After much speculation, Spark Therapeutics has confirmed the price it intends to charge for its sight-saving gene therapy Luxturna – and it’s at the high end of expectations.
Luxturna (voretigene neparvovec-rzyl) became the first gene therapy approved by the FDA last month and will have a wholesale acquisition cost of $425,000 per one-off treatment, ie $850,000 if a patient has the procedure on both eyes. Analysts had previously suggested the price would be in the $500,000 to $1m bracket per patient.
Luxturna is used to treat a sight-robbing disorder called biallelic RPE65-mediated inherited retinal disease (IRD), which affects somewhere between 1,000 and 2,000 people in the US with around 10 new cases per year, and a similar number in the EU.
While the overall price tag is capturing the headlines, drilling down into the story reveals the approach adopted by Spark and CEO Jeffrey Marazzo is more subtle, and gives the industry its first real-world model to consider as it tries to work out how to price and deliver new gene therapies that deliver significant clinical benefits.
In tandem with the pricing announcement, Spark has also revealed a rebate deal with US health service firm Harvard Pilgrim, which ties the cost to a successful clinical outcome. Specifically, it will pay back around a quarter of the price in the form of a rebate if visual improvement objectives are not reached.
“This outcomes-based rebate arrangement is truly innovative, as it ties payment for the therapeutic not only to a short-term goal, but also to a longer-term, 30-month assessment of efficacy,” commented Michael Sherman, Harvard Pilgrim’s chief medical officer.
The company has also submitted a proposal to the US Centers for Medicare & Medicaid Services (CMS) to allow Luxturna payments to be spread out over time. And thirdly, it will provide the option of buying the gene therapy directly from Spark, with the company handling all handling costs and insurers therefore able to sidestep hospital mark-ups.
With all this in mind, Spark estimates that after discounts and rebates the average net price for the treatment will be closer to $638,000 per patient.
“We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians and treatment centres,” commented Marazzo.
The biotech has previously drawn comparisons with GlaxoSmithKline’s ADA-SCID gene therapy Strimvelis – just approved for coverage by the UK health technology assessment agency NICE – which has a list price of around $714,000. It points out that with ADA-SCID there are alternative treatments available (stem cell transplant), while for patients with RPE65-IRD Luxturna is the only option.