ICER Publishes Final Evidence Report on Fezolinetant for Vasomotor Symptoms Associated with Menopause

— Independent appraisal committee voted that evidence is not yet adequate to demonstrate a net health benefit for fezolinetant when compared to no pharmacological treatment —

—  Using point estimates from short-term clinical trials, analyses suggest this drug would achieve common thresholds for cost-effectiveness if priced between $2,000 – $2,600 per year for women who cannot or choose not to take menopausal hormone therapy —

— All stakeholders have a responsibility and an important role to play in ensuring that women have access to effective new treatment options for symptoms of menopause—

BOSTON, January 23, 2023 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of fezolinetant (Astellas Pharma Inc.) for moderate-to-severe vasomotor symptoms (VMS) associated with menopause.

ICER’s report on this therapy was reviewed at the December 2022 public meeting of the 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

“While menopause is a part of normal aging for women, symptoms associated with menopause can last years and be disruptive,” said David Rind, MD, ICER’s Chief Medical Officer. “Vasomotor symptoms include flushing and sweating, and when frequent and/or severe, or when sleep is affected, can harm quality of life. Menopausal hormone therapy (MHT) can often effectively treat symptoms of menopause including vasomotor symptoms, but some women have contraindications to MHT and others are concerned about side effects of MHT, including an increased risk of breast cancer. There is an important need for new effective and safe options. While fezolinetant appears to be a promising treatment for women who cannot or do not wish to take MHT, there are still uncertainties about the magnitude of the benefit it provides and its long-term safety. All of the fezolinetant Phase III trials need to be peer reviewed and published.”

ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations

For women seeking relief from vasomotor symptoms associated with menopause:

A majority of panelists (11-1) found that current evidence is not adequate to demonstrate a net health benefit of fezolinetant compared to no pharmacological treatment.

All panelists (12-0) found that current evidence is not adequate to distinguish a net health benefit of fezolinetant compared to menopausal hormone therapy.

During their deliberations, panel members also weighed the therapy’s other potential benefits, disadvantages, and contextual considerations. Voting highlighted the following as particularly important for payers and other policymakers to note:

Magnitude of the lifetime impact on individual patients;
Patients’ ability to achieve major life goals related to education, work, or family life;
Society’s goal of reducing health inequities.

ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money

Fezolinetant is not yet approved by the FDA, and its manufacturer has not yet announced what the treatment’s US price will be if approved.  Using point estimates from short-term clinical trials to generate longer-term analyses of cost-effectiveness, ICER has calculated a health-benefit price benchmark (HBPB) for fezolinetant to be between $2,000 – $2,600 per year for women who cannot or who chose not to take MHT.   

Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for fezolinetant, the panel did not take a separate vote on the treatment’s long-term value for money.

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 manufacturer, 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:

Given that there are other treatment options available for many women, payers can use evidence to create coverage criteria for fezolinetant that reflect whether the manufacturer prices the drug in fair alignment with its benefits for patients. 

Given that many patients may benefit from readily available, effective, and low cost MHT, clinical experts agreed that it would be reasonable for payers to require prescriber attestation that patients are not appropriate candidates for MHT prior to prescribing fezolinetant.

Although an argument could be made to limit prescribing to experts in women’s health given the importance of full shared decision-making regarding the relative risks and benefits of treatment options for vasomotor symptoms, the vast majority of women receive their care from generalist physicians, and any attempt to narrow the providers able to prescribe would also disproportionately worsen access to care for patients with more limited economic resources.  Provider restrictions for fezolinetant would therefore not be a reasonable element of insurance coverage.   

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.

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.