There’s probably going to be a new most expensive drug ever
By the end of the week, the FDA is expected to approve BioMarin Pharmaceutical’s gene therapy for hemophilia A, a one-time medicine that promises to revolutionize treatment of the rare bleeding disorder. It also promises to cost millions of dollars, reinvigorating the years-long conversation about how to value life-changing medicines.
The treatment, called Roctavian, demonstrated a 95% reduction in bleeding episodes in a four-year trial, and it also led to a 96% improvement in patients’ need for drugs that improve blood clotting. That latter point is important. Treating hemophilia A generally costs about $300,000 a year for a lifetime, a figure that doesn’t include the hospitalizations and surgeries many patients come to need.
BioMarin hasn’t committed to a price but has said its gene therapy would be cost-effective at $3 million. That would make it the most expensive drug in the world, topping the $2.1 million cost of Novartis’s gene therapy Zolgensma. BioMarin believes it has enough data to make the case to payers that Roctavian is a long-term bargain. We’ll find out once the treatment is approved.
The treatment, called Roctavian, demonstrated a 95% reduction in bleeding episodes in a four-year trial, and it also led to a 96% improvement in patients’ need for drugs that improve blood clotting. That latter point is important. Treating hemophilia A generally costs about $300,000 a year for a lifetime, a figure that doesn’t include the hospitalizations and surgeries many patients come to need.
BioMarin hasn’t committed to a price but has said its gene therapy would be cost-effective at $3 million. That would make it the most expensive drug in the world, topping the $2.1 million cost of Novartis’s gene therapy Zolgensma. BioMarin believes it has enough data to make the case to payers that Roctavian is a long-term bargain. We’ll find out once the treatment is approved.
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