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New million-dollar eye treatment

It’s been announced a recently approved revolutionary gene therapy drug aimed at treating a rare type of inherited retinal dystrophy will come with an eye watering price tag.

US-based Spark Therapeutics’ Luxturna, which the company claims yields remarkable results after just a single treatment, will cost patients US$425,000 (AU$533,800) for each affected eye. However, despite raising eyebrows, CEO Mr Jeff Marrazzo is standing by the company’s valuation of the drug.

“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,” he told Bloomberg.

Perhaps in an effort to reassure patients the treatment is value for money, Spark has devised an innovative payment model that will provide discounts in the form of rebates should the drug not prove initially successful, or if its effectiveness wanes over time.

"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."
Jeff Marrazzo, Spark Therapeutics CEO

The one-time nature of the drug has also presented a challenge for insurers, which are used to paying for medicine over the course of a disease or over a patient’s life.

However, the way Luxturna is administered means they are at risk of paying outright for a patient’s treatment, only to have them potentially change providers soon after, making it impossible to recover much of the money spent.

To protect against this eventuality, Spark is negotiating with insurance companies to establish programs that spread out payments over a number of years.

Another proposed plan is for Spark to sell the gene therapy directly to insurance companies or specialty pharmacies, avoiding the current process that requires hospitals or healthcare providers to buy the therapies up front.

One of the companies negotiating such an arrangement with Spark is Express Scripts Holding, which in the past has been critical of exhorbitantly priced drugs.

However, despite the high cost of the drug, Express Scripts chief medical officer Dr Steve Miller said the fact that Luxturna is a one-time drug meant it should be considered as an exception.

“Many people were anticipating this would be more than a million dollars but in the end, this is a revolutionary product, and I think in most plans this will be covered,” Miller said.


However, not everyone agrees with this assessment.

A non-profit organisation that evaluates the clinical and cost effectiveness of new drugs, the Institute for Clinical and Economic Review (ICER), has said Luxturna is priced far too high.

According to Reuters, ICER believes in most cases the drug should be priced 50–75% less than its current cost.

“We believe it’s a scientific milestone, but that for the majority of patients being treated, the cost is not in line with what’s considered cost-effective,” ICER chief medical officer Dr David Rind said.

Figures released by ICER that take into account the associated benefits and saving to society of a person regaining their vision suggest the price for treating both eyes should be closer to US$400,000 (AU$501,000).

Luxturna was approved by the FDA in December.

George & Matilda Eyecare

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