Long-awaited gene therapy for blindness will cost $850000

Cost of treating rare genetic blindness: $425000 per eye

Cost of treating rare genetic blindness: $425000 per eye

Spark's Luxturna was approved by the U.S. Food and Drug Administration in December for patients who have a genetic mutation in the RPE65 gene, which causes degenerative retinal disease.

The first commercial gene therapy available in the USA will cost $850,000, Endpoints News reports.

The condition affects a few thousand people in the US and because of this the therapy has only been tested on a handful of people.

Consternation over skyrocketing drug prices, especially in the USA, has led to intense scrutiny from patients, Congress, insurers and hospitals. There is often little explanation from the drug's manufacturer about why it would need to cost that much, save for them claiming that it's very expensive to develop and test the drugs. 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". Spark Therapies says $850,000 with a money-back guarantee for its Luxturna gene therapy. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres. The hope is that such treatments could be cures, but at the moment, no one knows exactly how long the gene therapy's effects-which do not fully restore vision-will last. The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes.

As questions swirl around the cost and effectiveness of gene therapy, it could not be doubted that Luxturna is a breakthrough in the development of this kind of medicine. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

True to his meticulous nature, Marrazzo wants to carefully explain the multi-tier payment model Spark's team has been crafting and the objective behind it all: Not just steering the first gene therapy to an approval, but making sure that payers will cover it so that patients will be able use it to save their vision.

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients", said Spark Therapeutics' chief executive, Jeff Marrazzo. However, Novartis said this price will not be charged to patients who do not respond within a month of treatment. "We are also eager to work with CMS to enable more meaningful rebates as part of the pay-for-performance model".

Steve Miller, CEO of Express Scripts, is partnering with Spark Therapeutics to help make Luxturna more accessible to insurers and individuals.

"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 to health website STAT. "I do applaud them for thinking through the payment schemes".

However, even though the cost is lower than the $1 million price tag that analysts were expecting, there remain concerns that the drug is overpriced.

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