The Boston-based Institute for Clinical and Economic Review (ICER) made the determination using a commonly cited cost-effectiveness threshold that values each “quality-adjusted life year” (QALY) gained at $100,000 to $150,000.
If each QALY gained were assessed at $500,000, ICER found the gene therapy, Zolgensma, would be cost effective at just over $5 million.
Novartis has said the price will be determined in negotiations with health plans, but it believes the gene therapy would be cost-effective at $4 million to $5 million as a one-time treatment.
The company said in an emailed statement on Friday that “both the rare disease community and various governmental bodies” suggest that $500,000 per QALY “is the appropriate standard to protect vulnerable populations and allow access to innovative, transformational therapies.”
SMA can lead to paralysis, breathing difficulty and death. It is the leading genetic cause of death in infants.
Gene therapies use engineered viruses to carry healthy genetic material into a person’s cells to replace faulty or mutated genes that cause a disease or condition.