An incomplete and poorly conceived report from the controversial Institute for Clinical and Economic Review 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 FDA approved; the other is in clinical trials with FDA review anticipated this spring.
But perhaps two treatment options is too many for ICER.
In its latest evidence report, the group sets out to determine whether the two spinal muscular atrophy treatments are cost-effective. As with many of ICER’s previous reports, the exercise is premature. Neither therapy has long-term data. And the newer therapy does not yet have a price. But that doesn’t stop ICER.
ICER concludes that neither the existing therapy, called “nusinersen,” nor a new gene therapy meets ICER’s cost-effectiveness threshold. But ICER also effectively pits the two drugs against each other, suggesting that the new gene therapy could at least be more cost-effective than existing treatment – if it assumes the price point concocted by ICER.
Comparing the two drugs is hardly apples to apples. Even ICER admits it is “naïve.”
The existing treatment, nusinersen, is a periodic spinal injection that boosts the body’s production of a vital protein. It’s treated thousands of patients since first approved in 2017.
AfPA member Safi Shareef, MD, a pediatric neurologist, recalled seeing a baby girl with spinal muscular atrophy who began treatment with nusinersen. “Suddenly, she started to meet her milestones. She was starting to sit up, starting to stand up,” Dr. Shareef recalled, calling the progress “miraculous.”
The newer treatment is a gene therapy, a one-time infusion that replaces the defective gene by inserting an inactivated virus into the body. The data used by ICER is from the drug’s Phase I clinical trial, conducted on 15 patients. Early results show promise, and the possibility of treatment by a single infusion as opposed to periodic injections is an attractive possibility.
But neither the disease nor its treatment is one-size-fits-all. Many families would welcome a single-infusion treatment, Dr. Shareef hypothesized, though others may choose differently. “Families may elect not to do gene therapy if, for example, their child is prone to severe complications from viral illness,” Dr. Shareef explained.
The decision between treatments rightfully belongs to a patient’s family and their health care provider. But families may not have a say in the matter if insurers listen to ICER. In the past, the organization’s findings have influenced both Medicaid and commercial health plan coverage, with CVS Health announcing last year that its plan formularies could exclude drugs that didn’t meet ICER’s threshold.
Facing a devastating diagnosis, families of infants with spinal muscular atrophy don’t need ICER economists streamlining their already sparse treatment options. And they don’t need deeply personal medical decisions made for them based on what’s cost effective for the system.
They need options, and they need access.
ICER is accepting public comments on its report until January 31.
