BOSTON, May 25, 2023 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of resmetirom (Madrigal Pharmaceuticals, Inc.) and obeticholic acid (Ocaliva®, Intercept Pharmaceuticals, Inc.) for non-alcoholic steatohepatitis (NASH).
ICER’s report on these therapies was the subject of the April 2023 public meeting of the Midwest CEPAC (Midwest CEPAC) one of ICER’s three independent evidence appraisal committees.
Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations
“NASH is increasingly common and lacks good therapies,” said David Rind, MD, ICER’s Chief Medical Officer. “While many with NASH will remain asymptomatic, some individuals will progress to severe liver disease and experience the complications of cirrhosis, hepatocellular cancer, and/or require liver transplantation. NASH is also a marker for increased cardiovascular risk and one of these therapies, resmetirom, improves lipids, while the other therapy, obeticholic acid, worsens lipids and also causes itching in many patients. If these drugs receive FDA approval, while awaiting long-term liver and cardiovascular data, patients and doctors will need to balance the risks, burdens, and potential benefits of each of these therapies.”
ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations
For adults with NASH with significant fibrosis (i.e., stage 2 and stage 3 fibrosis) and not cirrhosis:
- A slight majority of panelists (8-7) found that current evidence is adequate to demonstrate a net health benefit for resmetirom when compared to lifestyle management alone.
- A majority of panelists (14-1) found that current evidence is not adequate to demonstrate a net health benefit for obeticholic acid when compared to lifestyle management alone.
During their deliberations, panel members also weighed potential benefits and disadvantages beyond the direct health effects, and broader contextual considerations. Voting highlighted the following as particularly important for payers and other policymakers to note:
- The magnitude of the lifetime impact of NASH on individual patients is substantial.
ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money
Both resmetirom and obeticholic acid are not yet approved by the FDA, and the manufacturers have not yet announced what the US prices will be if approved.
ICER has calculated a health-benefit price benchmark (HBPB) for resmetirom to be between $39,600 - $50,100 per year. ICER has calculated a health-benefit price benchmark (HBPB) for obeticholic acid to be between $32,600 - $40,400 per year.
Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for both treatments, the panel did not take separate votes on the treatments’ long-term value for money.
Access and Affordability Alert
At the placeholder prices for resmetirom and obeticholic acid, assuming a 20% uptake of resmetirom and obeticholic acid each year, approximately 6.5% and 1.2% respectively of US patients eligible for NASH treatment could be treated within five years without crossing the ICER potential budget impact threshold of $777 million per year.
ICER is issuing an access and affordability alert for resmetirom and obeticholic acid in the management of NASH. The purpose of an ICER access and affordability alert is to signal to stakeholders and policy makers that the amount of added health care costs associated with a new service may be difficult for the health system to absorb over the short term without displacing other needed services, creating pressure on payers to sharply restrict access, or causing rapid growth in health care insurance costs that would threaten sustainable access to high-value care for all patients.
Key Policy Recommendations
ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including the manufacturers, clinical experts, patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include:
- Life science companies should set initial prices within the bounds of independent value assessment and with further moderation in relation to the uncertainty of longer-term outcomes and the potential size of the eligible patient population.
- Payers should integrate coverage of drugs for NASH with coverage for obesity management, and may want to consider step therapy with lifestyle management efforts prior to providing coverage for NASH-specific drugs given that the advent of GLP-1 treatments for obesity may offer a new opportunity for many patients to achieve significant weight loss. If step therapy is required, then payers should cover intensive weight management programs that include nutritionists and drug therapy.
- Payers should develop coverage criteria based on non-invasive testing to foster equitable access to early detection and treatment across diverse communities.
- For NASH, both price-volume and outcomes-based agreements may be considered to manage the uncertainties surrounding the annual costs for these drugs.
ICER’s detailed set of policy recommendations, including comprehensive considerations for establishing evidence-based prior authorization criteria, is available in the Final Evidence Report and in the standalone Policy Recommendations document.