New England CEPAC

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SMA: Report-at-a-Glance

An at-a-glance summary of ICER’s full report.

NOTE: On May 24, 2019, ICER issued an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA). This update reflected the FDA approval of Zolgensma, as well as new clinical trial data for Zolgensma that suggest a substantial benefit for individuals with presymptomatic SMA, and at least a small benefit for individuals with Type II SMA. Stakeholders may prefer to consider the economic analyses of Drug X presented in the report when assessing the value of Zolgensma. Value-based price benchmarks for Drug X would range from $1.1 to $1.9 million (prices to reach $100,000 to $150,000 per QALY gained) and from $1.2 to $2.1 million (prices to reach $100,000 to $150,000 per life year gained). Additional details can be found in the full Final Evidence Report.

Other than the May 24 addendum, no other portions of this Report-at-a-Glance have been updated.


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