BACKGROUND: Chronic liver disease and cirrhosis are a leading cause of morbidity 
and mortality in the United States. Primary biliary cholangitis (PBC), 
previously known as primary biliary cirrhosis and which has been designated an 
orphan condition, is a chronic autoimmune disease resulting in the destruction 
of the small bile ducts in the liver. Without effective treatment, disease 
progression frequently leads to liver failure and death. Until May 2016, the 
only FDA-approved treatment for PBC was ursodiol (UDCA), an oral hydrophilic 
bile acid, which can slow progression of liver damage due to PBC. However, 1 out 
of 3 patients taking UDCA has an inadequate biochemical response, leading to 
increased risk of disease progression, liver transplantation, and mortality. 
Given this unmet clinical need, new therapies are in development for the 
treatment of PBC. To provide pharmacists with an overview of the latest research 
on the pathophysiology of PBC and potential new treatment options and to 
highlight medical and specialty pharmacy approaches to managing access to drugs 
to treat orphan diseases such as PBC, a 2-hour satellite symposium was presented 
in conjunction with the 2015 Academy of Managed Care Pharmacy (AMCP) Nexus 
meeting. Although obeticholic acid was approved by the FDA for the treatment of 
PBC in May 2016, this development occurred after the symposium presentation. The 
symposium was supported by an independent educational grant from Intercept 
Pharmaceuticals and was managed by Analysis Group. Robert Navarro, PharmD, 
moderated the CPE-accredited symposium titled "Medical and Specialty Pharmacy 
Management Update on Primary Biliary Cirrhosis." Expert panelists included 
Christopher L. Bowlus, MD; James T. Kenney, RPh, MBA; and Gary Rice, RPh, MS, 
MBA, CSP.
OBJECTIVE: To summarize the educational satellite symposium presentations and 
discussions.
SUMMARY: Autoimmune liver diseases, including PBC, are responsible for 15% of 
all liver transplants performed and an equal percentage of deaths related to 
liver disease. UDCA is the only FDA-approved therapy for treatment of PBC and is 
considered the standard of care. Nevertheless, many patients do not respond to 
UDCA, creating the need for new therapeutic options to improve clinical outcomes 
for PBC patients with inadequate response to treatment. While several agents are 
being studied in combination with UDCA, monotherapy with the novel agent 
obeticholic acid, a farnesoid X receptor agonist, has also shown promising 
results. Health plans are anticipated to assign any newly introduced therapy for 
the treatment of PBC to specialty pharmacy given its orphan disease status. This 
assignment enables the health plan to receive disease education, which is 
particularly important when new drugs are indicated for orphan diseases, and 
assistance with designing appropriate prior authorization criteria. The clinical 
value of any new therapeutic options that will inform formulary decisions and 
prior authorization criteria will be assessed based on evidence of efficacy, 
safety, and tolerability, among other factors, such as the potential to reduce 
or delay medical resource utilization (e.g., liver transplant). Key 
considerations for prior authorization of a new therapy will be determining 
which PBC patients are appropriate candidates for the new therapy and developing 
criteria for that determination. These are likely to include clinical diagnostic 
criteria and degree of response to prior treatment with UDCA. Initially, any new 
therapy would likely be positioned as noncovered until appropriate prior 
authorization criteria are established.
CONCLUSIONS: PBC is a chronic liver disease with significant morbidity and 
mortality, as well as a significant burden on the health care system if the 
disease progresses to the point at which a liver transplant is needed. Although 
UDCA, the current standard of care, has improved outcomes for many patients, 
others have an inadequate response to this treatment. This symposium discussed 
these issues and also addressed the overall treatment paradigm for orphan drug 
therapies, key implications for patient management, and the role of specialty 
pharmacy management and any associated needs both in general and specifically 
for new therapeutic options for PBC.