ICER vs Infants in spinal muscular atrophy

Describing it as “an incomplete and poorly conceived  report from the controversial Institute for Clinical and Economic Review,” the Institute for Patient Access (IfPA) says the ICER has set its sights on a most unbecoming target: infants with a degenerative, life-threatening neurological condition.

Once dubbed “floppy baby syndrome,” spinal muscular atrophy occurs when a genetic mutation disrupts the body’s supply of motor neurons. Affected infants slowly lose muscle tone and movement. They miss milestones like sitting up or rolling over. And as their movement and mobility wanes, many are bound to strollers or wheelchairs. Some require mechanical ventilation to breathe and feeding tubes for nutrition as they lose the ability to swallow.

While many rare diseases have no effective treatment, the families of spinal muscular atrophy patients will soon be fortunate enough to have two. One is Food and Drug Administration approved; the other is in clinical trials with FDA review anticipated this spring.