Orphan drug policies: implications for the United States, Canada, and developing countries.

The United States’ Orphan Drug Act (“ODA”) has been hailed as “one of the most successful US legislative actions in recent history”. Although many countries, and governing bodies, have enacted Orphan Drug legislation in the past decade, the United States (US) was the first country to implement an official policy, primarily because of public pressure due to the lack of treatment options for rare disease sufferers in the US. For everyone who has espoused the virtues of the ODA in encouraging research into rare disease treatments, there are equal numbers of detractors who claim that it is a policy that serves mainly to promote private industry by allowing companies to charge outrageous prices for products that rare disease sufferers have no option but to purchase. While these are wildly divergent opinions, the true value of the ODA likely resides somewhere in between.