A CROSS-NATIONAL COMPARISON OF ORPHAN DRUG POLICIES - IMPLICATIONS FOR THE US ORPHAN DRUG-ACT

Six countries-Canada, France, Japan, Sweden, the United Kingdom, and t he United States-were studied to compare public policies affecting the development and marketing of pharmaceuticals for rare diseases (i.e., orphan drugs). Information was obtained from a variety of published a nd unpublished sources, including interviews with public policy and ph armaceutical experts in each country. This article presents different approaches to encouraging the development of orphan drugs while ensuri ng access by regulating their prices. Additionally, the article descri bes access to orphan drugs as promoted by special coverage for populat ion subgroups, disease categories, and/or specific drugs. Not all effo rts to increase access to orphan pharmaceuticals have been the result of government action, as illustrated by the proliferation of for-profi t organizations that specialize in orphan drugs. The many policy optio ns from other countries identified in this study are especially releva nt, given increasing calls for reform of the U.S. Orphan Drug Act.