AMCP 2022: Patients with Rare Liver Disease Need Treatment Adherence Support

Although patients had insurance coverage for Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis, a rare live disease, many patients were not adherent to the treatment regimen, according to a poster presented at the annual meeting of the Academy of Managed Care Pharmacy held in Chicago, from March 29, 2022, to April 1, 2022.

Analysts at AllianceRx Walgreens Prime evaluated Ocaliva, developed by Intercept Pharmaceuticals, in patients diagnosed with primary biliary cholangitis for patient-reported adverse events, cost, and utilization. Ursodiol is first-line therapy for primary biliary cholangitis; but about 40% of patients do not have sufficient improvement in their liver function while on therapy and need another option. The FDA approved Ocaliva in May 2016 for patients who don’t have a response to ursodiol.

Primary biliary cholangitis is a rare, chronic autoimmune disease primarily affecting women that causes the small bile ducts in the liver to become inflamed, damaged and ultimately destroyed. This causes bile to remain in the liver, which damages the liver cells over time, and results in cirrhosis.

In this evaluation, the research, done in conjunction with Duquesne University School of Pharmacy, measured adherence by using proportion of days covered (PDC) over a 12-month period, beginning from each patient’s index date.

Researchers found that between September 1, 2019, to December 31, 2021, a total of 982 patients received a dispensed prescription for Ocaliva. Of those, 715 patients met the study inclusion criteria.

Kajal Patel, Pharm.D.

The out-of-pocket copay was lowest for patients with commercial insurance, accompanied with a patient assistance program. Although the average wholesale price of Ocaliva is costly ($9,653.41 for a 30-day supply), most patients (94.1%) had an average copay of less than $50. looking at adherence, most patients (449/715) had a mean proportion of days covered greater than 80% with an overall proportion of days covered of 77.1%.

Additional research is required, researchers said, because this study did not examine discontinuation rates and reasons.

“It’s possible the overall PDC may have been impacted by patient discontinuations as well as changing of insurance and specialty pharmacy provider during the 12-month measurement period from their index date,” Kajal Patel, Pharm.D., PGY-1 resident at AllianceRx Walgreens Prime and lead author/analyst of the study, said in a press release. “In future studies, it may be important to investigate additional factors to have a better understanding of potential barriers associated with adherence."

The most common side effects seen were itching (47%) and fatigue (13%), which were also common in the clinical trials.