Six countries—Canada, France, Japan, Sweden, the United Kingdom, and the 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 Information was obtained from a variety of published and unpublished sources, including interviews with public policy and pharmaceutical experts in each country. This article presents different approaches to encouraging the development of orphan drugs while ensuring access by regulating their prices. Additionally, the article describes access to orphan drugs as promoted by special coverage for population subgroups, disease categories, and/or specific drugs. Not all efforts to increase access to orphan pharmaceuticals have been the result of government action, as illustrated by the proliferation of for-profit organizations that specialize in orphan drugs. The many policy options from other countries identified in this study are especially relevant, given increasing calls for reform of the U.S. Orphan Drug Act.

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