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Gene Therapy Cure For Blindness Gets $850K Price Tag

12 January 2018

A few months later the FDA approved another gene therapy, this time for a rare inherited retinal disease, the drug is called Luxturna. Between 1,000 and 2,000 people in the United States are thought to be affected by the mutated gene. Misa was 4-yearsold when he received his gene therapy treatment.

Dr Peter Bach, director of a policy centre at Memorial Sloan Kettering Cancer Center in NY quoted regarding the issue, "The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable".

Luxturna is just the first of many one-time gene therapy treatments likely to hit in the market over the coming years, and therefore will necessitate changes in how payment and reimbursement are conducted. The manufacturers say the price is a bargain. While the staggering cost may be hard to bear for some patients, medical analysts are actually saying the price of the treatment is less than expected.

Approved for inherited retinal disease caused by a defective RPE-65 gene, Luxturna is a single injection that works by delivering 150 billion viral vector particles containing a correct copy of the gene to retinal cells.

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Spark, in fact, says there will be three distinct "payer programs" to ensure that US patients who need treatment can get it: "an outcomes-based rebate arrangement with a long-term durability measure, an innovative contracting model and a proposal to [Centers for Medicare and Medicaid Services] under which payments for Luxturna would be made over time". The company's CEO, however, has said the effects of treatment could be "life-long".

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.

One insurer, Harvard Pilgrim Health Care, which covers 2.7 million people in New England, has already signed on to pay for Spark's therapy.

Although Spark's Luxturna is the most expensive drug, the overall cost for patients and insurers may be less than for other expensive drugs which have to be taken multiple times.

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And it's in negotiations with the Centers for Medicare and Medicaid Services, the federal government health insurance agency, to come up with ways to let insurers pay by installments.

The company also plans to arrange for a new type of contracting model with payers to help ease the potential burden of upfront costs on providers. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.

"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".

This restores the patient's ability to make the missing enzyme - dramatically improving their eyesight and restoring a large degree of independence, although not offering 20/20 vision. "The answer to that is certainly not the $850,000 price tag announced today".

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Gene Therapy Cure For Blindness Gets $850K Price Tag