Reporters Sarah Jane Tribble and data specialist Sydney Lupkin revealed how drugmakers have been manipulating an FDA program designed to help patients with rare diseases get access to new life-saving medicines. Instead, the program has been corrupted by pharmaceutical giants looking to maximize profits on existing drugs and rake in billions of dollars in federal incentives, the investigation revealed. An exclusive data analysis showed that at least a third of the 450 orphan drugs approved by the FDA since 1983 are not “new” products whatsoever.
More than 70 were first approved for mass-market use, including cholesterol blockbuster Crestor, Abilify for psychiatric conditions, cancer drug Herceptin and rheumatoid arthritis drug Humira, the best-selling medicine in the world. More than 80 other orphans won FDA approval for more than one rare disease and, in some cases, multiple rare diseases, the data analysis showed. Yet, in each instance, drugmakers received a rich package of financial incentives including – most importantly – seven years of exclusive rights to treat each rare disease, or a virtual monopoly.
The result is that they can charge sky-high prices for the drugs which often exceed $100,000 to $300,000 a year, per patient. That’s critical because orphan drugs now account for about half the drugs approved by the FDA each year, at a time when Americans are outraged over soaring drug prices.