New comparator
Canadian regulated public prices drop
by 20% - TO THE NEW PMPRB11 INTL
MEDIAN
Further reductions of net (confidential)
prices bring total regulated reductions to as
much as 50% off Intl median price
Potential Impact of PMPRB Regulations / Guidelines
PMPRB 11 median = 0.80
Source: From Figure 21 of the PMPRB Annual Report 2017; MIDASTM database, 2017, IQVIA.

22. Potential PMPRB Guidelines for New Medicines
22©2019 PDCI Market Access
Median International Price
(PMPRB-11)
New Economic
Factors
Median Therapeutic
Class Test
Health Economics
$/QALY
Market Size
(> $20M / Yr)
Up to
50% ↓
from Median
Intl Price
Rebated Price of
Brand Comparators
Generic Prices of
Comparators
High Priority Low Priority
20% ↓
list prices
Illustrative only - impact will vary by individual medicine. The estimated average impact is based on assumptions in the RIAS and previously proposed PMPRB
methodologies – analysis & illustration will need to be updated once draft Guidelines are available from PMPRB.

23. Range of Potential Impact on Drug Expenditures
• The RIAS Cost benefit analysis baseline scenario is that the impact will be
reduction of $8.8Billion over tens years.
• The upper estimate is $36.7B, more than 4 times higher
• There will be greater clarity once the draft guidelines are released (mid
September)
23©2019 PDCI Market Access

24. Impact on Drugs for Rare Diseases
• Base case scenario assume an average 33.4% price reduction for expensive
drugs for rare diseases (EDRD)
24©2019 PDCI Market Access

25. Canada risks moving to 3rd or 4th wave of product launches
25©2019 PDCI Market Access
Source: Cutting Edge Information, 2016

26. Impact of External Reference Pricing (2017)
• Systematic Literature Review of Impact of External Reference Pricing (ERP)
• Trends observed from ERP:
– health expenditures can be reduced at least in the short-term because prices
are more likely to decrease
– availability of pharmaceuticals, equitable access to medicines and the
stimulation of industrial policy can be undermined
– the impact of ERP on the affordability of medicines is ambiguous.
• ERP can trigger spillover effects, resulting in
– price instability, leading to launch delays
– unwillingness of manufactures to launch in low price countries,
– promoting price convergence towards the international average
26©2019 PDCI Market Access

27. Impact of PMPRB Reforms on Drugs for Rare Diseases
• The PMPRB11 median may be less impactful for drugs for rare diseases and high cost
drugs
– Strict adherence to international pricing corridors
• $/QALY and “market size” likely to be the most impactful for rare disease drugs
– Rare disease drugs almost always significantly exceed traditional cost effectiveness thresholds
- in all markets
– Rare disease drugs would require a 33% reduction in price on average (base case scenario)
• Overall impact is that the average transaction price of rare disease drugs will be forced
significantly lower: between the lowest and median international price
• Significantly lower prices (and pricing uncertainty) will likely result in launch delays in
Canada
• Clinical trials will be impacted (unlikely that manufacturers will conduct clinical trials in
countries they don’t expect to enter)
• Patient support programs for new drugs could be at risk
27©2019 PDCI Market Access

28. Fact #1: PMPRB introducing new regulations and
guidelines
¤ MYTH #1:
New PMPRB regulations and guidelines are needed
to make medicines more affordable to Canadians
¤ REALITY #1:
- Mandate of the PMPRB is to ensure drug prices to Canada
are not excessive (relative to similar countries and to similar
therapeutic products), not affordability.
- Affordability should be determined by the “purchaser” and
may reflect the value of the product (to the purchaser), the
resources that could be allocated to the product (from
competing interests), and alternatives available to the
purchaser.
28

29. Fact #2: PMPRB will impose price control measures that change the
reference “basket” of countries and introduce pharmacoeconomic
factors
¤ MYTH #2:
- Canadian prescription drug prices are too high.
- PMPRB new price control measures will bring Canada in
line with other OECD countries.
¤ REALITY #2:
- Canadian negotiated drug prices are currently
comparable to OECD international average/median.
- PMPRB regulations impose price control measures that
are not used by any other OECD country.
29

30. Fact #3: The impact of PMPRB Regulations on
access to new medicines has been considered
¤ MYTH #3:
- There is no evidence of a link between pricing, research & development (R&D),
and access to medicines.
- “… these changes need not and will not hamper the ability of Canadian [rare
disease] patients to participate in well-designed clinical trials”(physicians).
¤ REALITY #3:
- Countries, such as Australia, New Zealand, South Korea, Spain, and all of Latin
America that have considerably lower list prices, also have slower drug approval,
barriers to reimbursement, and poorer access to clinical trials or early market
launch.
- PMPRB calculations project list price reductions by 30% and up to 90%, which puts
Canada at considerable disadvantage. There has been no analysis conducted
on the proposed changes to evaluate its impact on patient access despite over
100 patient groups calling for this.
- Companies developing drugs for rare diseases have openly expressed concerns
30
Drug-price reforms need not affect research on rare
diseases – or the Canadians who have them
CHERYL ROCKMAN-GREENBERG, ALEX MACKENZIE AND SANDRA SIRRS
August 26, 2019

31. Fact #4: Open and transparent consultations
¤ MYTH #4:
- The PMPRB is committed to an open and transparent
dialogue with Canadians on the new regulatory changes
and related Guideline changes.
- The PMPRB has been consulting with Canadians since 2016
on reform of its regulatory framework with the publication
of the PMPRB Guidelines Modernization – Discussion Paper
– June 2016.
¤ REALITY #4:
- No meaningful consultations have been held.
- Health Canada’s reference to the Dodge Report (which
was never released to the public) is an example of
selective reading.
31

32. 32
Discussion / Q&A

33. Thank you!
For more information on CORD, please visit:
www.raredisorders.ca
33