ABOVE: ©ISTOCK, JOHNNYGREIG
The US Food and Drug Administration recently approved two drugs that target the genetic error underlying spinal muscular atrophy (SMA), a muscle-wasting disorder, often fatal before the age of two, that was previously untreatable. The treatments—Zolgensma from Novartis Gene Therapies and Spinraza from Biogen—come with hefty price tags, yet a report in Science Translational Medicine today (November 11) argues that they offer only modest benefits with possible health risks, leaving patients with unmet medical needs and possibly with substantial healthcare bills.
“This paper highlights the complexity of developing treatments for rare diseases and paying for those treatments,” health policy researcher Stacie Dusetzina of Vanderbilt University who was not involved in the research writes in an email to The Scientist. “The review of outcomes for SMA treatments are particularly sobering, with small but meaningful gains in motor response outcomes for some infants with SMA . . . but ...
