Study says U.S. Sanfilippo societal cost will top $2B over 20 years
Treatment improvements could reduce burden, researchers say
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The total economic burden of Sanfilippo syndrome in the U.S. is on track to exceed $2 billion in the next 20 years, according to a new analysis.
The figure accounts for the direct costs of managing the condition, along with lost productivity for patients and their families. While Sanfilippo is rare, the overall impact on society is significant, so treatments that could improve the lives of affected families could generate substantial value, the researchers said.
“If treatment plans could recover just 25% of morbidity and mortality associated with the burden of disease for [Sanfilippo], it would potentially be collectively valued at tens of millions of dollars per year based on our estimates,” the researchers wrote.
The study, “Economic burden of Sanfilippo syndrome in the United States,” was published in the Archives of Public Health. The National Institutes of Health funded the work.
Sanfilippo syndrome is a genetic disorder that causes progressive neurological damage, resulting in a range of developmental and cognitive issues. There are no treatments available for the life-limiting disorder.
Costs to families run beyond medical bills
Rare diseases like Sanfilippo syndrome are often costly to manage. And the direct costs associated with managing the disease aren’t the only impact: People with Sanfilippo usually have minimal ability to participate in the workforce, and caring for a child with Sanfilippo takes a logistical and mental health toll on the child’s parents.
Researchers at the University of Florida conducted an analysis to estimate the total economic burden of Sanfilippo syndrome, including direct costs of disease care and caregivers’ lost productivity. The scientists noted that few studies have attempted to put a dollar figure on the societal costs of rare diseases like Sanfilippo.
“Understanding the value of the disease can help inform policy and funding decisions,” the researchers wrote.
The analysis estimated that the total economic burden of having a child with Sanfilippo syndrome is roughly $8 million, with some variation among the different types of Sanfilippo.
“Each family who bears a child afflicted with [Sanfilippo] can expect to lose on average a present value between $0.69-$1.07 million in lost labor value, $40.1-$56.0 thousand in medical out-of-pocket expenses, and $253-$363 thousand in non-medical costs,” the researchers wrote. “The family will also lose between $3.10 and $4.60 million present value in health – for both patient and caregiver.”
Based on birth rates and Sanfilippo prevalence, the researchers estimated the total economic burden for all Sanfilippo patients in the U.S. from 2023 to 2043. They calculated the disease’s total burden as $2.19 billion.
A notable caveat of the analysis is that the researchers’ models assumed that care for Sanfilippo in 2043 will be the same as it is today, which is unlikely to be the case, as treatments are in development and supportive care tends to improve over time.
Hypothetically, a treatment given at birth that fully cures all aspects of the disease would deliver roughly $8 million in value to society, the researchers said. But they stressed that this figure is “a representation of the total potential present value of curative treatment from the point of childbirth, not a price target.”
