ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy

–ICER’s update reflects the gene therapy’s new clinical data and FDA label–

BOSTON, May 24, 2019 — In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, the Institute for Clinical and Economic Review (ICER) today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA).

Since the publication of ICER’s Final Evidence Report on April 3, 2019, early results of a Phase III single arm trial (SPR1NT) have been encouraging for the use of Zolgensma in patients with presymptomatic SMA. ICER’s Final Evidence Report had included a hypothetical scenario analysis that modeled the cost-effectiveness of Zolgensma assuming a level of efficacy in the presymptomatic population that may be similar to what is now observed in early results from the SPR1NT trial:

  • To reach commonly cited cost-effectiveness thresholds of $100,000 to $150,000 per quality-adjusted life year (QALY) gained, a value-based price benchmark for Zolgensma would be between $1.1 million to $1.9 million per treatment.
  • To reach the alternative thresholds of $100,000 to $150,000 per life year gained (LYG), a value-based price benchmark for Zolgensma would be between $1.2 million to $2.1 million.

“Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given the new efficacy data for the presymptomatic population, the price announced today falls within the upper bound of ICER’s value-based price benchmark range,” said Steven D. Pearson, MD, MSc, President of ICER. “Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million. It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families.”

This updated value-based price benchmark for Zolgensma assumes that the treatment’s effectiveness will be long lasting, and that the US widely and rapidly adopts the recommendation to add screening for SMA to routine newborn screening. Once additional data are available, ICER may perform a New Evidence Update for both Zolgensma and nusinersen (Spinraza®, Biogen).

ICER’s commentary on this new evidence is now included within the executive summary of 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.