Spark Therapeutics Hopes to Cure a Rare Blindness for $850000 a Pop

Cost of treating rare genetic blindness: $425000 per eye

Spark Therapeutics Inc said on Wednesday it would charge $850,000 per patient for its gene therapy to treat a rare form of blindness, lesser than analysts' expectation of around $1 million.

Pricing it was entering a bit of a courageous new world: NBC News reports Luxturna is the first "true gene therapy" that's gotten the green light here for an inherited disease, and unlike other pharmaceutical companies' workhorses-drugs that patients take for weeks, months, or even a lifetime-Luxturna is expensive to create and delivered just once. 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.

"Our work is not done, but we believe that the offerings we are announcing today will help ensure that eligible USA patients have the coverage and financial support they need to gain access to both Luxturna and the specialized medical care required to deliver the product at treatment centers".

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".

"I feel like we made the right middle ground decision", Marrazzo said in an interview, balancing the company's desire to capture the economic value of Luxturna while ensuring patients will have access to the therapy.

"The challenge is like going from being an apartment renter to a condo buyer and being shocked at [the] purchase price", he says.

Like most prescription medicines in the US, most of the immediate costs of Luxturna will be borne by insurers - not patients - including private plans and government programs. The drug was approved in December, the first gene therapy for an inherited disease to be approved by US regulators.

The drug received approval from the U.S. Food and Drug Administration in December, and given its success, most insurers are likely to cover the treatment. The treatment was expected to cost $1 million, but the company said it brought prices down over concerns of accessibility to the drug. He cited CAR-T therapies for cancer, which cost hundreds of thousands of dollars, and newfangled immuno-oncology treatments with similar price tags. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly. 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. However, Novartis said this price will not be charged to patients who do not respond within a month of treatment. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.

Spark said it is also discussing a proposal in which insurers would pay for the drug in installments over several years.

At least one gene therapy sold overseas has already crossed the $1-million price threshold, a treatment for a rare protein disorder launched in Europe. "We're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs", Express Scripts' Chief Medical Officer Steve Miller told CNBC.



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