Are They Really Orphans? New Report Highlights Misuse of Orphan Drug Designation Driving High Prices

A study recently published in Health Affairs examines whether or not orphan drugs fit the criteria allowing them special privileges with the FDA. The authors report widespread misclassification, leading to excessively high prices. A quarter of 2017 drug spending in the US came from these so-called orphan drugs, with over 70 percent of drug spending intended for orphan drugs in fact paying for drugs outside the criteria.

According to Dr. Ge Bai at Johns Hopkins University’s Carey Business School and Bloomberg School of Public Health, “They [orphan drugs] treat a small number of patients, but at the same time they can be used to treat much more common diseases. So they [drugmakers] can still charge a high price and take the tax credit, while at the same time enjoying their impressive commercial success.” Read more here.

(Source: Peter Johnson, MMIT, 4/8/21)