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Abstract 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 Information was obtained from a variety of published and unpublished sources, including interviews with public policy and pharmaceutical experts in each country.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.
They are like children who have no parents, and they require special effort.-Congressman Henry Waxman
Rare diseases are estimated to affect up to twenty million people in the United States alone and countless others worldwide (U.S. House 1995a). Approximately 5,000 rare diseases are known to exist, with hereditary conditions comprising 80 percent of these diseases (Baudrihaye 1994). Included among rare diseases are HIV-related conditions such as Pneumocystis carinii pneumonia (PCP), cystic fibrosis, hairy cell leukemia, ovarian cancer, tuberculosis, and end-stage renal disease. The designation of drugs that treat rare diseases as "orphan drugs" is somewhat ambiguous. Criteria for the determination of orphan drug status vary by country, but the commonly accepted standard for a drug to be deemed orphan is that it is used to treat a disease that affects a population of approximately 650 to 1,000 per million (Baudrihaye 1994).
Despite the enormous number of rare diseases, incentives for pharmaceutical companies to invest time, effort, and resources into the development of drugs to treat such limited populations are few compared to developing drugs to treat more prevalent diseases. The development of pharmaceuticals for rare diseases (drugs termed orphans) can thus be a precarious venture that companies hesitate to pursue. Since orphan drugs are theoretically developed for the treatment of rare disorders, most of them have a narrow clinical scope and limited economic prospects. These features...