How Much Is A Miracle Worth?

- Caroline Chen

Sofia Priebe, 14, is slowly going blind. Her parents were devastated when they were told there’s no treatment for the genetic mutation that’s causing her retinas to deteriorate. For the dozen years since Sofia received that diagnosis, her mother has lived every parent’s nightmare—being powerless to help her suffering child.

Now a gene therapy for a similar form of blindness is expected to receive U.S. Food and Drug Administration approval this year, and Laura Manfre, Sofia’s mom, is holding out hope that her daughter may soon get treatment as well. “We don’t really care what it costs,” she says.

But how much is a miracle really worth? A million dollars? Five million? More? And who will pay and how? It’s one of the most vexing challenges confronting drug and insurance companies as modern medicine advances, spurred by research on the human genome. Spark Therapeutics Inc., which developed the gene therapy to cure a rare form of childhood blindness called RPE65-mediated inherited retinal disease, is among the first to face this question. Spark’s treatment, voretigene neparvovec, delivers a functioning piece of DNA directly to the eyes to preserve remaining sight and even restore some vision. Other companies, including GlaxoSmithKline Plc and BioMarin Pharmaceutical Inc., have also been grappling with the pricing problem.