Fighting Spinal Muscular Atrophy: How Much Is a Child’s Life Worth?

Drug with a $750,000 price tag creates ethical dilemmas

Spinraza treats spinal muscular atrophy (SMA), a disease that saps muscle strength, with sufferers not being able to sit or stand or even, as it progresses, breathe. A year of treatment costs $750,000. A Kaiser Health News article on the headaches and heartaches engendered by the hope that such a medication creates begins with the tale of two children afflicted with SMA. One has private insurance and can get the drug. The other child is covered by Medicaid, which chooses not to cover so long as the child can breathe on a ventilator.

About 10,000 people across America suffer from SMA, and a good proportion of those are infants and children. Spinraza is an “orphan” drug. Orphan drug designation gives pharmaceutical companies motivation to develop medications for diseases that affect fewer than 200,000 people a year, providing a package of incentives for research and development.

The FDA offers seven years of patent protection if the drug proves effective, but does not rule on pricing. Spinraza is not a cure, but 40% of infants given the medication in one clinical trial did show improvement. The FDA approved the drug last December.

“Five days after getting the FDA’s approval to sell the drug in the U.S., Biogen announced the price: $125,000 a dose, or $750,000 for the first year,” Kaiser Health News reports. “Fewer doses in following years drop the total annual cost to $375,000. The drug must be taken for life.”

But there are questions. Will it work on infants who are on ventilators? How effective will it be for older children and adults? Just how to measure if the drug is actually stalling the disease’s progression? How long will it last?

Then, of course, there are the financial trade-offs. Jack Hoadley, health policy analyst at Georgetown University's Health Policy Institute, tells KHN: “Treating one of these patients may mean not treating 1,000 patients with some other, less expensive problem — or saying they have to raise more tax dollars. Private insurers have the same trade-off. Do we pay for this if it will ultimately raise our premiums?”

Source: Wall Street Journal