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12 January 2018, 10:26 | Joann Bryant
Spark Therapeutics offers medicine for treatment of rare inherited form of blindness
Maybe if Spark focused its attention on developing a gene-therapy treatment for a disease less rare than the blindness Luxturna is supposed to treat, the company wouldn't have to charge such an exuberant price.
Spark already has a program in place with Boston insurer Harvard Pilgrim Health Care, by which Spark will get a full payment immediately after treatment and offer a rebate if patients do not see an immediate benefit to their eyesight, measured at 30 days or 30 months. It's very rare, affecting only a few thousand people in the U.S.
The Alliance for Regenerative Medicine there are more than 70 regenerative medicine and advanced therapy products in late-stage development and hundreds more further down the pipeline.
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 include a treatment for haemophilia and another for so-called "bubble baby" syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag. 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".
"If the units sold is zero you have zero in the revenue line", says the CEO, whatever the price. Hemophilia treatments for example can now cost up to $300,000 per year, for the lifespan of a patient, so a single dose of a cure priced at $1 million dollars doesn't seem entirely unreasonable.
One more gene therapy squeaked in before year's end, not for cancer treatment, but an inherited form of vision loss that can lead to blindness called biallelic RPE65 mutation-associated retinal dystrophy.
These therapies are generally meant to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. The treatment was expected to cost $1 million, but the company said it brought prices down over concerns of accessibility to the drug.
The company said the list price of the therapy, which was approved by the FDA last month, would be $425,000 per dose. It says it may give insurers a rebate if patients' vision gets worse within two and a half years, and it is working with Medicare and Medicaid so that the US government can pay in installments.
Spark is also discussing a proposal for insurers to pay for the drug in instalments over several years. 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. "We are also eager to work with CMS to enable more meaningful rebates as part of the pay-for-performance model". "We're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs", Express Scripts' Chief Medical Officer Steve Millertold CNBC. "But the question that must be asked is this: What is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?"
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