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.