Orphan Drug Act

The Government Accountability Office found breakdowns in the way the Food and Drug Administration evaluates drugs for rare diseases. The analysis came after an investigation by Kaiser Health News.

Three decades ago, Congress set up a system to encourage drug companies to develop treatments for rare diseases. The law has worked, but at a high cost.

Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives by doing so. But nearly a third of the medicines aren't new or were repurposed many times for financial gain.