World’s Most Expensive Medicine For Rare Blindness Priced At $850,000

The first gene therapy in the US To treat a rare and inherited form of blindness, it now has a price tag: $ 850,000 And World’s Most Expensive Medicine.

That’s what makes the treatment, called Luxturna and manufactured by Spark Therapeutics, the World’s Most Expensive Medicine sold in the US. Classified by day price.

But the CEO of Spark, Jeff Marrazzo says the ability to restore Luxturna’s vision in a small number of people with a defective gene justifies the high cost, especially because gene therapy is injected only once for a profit in each eye, long term Many investors expected That Spark would charge $ 1 million or more for Luxturna, so the real price is considered cheap by some.

“I think we made the right interim decision,” Marrazzo said in an interview to reconcile the company’s desire to achieve the economic value of Luxturna while ensuring that patients have access to treatment.

To soften further reimbursement and silenced the inevitable criticism of the price, the commitment to provide Wednesday reimbursement of health insurance funds sparked when patients do not meet the limits given vision between 30 and 90 days and 30 months after treatment with Luxturna , Genetic therapies should be curative, but patients in clinical trials have not been followed enough to see if the benefits of treatment continue for decades.

With the charitable Harvard Pilgrim Health Plan, which secures 1.2 million people in New England, Spark has signed a Memorandum of Understanding for the first earnings-based reimbursement agreement. Other reimbursement contracts are being negotiated with other commercial insurance companies, Spark said.

It was not disclosed how much of the $ 850,000 in Luxturna costs would be reimbursed to insurers if patients did not benefit from the treatment.

Harvard Pilgrim and Express Scripts, the administrator of the country’s largest pharmacy benefits, also agreed to spank a new holding agreement to ensure patients’ access to Luxturna in return for reducing the financial burden and risk for healthcare providers and insurers

According to another proposal still in the design phase, Spark would allow insurers to distribute Luxturna payments for several years.

“In terms of price and structure to pay the price, I think everything is in line with what we see in other innovative therapies,” said Dr. Stuart Orkin, a pediatric oncologist at Dana-Farber Cancer. Institute and Boston Children’s Hospital. He cited CAR-T therapies for cancer that cost hundreds of thousands of dollars, and novel immuno-oncology treatments with similar prices.

The question for Orkin, who has written extensively about how the Society should pay for gene therapy, just as sparks, determines whether a patient treated with Luxturna has “failed” and therefore deserves a refund.

“But I appreciate you for thinking about the terms of payment,” Orkin said. “It’s better than having just taken one price and said they pay.”

The Food and Drug Administration approved Luxturna in December to treat people with a type of hereditary retinal disease caused by a mutation in a gene called RPE65. People born with mutated RPE65 genes suffer from severe vision problems, including night blindness. As the disease progresses, people lose all functional vision and eventually become completely blind.

The disease is rare, affecting between 1,000 and 2,000 people in the US. About 10-20 babies are born each year with RPE65 mutant retinal disease, according to Spark.

Wall Street analysts predict that Luxturna’s revenue will increase to $ 364 million by 2022, according to Evaluate. According to pharmaceutical industry standards, which combine the status of overproduction with products that generate $ 1 billion in sales, Luxturna is expected to have only a modest success. But that’s also one of the reasons why Spark has decided not to rate it more aggressively.

Prevents the commercial launch of Luxturna indignation insurers and suppliers with too high a price that will not serve Spark in the future, when their other gene therapies, major and lucrative diseases such as hemophilia targeting, hit the market, promising data of it Seen from today to approvals.

However, the biotech group said it participated in discussions about the development of a pilot rate plan with the US Centers for Medicare and Medicaid Services. UU., The federal agency that administers the medical attention financed by the taxpayers for people of low income.

If successful, the pilot project could serve as a prelude to regulatory changes that increase the frequency of drug payment plans.

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