A pair of new medicines designed to treat spinal muscular atrophy, a rare and often fatal genetic disease affecting muscle strength and movement, may be beneficial but appear to be priced too high to be considered cost effective, according to a preliminary analysis.
One drug, which is called Spinraza, costs $750,000 during the first year of treatment and $375,000 thereafter. But at that price, the Biogen (BIIB) drug fails to provide sufficient value based on QALY, or quality-of-life years, which measures both the quantity and quality of life generated by providing a treatment or some other health care intervention.
Using that benchmark, the QALY score for Spinraza was estimated at $728,000, which greatly exceeds the usual cost effectiveness thresholds of $100,000 or $150,000 in the U.S., according to the report by the Institute for Clinical and Economic Review, a nonprofit that has become an unofficial force in the country for assessing the economic benefits of new medicines.
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