There’s a possible cure for ‘bubble boy’ disease. It will cost $665,000

GlaxoSmithKline has developed what appears to be a cure for a rare and deadly disease, and it will cost $ 665,000 for a single dose.

This tag clouds the price – more than double the average price of a house – is likely to shake what is already a long simmering debate health care : how much is healing therapies will cost, and how society should pay for them?

GSK drugs, called Strimvelis, is a gene therapy severe immune deficiency combined , a congenital disease that causes children can not protect themselves against infection. It is the same condition that forced David Vetter live his life in a protective plastic sphere, depicted in the documentary “The Bubble Boy.”

The article continues after the announcement

Without treatment, children with the disease die before the age of 2. However, a single administration of therapy GSK maintains 100 percent of patients alive after three years in a small clinical trial, and that was enough to convince European regulators to approve it in May.

And those results are worth $ 665,000, according to the Italian Medicines Agency, which agreed to reimburse for the drug to GSK. SCID is otherwise treated with bone marrow transplants risk or replacement therapies enzymes that must be taken for life and can cost more than $ 4 million over the course of a decade, GSK said. Strimvelis, however, it could be a bargain.

Gilead Sciences uses a similar [19459018calculation] defend their treatments of hepatitis C , which carry a price tag of more than $ 80,000 for 12 weeks of treatment. The alternative, Gilead says, is hospitalization and liver transplantation, which cost much more than drug therapy in the long term.

But GSK and other developers of gene therapy will have to convince taxpayers to spend large sums of money, once for treatments whose long-term effects are still unknown.

Philadelphia Therapeutic Spark it is expected to get approval from the FDA for next year a treatment for a disease rare eyes, a drug that would become the first gene therapy of USA .. like GSK, Spark has shown excellent results in a small clinical trial, but no one knows how well your therapy take place throughout the patient’s life.

That could be a concern for insurers, who do not want to shell out big money for a cure alleged that disappears over time. In addition, although the gene therapies work as promised, reimbursement for them, it could present a sunk cost for payers. If a patient changes insurance providers after receiving a healing therapy, the insurer pays for it is essentially subsidizing a competitor.

Some argue the increased gene therapy requires new approaches to how drugs are reimbursed. One idea put forth in Science earlier this year, is requiring drug makers to issue a kind of guarantee, which promises to give a refund or pay an additional treatment if gene therapy stops working . Insurers could also negotiate a payment plan, the trial suggests Science, paying only the time the medication is doing its job.

Meanwhile, pharmaceutical companies and venture capitalists are investing billions of dollars in projects of gene therapy in the hope of coming and going more treatments at once on the market in the coming years. Pfizer, which has been associated with spark, just acquired a small biotechnology company to develop own genetic therapies. Bayer has invested in space, like Biogen, Sanofi and Novartis.

There are at least a warning on the books. Glybera, a gene therapy approved in Europe in 2012, spat on the commercial failure after reimbursement agencies balked at the cost of $ 1 million. Uniqure, the company that invented finally scrapped plans for a US request and sold the marketing rights for the drug to another company.

Despite its high cost, Strimvelis is unlikely to be a blockbuster for GSK. The disease is treated only affects about 15 people each year in Europe, the company said. In the US, where GSK intends to seek approval next year, that number is closer to 12.

The drugmaker plans to manage your gene therapy in the San Raffaele Hospital Milan, who helped develop the treatment. Children across the EU may obtain Strimvelis there thanks to the benefits of cross-border healthcare, GSK said, with their countries of origin reflect the cost.

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