LAUSR

Sodium oxybate, available in once-nightly and twice-nightly formulations, is indicated for treatment of cataplexy or excessive daytime sleepiness in narcolepsy. Once-nightly sodium oxybate extended-release oral suspension (ON-SXB; LUMRYZ™) may alleviate potential dosing challenges associated with immediate-release twice-nightly formulations. Human factors validation testing was conducted to confirm safe use of ON-SXB, using the product packaging, mixing cup, and Instructions for Use (IFU). Two summative studies evaluated simulated usability of ON-SXB in individuals (16–65 years of age) with narcolepsy, who were categorized as either experienced (having current or previous experience self-administering SXB) or naive (no current or previous experience). Both studies were conducted in lab settings modeled after a home bedroom environment. Study 1 (S1) assessed all critical and noncritical tasks related to storage, preparation, administration, and disposal of ON-SXB. After S1, IFU updates were implemented for additional risk control. Study 2 (S2) assessed tasks associated with these updates. Tasks were evaluated through observed performance or participant responses to a labeling comprehension activity. Of the S1 participants enrolled (mean [range] age, 37.0 [16–57] years; female, 77%), 77% (24/31) successfully prepared and administered the full ON-SXB dose. Critical use errors included failure to administer dose at bedside (experienced, 8/15; naive, 12/16) and preparing >1 dose packet (experienced, 1/15; naive, 1/16). Multiple critical use errors occurred during the label comprehension activity, which S1 participants attributed to difficulty in locating certain safety-critical warnings in the labeling. Using these data, the IFU was updated. In S2, 87% (26/30) of participants (mean [range] age, 35.8 [17–64] years; female, 83%) successfully prepared and administered the dose. Fewer S2 participants encountered critical errors observed in S1 (not administering dose at bedside [experienced, 4/15; naive, 5/15] and preparing >1 dose packet [none]), indicating that IFU updates effectively reduced the associated use risks. Only 2 use errors, which occurred with naive participants and were related to product storage, occurred in the S2 label comprehension activity. Validation to confirm that individuals with narcolepsy can follow labeled instructions for safe use and self-administration of oxybates is important and has been conducted with ON-SXB. Avadel Pharmaceuticals