ICER’s Assessment Finds Spinraza and Zolgensma Provide Substantial Health Benefits for People with Spinal Muscular Atrophy

–Current pricing of Spinraza would require a substantial discount to meet traditional cost-effectiveness ranges; Zolgensma’s value-based price range is between $310,000-$900,000 using standard methodology but as high as $1.5 million using alternative measures of health gain–

–As with all treatments for ultra-rare conditions, judgments of overall value require consideration of contextual issues and broader benefits for patients and families; these additional factors will be discussed at a March 7 public meeting–

BOSTON, February 22, 2019 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of nusinersen (Spinraza®, Biogen) and onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis) for the treatment of spinal muscular atrophy (SMA). Spinraza was approved in 2016 for treatment of SMA in both children and adults. Zolgensma is a gene therapy that has been studied in infants with Type I SMA, and an FDA decision is expected in the first half of 2019.

“Both of these treatments appear to dramatically improve the lives of children with SMA, as well as the families who take care of them,” said David Rind, MD, ICER’s Chief Medical Officer. “And while Spinraza has a broader body of evidence that provides more certainty around the health benefits patients may receive, the limited data on Zolgensma suggest that the gene therapy has the potential to deliver large benefits through a one-time treatment. Unfortunately, at its current pricing, Spinraza far surpasses common thresholds for cost-effectiveness. Among the various companies that are now bringing gene therapies to market, 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.”

This Evidence Report will be the subject of an upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), in Boston on March 7, 2019. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

Evidence provides high certainty that both Spinraza and Zolgensma provide a substantial net health benefit compared to prior standard care in patients with infantile-onset (Type I) SMA. Differences in studied populations and trial design pose challenges when distinguishing between the two treatments for Type I SMA; the clinical data for Spinraza are of much higher quality – including multiple randomized, placebo-controlled trials – than those for Zolgensma.

Evidence provides moderate certainty of Spinraza’s small-to-substantial benefit for patients with later-onset SMA. Spinraza also appears to be of benefit in presymptomatic SMA, with the evidence limited by the lack of published data. There are no data available to assess the net benefit of Zolgensma in presymptomatic or later-onset SMA populations.

Key Cost-Effectiveness Findings

ICER’s value-based price benchmarks suggest a price range, net of any discounts and rebates, that aligns fairly with a treatment’s added benefits for patients over their lifetime. The ranges reflect commonly cited cost-effectiveness thresholds of between $100,000 and $150,000 per Quality-Adjusted Life Year (QALY) gained.

  • Based on clinical trial results and increased newborn screening in the US, in the future Spinraza appears most likely to be used to treat individuals with presymptomatic SMA. For this population, ICER’s value-based price benchmark for Spinraza, inclusive of any mark-up for providers, is between $72,000-$130,000 for the first year of treatment when loading doses are required, and between $36,000-$65,000 for each successive year. Currently, excluding any mark-ups, the list price of Spinraza is $750,000 for the initial year and $375,000 per year thereafter.
  • ICER’s value-based price benchmark for Zolgensma, which is expected to be administered only once in a patient’s lifetime, is between $310,000-$900,000 per treatment in the infantile-onset (Type I) population – the only population in which the gene therapy has been studied. Although clinicians and families will likely consider using Zolgensma also in presymptomatic infants, the data are not yet available regarding its use in this population. The FDA has yet to approve the treatment, so the labeled indication remains unknown at this time.

Consistent with ICER’s commitment to provide a broader view of cost-effectiveness, this Evidence Report also highlights ICER’s value-based price benchmark based on a complementary measure of the ability of treatments to benefit patients: the Life Year Gained (LYG). To reach thresholds of $100,000 and $150,000 per LYG, Spinraza’s price for presymptomatic patients would need to be between $83,000-$145,000 during the initial year and $41,000-$72,000 per year thereafter*, and Zolgensma’s price for Type I patients would need to be between $710,000-$1.5 million per treatment.

We note that for treatments of ultra-rare disorders, insurers and other decision-makers often give added weight to contextual considerations that lead to acceptance of prices higher than those that would meet traditional cost-effectiveness ranges. Therefore, ICER’s report also includes multiple threshold price analyses for both drugs, ranging from $50,000-$500,000 per QALY and per LYG.

Register here to attend the New England CEPAC meeting in person or to watch by live webcast.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.

*NOTE: An earlier version of this press release, but not the Evidence Report, misstated the cost/LYG value-based prices for Spinraza. The Evidence Report remains accurate, and this has now been corrected within the press release. We regret any confusion. (Updated 2/22/2019)