OBJECTIVES: The objective of this review was to characterize the inclusion of migraine patient-reported outcomes (PROs) in FDA approvals subsequent the FDA’s release in 2018 of the Patient Focused Drug Development (PFDD) guidance.

METHODS: The PROLABELS database was searched over a 5-year period between June 2018 and June 2023 for migraine disorders with label claims referencing migraine-specific PROs. The PROLABELS database is a commercially available database for Clinical Outcome Assessment (COA) endpoint strategies that are used to demonstrate the efficacy and safety of drugs or devices in the market approval process.

RESULTS: Eleven drugs with migraine PROs in the label were identified. The primary and secondary endpoints were most often assessed using daily diaries measuring the number of monthly migraine days (n=5, 45%) or headache pain occurring two hours post dosing (n=5, 45%), use of rescue medication (n=3, 27%), most bothersome symptom freedom at two hours post-dosage (n=5, 45%), pain relief 2hrs post dose (n=5, 45%), migraine associated symptoms (n=4, 36%), headache severity diary (n=1, 1%), and activities of daily living and motor function due to migraine (n=1, 1%). Other validated PRO questionnaires included the labels assessed the impact of migraine (n=2, 18%), and migraine quality of life (n =1, 1%).

CONCLUSIONS: Only 11 migraine-related therapies were approved by FDA in the 5-year period since the FDA released its PFDD guidance that incorporated migraine-specific PROs in the product label. Data used to assess endpoints were most often gathered using daily dairies. However, only two migraine-related diaries included in label claims have been published to date. Future work should focus on further development of diaries for migraine symptoms meeting the labelling claims criteria. The availability of such diaries to the scientific community could facilitate timely access to effective migraine treatments.