Congress set up the orphan drug designation to help bring drugs affecting small numbers of patients to market. To entice companies, the government allows drugs that meet the sought-after orphan designation to get a fee waiver, a tax credit for the drug’s trials, support from the Food and Drug Administration and seven years of market exclusivity. Many of the drugs are expensive, with some reaching $70,000 a month.
KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.
KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.Some elements may be removed from this article due to republishing restrictions. If you have questions about available photos or other content, please contact khnweb@kff.org.
