A federal incentive for research into rare diseases for so-called orphan drugs is beloved by pharmaceutical companies. And during Donald Trump’s first two years in office, the Food and Drug Administration has been authorizing them at an unprecedented rate, according to Axios.
Rare diseases are those that affect 200,000 or fewer people in the country, according to the FDA. There are 7,000 known rare diseases but only a few hundred have federally approved treatments. As a way to encourage pharmaceutical companies to meet the needs to patients, Congress passed the Orphan Drug Act in 1983. It allows the FDA to grant additional exclusive selling time. Being a monopoly during that time, manufacturers can keep prices higher.
But there’s been criticism that drug companies manipulate the program to maximize profits. Often the drugs already exist with a mass-market use. By getting orphan drug status, the companies get millions of dollars in government incentives and a seven-year monopoly on treating the rare disease.
A November 2018 government report criticized the FDA for failing to meet the intent of the Orphan Drug Act. The Axios analysis found that the rate at which drug companies gained orphan status for their drugs has skyrocketed during the Trump administration. The FDA has approved 174 drugs under the program during Trump’s first two years. The number represents about 23% of all orphan drug designations during the program’s 35-year history.
Many of these designations were for existing drugs. One mentioned in the analysis, Keytruda, is a cancer drug that got six orphan approvals over the last two years for different types of cancers.
Trump has said that reduction of drug prices is a key goal of his administration.