Rare Daily Staff

The Institute for Clinical and Economic Review, an independent, nonprofit that analyzes the cost-effectiveness of therapeutics, issued a report that found Biogen’s SMA drug Spinraza is not cost-effective as priced and warned that Novartis’ experimental gene therapy Zolgensma, if priced as expected, would also be too pricey to be cost effective.

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. It is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The most severe form of SMA is type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients.

“Both Spinraza and Zolgensma dramatically improve the lives of children with SMA and that of their families,” said David Rind, ICER’s chief medical officer. “However, the current price of Spinraza far exceeds common thresholds for cost-effectiveness. The price of Zolgensma is not yet known, but there has been public discussion of prices above commonly accepted cost-effectiveness thresholds as well.”

Rind said while these treatments will be covered by U.S. insurers regardless of the pricing, the ripple effect of pricing decisions like these “threatens the overall affordability and sustainability of the U.S. health system.”

The organization said the clinical evidence is sufficient to show a net health benefit for Spinraza and Zolgensma in Type I SMA, and for Spinraza in the later-onset and pre-symptomatic populations. It noted testimony from families and clinical experts affirmed the benefits of both therapies, which included a gain in dignity and independence for patients from improvements in motor function; and greater freedom, reduced stress, and lower financial burden related to transportation and other costs for families.

Nevertheless, ICER found that Spinraza is priced in such a way that it represents a low long-term value for money. Spinraza costs $750,000 per patient in the first year and $375,000 each year thereafter. Using commonly cited cost effectiveness thresholds based on quality-adjusted life years gained, ICER said, Spinraza would need to be priced at between $72,000-$130,000 for the first year of treatment and between $36,000-$65,000 for each successive year to be cost-effective.

Using an alternative method based on life years gained, it said, Spinraza’s price for pre-symptomatic patients would need to be between $83,000-$145,000 during the initial year and $41,000-$72,000 for each successive year.

ICER did not offer a conclusion on the long-term cost-effectiveness of Zolgensma because the treatment’s price is not yet known. It did say, though, that it would need to be priced between $310,000 to $900,000 per treatment. To reach alternative thresholds based on life years gain, Zolgensma’s price for Type I SMA would need to be between $710,000-$1.5 million per treatment.

Novartis has said the therapy could justify a price of between $4 million and $5 million per patient, although it has not yet said how it will price the therapy.

Among its recommendations, ICER said payers should negotiate outcomes-based contracts under which a substantial portion of treatment cost is at risk should patients not receive adequate clinical benefit. It said outcomes measures should extend beyond death and permanent ventilation, which might not be able to capture near-term lack of benefit for some type I patients and are inadequate measures for treatment of later-onset or pre-symptomatic patients.

Photo: David Rind, ICER’s chief medical officer

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