Orphan drugs bring bounty for Big Pharma, at a steep price: Shots, NPR

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Summary: “Before Luke Whitbeck began taking a $300,000-a-year drug, the 2-year-old’s health was inexplicably failing. A pale boy with enormous eyes, Luke frequently ran high fevers, tired easily and was skinny all over, except his belly stuck out like a bowling ball,” Sarah Jane Tribble and Sydney Lupkin write at NPR in a piece about orphan drugs. “‘What does your medicine do for you?’ Luke’s mother, Meg, asked after his weekly drug treatment recently. ‘Be so strong!’ Luke said, wrapping his chubby fist around an afternoon cheese stick. Luke now spends days playing with his big brother, thanks to what he calls his ‘superhero’ medicine, a drug called Cerezyme, which has saved his life. For the Whitbecks, finding a way to pay for the drug has been a nerve-wracking struggle. Their family business and its insurer cough up hundreds of thousands of dollars per year, spreading the cost across the people it insures. Cerezyme is an orphan drug, which means it was created to treat a rare disease, one that affects fewer than 200,000 people in the U.S. The orphan drug program overseen by the Food and Drug Administration is loaded with government incentives and has helped hundreds of thousands of patients like Luke feel better or even stay alive. But the 34-year-old program has opened the door to almost unlimited price tags and created incentives for drugmakers to cash in, and to cash in repeatedly, a Kaiser Health News investigation shows. The explosion has burdened insurers as well as government programs like Medicare and Medicaid with drugs that cost up to $840,000 a year that they have almost no choice but to pay for.” Sarah Jane Tribble and Sydney Lupkin, Orphan Drug Bounty Comes At Steep Price,” Shots – Health News, NPR.