Spark Therapeutics' gene therapy for a rare blindness gets $850K price tag

Spark Therapeutics' Luxturna will cost $850,000 the company announced Wednesday

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.

Pricing it was entering a bit of a courageous new world: NBC News reports Luxturna is the first "true gene therapy" that's gotten the green light here for an inherited disease, and unlike other pharmaceutical companies' workhorses-drugs that patients take for weeks, months, or even a lifetime-Luxturna is expensive to create and delivered just once. In addition, it is working with stakeholders in the healthcare industry, including health insurers, for installment options and rebates. It can improve the vision of those with a rare form of blindness that is estimated to affect just a few thousand people in the U.S.

In some cases, the gene therapy will be available under an "outcomes-based rebate arrangement".

Drug prices are not regulated in the US, as they are in many other countries, so drugmakers can price their goods like any other manufacturer.

The company said the price tag for voretigene neparvovec-rzyl (Luxturna) reflects patient and insurer concerns about access and cost, according to Reuters.

"If you look at the history of biotech, the first approved products don't necessarily define what the market is", he says.

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David Mitchell, founder and president of the advocacy group Patients for Affordable Drugs, is concerned that the treatment will be out of reach for people with high-deductible health plans and would bankrupt those without insurance.

The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene. The treatment was expected to cost $1 million, but the company said it brought prices down over concerns of accessibility to the drug. But the firm said it had settled for the lower price of US$850,000, or US$425,000 per eye. Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of US$1.2millio. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly. The therapy was controversially priced at US$475,000 per treatment.

"As far as the price, and the structures to pay the price, I think it's all pretty much in line with what we're seeing in other innovative therapies", said Dr. Stuart Orkin, a pediatric oncologist at the Dana-Farber Cancer Institute and Boston Children's Hospital.

"Many were anticipating this was going to be over a million dollars because it's a small patient population", Steve Miller of Express Scripts told Forbes.

Luxturna is delivered to patients just once. But the Spark pricing team has worked it out so that under one model Spark can offer payers unspecified rebates at 30 days and 30 months - which is about the average amount of time a patient stays in a commercial plan - if Luxturna falls short of established efficacy goals on vision. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high".

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