How Migraine Trials Can Better Capture Patient-Centered Outcomes: Amaal Starling, MD, FAHS, FAAN

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“Migraine is not just a headache. It’s light sensitivity, sound sensitivity, nausea, brain fog—all of those symptoms determine whether a patient has a good day or a disabling day.”

Migraine clinical trials have traditionally relied on standardized endpoints such as reductions in monthly headache days (MHDs) to measure treatment benefit. While these metrics remain central to regulatory evaluation, they may not fully capture the broader burden patients experience, including symptoms like photophobia, nausea, and cognitive dysfunction that affect daily functioning.^1,2

The ongoing real-world INFUSE study has contributed to this discussion. Presented at the 2026 Headache Cooperative of the Pacific (HCOP) Annual Conference, the observational study evaluated intravenous eptinezumab (Vyepti; Lundbeck) in adults with migraine who had previously failed at least one calcitonin gene–related peptide (CGRP)–targeted preventive therapy. Among the 111 participants who completed six months of treatment, investigators reported improvements in monthly headache days as well as patient-reported outcomes such as the Patient Global Impression of Change (PGIC) and the number of “good days” experienced each month.³

These patient-centered endpoints provide a broader perspective on treatment benefit beyond traditional headache metrics. Participants in the INFUSE cohort entered the study with a substantial disease burden—averaging about 20 monthly headache days—and many had previously failed multiple CGRP-directed therapies. Despite this refractory population, treatment with eptinezumab was associated with reductions in migraine frequency alongside improvements in day-to-day functioning.

Following the meeting, Amaal Starling, MD, FAHS, FAAN, associate professor of neurology at Mayo Clinic College of Medicine and a study investigator, spoke with NeurologyLive^® about how migraine trials may continue to evolve to better reflect patient experiences. In the discussion, she addresses the growing role of patient-reported outcomes such as “good days,” the importance of correlating these measures with established endpoints like monthly headache days, and the broader shift toward elevating the patient voice in migraine research.

REFERENCES
1. Diener HC, Dodick DW, Goadsby PJ, et al. New therapeutic approaches for migraine prevention. Lancet Neurol. 2023;22(3):251-264. doi:10.1016/S1474-4422(22)00401-3
2. Lipton RB, Dodick DW, Silberstein SD, et al. Migraine burden and functional impairment. Headache. 2016;56(2):254-262. doi:10.1111/head.12735
3. Lundbeck. Six-month results from the INFUSE real-world study evaluating eptinezumab in adults with migraine who failed prior CGRP therapies. Presented at: Headache Cooperative of the Pacific Annual Conference; January 30–31, 2026.