Flygare, Julie

Abstract Introduction Immediate-release sodium oxybate (SXB) and mixed-salt oxybates require patients with narcolepsy to take a second nightly dose 2.5–4 h after the first. Extended-release, once-nightly SXB (ON-SXB; FT218) is an investigational treatment for adults with narcolepsy. Surveys evaluated patient and healthcare provider (HCP) perspectives on narcolepsy disease burden and satisfaction with current narcolepsy treatment options. Methods Individuals with narcolepsy and HCPs participated in 30-minute, web-based surveys. Participants were 1) adults with self-reported, physician-diagnosed narcolepsy for ≥1 year, and prior/current/no use immediate-release oxybates; and 2) board-certified/board-eligible HCPs (pulmonology, sleep medicine, neurology, psychiatry specialties); nurse practitioners; or physician assistants. Participants responded using 9-point scales; higher scores indicated greater severity/agreement/satisfaction/importance/preference. Results Mean patient participant (n=120) age was 40 years; most were white (81%), female (79%), and current/past users of twice-nightly SXB (n=86) or mixed-salt oxybates (n=56). Twenty-six were oxybate naive. Most HCPs (n=100; 68% male) had sleep medicine (37%) or neurology (30%) specialties; 91% and 83% had experience with twice-nightly SXB and mixed-salt oxybates, respectively. Patients and HCPs agreed that patients preferred narcolepsy treatments dosed fewer times (rated 6.7 and 7.7, respectively). Common symptoms patients experienced daily/almost daily at narcolepsy diagnosis were tiredness/fatigue (64%) and excessive daytime sleepiness (EDS; 68%). HCPs and patients expressed moderate-to-high satisfaction with mixed-salt oxybates (both 7.1) and twice-nightly SXB (6.8 and 6.6, respectively). Compared to HCPs, patients were less satisfied with modafinil (6.9 vs 4.5), armodafinil (6.9 vs 4.8), solriamfetol (6.8 vs 5.4), and pitolisant (6.6 vs 5.2). Twice-nightly SXB and mixed-salt oxybates received high ratings from patients and HCPs for reduction of cataplexy (patients: 7.3 and 7.4; HCPs, 6.7 and 6.8) and EDS (patients: both 7.0; HCPs, both 6.9). Lower satisfaction was reported for twice-nightly SXB and mixed-salt oxybates with dosing frequency (patients, 5.4 and 6.0; HCPs, 5.9 and 6.3) and medication taste (patients, 5.3 and 5.7; HCPs, 5.9 and 6.2). Conclusion While both individuals with narcolepsy and HCPs are relatively satisfied with current narcolepsy treatments, both groups are less satisfied with the dosing frequency of currently approved oxybate formulations. ON-SXB will be an additional treatment option that can address this unmet need. Support (If Any) Avadel Pharmaceuticals

Abstract Introduction Currently available immediate-release sodium oxybate (SXB) and mixed-salt oxybates are dosed twice nightly (second 2.5–4 hours after first). Extended-release, once-nightly SXB (ON-SXB; FT218) is in development. To characterize drivers of oxybate treatment attribute preferences, discrete choice experiments (DCEs) were conducted for both patients and healthcare providers (HCPs). Methods Thirty-minute web-based surveys were fielded to 1) adults with self-reported physician-diagnosed narcolepsy with/without prior/current use of oxybate, and 2) board-certified/board-eligible HCPs treating ≥5 narcolepsy patients in the last month. Choice sets of 2 hypothetical treatment profiles were generated combining attributes of twice-nightly SXB, mixed-salts oxybate, and ON-SXB. While viewing 12 choice sets, each participant indicated (1) their preferred product overall, (2) the product that would improve quality of life (QoL), and (3) the product that would result in less patient stress/anxiety. Results were analyzed using a mixed logit model. Results For patients (n=120) and HCPs (n=100), the most important attribute driving overall product choice was dosing frequency (relative attribute importance, 26.0 and 46.1, respectively), with once-nightly preferred over twice-nightly dosing (relative preference weight, ±25.6 and ±43.6); QoL (relative attribute importance, 28.7 and 41.7), with once-nightly preferred over twice-nightly dosing (relative preference weight, ±25.7 and ±38.5); and reducing patient anxiety/stress (relative attribute importance, 26.7 and 44.0), with once-nightly preferred over twice-nightly dosing (relative preference weight, ±23.9 and ±41.6). Other drivers of overall product choice were as follows: patients, clinical efficacy and sodium content (relative attribute importance, 23.5 and 20.8); HCPs, adverse reactions and sodium content (relative attribute importance, 19.7 and 18.6). Drivers of QoL preference were as follows: patients, clinical efficacy and sodium content (relative attribute importance, 28.3 and 20.9); HCPs, adverse reactions and clinical efficacy (relative attribute importance, 21.5 and 18.6). Drivers of reducing patient anxiety/stress were as follows: patients, clinical efficacy and sodium content (relative attribute importance, 17.4 and 17.3); HCPs, adverse reactions and sodium content (relative attribute importance, 18.2 and 14.2). Conclusion Dosing frequency was identified as the most important attribute driving preference for overall product choice, QoL, and reducing patient anxiety/stress for both patients and HCPs with once-nightly preferred over twice-nightly dosing. Support (If Any) Avadel Pharmaceuticals