ICER finds Novartis' gene therapy Zolgensma cost effective up to $900 000 based on commonly used threshold; Biogen, Ionis' Spinraza would need

According to an evidence report issued Friday from the Institute for Clinical and Economic Review (ICER), the value-based price range for Novartis' proposed spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) is between $310 000 and $900 000. ICER made its determination based on a commonly cited cost-effectiveness threshold of between $100 000 and $150 000 per quality-adjusted life year (QALY) gained. However, if each QALY gained were assessed at $500 000, Zolgensma would be cost effective at just over $5 million.

Using an alternate measure of life years (LY) gained at the threshold between $100 000 and $150 000, ICER found that Zolgensma would be cost-effective between $710 000 and $1.5 million. The figure rises to just over $7 million when the threshold price is at $500 000.

A final decision regarding FDA approval of Zolgensma is anticipated in May after the treatment was granted priority review for use in patients with infantile-onset type I SMA. Novartis has said the price will be determined in negotiations with health plans, but suggested that Zolgensma, previously known as AVXS-101, could be cost-effective at $4 million to $5 million per patient. ICER noted that its estimate applies to type 1 SMA patients, and while "clinicians and families will likely consider using Zolgensma also in pre-symptomatic infants, the data are not yet available regarding its use in this population."

Commenting on the news, Novartis spokesman Eric Althoff stated that "both the rare disease community and various governmental bodies" suggest that the cost-effectiveness threshold of $500 000 per QALY "is the appropriate standard to protect vulnerable populations and allow access to innovative, transformational therapies for a small number of patients."

Meanwhile, current pricing for Biogen and Ionis Pharmaceuticals' SMA drug Spinraza (nusinersen) "would require a substantial discount to meet traditional cost-effectiveness ranges," ICER said. The list price for Spinraza, which was cleared in 2016 and is the only FDA-approved treatment for SMA in the US, is $750 000 for the first year and $375 000 for each subsequent year of therapy. 

ICER stated that based on clinical data and increased newborn screening in the US, "in the future Spinraza appears most likely to be used to treat individuals with pre-symptomatic SMA." At thresholds of between $100 000 and $150 000 per QALY gained, cost-effectivesness for Spinraza in the pre-symptomatic population ranges from $72 000 to $130 000 for the first year of treatment, and between $36 000 and $65 000 for each successive year. At the QALY value of $500 000, Spinraza was seen by ICER as cost-effective at $264 000 per year.

Meanwhile, ICER said that to reach thresholds of $100 000 and $150 000 per LY gained, Spinraza's price for pre-symptomatic patients would need to be between $83 000 and $145 000 during the initial year of therapy, and between $41 000 and $72 000 per year after that. Based on an LY value of $500 000, Spinraza was deemed cost-effective at $289 000 per year, the report said.

ICER chief medical officer David Rind commented that "at its current pricing, Spinraza far surpasses common thresholds for cost-effectiveness." However, he said "Novartis has a real opportunity here to demonstrate both scientific and ethical leadership by setting the launch price of Zolgensma in line with the benefits patients will likely receive."

In December, ICER released a draft of the report indicating that Novartis' gene therapy could be more cost-effective than Spinraza once additional information became known about its US price and long-term success rates.

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