Abstract
BackgroundHealth Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians suffer from a rare disease. Drugs treating rare diseases (DRDs) are also known as orphan drugs. While Canada is currently developing an orphan drug framework, in the United States (US), the Orphan Drug Act (ODA) of 1983 established incentives for the development of orphan drugs.This study measured total annual expenditure of orphan drugs in Canada (2007–13) and estimated future (2014–18) orphan drug expenditure.MethodsOrphan drugs approved by the US Food and Drug Administration (FDA) in the US were used as a proxy for the orphan drug landscape in Canada. Branded, orphan drugs approved by the FDA between 1983 through 2013 were identified (N = 356 unique products). Only US orphan drugs with the same orphan indication(s) approved in Canada were included in the analysis. Adjustment via an indication factoring was applied to products with both orphan and non-orphan indications using available data sources to isolate orphan-indication sales. The IMS Health MIDAS database of audited biopharmaceutical sales was utilized to measure total orphan drug expenditure, calculated annually from 2007–2013 and evaluated as a proportion of total annual pharmaceutical drug expenditure (adjusted to 2014 CAD).ResultsBetween 2007 and 2013, expenditure was measured for a final N = 147 orphan drugs. Orphan drug expenditure totaled $610.2 million (M) in 2007 and $1,100.0 M in 2013, representing 3.3– 5.6 % of total Canadian pharmaceutical drug expenditure in 2007–2013, respectively. Future trend analysis suggests orphan drug expenditure will remain under 6 % of total expenditure in 2014–18.ConclusionsWhile the number of available orphan drugs and associated expenditure increased over time, access remains an issue, and from the perspectives of society and equity, overall spending on orphan drugs is lower relative to the number of patients affected with an orphan disease in Canada. The overall budget impact of orphan drugs is small and fairly stable relative to total pharmaceutical expenditure. Concerns that growth in orphan drug expenditure may lead to unsustainable drug expenditure do not appear to be justified.
Highlights
Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons)
Of the 356 branded products identified from the Food and Drug Administration (FDA) website, MIDAS captured sales for 147 unique orphan drugs in Canada between 2007 and 2013, of which, 39 (26.5 %) were partial orphan drugs
Reasons for sales data not being available include the drug not having been approved in Canada, the orphan indication in the United States (US) not being approved in Canada, funding for the drug being unavailable in Canada, the product no longer being marketed in Canada or other reasons, such as MIDAS coverage limitations
Summary
Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). While Canada is currently developing an orphan drug framework, in the United States (US), the Orphan Drug Act (ODA) of 1983 established incentives for the development of orphan drugs. Health Canada has defined a rare disease as a lifethreatening, seriously debilitating, or serious and chronic condition affecting a relatively small number of patients: 1 in 2,000 persons [1]. Almost all developed countries have a regulatory framework for orphan drugs, i.e., legislative policy or regulations that set the criteria and requirements for orphan drug designation, market authorization, post-marketing monitoring and financial incentives, etc. The United States (US) was the first nation to introduce orphan drug legislation with the Orphan Drug Act (ODA) of 1983 [4]
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