CADTH 2015 Symposium

1. 1
The Budget Impact of Drugs Treating Rare Diseases in
Canada: A 2007-2013 MIDAS Sales Data Analysis
April 14, 2015
Oral Presentation at the 2015 CADTH Symposium; Concurrent Session D1, 10:15 – 11:45
Victoria Divino1, Mitch DeKoven, MHSA1, Tony Kim, MA2, Michael Kleinrock, MA1, Rolin L. Wade, RPh,MS1 and
Satyin Kaura, MSci, MBA3
1IMS Health, Fairfax, VA USA; 2Celgene, Mississauga, ON, Canada; 3Celgene, Summit, NJ USA

2. 2
Study Background
• Orphan drugs treat rare diseases that affect a very small
number of persons (~5 in 10,000 persons). An estimated 1
in 12 Canadians, approximately 3M or 9% of Canadians
suffer from a rare disease.1
• There is currently no official “orphan disease” status in
Canada; Health Canada announced in 2012 the development
of a modern orphan drug framework for Canada.
• Orphan drug policies in other countries have dramatically
increased the development and approval of orphan drugs,
such as the Orphan Drug Act (ODA) of 1983 in the US
• Little evidence/data has been generated as to the actual
burden of orphan drug expenditures in Canada.
Background
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
1. http://www.rarediseasefoundation.org/about

3. 3
Study Objectives
To measure the total annual expenditures on branded
orphan drugs between 2007-2013 in Canada. The specific
objectives were:
• To estimate total orphan drug expenditures in Canada
from 2007-2013 and to evaluate total orphan drug
expenditures as a proportion of total pharmaceutical
expenditures.
• To evaluate future orphan drug expenditures (2014-
2018) and as a proportion of total future
pharmaceutical expenditures.
Objectives
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

4. 4
Methods
• Branded drugs approved by the FDA and granted orphan
status between 1983 and 2013 in the US (N=356) were
used as a proxy for the orphan drug landscape in Canada.
82% of the 356 were “orphan only” and 18% “partial
orphans.”
• The analysis only considered those products which received
approval in Canada, and for which the US orphan
indication(s) was also approved.
• The IMS Health MIDAS database of audited
biopharmaceutical sales volume was used to assess 2007-
2013 orphan drug and total drug expenditures in Canada.
• Expenditures for partial orphans (both orphan and non-
orphan indications) were adjusted. In-depth research was
conducted to determine a ‘disease factor’ to apply to
consider orphan indication sales only.
Methods
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

5. 5
Analyses
• Total expenditures for the branded orphan drugs, captured
and measured in the MIDAS Canadian dataset, were
calculated annually from 2007-2013.
• Orphan drug expenditures were further evaluated as a
proportion of total annual 2007-2013 Canadian
pharmaceutical drug expenditures sourced from MIDAS.
• Expenditures were recorded in Canadian Dollars (CAD) at
the time of the sale, and adjusted to 2014 CAD using the
Canadian Consumer Price Index.
• A future trend analysis estimated orphan drug sales from
2014-2018, and evaluated sales out of projected future total
drug sales from IMS Health Market Prognosis forecasts.
Analyses
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

6. 6
Results
“Orphan Only” drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations.
“Partial Orphan” drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures.
Total Orphan drugs = (“Orphan Only” drugs + adjusted “Partial Orphan” drugs)
Table 1. 2007-2013 Orphan Drugs Captured in IMS database
2007 2008 2009 2010 2011 2012 2013
# “Orphan only”
drugs captured
70 74 73 79 81 91 99
# “Partial Orphan”
drugs captured
25 26 27 28 30 31 34
# Total Orphan drugs
captured
95 100 100 107 111 122 133
2007-2013 Orphan Drug Landscape in Canada
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

