Gene therapy for rare form of blindness: $850K

The Most Expensive U.S. Medicine Now Has an Official Sticker Price

Luxturna was approved by the Food and Drug Administration in December to treat a rare, inherited retinal disease that can lead to blindness. In addition, it is working with stakeholders in the healthcare industry, including health insurers, for installment options and rebates.

The therapy treats the genetic variation which affects some of thousand people in the US.

Gene Therapy: The Future Of Medicine?

Consternation over skyrocketing drug prices, especially in the US, has led to intense scrutiny from patients, Congress, insurers and hospitals. However, "there are very few such patients, and the price of every treatment matters a great deal".

In a phone interview with Bloomberg, Spark CEO Jeff Marrazzo said that "We believe that this price reflects not only the breakthrough, life-altering value of one-time Luxturna, but it will enable us to continue to invest and build on the revolutionary science that supports not only Luxturna but the rest of our pipeline".

"New technologies such as gene and cell therapies hold out the potential to transform medicine", FDA Commissioner Dr. Scott Gottlieb-said in a statement announcing the August approval of Kymriah, considered to be the first gene therapy to be approved for the U.S. market.

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

Another company making pricey treatments has a plan to offer rebates if patients don't get better. The drug was approved in December, the first gene therapy for an inherited disease to be approved by US regulators.

"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. Spark backs up its price decision by saying that Luxturna is meant to be a lifelong solution, while many other drugs have to keep being renewed annually, meaning that ultimately they could end up costing you more.

The company has opted for a slightly lower price of $425,000 per eye and has announced three programmes making the one shot per eye treatment more manageable for U.S. payers.

Most prescription drugs in the United States are paid for by insurers, including private plans and government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.

The question for Orkin, who has written extensively about how society should pay for gene therapy, will be just how Spark determines whether an individual patient treated with Luxturna has "failed" and thus merits a rebate. Steve Miller, Express' chief medical officer, said that "Many people were anticipating this [retinal disease treatment] would be more than a million dollars..." "Gene therapy is also being used to correct deficiencies in the production of dopamine, such as in Parkinson's disease, correct some immune system problems, and restore components needed for normal blood cell function in those with certain blood diseases, such hemophilia and beta-Thalassemia. But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?" The treatment was expected to cost $1 million, but the company said it brought prices down over concerns of accessibility to the drug. "The answer to that is certainly not the $850,000 price tag announced today", he added.

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