If it’s your loved one, “priceless.” In medical malpractice litigation, a “wrongful death” in a non-wage earner is valued at $200,000. “Payors” of health insurance claims generally approve $100,000 for a multiple sclerosis medication that provides a quality-of-life year (QALY). But what about a drug that prevents an otherwise certain death?

This is the dilemma of the new high priced spinal muscular atrophy (SMA) drugs. How is society going to pay for them? SMA, a rare genetic disorder with 500 new cases diagnosed each year, is the most common cause of death in infants. There are now 1100 infants under age 2 diagnosed with SMA Type 1 and destined to die or require permanent ventilation by age 2. The milder Type 2 form affects toddlers who never walk independently but can survive into adulthood with intensive supportive care.

The FDA has recently approved 2 medications for SMA-1. The first is Spinraza administered via lumbar puncture (spinal tap) four times in the first 2 months and every 4 months thereafter. The earlier studies extended only to age 5 but children up to age 12 are now being treated and there is no consensus as to how long to continue treatment. It is a disease modifying therapy, not a cure, and costs $750,000 for the first year and $375,00 for subsequent years.

The second is Zolgensma, a gene therapy, and costs $2.1 million for a single dose. Long-term efficacy is unknown but it may prove to be cheaper than Spinraza. Some families are requesting treatment with both drugs simultaneously.

The medications pose a serious financial risk for institutions and hospitals that administer them. They must “buy and bill” and hope that insurers will reimburse them. Incredibly, prior authorization does not guarantee payment!

See Neurology Today, August 22, 2019.

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