An experimental gene therapy from Novartis (NVS) will be more cost effective than Biogen (BIIB) and Ionis Pharmaceuticals‘ (IONS) treatment for a muscle-wasting disease, experts said this week.
Biogen and Ionis grabbed approval for Spinraza in late 2016. So far, it’s the only drug on the market in the U.S. to treat spinal muscular atrophy, a genetic disease of the nerves that control muscles. But Novartis is hoping to follow in 2019 with its gene therapy called Zolgensma.
If priced at the $2 million mark, Zolgensma is more cost effective than Spinraza, experts with the Institute for Clinical and Economic Review said in a report late Thursday.
“These findings could offer support for Novartis in pricing and access negotiations for Zolgensma ahead of the 2019 estimated approval,” Leerink analyst Geoffrey Porges said in a report to clients on Friday.
Can Gene Therapy Reduce Cost Over Time?
The report measures disease burden over time. It uses a metric called a quality-adjusted life year. One quality-adjusted life year is equal to one year of life in perfect health.
Novartis acquired Zolgensma in an $8.7 billion takeover of AveXis earlier this year. The company suggests its gene therapy could yield 13.3 quality-adjusted life years in patients who show symptoms in the first six months of life, known as type 1.
Zolgensma “could be priced to $4 million to $5 million based on the relationship of 10-year cost of approved drugs for ultrarare diseases and their corresponding incremental quality-adjusted life years gained,” Porges said.
Gene therapy is meant to be administered just one time, which would cut the cost of chronic treatment over time. But the ICER report suggests Zolgensma would provide just 11.33 quality-adjusted life years at a placeholder price of $2 million.
Porges estimates Novartis’ gene therapy will net $1.57 million per patient across all types of spinal muscular atrophy. This is equivalent to five years of Spinraza net pricing.
Spinraza Costs Add Up Over Time
ICER estimated the real-world cost of Spinraza would equal $11.94 million to gain 21.54 years of perfect health. This would be in patients who don’t yet show the symptoms. This group benefits the most in that Spinraza could prevent their symptoms.
At this measure, Spinraza costs $728,000 for each additional year of perfect health. The report didn’t offer a similar look for Zolgensma. But at $2 million, Zolgensma would cost $247,000 per added quality-adjusted life year in patients who show symptoms as newborns.
The report suggests a one-time drug as effective as Spinraza would cost $161,000 per added year of perfect health. That’s a nearly 80% discount to Spinraza in presymptomatic patients, Porges said.
“The high cost/quality-adjusted life year of Spinraza in type 1 spinal muscular atrophy patients offers Novartis bargaining power with payers if the company can argue Spinraza use could be reduced or eliminated to offset total costs,” he said.
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