Check out all the drugs the FDA has approved to treat rare diseases. You can search by brand name, or by disease, and see familiar names that were first sold on the mass market or all the drugs that won FDA approval to treat more than one rare disease. Each approval gives the drugmaker seven years of exclusive rights to the market. Drugmakers are companies that sought orphan approval, not necessarily current drug owners. Some drugs appear in more than one tab.
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Approved Orphan Uses
We relied primarily on the FDA’s orphan drug designations and approvals database because it has the most detailed information about orphan drug approvals, disease indications and dates. We included only orphan designations that led to approvals, and cleaned up and standardized many of the drug names manually because they had either changed or had never been entered.
We also used the orphan drugs database to look for drugs approved for multiple orphan indications. Occasionally, a drug had more than one approval on the same day. Sometimes, they were months apart, and other times they were years apart.
Determining whether a drug was on the mass market before its orphan use was approved required pulling initial drug approval dates from additional FDA datasets, including the Drugs@FDA database as well as lists of biologic drugs and fractionated plasma products that didn’t appear in Drugs@FDA. If a drug had an initial approval date before its first marketing approval date in the orphan database, we put it in the mass market first category.
Our analysis does not include a list of drugs that were first approved for orphan indications and later received approval for mass market indications. Although the FDA tried to help us with this, we determined that data was not consistent enough for our project.
Our analysis does not examine the medical or cost effectiveness of these drugs.
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KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.