When Giving ‘Hope To The Hopeless’ Comes At A Sky-High Price
Orphan drugs are helping patients who otherwise would be facing a lifetime of suffering, but their price tags can be astronomical.
Orphan Drugs Offer Hope For Treating Rare Diseases, Despite High Price Tags
At Sutter Children’s Center in Sacramento this week, the 7-year-old was given an injection of Spinraza, the first drug approved in the U.S. to treat spinal muscular atrophy, or SMA. ... With Spinraza, each thumb-sized 5-milliliter vial – about a teaspoon’s worth – costs $125,000. The recommended dosage is four “loading” injections within about two months, followed by maintenance shots every four months for life. For families such as the Goepperts, that price tag is staggering: roughly half a million dollars just for the initial series of Spinraza shots and $375,000 a year thereafter. (Buck, 2/20)
Related California Healthline coverage: The Orphan Drug Machine