Drug firm Spark Therapeutics is charging $850000 for its new blindness treatment

A research scientist works in Spark Therapeutics’ laboratory.				Spark Therapeutics Inc

A research scientist works in Spark Therapeutics’ laboratory. Spark Therapeutics Inc

Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children's Hospital of Philadelphia.

Gene therapy can be especially risky and expensive to develop, but the company got tax breaks, Public Citizen noted.

A first-of-its kind genetic treatment for blindness will cost $850,000.

Consternation over skyrocketing drug prices, especially in the USA, has led to intense scrutiny from patients, Congress, insurers and hospitals.

The company said the price tag for voretigene neparvovec-rzyl (Luxturna) reflects patient and insurer concerns about access and cost, according to Reuters.

The first gene therapy in the USA, approved last month to treat a rare, inherited form of blindness, now has a price tag: $850,000. The disease, which affects up to 2,000 people in the United States, is caused by a genetic mutation that gradually disrupts the cells in the back of the eye forming the retina.

At $850,000 for a treatment of Luxturna, the medicine appears to be grossly expensive.

"We believe these pricing initiatives for a one-time treatment may widen the availability of the drug for patients", Justin Kim, an analyst at Cantor Fitzgerald, said in a research note.

The previous two are custom-made treatments for forms of blood cancer.

Many costly drugs need to be purchased year after year. Both cost around $750,000. For instance, cholesterol-lowering drugs must be taken daily for life and they are taken by millions of people.

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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".

Still, the price tag is higher than any other such treatments on the market.

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 estimate by the non-profit Institute for Clinical and Economic Review assumes the drug would maintain patients' vision for 10 years. However, Spark has only monitored the results of Luxturna in patients for a maximum of four years, which isn't exactly the same as a lifetime, so really, there's no telling how long the effects of Luxturna will actually last.

"Over these past few months, we have been working with health insurers to create innovative pathways for access to Luxturna that may serve as models for other one-time administered gene therapies in the future", Marrazzo said in a statement. The therapy just got approved last month. It was never approved in the U.S.

Spark said that government regulations meant it could not offer to sell the drug under instalment plans that would allow insurers and patients to bear the cost over time.

"If they decided not to cover it they would immediately have to face negative publicity", said Meredith Rosenthal, a professor of health economics at Harvard University to the Toronto Star.

But the starting price of some new drugs has soared.

Spark said it will offer rebates to insurers if the treatment doesn't work.

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