inside the paradoxical world of drug pricing, the first U.S. cost tag exceeding $1 million for a medicine is usually being contemplated as the nation’s agita over the cost of prescription drugs climbs ever higher.
brand new gene therapies in which aim to cure hemophilia, a disease affecting the blood’s ability to clot, may carry prices of $1.5 million or more, analysts at Leerink wrote in a research note Monday.
Gene therapies deliver a healthy copy of a gene to make up for a defective one in which causes disease, aiming to cure — or at least significantly improve — the malady in just one treatment. Such therapies for hemophilia are in development at drugmakers BioMarin, Spark Therapeutics as well as UniQure.
They’re changing the way we think about delivery — as well as pricing — of medicine. The first gene therapy was approved inside the U.S. in December, as well as received a cost tag of $850,000 in January. Called Luxturna, This particular treats a rare form of blindness as well as is usually made by Spark.
A cost of $1.5 million or more could set a brand new paradigm inside the U.S.
“This particular appears the seemingly impervious million-dollar threshold may be breached with hemophilia gene therapy, which could do so while still creating value for society by reducing the cost of factor replacement therapy,” Leerink analysts Joseph Schwartz as well as Dae Gon Ha wrote in their research note.
Factor replacement therapy is usually the current treatment for people with hemophilia, as well as is usually estimated to cost between $580,000 as well as $800,000 a year for adult patients, according to Leerink. For some patients, costs can approach $1 million a year, or even more. About 20,000 Americans have hemophilia.
For in which reason, a potential one-time $1.5 million cost is usually perceived by many to be a bargain compared using a lifetime of chronic therapy.
Others, though, like Walid Gellad, director of the Center for Pharmaceutical Policy as well as Prescribing at the University of Pittsburgh, see This particular as excessive.
This particular will be the first major example of when cost effective/cost saving fails us in pricing. Kidney transplants are life saving however the surgeon doesn’t charge $1.5million.
The Leerink analysts took the cost of Spark’s gene therapy, Luxturna, into consideration in coming to their cost estimate, noting “one of the top questions about gene therapy treatments concerns the cost which the market will bear,” taking into account direct as well as indirect costs “as well as societal cost burden of the affliction.”
They said there may even be “some headroom” above $1.5 million for hemophilia gene therapies, with the potential for the medicines, if approved, to be priced at $2 million per patient.