- Spark Therapeutics Inc.'s newly approved gene therapy for a rare form of blindness is too expensive to be considered cost-effective, according to a new report from a research organization known for its critiques of industry pricing practices.
- Currently priced at a wholesale acquisition cost of $850,000 per patient, Luxturna would need to be discounted by between 75% and 82% to meet the cost-effectiveness thresholds used by the Institute for Clinical and Economic Review (ICER).
- Luxturna is the first gene therapy for an inherited disorder to be approved in the U.S. — making it an important milestone for a field expected to deliver more treatments aimed at correcting genetic errors responsible for hereditary diseases like hemophilia or beta thalassemia. Luxturna's price, therefore, has received close scrutiny as a potential sign of what's to come.
Spark's clinical evidence for Luxturna (voretigene neparvovec) impressed both independent experts and the Food and Drug Administration, showing the therapy improved sight and mobility in individuals with a type of inherited retinal dystrophy caused by defects in the RPE65 gene.
ICER largely agreed, citing high certainty that Luxturna delivered at least a small net health benefit to patients. In the principal clinical study used by Spark to support approval, treatment with Luxturna helped nearly all patients complete an obstacle course at low light levels faster and with better accuracy.
Unsurprisingly for a gene therapy designed to be given once, ICER's chief clinical concern focused on whether Luxturna leads to a durable, long-term benefit. "Even if improvements persist in treated cells, it remains unclear whether long-term retinal degeneration is impacted by gene therapy," the group wrote in its report.
The main question around Luxturna, however, has centered on how much Spark would charge. The biotech released details on pricing earlier this month, announcing planned outcomes-based contracts to help ameliorate the potential burden on payers as well as support for patient access.
An agreement in principle with Harvard Pilgrim, for example, would have Spark pay rebates if Luxturna's efficacy falls short of certain thresholds, measured on both a short-term horizon of 30 to 90 days and on long-term durability of 30 months.
At $425,000 per eye, Luxturna's price makes it among the most expensive treatments currently available. And, according to ICER, that cost falls well above its cost-effectiveness thresholds of between $100,000 and $150,000 per quality-adjusted life year.
When only considering direct medical care costs for patients treated at age 15, ICER calculated Luxturna would need to cost between 75% and 82% less to be cost effective. Taking societal benefits such as lower caregiver burden or greater productivity into account improved the cost picture somewhat. Still, a discount of between 50% and 57% would be required to bring Luxturna's cost into alignment with ICER's benchmark.
Spark has argued the positive effect Luxturna has on patients' quality of life and ability to participate more fully in life is integral to considering the value of the treatment.
The biotech disagrees with ICER's base-case model, although it finds more common ground with the group's second assessment.
"ICER's modified societal perspective model more accurately assesses the cost-effectiveness of voretigene neparvovec, as it incorporates the significant indirect costs associated with the disease and non-medical benefits associated with voretigene neparvovec," Spark said in an emailed statement. "In both cases, however, ICER’s analysis still fails to account for any benefit of treatment on quality of life for caregivers/family members."
Importantly, ICER only assumed a treatment benefit from Luxturna would last between 10 and 20 years. If Luxturna's effect on sight actually lasts for a lifetime, the therapy would be almost — but not quite — cost-effective by ICER's measure.
Luxturna's precedent-setting nature is also on ICER's mind.
"Policymakers also will need to consider how to manage pricing and payment arrangements for voretigene neparvovec to ensure long-term affordability, as well as patient access to the coming wave of genetic therapies for other conditions," said David Rind, chief medical officer at ICER, in a Jan. 12 statement.
Editor's note: This article has been updated to include a statement from Spark Therapeutics Inc., which returned a request for comment after publication.