Orphan Drug Launch Monitor (ODLM)
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Background and Approach
To stimulate drug development, a number of countries have adopted orphan drug regulatory frameworks. These include the United States (US) (1983), Singapore (1991), Japan (1993), Australia (1997), the European Union (EU) (1999/2000), Taiwan (2000) and South Korea (2003).
The Orphan Drug Launch Monitor analyzes the international approval of designated orphan drugs and assesses their availability in Canada. This analysis focuses on a number of select orphan drugs reported at the active-substance level and identified based on the following criteria:
- drugs that received orphan designation and approval in the US and/or EU over the last decade (2005–2014); and
- drugs that only have orphan indications.
The international market analyzed includes Canada and the seven PMPRB comparator countries (PMPRB7): France, Germany, Italy, Sweden, Switzerland, the United Kingdom (UK) and the US. The results report on sales and prices for 2014.
Based on the selection criteria, 134 orphan drugs were identified, accounting for 2.9% of the international pharmaceutical sales in 2014.
Methods
The analysis used the US Federal Drug Administration (FDA) Orphan Drug Product Designation Database and the European Community Register of Orphan Medicinal Products to identify the list of orphan drugs.
The IMS Health MIDAS™ Database was used to report on drug availability, market utilization, sales and pricing for the select orphan drugs. While this database is comprehensive, sales information for products with a low utilization may not always be available. This limitation impacts the analysis and may underestimate the sales of the select orphan drugs, especially in smaller markets. The drug availability for Canada was further validated based on other data sources, including the Health Canada Notice of Compliance Database.
The analysis does not assess the timing of market availability or the extent of the utilization across markets.
Key Findings
1. More drugs are designated and approved for orphan indications in the US than in the EU
The majority of the select orphan drugs were granted orphan designations and market approval only in the US (54%; n = 72); a sizable proportion were approved in both the US and the EU (36%; n = 49); and a small proportion of drugs were only approved in the EU (10%; n = 13).
In terms of sales, drugs that were granted orphan designations in both the US and the EU dominate the market (60%), given the broad international approval. Drugs granted orphan designations only in the US account for a sizable share of the international sales (39%); while drugs granted orphan designations only in the EU have negligible sales (1%) given their limited international use, especially in the US.
Section 1
Click on image for larger view
Figure description
These two pie charts give (1) the share of orphan drugs analyzed and (2) their respective share of sales for Canada and the PMPRB7 in 2014 according to the source of market approval: in both the United States and the European Union; only in the European Union; and only in the United States.
The left-hand pie chart depicts the share of drugs.
Both the United States and the European Union: 36% of the orphan drugs analyzed; European Union only: 10%; United States only: 54%.
The right-hand pie chart depicts the share of sales, which includes Canada and the PMPRB7 countries: France, Germany, Italy, Sweden, Switzerland, the United Kingdom and the United States.
Both the United States and the European Union: 60% of sales; European Union only: 1%; United States only: 39%.
2. Many orphan drugs are approved for use in Canada
While Canada currently does not have an orphan drug regulatory framework in place, 57% of the select orphan drugs received market approval by the end of 2014 through the regular submission processes. These drugs accounted for 89% of international sales, indicating that they have a relatively high utilization and/or cost. While Canadians are also able to access some of the unavailable/ unapproved orphan drugs through Health Canada’s Special Access Programme (SAP), these drugs are not captured in the results.
The availability and respective international sales for the select orphan drugs in Canada are in line with those in European markets, where the drug availability is in the range of 38% to 64% and the corresponding sales are in the range of 67% to 92%. Given that
the sales information for products with low utilization may not always be available, the graph also reports the additional EU-approved orphan drugs without sales in the respective European countries.
The US market has the greatest availability and sales coverage for the select orphan drugs, with 91% of them approved by the FDA, accounting for 99% of the sales.
Section 2
Click on image for larger view
Figure description
This bar graph and accompanying table depict the availability and sales of the 134 orphan drugs selected for analysis for Canada and the PMPRB7 in 2014.
|
Canada |
France |
Germany |
Italy |
Sweden |
Switzerland |
United Kingdom |
United States |
| Number of drugs with available sales |
76 |
75 |
86 |
70 |
76 |
51 |
80 |
122 |
| Share of drugs with available sales |
57% |
56% |
64% |
52% |
57% |
38% |
60% |
91% |
| Share of international† sales |
89% |
89% |
92% |
87% |
88% |
67% |
92% |
99% |
| Additional European Union approved drugs with no available sales in the respective country |
|
17 |
7 |
19 |
15 |
35 |
13 |
|
| Population (millions) |
36 |
65 |
83 |
61 |
10 |
8 |
63 |
323 |
The sales and drug availability by country was determined based on the IMS Health MIDAS™ Database. The drug availability information was supplemented with information from the following sources: US FDA Orphan Drug Product Designation Database; the European Community Register of Orphan Medicinal Products; and Canadian data sources, including the Health Canada Notice of Compliance Database. The sales reported include Canada and the PMPRB7 countries.
3. Canada approved 9 of the top 10 select orphan drugs
In 2014, the 10 top-selling orphan drug products accounted for 50% of the orphan-only drug market. These were predominantly oncology and/or biologic drug products. Only one of the drugs was not available in Canada as of the end of 2014: coagulation factor VIIa (recombinant) – used to treat and prevent bleeding episodes in patients with hemophilia A or B.
