Biogen SMA drug price, Novartis estimates for its treatment far too high: U.S. group

Biogen Inc should slash the price of its spinal muscular atrophy (SMA) drug, and the $4 million to $5 million Novartis has said its experimental gene therapy for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said on Wednesday.

Assessments by the nonprofit Institute for Clinical and Economic Review (ICER) have become increasingly influential in U.S. drug pricing and are taken into consideration by health insurers and pharmacy benefit managers making decisions about payments and patient access to treatments.

Boston-based ICER has been looking at Biogen`s Spinraza and Novartis` Zolgensma since last year. The extremely high cost of new treatments for rare conditions has placed SMA at the centre of the drug affordability debate.

In its final report that largely mirrors a draft issued in February, ICER urged the drugmakers to balance innovation with affordability for health systems, which translates to significantly lower prices.

The group said Spinraza`s list price of $750,000 for the initial year and $375,000 annually thereafter needs to be "far lower" to be cost effective.

Spinraza, an important growth driver for Biogen, took in $1.7 billion in 2018 sales.

To determine value, ICER used a measure known as "quality-adjusted life year” (QALY), in which each year of healthy or near-healthy life resulting from the treatment is worth $100,000 to $150,000.

SMA can lead to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form of the disease. It is the leading genetic cause of death in infants.

Novartis has not set a price for Zolgensma, which could get U.S. approval next month. The Swiss drugmaker has said its one-time treatment would be cost effective at up to $5 million.

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 price for Zolgensma should be lower than the hypothetical $4-$5 million price the manufacturer has suggested could be justified," ICER concluded.

Using the QALY benchmark, ICER said Spinraza would need to cost between $72,000 and $130,000 for the first year of treatment, and cost $36,000 to $65,000 per year after that, for infants not yet showing symptoms of the disease.

With an alternative benchmark, known as life-year gained (LYG) based on the additional number of years a person lives due to a treatment, Spinraza is worth $83,000 to $145,000 in year one, and $41,000 to $72,000 annually thereafter, ICER determined.

Zolgensma would be worth $310,000 to $900,000 for Type 1 SMA patients based on the QALY assessment, or $710,000 to $1.5 million using the LYG calculation, ICER said.

"These treatments will be covered by U.S. insurers regardless of the pricing, but the ripple effect of pricing decisions like these threatens the overall affordability and sustainability of the U.S. health system," said ICER Chief Medical Officer Dr. David Rind.

Novartis contends the rare disease community believes that $500,000 per QALY is a more-appropriate standard for transformational therapies. At that level, Zolgensma would be cost effective at $5 million.

"The value measures and thresholds employed by ICER in this report are designed around the status quo of chronic care management and cannot possibly capture the full benefits of disease-modifying treatments delivered as a one-time administration," Novartis said in a statement.

Biogen, in an emailed statement, said Spinraza is the standard of care for SMA and has benefited more than 6,600 people. It noted that Novartis has so far reported Zolgensma results for only 15 patients.

ICER also recommended providers, payers, and manufacturers cooperate on so-called "outcomes-based contracts." Such arrangements pay drugmakers based on the effectiveness of their treatments or offer refunds if they do not work.

(This article has not been edited by Zeebiz editorial team and is auto-generated from an agency feed.)