The Institute for Clinical and Economic Review (ICER) on Wednesday issued a final report indicating that Biogen and Ionis Pharmaceuticals' spinal muscle atrophy (SMA) therapy Spinraza (nusinersen) is not cost-effective at its current price. The group also noted that Novartis' SMA treatment Zolgensma (onasemnogene abeparvovec), for which an approval decision by the FDA is expected next month, should be priced below the drugmaker's hypothetical value of $4 million to $5 million. 

"Both Spinraza and Zolgensma dramatically improve the lives of children with SMA and that of their families," commented ICER chief medical officer David Rind, adding "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."  

Specifically, ICER concluded that using cost-effectiveness thresholds of $100 000 to $150 000 per quality-adjusted life year (QALY), Spinraza should carry a cost of $72 000 to $130 000 for its first year and $36 000 to $65 000 for each successive year. Using the alternative thresholds of $100 000 to $150 000 per life year gained (LYG), the drug should be priced at $83 000 to $145 000 in its first year and $41 000 to $72 000 for each additional year. Upon its approval in 2016, Biogen and Ionis priced the drug at $750 000 for its first year and at $375 000 per patient in subsequent years of therapy. 

Meanwhile, at cost-effectiveness thresholds of $100 000 to $150 000 per QALY, ICER concluded that Zolgensma should be priced at $310 000 to $900 000 per treatment in patients with type I SMA. At thresholds of $100 000 to $150 000 per LYG, the group estimated that Zolgensma should carry a per-treatment cost of $710 000 to $1.5 million. 

In addition, ICER also called for providers, payers and manufacturers to enter into "outcomes-based contracts" regarding funding for the drugs. The group noted that outcome measures should extend beyond death and permanent ventilation, arguing that the measures may not capture short-term lack of benefit in some patients with type I SMA and that they are not suitable for assessing patients with later-onset or pre-symptomatic disease. 

ICER previously issued guidance calling for a "substantial discount" to Spinraza, while estimating that Zolgensma could be cost-effective at up to $900 000 using standard thresholds. The institute also suggested that Zolgensma could be more cost-effective than Spinraza once more information about its US price and success rates became available.

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