Gene therapy for inherited blindness to cost $850000

Gene therapy for inherited blindness to cost $850000

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 Therapeutics Inc. has priced its new gene therapy for a rare form of blindness at $850,000.

Anticipating criticism, Spark promised to reimburse patients if the treatment proves not to be effective, and said it expects USA health authorities to authorize payment in stages.

The condition affects a few thousand people in the USA and because of this the therapy has only been tested on a handful of people. Misa was 4-yearsold when he received his gene therapy treatment.

In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy.

At the end of past year, the CEO of Spark Therapeutics, Jeffrey Marrazzo, said he thought the medicine price will be over 1 million United States dollars, but after insurers' responses, he cut it by 150,000 USD. 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". 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.

It's a small pool of potential patients: only 2,000 people in the US have the disease, which is caused by a mutation in the RPE65 gene.

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Mark Trusheim, who directs MIT's New Drug Development Paradigms program, says gene therapies are moving medicine from a model of "renting" treatments to one of "buying" long-term health improvements. The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes.

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.

Most prescription drugs in the U.S. are paid for by insurers, including private plans and government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

The company has opted for a slightly lower price of $425,000 per eye and has announced three programmes making the one shot per eye treatment more manageable for USA payers. "The therapy was controversially priced at $475,000 per treatment", Rich Haridy reminds us on New Atlas. The gene therapy is widely expected to be the first in a wave of cutting-edge treatments that are targeted at fixing the causes of a wide range of genetic diseases. Having said that, the price is still a bit hard to come to terms with.

Spark said it had also designed a scheme that would allow the drug to be sold directly to insurers or special pharmacies, rather than hospitals or treatment centres, which tend to charge big mark-ups that can prove costly in the case of expensive drugs such as Luxturna.

Luxturna is delivered to patients just once. "The answer to that is certainly not the $850,000 price tag announced today".

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