Skipping PBC treatment leads to longer hospital stays, higher costs: Study
Timely care for newly diagnosed patients urged for reducing healthcare burden
Written by |
People with primary biliary cholangitis (PBC) who don’t start treatment after being diagnosed with the liver disease end up hospitalized more often and for longer periods than those who do, according to a new U.S. study.
Moreover, newly diagnosed PBC patients who skip treatment ultimately face substantially higher medical costs than those who start care, the researchers noted. Their study involved claims data spanning more than five years, from 2016 to 2022.
“These findings emphasize the importance of timely treatment in reducing the healthcare burden of PBC,” the researchers wrote.
Still, patients who started treatment also used significantly more healthcare resources over time, the team found. Specifically, more outpatient visits were reported for those on first-line treatment with ursodeoxycholic acid (UDCA), with longer hospitalizations for individuals on second-line or later therapies after UDCA.
This “highlights the need for new primary biliary cholangitis medications for patients who do not improve with initial treatments,” the researchers wrote. Overall, the study found a “substantial” economic burden for PBC patients.
The findings were detailed in “Economic Burden of Primary Biliary Cholangitis by Line of Therapy in the United States,” a study published in the journal Advances in Therapy. The work was funded by Ipsen, the developer of Iqirvo (elafibranor), which was approved as a second-line therapy for PBC in 2024, after the time period covered by this study.
PBC is a chronic form of cholangitis characterized by inflammation of the bile ducts, the tubes that carry the digestive fluid bile from the liver to the intestines. In patients, bile accumulates to toxic levels in the liver, damaging the organ, and leaks into the bloodstream, resulting in symptoms like itching.
UDCA treatment doesn’t work for about 40% of PBC patients
The first-line PBC treatment is UDCA, a medication sold as Urso and Actigall (with generics available) that improves bile flow. However, in about 40% of patients, the disease does not respond adequately, and second-line treatment is often needed.
Until 2024, Ocaliva (obeticholic acid) was the only approved second-line therapy for PBC in the U.S. However, it was withdrawn from the U.S. market last year due to safety concerns. Also, a type of therapy called fibrates has been used off-label among some patients with incomplete responses to UDCA.
A lack of or delayed treatment in people with PBC is associated with poor outcomes for these patients. This can involve disease progression to cirrhosis, marked by irreversible liver scarring, and liver failure. Such outcomes contribute to increased disease burden and healthcare resource use for patients.
While previous research has shown that people with PBC “have high healthcare resource use (HRU) and healthcare costs … research on HRU and costs across lines of therapy is lacking,” the scientists wrote.
To fill this knowledge gap, a research team turned to healthcare insurance claim data. The team conducted an analysis of resource use and healthcare costs across several treatment lines in PBC patients over six years.
The study involved 2,449 adults with PBC: 609 with newly diagnosed, untreated disease; 1,659 who started treatment with UDCA only, dubbed the 1L group; and 181 who started treatment with Ocaliva or fibrates after UDCA, who were identified as the 2L group.
Across the three groups, the patients had a mean age of 52.9 to 54.5 years. More than 77% were women. Those in the 2L-plus group had the highest burden of coexisting health conditions and the highest rates of itching, followed by the 1L and untreated groups.
“The proportion of patients with common PBC-related complications, [simultaneous conditions], and symptoms … increased from the untreated cohort to 1L and [2L-plus groups], highlighting the progressive nature of PBC,” the researchers wrote.
During follow-up, untreated patients were hospitalized most
During a median follow-up of 15 to 18.5 months, untreated patients had the highest hospitalization rates (24.6% vs. 16% in the 2L group and 12.4% in the 1L group). These patients were also hospitalized more often (a mean of 0.93 visits per year) and for longer periods (a mean of 7.45 days) than the 1L group (0.16 visits; 1.12 days) and 2L group (0.19 visits; 1.77 days).
These results were “likely explained by the worse prognosis and poorer survival associated with lack of treatment … [and] emphasize the importance of timely treatment in reducing the healthcare burden of PBC,” the researchers wrote.
The data showed that all groups experienced a significant increase in healthcare costs during follow-up: by $39,015 in the untreated group, $5,312 in the 1L group, and $58,492 in the 2L group.
For untreated patients, this increase was mainly driven by higher medical costs, particularly hospitalizations (by $32,088). For those in the 2L group, it was tied to increased PBC treatment costs (by $51,922).
[This study’s results] emphasize the importance of timely treatment in reducing the healthcare burden of PBC.
Overall, untreated patients had higher medical costs than the 1L and 2L groups during the follow-up period, not only in hospitalizations ($37,974 vs. $5,854 vs. $6,898) but also in outpatient ($14,298 vs. $9,377 vs. $9,613) and emergency department costs ($1,855 vs. $887 vs. $1,052). In contrast, the 2L group had the highest PBC medication costs ($53,698 vs. $2,251 in the 1L group vs. $0 in the untreated group).
These results show a “substantial economic burden in patients with PBC across different lines of treatment, including those not being treated,” the team wrote.
The researchers noted that “the development of novel, effective PBC treatments could improve patient outcomes and reduce HRU and medical costs.”
However, the team also stressed that “it is important to recognize other factors that may influence this burden, such as the effect of socioeconomic factors on access to treatment.” To that end, future study is needed, the researchers concluded.
