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)|
|Best supportive care||n/a||789||789||0.46||n/a|