As the topic of managing drug costs becomes more of a talking point in Washington, the FDA just approved the most expensive drug in the world. According to a recent CNN Health article, Hemgenix is a gene therapy that treats adults with hemophilia B, a bleeding disorder where people don’t produce the protein that creates blood clots. Current treatment options include regular intravenous infusions to maintain levels of the missing clotting protein, but Hemgenix is meant to be a one-time IV infusion. That said, it does cost $3.5 million per treatment.
A cost-effectiveness analysis conducted by The Institute for Clinical and Economic Review that weighs health benefits against offset costs suggested a fair price is between $2.93 and $2.96 million. The approval of Hemgenix makes it the most expensive drug in the world, passing up Zolgensma, a spinal muscular atrophy drug approved in 2019 that costs $2.1 million for a course of treatment.