Divino Victoria, DeKoven Mitch, Kleinrock Michael, Wade Rolin L, Kim Tony, Kaura Satyin
IMS Health, 8280 Willow Oaks Corporate Drive, Suite 775, Fairfax, VA, 22031, USA.
IMS Institute for Healthcare Informatics, One IMS Drive, Plymouth Meeting, PA, 19462, USA.
Orphanet J Rare Dis. 2016 May 21;11(1):68. doi: 10.1186/s13023-016-0450-y.
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). 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.
Orphan 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).
Between 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.
While 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.
加拿大卫生部将罕见病定义为危及生命、严重致残或影响极少数患者（约每2000人中1例）的严重慢性病。据估计，9%的加拿大人患有罕见病。治疗罕见病的药物（DRDs）也被称为孤儿药。虽然加拿大目前正在制定孤儿药框架，但在美国，1983年的《孤儿药法案》（ODA）为孤儿药的开发设立了激励措施。本研究衡量了加拿大2007 - 2013年孤儿药的年度总支出，并估计了未来（2014 - 2018年）的孤儿药支出。
美国食品药品监督管理局（FDA）批准的美国孤儿药被用作加拿大孤儿药情况的替代指标。确定了1983年至2013年期间FDA批准的品牌孤儿药（N = 356种独特产品）。分析仅纳入在加拿大获批有相同孤儿适应症的美国孤儿药。对于具有孤儿和非孤儿适应症的产品，通过适应症因子调整，利用可用数据源分离出孤儿适应症的销售额。利用艾美仕市场研究公司（IMS Health）经审计的生物制药销售MIDAS数据库来衡量孤儿药总支出，该支出按2007 - 2013年逐年计算，并评估为年度药品总支出的比例（调整为2014年加元）。
2007年至2013年期间，对最终确定的N = 147种孤儿药进行了支出衡量。2007年孤儿药支出总计6.102亿加元，2013年为11亿加元，分别占2007 - 2013年加拿大药品总支出的3.3% - 5.6%。未来趋势分析表明，2014 - 2018年孤儿药支出将保持在总支出的6%以下。
虽然可用孤儿药数量和相关支出随时间增加，但可及性仍是一个问题，并且从社会和公平的角度来看，相对于加拿大患罕见病的患者数量，孤儿药的总体支出较低。相对于药品总支出，孤儿药的总体预算影响较小且相当稳定。关于孤儿药支出增长可能导致药品支出不可持续的担忧似乎没有依据。
