Expensive gene therapy can cure the rarest blindness

Expensive gene therapy can cure the rarest blindness

The FDA in December approved Luxturna, a new gene therapy that can treat the progressive blindness, and on Wednesday, its maker revealed the staggering cost: $425,000 per eye.

Luxturna, the first gene therapy for an inherited disease in the USA to be injected into each eye, was originally going to be priced at $1 million, but Spark decided against this in the end due to comments from health insurers about their concerns over being able to cover that amount - not that $850,000 is much more manageable.

Despite Spark's efforts to offer alternative payment options (while still bringing in a profit), patient groups are already up in arms about the cost of Luxturna. Between 1,000 and 2,000 people in the United States are estimated to have the disease. But gene therapies are given only once, with potentially permanent effects. Consternation over skyrocketing drug prices in the US has led to intense scrutiny from patients, Congress, insurers and hospitals.

Drug prices are not regulated in the US, as they are in many other countries, so drugmakers can price their goods like any other manufacturer.

In an article, Bloomberg's Michelle Cortez noted that the schemes are very different from the approach offered by pharma companies, and could set a precedent for how expensive gene therapies could get to market in the future.

How much of the $850,000 cost of Luxturna would be returned to insurers if patients don't benefit from treatment was not disclosed. For instance, Spark wants to offer discounts based on whether or not the drug initially works and remains effective; there are roughly 1,000 to 2,000 patients in the USA who suffer from this kind of inherited retinal disease caused by a mutant gene.

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Just like two other gene therapies which got approved previous year in the in the US, Luxturna is more expensive than nearly every medicine across the global market.

Spark said it is also discussing a proposal in which insurers would pay for the drug in installments over several years. The product works by delivering, via an eye injection, some 150 billion viral vector particles containing a correct copy of the RPE65 gene to retinal cells, restoring their ability to make the needed enzyme. It says it may give insurers a rebate if patients' vision gets worse within two and a half years, and it is working with Medicare and Medicaid so that the USA government can pay in installments. Ultimately, if you price it at a point that is too high, and you don't have access...you don't have patients who get therapy and get access to this one time treatment.

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients", said Spark Therapeutics' chief executive, Jeff Marrazzo. While it's less than the $1 million price tag that analysts had expected, it's still among the most expensive genetic therapies in the world.

Marrazzo's not saying how much he's offering in rebates, but when I asked him why not a full refund for patients who don't respond adequately, he said that's not possible.

"Many were anticipating this was going to be over a million dollars because it's a small patient population", Steve Miller of Express Scripts told Forbes.

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