Revised Icer report says SMA drugs are still too expensive

Spinraza is too expensive and Zolgensma is expected to be too expensive to meet value-based pricing benchmarks in infantile-onset spinal muscular atrophy, according to the report Icer will present to an expert voting panel 10 days from now. Spinraza, at $382,500 annually for the maintenance dose, costs $1.1m per quality-adjusted life year (QALY), and the gene therapy Zolgensma, at the assumed price of $2m for a one-time dose, will cost $243,000 per QALY. Icer’s calculation of Spinraza’s cost per QALY has declined from $1.6m in a draft analysis published in December, although it is still above the generally accepted $150,000 per QALY value benchmark. On the basis of life years gained rather than QALYs, Zolgensma hits the $150,000 threshold at a price of $1.5m, which SVB Leerink analyst Mani Foroohar said would represent a US price floor; Novartis has claimed that a price tag of $4-5m would be cost-effective. One edge Spinraza holds is in pre-symptomatic patients, where it has reported positive data and Zolgensma has, as yet, not. Spinraza costs $709,000 per QALY in that population, however. Both agents look worse using a conservative scenario in which those who achieve motor milestones such as sitting or walking regress; in that case, Spinraza would cost $1.5m and Zolgensma $406,000 per QALY.

Spinraza and Zolgensma against usual care in SMA
  Drug costs ($'000) Non-drug treatment costs ($'000) Total costs ($,000) QALYs Cost/QALY gained ($'000)
Spinraza 2,231 1,653 3,884 3.24 1,112
Zolgensma 2,000 1,657 3,657 12.23 243
Best supportive care n/a 789 789 0.46 n/a
Source: Icer.

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