Gilead Science's remdesivir, an antiviral medicine being tested for treatment of COVID-19, would get a seven-year monopoly if approved by the Food and Drug Administration.
Changes in the way the Food and Drug Administration reviews new medicines means that there are more cures and treatments on the market. But there's also less proof the drugs are safe and effective.
Food and Drug Commissioner Scott Gottlieb says he wants to know why drugmakers churn out medicines for some rare diseases but not others.
The Republican tax plan could reduce or eliminate federal tax credits that reward companies for developing drugs to treat rare diseases. People who benefit from the medicines have objected.
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.
There's a genetic test for ALD, the inherited disorder portrayed in the movie Lorenzo's Oil, and the federal government recommends it for all newborns. But only a handful of states offer it routinely.
A federal drug program blocks rural hospitals from getting discounts on rare-disease drugs, forcing staff to cut back on supplies of lifesaving medicines.
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.
The Orphan Drug Act was created to help patients with rare diseases get life-saving medications. But soaring prices suggest the law is being manipulated to increase profits.