A new drug, Luxturna, literally allows blind people to see. It does it by using a virus to insert new genes into patients’ eyes. This morning, Luxturna’s maker, Spark Therapeutics, is announcing the cost of this medical miracle: $425,000 per eye, or $850,000 for most patients.
That figure is actually less than investors and outside experts expected. In December, the investment bank Jefferies had forecast a $1 million price. At the same time as it is announcing the wholesale price, which will be discounted in many cases and should be paid mostly by insurers and government agencies, not individuals, Spark is also announcing a series of steps to help hospitals, insurers, and patients deal with the complications of this high one-time cost.
“Many were anticipating this was going to be over a million dollars because it’s a small patient population,” says Steve Miller, the chief medical officer of Express Scripts, who has often criticized high drug prices. “To be very frank, they’ve hit on a responsible price. Is it inexpensive? Absolutely not. But it’s responsible.”
Express Scripts is working with Spark on some of the programs it is announcing, which will allow insurers to have an option to receive rebates two-and-a-half years after Luxturna is giving if patients improvements in vision aren’t maintained. (In clinical trials, the benefit endured that long in more than 9 out of 10 patients.)
Luxturna treats a rare form of blindness, called retinal dystrophy, that occurs when patients inherit a mutation of a non-working form of a gene called RPE65 from each parent. There are about 1,000 cases in the U.S., and there are expected to be 10 to 20 new cases a year. The treatment does not always result in a full restoration of vision, but in a clinical trial allowed most patients to get much better at navigating an obstacle course in low light.
The price is not out of the realm of normal for rare disease drugs. It can cost $1 million a year to treat hemophilia in rare cases where patients develop antibodies to the drugs used to prevent clotting. Spinraza, a drug for a deadly childhood disease called spinal muscular atrophy sold by Ionis Pharmaceuticals and Biogen, can cost $750,000 in its first year, but half as much thereafter. Soliris, made by Alexion Pharmaceuticals, costs a similar amount at doses used to treat a rare kidney disease. Glybera, a gene therapy for another rare disease, was available in Europe at a $1 million price; it didn’t sell and its maker, UniQure, allowed the product’s marketing clearance to lapse.
Jeff Marrazzo, Spark’s chief executive, said that his goal was to create a “menu” of options for insurers, called “payers” in health-care-speak, that would allow Spark to stand behind the company’s faith in Luxturna’s efficacy, present an alternative to the current “buy and bill” model in which drugs are always purchased for a one-time cost, and address the problem of “cost density,” that is, if you’re a hospital, it’s pretty tough to purchase an $850,000 treatment, or, even worse, to keep such a treatment in stock, even if it is worth the money because it represents such a big capital cost. (For the same amount, you could buy a DNA sequencer or two.)
Here are Spark’s solutions:
Express Scripts Miller, while praising the current price, said that he hopes future gene therapies will have lower prices, pointing to the hemophilia treatments Spark is developing. “There will obviously be more patients to treat at that point in time,” Miller says.