Spark Therapeutics on Wednesday announced that its recently approved  gene therapy Luxturna (voretigene neparvovec-rzyl) will carry a price of $425 000 per eye, or $850 000 in total, in the US, below earlier expectations of around $1 million. The drugmaker also said it reached an agreement in principle with health services company Harvard Pilgrim to make the one-time treatment available under an outcomes-based rebate programme, while a proposal to the US Centers for Medicare & Medicaid Services (CMS) would see Luxturna payments made over time.
Luxturna was authorised in December for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy as the first directly administered gene therapy cleared in the US that targets a disease caused by mutations in a specific gene. Spark CEO Jeffrey Marrazzo remarked "over these past few months, we have been working with health insurers to create innovative pathways for access to Luxturna that may serve as models for other one-time administered gene therapies in the future."
Commenting on the price of Luxturna, Marrazzo noted "it came down to the value we believed was inherent in the therapy." Marrazzo said "we also heard and did market research with payers…and wanted to take into account the questions and challenges they had about affordability," adding "ultimately this is about access."
Specifically, Spark noted that it will share risk with certain health insurers by paying rebates if Luxturna fails to achieve thresholds for short-term efficacy and long-term durability as determined using full-field light sensitivity threshold testing scores. While the drugmaker declined to clarify the size of the rebate, Marrazzo said they would not exceed the 23-percent mandatory rebate paid by other companies to Medicaid.
Along with the deal with Harvard Pilgrim, Spark also reached an agreement in principle with affiliates of Express Scripts to enable a contracting model. Under the scheme, the drugmaker would enter into an agreement with commercial payers under which the payer or payer's specialty pharmacy, rather than the treatment centre, purchases Luxturna.
Commenting on the news, Express Scripts chief medical officer Steve Miller remarked "many were anticipating this was going to be over a million dollars because it's a small patient population," continuing "to be very frank, they've hit on a responsible price. Is it inexpensive? Absolutely not. But it's responsible." Miller noted "we're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs," adding "there are going to be more of these drugs coming to the marketplace for even bigger populations."
Additionally, Spark noted that while it has been seeking arrangements that would permit patients to pay for Luxturna in instalments over several years, current government price reporting requirements preclude this option. The company indicated in addition to its proposal seeking to permit instalment payments, the CMS proposal would also allow it to offer larger rebates tied to clinical outcomes. "We are encouraged by CMS' willingness to engage with us in exploring a new model," Marrazzo remarked, adding "we are also eager to work with CMS to enable more meaningful rebates as part of the pay-for-performance model."
For related analysis, read ViewPoints: Spark’s payment plans reassure investors about Luxturna access .