Advertisement

THE UTILITY OF DISCRETE CHOICE EXPERIMENT IN EVALUATING TREATMENT PREFERENCES AMONG PATIENTS WITH NARCOLEPSY

      TOPIC: Sleep Disorders
      TYPE: Original Investigations
      PURPOSE: Twice-nightly, immediate-release sodium oxybate (SXB) has been available to treat narcolepsy since 2002 but requires patients to awaken for a second dose 2.5–4 h after the first dose. FT218, is an investigational, once-nightly SXB formulation (ON-SXB) in development for the treatment of narcolepsy. A discrete choice experiment (DCE) was undertaken to evaluate patient perspectives and understand drivers of patient preference for attributes associated with treatments of narcolepsy, including those for ON-SXB.
      METHODS: An IRB-approved, 30-min Web-based survey was fielded. Adults with self-reported physician-diagnosed narcolepsy for ≥1 year and prior/current use of twice-nightly SXB were eligible. The survey captured patient experiences and a DCE. Participants responded using 9-point scales, with higher scores indicating greater severity/agreement/satisfaction/importance/preference. In the DCE, hundreds of profiles were generated, each combining attributes of twice-nightly SXB and a once-nightly SXB formulation based on phase 3 clinical trial data of the 9-g dose of ON-SXB. Participants viewed 20 product profiles each. DCE was analyzed using a hierarchical Bayesian model. Differences in metrics captured in other sections of the survey were compared with t-tests (mean) and chi-square tests (proportion).
      RESULTS: From June to September 2020, 75 patients were surveyed (50 current and 25 past users of twice-nightly SXB; mean age, 39 y; 77% non-Hispanic white; 83% women). Dosing frequency was the single most important attribute of a narcolepsy treatment (relative attribute importance, 33.2), with once-nightly being significantly more preferred as compared to twice-nightly. The most common reasons for overall product preference were lack of need to wake up in the middle of the night for a second dose (48%), fewer side effects (46%), and ease of taking/handling (32%). The number of nightly doses was also the most important driver of taking the medication exactly as directed (relative attribute importance, 42.3) and reduced anxiety/stress (relative attribute importance, 35.5); once-nightly dosing was strongly preferred over twice-nightly dosing. When shown blinded profiles of the currently available twice-nightly SXB and investigational ON-SXB, patients were significantly more likely to prefer the once-nightly profile (mean rating, 7.5 vs. 4.3; P<0.05).
      CONCLUSIONS: Dosing frequency was the most important attribute when considering patient preference for overall product choice, likelihood to take the medication exactly as directed, and reducing anxiety/stress. Specifically, ON-SXB was significantly preferred over twice-nightly SXB.
      CLINICAL IMPLICATIONS: DCE provides critical insight about the preferences for various treatment options and potential trade-offs that individuals are willing to make. Once approved, FT218, a once-nightly SXB formulation for managing narcolepsy, may result in better patient adherence and lower anxiety/stress related to taking medication.
      DISCLOSURES: Consultant relationship with Avadel Please note: current Added 05/05/2021 by Amod Athavale, source=Admin input, value=Consulting fee
      Consultant relationship with Avadel Please note: 02/01/2020- Present Added 04/29/2021 by Alon Avidan, source=Web Response, value=Consulting fee
      Consultant relationship with Jazz Pharmaceuticals Please note: 04.22.2021 Added 04/29/2021 by Alon Avidan, source=Web Response, value=Consulting fee
      Speaker/Speaker's Bureau relationship with Harmony Biosciences Please note: 03.01.2019- Present Added 04/29/2021 by Alon Avidan, source=Web Response, value=Honoraria
      Speaker/Speaker's Bureau relationship with Jazz Please note: current Added 05/05/2021 by Bruce Corser, source=Admin input, value=Honoraria
      Advisory Committee Member relationship with Avadel Please note: current Added 05/05/2021 by Bruce Corser, source=Admin input, value=Consulting fee
      Speaker/Speaker's Bureau relationship with Harmony Please note: current Added 05/05/2021 by Bruce Corser, source=Admin input, value=Honoraria
      Speaker/Speaker's Bureau relationship with Merck Please note: current Added 05/05/2021 by Bruce Corser, source=Admin input, value=Honoraria
      Speaker/Speaker's Bureau relationship with Eisai Please note: current Added 05/05/2021 by Bruce Corser, source=Admin input, value=Honoraria
      Employee relationship with Paragon Biosciences Please note: current Added 05/05/2021 by Jordan Dubow, source=Admin input, value=Salary
      Employee relationship with Abbvie Please note: current Added 05/05/2021 by Jordan Dubow, source=Admin input, value=Salary
      Removed 05/05/2021 by Jordan Dubow, source=Web Response
      Consultant relationship with Avadel Pharmaceuticals Please note: current Added 05/05/2021 by Jordan Dubow, source=Admin input, value=Consulting feestockholder relationship with Avadel Pharmaceuticals Please note: current Added 05/05/2021 by Jordan Dubow, source=Admin input, value=dividends
      Employee relationship with Curry Rockefeller Group Please note: 1 Added 03/08/2021 by Elizabeth Guida, source=Web Response, value=Salary
      Consultant relationship with Avadel Pharmaceuticals Please note: current Added 05/05/2021 by Clete Kushida, source=Admin input, value=Consulting fee
      Consultant relationship with XW Pharma Please note: current Added 05/05/2021 by Clete Kushida, source=Admin input, value=Consulting fee
      Employee relationship with Avadel Pharmaceuticals Please note: 8/19-present Added 05/04/2021 by David Seiden, source=Web Response, value=Salary
      Stock holder relationship with Avadel Pharmaceuticals Please note: 8/19-present Added 05/04/2021 by David Seiden, source=Web Response, value=Stock