When Everyone Is an Orphan: Against Adopting a US-Styled Orphan Drug Policy in Canada

作者：Herder Matthew^*

来源：Accountability in Research, 2013, 20(4): 227-269.

DOI：10.1080/08989621.2013.793120

• health innovation policy
• orphan drug
• pharmaceutical industry
• rare disease

Putting aside whether diseases that affect only small numbers of people (rare diseases) should be prioritized over diseases that are otherwise orphaned, in this article I argue that a new approach to rare, orphan diseases is needed. The current model, first signaled by the United States%26apos; Orphan Drug Act and subsequently emulated by several other jurisdictions, relies on a set of open-ended criteria and market-based incentives in order to define and encourage drug therapies for rare, orphan diseases. Given a) the biopharmaceutical industries%26apos; growing interest in orphan diseases, b) progress in the sphere of personalized medicines enabling more and more common diseases to be reclassified as rare, and c) empirical evidence suggesting that the most orphan drugs target only a limited, lucrative subset of rare diseases, I argue that Canada, which recently announced plans to develop its own orphan drug framework should not follow the United States%26apos; orphan drug model.

• 出版日期2013-7-4