Spark Therapeutics CEO Jeff Marrazzo reassured patients and the medical community he has an obligation to ensure access to the novel gene therapy.
When the US Food and Drug Administration (FDA) last month approved Luxturna, the first-ever therapy for a genetic disease, excitement abounded. Following the announcement, FDA commissioner Scott Gottlieb, MD, said he believed gene therapies like Luxturna will soon be mainstays “in treating, and maybe curing, many of our most devastating and intractable illnesses.”
Clinical trial evidence suggested that the novel gene therapy holds that potential for patients with biallelic RPE65 mutation-associated retinal dystrophy, which often progresses to blindness. Still, a big question remained: what would it cost?
Many experts predicted Luxturna (voretigene neparvovec-rzyl) would ring in at about $1 million because it’s designed for a rare genetic disease and a small patient population. They weren’t far off. On January 3, Luxturna’s maker Spark Therapeutics gave the medical community its answer: $850,000 for a complete, one-time operation, or $425,000 per eye.
A blog post co-authored by Spark Therapeutics CEO Jeff Marrazzo said that high gene therapy prices like the one for Luxturna are driven by high early-stage failure rates, lack of formal guidance for good manufacturing practices and supply chain management strategies, and the need for highly specialized treatment centers.
However, on the same day as the pricing announcement, Marrazzo said in a statement that Spark would harness “the same level of innovation applied in development of the treatment to the delivery of, and access to, the treatment.”
“The obligation, as I see it, is to ensure that you have access. Ultimately, if you price it at a point that is too high, and you don’t have access…you don’t have patients who get therapy and get access to this one time treatment. You’re not maximizing anything when it comes to generating revenue and topline for business,” Marrazzo said at the Forbes 2017 Healthcare Summit. “I think that question of access is a really important one, and has, ultimately, a lot of different components to it in the context of these one-time treatments.”
In the statement, Spark summarily outlined 3 new payer programs to facilitate access to Luxturna:
“We are encouraged by CMS’ willingness to engage with us in exploring a new model,” Marrazzo said. “We are also eager to work with CMS to enable more meaningful rebates as part of the pay-for-performance model. We are confident in the clinical meaningfulness of Luxturna and willing to stand behind the product’s efficacy and durability from a single dose.”
According to the statement, Harvard Pilgrim and affiliates of Express Scripts have agreed in principle to participate in Spark’s facilitated access programs, and discussions are underway with additional commercial insurers.
Express Scripts Chief Medical Officer Steve Miller, who is well known for his critiques of high drug prices, called Luxturna’s $850,000 price tag “responsible.”
“To be very frank, they’ve hit a responsible price. Is it inexpensive? Absolutely not. But it’s responsible,” Miller told Forbes.