Gene therapy for blindness will cost $850,000
- The FDA approved the first gene therapy for a rare inherited form of blindness affecting between 1,000 and 2,000 Americans last month
- After uproar over the drug’s expected $1 million price tag, its maker lowered the cost to $850,000
- The therapy is still more expensive than any of the
Natalie Rahhal For Dailymail.com
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A first-of-its kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but it’s still among the most expensive genetic therapies in the world.
The new therapy was approved last month to treat a rare genetic mutation that causes blindness in between 1,000 and 2,000 people in the US.
Spark Therapeutics, which makes the drug, says it decided on the lower price tag for Luxturna after hearing concerns from health insurers about their ability to cover the injectable treatment.
Consternation over skyrocketing drug prices, especially in the US, has led to intense scrutiny from patients, Congress, insurers and hospitals.
Dr Albert Maguire (right) checked the eyes of Misa Kaabal (left), eight, in October 2017, four years after he received Luxturna to treat his genetic blindness. The drug is now on the market for $850,000, making it one of the world’s most expensive genetic therapies
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‘We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,’ said CEO Jeffrey Marrazzo, in an interview with The Associated Press.
Luxturna is still significantly more expensive than nearly every other drug on the global market, including two other gene therapies approved earlier last year in the US.
America’s Got Talent semi-finalist Christian Guardino got his sight back after participating in a clinical trial for Luxturna more than five years ago
It is the nation’s first gene therapy for an inherited disease. 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 US.
Before the drug was even approved, it made headlines when it restored sight to America’s Got Talent semi-finalist Christian Guardino, who took part in a Luxturna clinical trial.
Luxturna is an injection – one for each eye – that replaces a defective gene in the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425,000 per injection.
The treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.
There are questions about the wisdom of devoting so much energy to specialty drugs, which are used to treat so few people, but still account for a growing slice of overall health care costs.
Drug-makers have historically offered little explanation for the prices they charge. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.
Spark Therapeutics, based in Philadelphia, has said that the cost for a lifetime of blindness – including lost earnings and caregiver wages – can easily exceed $1 million.
Not everyone agrees with that argument. A preliminary analysis by one group found the drug would have to be priced significantly lower ‘to be a cost-effective intervention.’
The rare genetic mutation Luxturna treats
Luxturna injections treat mutations that occur in the RPE65 gene.
Mutations of the gene disrupt the production of a key protein to vision.
RPE65 mutations can cause three forms of vision loss:
Leber congenital amaurosis, which causes serious vision impairment early in life.
Fundus albipunctatus, which makes seeing in low light difficult and is sometimes called ‘night blindness.’
Retinitis pigmentosa, which damages the retina, causing progressive vision loss.
The estimate by the non-profit Institute for Clinical and Economic Review assumes the drug would maintain patients’ vision for 10 years. However, Spark expects the drug’s effect to be long-lasting, if not lifelong, though it has only tracked patients for about four years.
At least one gene therapy sold oversees has already crossed the $1 million price threshold.
The treatment for a rare protein disorder launched in 2012 with a price of $1.2 million. Manufacturer uniQure stopped selling the drug earlier last year after seeing a lack of demand. The drug was never approved in the US.
Like most prescription medicines in the US, most of the immediate costs of Luxturna will be borne by insurers, including private plans and government programs. For patients, Spark said it would cover all out-of-pocket expenses needed to obtain the medication, including transportation to hospitals trained to administer the injections.
Spark will try to deflect some pricing concerns by offering unconventional payment plans to insurers. Under one arrangement with the non-profit insurer Harvard Pilgrim, Spark will repay some of Luxturna’s costs if patients don’t experience the expected improvements in vision. The company did not disclose how much money would be returned to the insurer, which covers more than a million people in New England.
Spark said it is also discussing a proposal in which insurers would pay for the drug in installments over several years. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.
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