Boston, Mass., June 23, 2016 – The Institute for Clinical and Economic Review (ICER) has released two Evidence Reports assessing the comparative clinical effectiveness and value of obeticholic acid (OCA) (Ocaliva™, Intercept Pharmaceuticals, Inc.): one examining its use for primary biliary cholangitis (PBC), and the other examining its use for nonalcoholic steatohepatitis (NASH). Draft versions of these reports were open for a two-week public comment period. The updated Evidence Reports reflect changes based on comments received from patient groups, clinicians, and the manufacturer of the drug.

OCA was recently approved by the FDA for treatment of PBC, a rare autoimmune liver disease that can lead to cirrhosis and ultimately liver failure. There is also expected to be significant interest in using OCA for indications beyond those that were part of initial FDA approval, including for treatment of patients with NASH, a liver condition that can lead to cancer and liver failure and affects as many as 15 million adults Americans.

The ICER reports provide analyses of the clinical effectiveness, long-term cost-effectiveness, and potential budget impact of OCA, and the results of these analyses were used to calculate a value-based price benchmark for the treatment of PBC. Price benchmarks suggest a price range that aligns the price of a new drug with estimates of how much better it is at improving patient outcomes over both the short and the long term.  The potential budget impact analysis provides additional information about whether the potential use of a new drug, based on its added costs and the number of patients who would be eligible for it, could lead to short-term rapid increases in spending that would push overall health care cost growth past a national affordability threshold.

ICER concludes that for treatment of PBC, the annual price of OCA would need to be discounted by 64%-73% to be better aligned with the added value it brings to patients. Due to the preliminary nature of data and ongoing trials of OCA for NASH, a value-based price benchmark was not calculated for this use of the drug.

“As it stands, patients with PBC or NASH have very few treatment options available to them, and Ocaliva represents an important advance,” noted Steven Pearson, MD, MSc, President of ICER. “Our reports analyze the strength of the evidence behind Ocaliva’s use for different kinds of patients.  Even with its potential benefits, our analyses suggest that the list price for Ocaliva chosen by the manufacturer is too high to align well with how much added value it brings to patients with PBC. The fact that there are millions of Americans with NASH who may also be eligible soon for treatment with this drug raises the stakes further in thinking about Ocaliva’s clinical effectiveness and how its pricing may affect patient access.”

Primary Biliary Cholangitis

For patients with an inadequate response to ursodeoxycholic acid (UDCA), the current standard of treatment for PBC, ICER’s review found that there is moderate certainty that treatment with OCA in combination with UDCA provides incremental or better net health benefit for patients in early stages of PBC as compared to UDCA alone. Evidence for OCA taken as monotherapy in patients unable to tolerate UDCA was found to be promising but inconclusive, due to limited data on outcomes for patients using OCA as monotherapy. A cost analysis found that OCA exceeds commonly cited thresholds for cost-effectiveness, and would need to be priced between $18,400 and $25,300 to fall below these widely accepted thresholds. This price represents a 64%-73% discount from its current list price of $69,350 per year of treatment.

Nonalcoholic Steatohepatitis

For treatment of NASH, ICER’s review found the clinical evidence to be insufficient to draw a meaningful conclusion on its effect for patients. A Phase III trial is currently enrolling patients and will help provide more clarity on the utility of this treatment for patients with NASH. Given that clinical trials are ongoing, ICER determined that the introduction of a value-based price benchmark for OCA in NASH would be premature at this point in the drug’s development. Due to the significant interest in using OCA for treatment of NASH, however, we produced the review of clinical effectiveness and cost-effectiveness without these measures and hope the review of currently-available information will be useful in decision-making.

ICER is committed to a transparent public engagement process to ensure that all stakeholders have the opportunity to provide input to our reports. As noted above, after ICER’s Draft Evidence Report was released on May 25th, interested stakeholders had a two-week period to provide comments. ICER staff considered these comments and made revisions to the Evidence Report as needed. The Evidence Report, as well as the accompanying voting questions, public comments, and ICER’s written response to comments, are available on the ICER website.

The Evidence Report will be the subject of the July 15th public meeting of the New England CEPAC, during which the independent Council will vote on key questions raised in the report, and a policy roundtable will discuss recommendations to apply the evidence to policy and practice. During the meeting, there will be further opportunity for public comment; a limited amount of time will be available for interested stakeholders to provide a brief oral comment on the report. Requests to make oral comments should be submitted to by 5:00 PM ET on Friday, July 8, 2016.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.