— Independent appraisal committee notes family testimony in votes confirming broad benefits of both treatments; however, committee votes unanimously that price for Spinraza is too high to align fairly with these benefits, and urges fair pricing for emerging gene therapy Zolgensma to support sustainable access to innovation —

BOSTON, April 3, 2019 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance 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. ICER’s assessment was reviewed at the March 2019 public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.

“Both Spinraza and Zolgensma dramatically improve the lives of children with SMA and that of their families,” said David Rind, MD, ICER’s Chief Medical Officer. “However, the current price of Spinraza far exceeds common thresholds for cost-effectiveness. The price of Zolgensma is not yet known, but there has been public discussion of prices above commonly accepted cost-effectiveness thresholds as well. These treatments will be covered by US insurers regardless of the pricing, but the ripple effect of pricing decisions like these threatens the overall affordability and sustainability of the US health system.”

Independent Voting on Clinical Benefits, Contextual Considerations, and Economic Value

The New England CEPAC unanimously voted that the clinical evidence is sufficient to show a net health benefit for Spinraza and Zolgensma in Type I SMA, and for Spinraza in the later-onset and pre-symptomatic populations. During their deliberation, the Council reflected on the testimony from families and clinical experts documented within the ICER report and provided in-person at the public meeting. Council members voted to affirm several important broader benefits of both therapies, including the ability of patients to gain dignity and independence even from very small improvements in motor function, and broader effects on families, including greater freedom, reduced stress, and lower financial burden related to transportation and other costs. Council members also unanimously recognized that Zolgensma, administered in a one-time dose, offers less complexity than Spinraza.

While acknowledging these substantial clinical and other benefits of Spinraza, the Council voted unanimously that the treatment is priced in such a way that it represents a low long-term value for money. To reach commonly cited cost-effectiveness thresholds of $100,000-$150,000 per Quality-Adjusted Life Year (QALY) gained, Spinraza’s price for patients with presymptomatic SMA would need to be between $72,000-$130,000 for the first year of treatment and between $36,000-$65,000 for each successive year. To reach alternative thresholds of $100,000-$150,000 per Life Year Gained (LYG), Spinraza’s price for pre-symptomatic patients would need to be between $83,000-$145,000 during the initial year and $41,000-$72,000 for each successive year. However, the current list price of Spinraza, excluding any mark-ups for providers, is $750,000 for the initial year and $375,000 per year thereafter.

The Council did not vote on the long-term value for money of Zolgensma because the treatment’s price is not yet known. To reach commonly cited cost-effectiveness thresholds of $100,000-$150,000 per QALY gained, Zolgensma’s price for Type I SMA would need to be between $310,000-$900,000 per treatment. To reach alternative thresholds of $100,000-$150,000 per LYG, Zolgensma’s price for Type I SMA would need to be between $710,000-$1.5 million per treatment.

For treatments of ultra-rare disorders like SMA, 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.

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including clinicians, patient advocates, and manufacturer and payer representatives – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • To align reasonably with the benefits for patients and families, the price for Spinraza should be far lower than it is, and the price for Zolgensma should be lower than the hypothetical $4-5 million price the manufacturer has suggested could be justified. To achieve the needed balance between incentives for innovation and health system affordability, all manufacturers should exercise their monopoly pricing power responsibly, setting prices that do not exceed a reasonable cost-effectiveness threshold.
  • Payers should negotiate outcomes-based contracts under which a substantial portion of treatment cost is at risk should patients not receive adequate clinical benefit. Outcomes measures should extend beyond death and permanent ventilation, which might not be able to capture near-term lack of benefit for some Type I patients and are inadequate measures for treatment of later-onset or presymptomatic patients.
  • Biogen’s high-quality clinical trials of Spinraza should provide a model for others investigating treatments for ultra-rare conditions.
  • Patient organizations should view their longer-term mission in support of patients to include active engagement with manufacturers to demand reasonable value-based pricing of the therapies that patients and their families helped bring to the market.
  • Individual clinicians and clinical specialty societies should work to address insurance barriers to inappropriate care, be vocal witnesses to the negative effects of excessive prices on patients and their families, integrate considerations of value into clinical guidelines, and work towards a health system that improves access and affordability while continuing to incentivize innovation.

ICER’s detailed set of policy recommendations, including considerations for establishing prior authorization criteria, is available in the Final Evidence Report.

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.