The median foreign prices for most of these drugs are at the same level or higher than Canadian prices, except for bendamustine hydrochloride (Treanda), which has a notably higher price in Canada.
Ten top-selling† select orphan drugs, Canada and the PMPRB7, 2014
| Active substance (Trade name) |
Share of international sales for orphan drugs* |
Form and strength |
Canadian price |
Median foreign price** |
| Lenalidomide (Revlimid)O,M |
9.0% |
Capsule, 10 mg |
Not available |
$387 |
| Ipilimumab (Yervoy)O,B |
7.1% |
Vial, 5 mg/mL |
$9,256 |
$12,653 |
| Dasatinib (Sprycel)O,M |
6.4% |
Tablet, 100 mg |
$147 |
$196 |
| Nilotinib (Tasigna)O |
5.9% |
Capsule, 150 mg |
$28 |
$37 |
| Bendamustine hydrochloride (Treanda)O,M |
5.2% |
Vial, 100 mg/vial |
$1,212 |
$439 |
| Coagulation factor VIIa (recombinant) (Novoseven)B,M |
3.7% |
Vial, 5 mg/mL |
Not available in Canada |
$4,414 |
| Eculizumab (Soliris)B,M |
3.6% |
Vial, 10 mg/mL |
Not available |
$6,248 |
| Azacitidine (Vidaza)O,M |
3.3% |
Vial, 100 mg/vial |
Not available |
$511 |
| Deferasirox (Exjade)M |
3.0% |
Tablet, 500 mg |
$43 |
$39 |
| Ibrutinib (Imbruvica)O,M |
2.9% |
Capsule, 140 mg |
$92 |
$97 |
| Total |
50% |
|
|
|
O, oncology; B, biologic; M, multiple orphan indications.
* The share of sales reported are for the markets analyzed, including Canada and the PMPRB7 countries: France, Germany, Italy, Sweden, Switzerland, the UK and the US.
** The foreign price is the median across the PMPRB7 countries.
4. Orphan drugs not available in Canada represent a small share of the international market
While the select orphan drugs that are not available in Canada account for a sizable share of the drugs in this market (43%, n = 58), they only account for a small share of the international sales (11%). However, some of them (n = 21) have been accessed in Canada through the Special Access Programme (SAP), while others (n = 9) were approved in Canada in 2015, given the usual lags in new drug product launches. It is important to note that alternative treatment options may be available for some orphan drugs not available in Canada.
Sales for the drugs that are not available in Canada are highly concentrated, with the top 10 accounting for a considerable sales volume (9.4%). The table reports the top 10 select orphan drugs not available in Canada. Most of these drugs are only available in the US, while two are approved in the EU. Half of these drugs are biologic therapies, and only two are for cancer treatment. Generally these drugs come at a very high price tag.
Ten top-selling† select orphan drugs not available in Canada, Canada and the PMPRB7, 2014
| Active substance |
ATC* description |
Share of international sales for orphan drugs** |
Orphan designation |
US/EU market approval year |
Median foreign price*** |
| Coagulation factor VIIa (recombinant)B,M |
Blood and blood forming organs |
3.7% |
US |
2014 |
$4,414 (vial, 5 mg/mL) |
| CarfilzomibO |
Antineoplastic and immunomodulating agents |
2.3% |
US |
2012 |
Not available |
| DecitabineO,M |
Antineoplastic and immunomodulating agents |
1.6% |
US and EU |
2006/2012 |
$1,627 (vial, 50 mg/vial) |
| Prothrombin Complex Concentrate (Human)B |
Blood and blood forming organs |
0.4% |
US |
2013 |
Not available |
| Hydroxyprogesterone caproate |
Genito-urinary system and sex hormones |
0.3% |
US |
2011 |
Not available |
| Benzoate/Phenylacetate |
Various |
0.3% |
US |
2005 |
Not available |
| BelataceptB |
Antineoplastic and immunomodulating agents |
0.2% |
US |
2011 |
$676 (vial, 250 mg/vial) |
| EcallantideB |
Blood and blood forming organs |
0.2% |
US |
2009 |
Not available |
| Ziconotide |
Nervous system |
0.2% |
EU |
2005 |
$798 (vial, 100 mcg/mL) |
| PegloticaseB |
Musculo-skeletal system |
0.2% |
US |
2010 |
Not available |
| |
Total |
9.4% |
|
|
|
O, oncology; B, biologic; M, multiple orphan indications.
* World Health Organization Anatomical Therapeutic Classification.
** The share of sales reported includes markets in Canada and the PMPRB7: France, Germany, Italy, Sweden, Switzerland, the UK and the US.
*** The foreign price is the median across PMPRB7 countries.
Drugs accessed in Canada through the Special Access Programme (SAP): decitabine, hydroxyprogesterone caproate, sodium benzoate/sodium phenylacetate, belatacept, and pegloticase; drugs approved in 2015: carfilzomib.
Data Source: †MIDASTM Database, Manufacturer Ex-factory Sales, IMS AG. All Rights Reserved.
Population information was obtained from Worldometers.
Disclaimer: Although based in part on data provided under license by the IMS AG’s MIDAS™ Database, the statements, findings, conclusions, views and opinions
expressed in this report are exclusively those of the PMPRB and are not attributable to IMS AG.
NPDUIS is a research initiative that operates independently of the regulatory activities of the PMPRB.