Gene therapy for inherited blindness to cost $850000

Gene therapy for inherited blindness to cost $850000

Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of US$1.2millio.

Some patient groups are already pushing back.

Novartis has offered a similar scheme giving discounts if its expensive CAR-T therapy does not produce results in an aggressive form of blood cancer.

These therapies are generally meant to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. Drugmakers have historically offered little explanation for the prices they charge, other than to cite the high cost of developing a drug and the fact that so many drugs fail during trials and must be abandoned. But gene therapies are given only once, with potentially permanent effects.

Across the pond, "Luxturna could potentially become available free on the [National Health Service] in the United Kingdom after being submitted for approval by the European Medicines Agency (EMA)".

The biotech said it has reached an agreement in principle with New England-based insurer Harvard Pilgrim that would base pricing on outcomes. Should the therapy not perform as expected and meet certain undisclosed criteria after the one-time administration at those specified time points, Spark will pay rebates to payers. Kymriah, too, carries a guarantee that there will be no charge if the treatment does not work, however.

Spark is also discussing a proposal for insurers to pay for the drug in instalments over several years. Current government price reporting requirements do not allow for payment models of this type, but Spark is hoping to change the paradigm. Following the announcement, FDA commissioner Scott Gottlieb, MD, said he believed gene therapies like Luxturna will soon be mainstays "in treating, and maybe curing, many of our most devastating and intractable illnesses".

"We are encouraged by CMS' willingness to engage with us in exploring a new model", Marrazzo said.

Express Scripts is partnering with Spark for distribution of Luxturna, Terrell reports.

The treatment is expensive because of the various unique reasons such as the therapy represents the medical discovery, and it can treat a small number of patients.

Luxturna is delivered to patients just once. "But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?"

However, even though the cost is lower than the $1 million price tag that analysts were expecting, there remain concerns that the drug is overpriced. "We are confident in the clinical meaningfulness of Luxturna and willing to stand behind the product's efficacy and durability from a single dose".

It's a gradual and cruel fate: an inherited form of vision loss that typically ends in blindness and has had no cure.

Breazzano said Spark is "clearly focused on moving away from this one-time upfront payment to an annuity-like model with payments over multiple years".

These new prospective models are situated inside a greater debate that is now occurring as more of these breakthrough gene therapies become real.

The next year will be critical in showing that gene therapies are marketable.

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