Why Real-World Evidence Matters for Nonhormonal Menopause VMS Therapy

Welcome back to another Contemporary OB/GYN Investigator Perspectives series. In this opening segment, Pauline M. Maki, PhD, professor of psychiatry, psychology, and obstetrics and gynecology at the University of Illinois Chicago, where she directs the Center for Health Awareness and Research on Menopause, discusses real-world evidence for nonhormonal treatment of vasomotor symptoms (VMS) of menopause.

Maki introduces OPTION-VMS, which she describes as the first real-world evidence to include fezolinetant (Veozah) among nonhormonal menopausal therapies. She outlines what the study reveals about VMS bother, mood, and sleep; how its preliminary data and updated data cuts connect; why objective actigraphy adds a dimension to sleep assessment beyond patient-reported outcomes; and what the findings mean for counseling patients in everyday ob-gyn practice.

She begins by explaining why the study was needed. The randomized, placebo-controlled trial remains the gold standard for understanding the effects of a new medication, Maki notes, because the placebo arm shows what improvement can be expected by chance for outcomes such as hot flashes, mood, and sleep. That design establishes whether a medication works, the magnitude of improvement, and the health indications it improves. The SKYLIGHT trials took this approach and showed the efficacy of fezolinetant on VMS and sleep.

The next question, Maki explains, is how those results translate to the women practitioners treat in their clinics. Clinical trials must select relatively healthy participants, who may differ from the typical patients clinicians see, and many groups of women do not participate in clinical trials for a variety of reasons. This raises the question of external generalizability: whether findings observed in a clinical trial hold for the types of women who walk through a clinic door. That, she says, is what OPTION-VMS was designed to test.

In the next segment, Maki reviews what the first OPTION-VMS data cuts show about hot flash bother, work productivity, depression, and anxiety.