orphan drugs

Patients who depend upon special drugs to treat rare diseases are caught in the crossfire as drugmakers and the FDA battle over regulations that reward companies for developing treatments for relatively small pools of patients.

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.

For more than three decades, pharmaceutical companies have claimed a 50 percent tax credit for the cost of clinical trials of drugs for rare diseases. The credit is now in jeopardy.

Following an investigation by Kaiser Health News and NPR, the Food and Drug Administration has moved to close a loophole that lets drugmakers skip pediatric testing of drugs to treat rare diseases.

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.