Cost of SMA Drug Is Misleading, Study Suggests

Characterizing the one-time gene therapy onasemnogene abeparvovec for infants with spinal muscular atrophy (SMA) as “the most expensive ever” is misleading, argues a paper recently published in the Journal of Managed Care and Specialty Pharmacy.

“Immediately following its approval by the US Food and Drug Administration in May 2019, a National Public Radio story affirmed this with the headline: ‘At $2.1 Million, New Gene Therapy Is the Most Expensive Drug Ever,’” wrote Louis P Garrison, Jr, PhD, and Boshen Jiao, MPH, both of the University of Washington, along with Omar Dabbous, MD, of Novartis Gene Therapies Inc. “However, the article also (correctly) indicated that babies with the most severe form of the disorder (SMA type 1) typically do not live past their second birthdays.”

According to the piece, the Institute for Clinical and Economic Review estimates that lifetime costs of factor VIII for a patient with hemophilia A average between $15 million and $100 million, which is as many as 20 times the projected lifetime costs of a patient with SMA treated with onasemnogene abeparvovec.

What’s more, describing upfront costs with the tag “expensive” suggests poor value for the money. That suggestion, researchers advised, is flawed.

“To accurately assess treatment value, one must understand the return on a $2.125-million upfront investment in a baby with SMA type 1, a calculation complicated by many factors, including the expected lifetime productivity of a patient with SMA treated with onasemnogene abeparvovec, downstream medical costs, and the likely spillover effects on caregivers and family members,” the authors wrote.

The paper also points out that costs comparisons are generally conducted on a lifetime basis. Oncology drugs often run $100,000 to $250,000 a year, for example, and can be used for several years. Taking a long view when it comes to medication costs provides a more level field for comparison.

“From a scientific standpoint,” the authors wrote, “value assessment should be made based on costs and health benefits over patients’ lifetimes,” and not the upfront price.

Reference:
Garrison LP Jr, Jiao B, Dabbous O. Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies. J Manag Care Spec Pharm. 2021;27(5):674-681. doi:10.18553/jmcp.2021.27.5.674