ICER Releases Final Reports on Obeticholic Acid for Treatment of PBC and NASH

Boston, Mass., July 26, 2016– The Institute for Clinical and Economic Review (ICER) has released two Final Reports, the first titled Obeticholic Acid for the Treatment of Primary Biliary Cholangitis: Comparative Clinical Effectiveness, Value, and Value-Based Price Benchmarks, and the second titled Obeticholic Acid for the Treatment of Nonalcoholic Steatohepatitis: Comparative Clinical Effectiveness and Value. The Final Reports, which review the comparative clinical effectiveness and value of obeticholic acid for treatment of these two liver diseases, incorporate a summary of votes taken on the evidence during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), as well as key policy recommendations stemming from discussion with a panel of experts during the meeting.

The Final Reports are accompanied by a “Report-at-a-Glance” document, which summarizes key points of the evidence review and economic model, voting results, and policy recommendations. The Report-at-a-Glance focuses primarily on the report addressing treatment of PBC, due largely to the preliminary nature of the data on use of OCA in NASH.

A key goal of ICER’s reports is to ensure that patients, providers, insurers, and policymakers have the information they need to support efforts to improve the quality and value of care.

“Patients with PBC have very limited options when it comes to treatments,” noted Steven D. Pearson, MD, MSc, President of ICER. “We’re pleased that our report was able to shed light on the potential health benefit of OCA for people with PBC who aren’t benefiting from currently available treatments. We hope that our findings on value will contribute to constructive discussions about pricing to ensure that as many patients as possible who can benefit from this drug are able to access it.”

Highlights of the meeting, as summarized in the Final Evidence Reports, include:

Comparative Clinical Effectiveness and Value

A majority of the Council voted that for patients with PBC who do not have an adequate response to usual care with ursodeoxycholic acid (UDCA), the evidence demonstrates that OCA provides a net health benefit. When voting on the care value of the drug, a measure which considers the clinical effectiveness of the drug, its cost, contextual considerations, and added benefits or disadvantages of the treatment, a majority of the Council found OCA to represent a low care value, citing the high cost of the drug as the primary reason for their vote. Some members, voting for an intermediate value, indicated that the high percentage of patients who do not respond to UDCA, along with possible productivity gains for a patient population that includes predominantly working-age women, represent important contextual considerations that contribute to the overall care value of the drug.

When voting on the clinical effectiveness of OCA for treatment of NASH, the Council voted unanimously that evidence is not adequate to demonstrate a net health benefit based on the preliminary nature of the data.

A more detailed review of the votes can be found in found in the full reports.

Policy Recommendations

During the meeting, the Council discussed policy implications of their evidence votes with a panel including a patient representative, clinical experts with extensive experience treating PBC and NASH, a representative of the drug’s manufacturer, and representatives of both public and private payers. Several key policy themes emerged from this discussion, which are detailed in full in the final report. Among the key recommendations are the following:

  • Increase efforts to include patient-relevant outcomes in clinical studies.
  • Employ strategies to improve medication adherence to UDCA and minimize side effects.
  • Assess the use of non-invasive tests as an alternative to liver biopsy for obtaining information on disease status by examining both types of tests in clinical trials of treatments of treatments for PBC and NASH.
  • Refrain from administering OCA to patients with NASH outside of the clinical trial environment.

A more detailed discussion of the recommendations can be found in the full reports. The final report for PBC and for NASH can be found on the ICER website, along with the Report-at-a-Glance.

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.