Novartis gene therapy is cost effective at up to $900,000: U.S. group

An experimental gene therapy for spinal muscular atrophy (SMA) developed by Swiss drugmaker Novartis AG would offer value at a price of $310,000 to $900,000, according an independent U.S. nonprofit organization that reviews the value of drugs and medical treatments.

The report from Boston-based Institute for Clinical and Economic Review (ICER) also found that Spinraza, Biogen Inc’s treatment for SMA – a rare, often fatal, neuromuscular condition – would require a substantial price discount to be considered cost effective.

Spinraza’s list price is $750,000 for the initial year and $375,000 per year thereafter. The Novartis therapy is a one-time treatment.

SMA can lead to paralysis, breathing difficulty and death. It is the leading genetic cause of death in infants.

ICER’s assessments based on a measure known as “quality-adjusted life years” have become increasingly influential in U.S. drug price negotiations and are taken into consideration by insurers making decisions about patient access to treatments. Some drugmakers have begun consulting ICER before setting prices for new medicines.

Using an alternate measure of life years gained, ICER found that the Novartis gene therapy, Zolgensma, would have a value-based price as high $1.5 million.

Spinraza was approved in 2016 to treat SMA in both children and adults.

The U.S. Food and Drug Administration is expected to decide in the first half of this year whether to approve Zolgensma, which has been studied in infants with SMA.

ICER said its SMA report will be the subject of an upcoming meeting of the New England Comparative Effectiveness Public Advisory Council in Boston.

https://www.reuters.com/article/us-novartis-biogen-sma/novartis-gene-therapy-is-cost-effective-at-up-to-900000-u-s-group-idUSKCN1QB27K