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

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

Novartis, which did not immediately respond to a request for comment, has said the therapy’s price will be determined in negotiations with health plans. But the drugmaker has said it believes the gene therapy would be cost-effective at $4 million to $5 million as a one-time treatment. [L8N1YO5Y8]

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.

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 decrease to be considered cost effective.

Spinraza’s list price is $750,000 for the initial year and $375,000 per year thereafter, versus a likely one-time cost for the Novartis therapy with a proposed brand name of Zolgensma.

Biogen, in an emailed statement, said ICER’s analysis fails to account for the fact that Zolgensma is an experimental therapy with reported results to date for only 15 patients, compared with Spinraza’s track record of use by more than 6,600 patients. Spinraza is considered by Wall Street to be one of Biogen’s most important growth drivers.

ICER derived its proposed value of Zolgensma using a commonly cited cost-effectiveness threshold of between $100,000 and $150,000 per “quality-adjusted life year” (QALY) gained.

The group’s assessments 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 known as “life years gained” (LYG), ICER found the Novartis gene therapy would have a value-based price of between $710,000 and $1.5 million.

ICER found that Spinraza, when used for patients who do not yet show symptoms, would have value at $72,000 to $130,000 for the first year and between $36,000 and $65,000 for each successive year. Based on the LYG measurement, the price would need to be $72,000 to $82,000 during the initial year and $36,000 to $41,000 per year thereafter, according to the ICER report.

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

Spinraza was approved in 2016 to treat SMA in 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.