Cadth 2015 d1 budget impact of drugs for treating rare diseases in canada cadth presentation 2015

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The Budget Impact of Drugs Treating Rare Diseases in Canada: A 2007-2013 MIDAS Sales Data AnalysisApril 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

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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

http://www.rarediseasefoundation.org/about

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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


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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


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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


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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


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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%



Table 2. 2007-2013 “Orphan Only”, “Partial Only” and Total Orphan Drug Expenditures (2014 CAD)

2007-2013 Orphan Drug Expenditures


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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%



Table 2. 2007-2013 “Orphan Only”, “Partial Only” and Total Orphan Drug Expenditures (2014 CAD)

2007-2013 Orphan Drug Expenditures


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Results

2007 2008 2009 2010 2011 2012 20130

4000000000

8000000000

12000000000

16000000000

20000000000

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%

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)



Total Pharmaceutical

Expenditure

‘

‘

’

’


$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

2007 2008 2009 2010 2011 2012 20130

200,000,000

400,000,000

600,000,000

800,000,000

1,000,000,000

1,200,000,000

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%

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)


Total Orphan Drug

Expenditure

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Results

Future trends (2014-2018)


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)


Source: Total Canadian Pharmaceutical Sales estimated from MIDAS (2007-2013) & IMS Health Market Prognosis Forecast (2014-2018)

2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 20180

5,000

10,000

15,000

20,000

25,000

30,000

18,23419,598 20,514 20,629 19,976 19,746 19,666

21,85923,841

24,99626,307

27,550

610 669 744 818 880 990 1,100 1,151 1,231 1,312 1,392 1,472

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%).


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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


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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


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Thank You

Cadth 2015 d1 budget impact of drugs for treating rare diseases in canada cadth presentation 2015

Health & Medicine

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