7. 7
Results
Measure 2007 2008 2009 2010 2011 2012 2013
“Orphan Only” Drug Expenditures
($M)
537.6 586.7 649.2 692.6 729.3 786.8 868.0
“Partial Orphan” Drug Expenditures
($M)
72.6 82.5 94.5 125.5 151.2 202.8 232.0
Total Orphan Drug Expenditures ($M) 610.2 669.2 743.7 818.1 880.5 989.6 1,100.0
% “Orphan Only”/ Total Orphan 88.1% 87.7% 87.3% 84.7% 82.8% 79.5% 78.9%
% “Partial Orphan”/ Total Orphan 11.9% 12.3% 12.7% 15.3% 17.2% 20.5% 21.1%
Total pharmaceutical expenditure ($M) 18,233.6 19,598.1 20,514.4 20,628.6 19,976.3 19,746.0 19,665.7
% “Orphan Only”/ Total
pharmaceutical expenditure
2.9% 3.0% 3.2% 3.4% 3.6% 4.0% 4.4%
% “Partial Orphan”/ Total
pharmaceutical expenditure
0.4% 0.4% 0.4% 0.6% 0.8% 1.0% 1.2%
% Total Orphan/ Total pharmaceutical
expenditure
3.3% 3.4% 3.6% 4.0% 4.4% 5.0% 5.6%
“Orphan Only” drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations.
“Partial Orphan” drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures.
Total Orphan drugs = (“Orphan Only” drugs + adjusted “Partial Orphan” drugs)
Table 2. 2007-2013 “Orphan Only”, “Partial Only” and Total Orphan Drug
Expenditures (2014 CAD)
2007-2013 Orphan Drug Expenditures
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

8. 8
Results
Measure 2007 2008 2009 2010 2011 2012 2013
“Orphan Only” Drug Expenditures
($M)
537.6 586.7 649.2 692.6 729.3 786.8 868.0
“Partial Orphan” Drug Expenditures
($M)
72.6 82.5 94.5 125.5 151.2 202.8 232.0
Total Orphan Drug Expenditures ($M) 610.2 669.2 743.7 818.1 880.5 989.6 1,100.0
% “Orphan Only”/ Total Orphan 88.1% 87.7% 87.3% 84.7% 82.8% 79.5% 78.9%
% “Partial Orphan”/ Total Orphan 11.9% 12.3% 12.7% 15.3% 17.2% 20.5% 21.1%
Total pharmaceutical expenditure ($M) 18,233.6 19,598.1 20,514.4 20,628.6 19,976.3 19,746.0 19,665.7
% “Orphan Only”/ Total
pharmaceutical expenditure
2.9% 3.0% 3.2% 3.4% 3.6% 4.0% 4.4%
% “Partial Orphan”/ Total
pharmaceutical expenditure
0.4% 0.4% 0.4% 0.6% 0.8% 1.0% 1.2%
% Total Orphan/ Total pharmaceutical
expenditure
3.3% 3.4% 3.6% 4.0% 4.4% 5.0% 5.6%
“Orphan Only” drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations.
“Partial Orphan” drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures.
Total Orphan drugs = (“Orphan Only” drugs + adjusted “Partial Orphan” drugs)
Table 2. 2007-2013 “Orphan Only”, “Partial Only” and Total Orphan Drug
Expenditures (2014 CAD)
2007-2013 Orphan Drug Expenditures
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

9. 9
Results
96.7%
96.6% 96.4% 96.0% 95.6% 95.0% 94.4%
2.9%
3.0%
3.2%
0.6%
0.8%
4.0% 4.4%0.4%
0.4%
0.4%
3.4%
3.6%
1.0% 1.2%
0
4E+09
8E+09
1.2E+10
1.6E+10
2E+10
2007 2008 2009 2010 2011 2012 2013
Partial
Orphan
(Adjusted)
Orphan Only
Non-Orphan
(Adjusted)
$18.2B $19.6B $20.5B $20.6B $19.7B $19.7B
Figure 1. 2007-2013 Orphan Drug Expenditures Out Of Total Canadian Drug
Expenditures (2014 CAD)
“Orphan Only” drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations.
“Partial Orphan” drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures.
Total Orphan drugs = (“Orphan Only” drugs + adjusted “Partial Orphan” drugs)
Total
Pharmaceutical
Expenditure
‘
‘
’
’
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
$20.0B
$16.0B
$12.0B
$8.0B
$4.0B
$0
$20.0B

