• Researchers conducted a study analyzing the financial impact of spinal muscular atrophy (SMA).
• The study authors combined data from 15 studies that analyzed how much money is typically spent on SMA treatments and other costs.
• The cost of health care and specific SMA drug treatments can be very high, and some treatments may not be cost-effective.

Cost often plays a role when it comes to deciding on medical treatments. Getting a clearer picture of the financial impact of SMA can help families plan ahead. It can also help doctors better understand what families are going through. Finally, pharmaceutical companies and insurance companies use economic studies to help determine how much to charge and reimburse for medications.

A study published on Jan. 23 in Orphanet Journal of Rare Diseases sheds light on the economic burden that often comes with SMA. The authors found nine previously published studies that analyzed the cost of SMA care, and six additional studies that looked at whether SMA treatments were cost-effective.

When analyzing expenses related to SMA, studies looked at health care costs in addition to other types of spending. Some studies analyzed nonmedical costs, such as making the home more accessible, while others also collected data about indirect expenses. These indirect costs included things like loss of income when a parent had to miss work to provide care.

The authors found that expenses varied based on the type of SMA. Costs for treating the most common type of SMA, called SMA type 1 or Werdnig-Hoffmann disease, ranged from $75,047 to $196,429 per year. Expenses for SMA type 2, SMA type 3, and SMA type 4 fell between $27,157 and $82,474 per year.

Drugs for SMA can be extremely expensive. For example, Zolgensma (onasemnogene abeparvovec-xioi) costs $2.1 million for a one-time injection in the United States. However, some research suggests that other health care costs may decrease when these drugs are used.

This study also aimed to analyze whether SMA treatments were cost-effective. The authors compared the cost of a drug to the health outcomes that the drug could provide. Although there are medications that can reduce symptoms and improve quality of life for people with SMA, the authors concluded that these treatments could not be considered cost-effective because they were so expensive. However, they also noted that cost was not the only important factor. These treatments have other benefits, such as improving symptoms, reducing hospital admissions, and lessening the burden on caregivers and family.

The researchers speculated that as companies develop additional SMA drugs, prices may become lower over time. “With the arrival on the market of three therapies, prices should tend to decrease, which could then make the prices more acceptable,” the authors wrote.