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Results
Oncology was the most common therapeutic class, with approx. 40%
of orphan drugs identified with an orphan oncology indication
37.3% 36.7%
36.9%
37.9%
39.7%
40.0%
43.2%
62.7%
63.3%
63.1%
62.1%
60.3%
60.0%
56.8%
0
200,000,000
400,000,000
600,000,000
800,000,000
1,000,000,000
1,200,000,000
2007 2008 2009 2010 2011 2012 2013
All Other
Oncology-
Related
$1,200.0M
$1,000.0M
$800.0M
$600.0M
$400.0M
$200.0M
$0
$610.2M $669.2M $743.7M $818.1M $989.6M $1,100.0M$880.5M
Figure 2. 2007-2013 Orphan Drug Expenditures By Therapeutic Class
(2014 CAD)
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
Total Orphan
Drug
Expenditure

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Results
Future trends (2014-2018)
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
A linear trend line was created applying 2007-2013 sales to
estimate orphan drug expenditures for 2014-2018.

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Results
Future trends (2014-2018)
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
Source: Total Canadian Pharmaceutical Sales estimated from MIDAS (2007-2013) & IMS Health Market Prognosis Forecast (2014-2018)
18,234
19,598
20,514 20,629 19,976 19,746 19,666
21,859
23,841
24,996
26,307
27,550
610 669 744 818 880 990 1,100 1,151 1,231 1,312 1,392 1,472
0
5,000
10,000
15,000
20,000
25,000
30,000
2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
Total Pharmaceutical Sales Total Orphan Drugs Sales
Total Sales (CAD millions)
Orphan drug expenditures for 2014-2018 are anticipated to
grow, but will remain below 6% as a proportion of total
Canadian pharmaceutical expenditures.

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Discussion
Discussion
• While a select orphan drugs may have high costs, the population
using these orphan drugs is small so total orphan drug
expenditures, when considered out of total drug expenditures, is
relatively small.
• For the analysis of “orphan only” drugs, expenditures
represented 2.9-4.4% of 2007-2013 total Canadian
pharmaceutical expenditure.
• Adjusted “partial orphan” drug sales, accounted for 0.4-1.2%
of 2007-2013 total Canadian pharmaceutical expenditure
• Total orphan drug expenditures represented 3.3-5.6% of total
Canadian drug expenditures, in 2007-2013.
• Future trend analysis suggests that while orphan drug
expenditures in 2014-2018 will increase, growth is slowing down
and will remain fairly stable as a proportion of total drug
expenditures (below 6%).
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

14. 14
Discussion (continued)
Discussion
• According to the Canadian Organization for Rare Disorders,
an estimated 1 in 12 Canadians, approximately 3M or 9% of
Canadians suffer from a rare disease.1
• Interestingly, expenditure on orphan drugs represents
less than 6% of total pharmaceutical expenditures
• Overall, these results are consistent with analyses conducted
in EU and US:
• EU: orphan drug expenditures represent 3-6% of total
pharmaceutical expenditures in 2007-2013.
• US: orphan drug expenditures represent 5-9% of total US
pharmaceutical expenditures.
• Future tend analysis suggest orphan drugs expenditures
will remain fairly stable as a proportion of total
pharmaceutical expenditures.
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium
1. http://www.rarediseasefoundation.org/about

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Limitations
• There are a couple of limitations to note with this analysis.
• This cost analysis does not highlight or recognize the
clinical value that orphan drugs provide to patients and
society.
• IMS MIDAS database coverage is limited with respect to
certain products and/or manufacturers.
• There is the potential for off-label use of orphan drugs.
Limitations
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

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Conclusions
• The US Orphan Drug Act (ODA) led to an increase in the
number of approved orphan drugs in the US, which led to an
associated increase in the number of drugs for rare diseases
in Canada.
• While associated orphan drug expenditures have increased,
these drugs benefit many people with previously underserved
rare conditions.
• The annual expenditures on orphan drugs are relatively small
(3.3-5.6%) and have remained fairly stable relative to total
pharmaceutical drug expenditures.
• Future trend analysis suggests growth is slowing down.
• Concerns that growth in orphan drug expenditures may lead
to unsustainable drug expenditures do not appear to be
justified.
Conclusions
Budget Impact of Drugs Treating Rare Diseases in Canada, Presentation at the 2015 CADTH Symposium

17. 17
Thank You