Individualized Treatment Patterns for Patients with Narcolepsy Treated with Oxybate: A Clinical Practice Perspective

Abstract

Purpose

Real-world data regarding divided nightly dosing of oxybate and individualized prescribing in patients with narcolepsy are limited. Study objectives were to understand oxybate prescribing practices, including optimizing dose regimens and adjusting dosing per occasional changes in patients’ routines, and physician recommendations for representative patient scenarios.

Patients and Methods

A cross-sectional, web- and audio-based survey of physicians treating ≥2 patients with narcolepsy, prescribed nightly oxybate (sodium oxybate) dosing for ≥6 months, was conducted. Physicians were surveyed on patients’ usual oxybate dosing regimens, frequency of and reasons for oxybate dosing-related discussions, and preferred methods for and perceptions of adjusting oxybate dosing. Physicians provided dosing-related guidance for 4 representative scenarios.

Results

Participating physicians (N=25) were neurologists (52%), psychiatrists (44%), and neuropsychiatrists (4%). Individualized oxybate prescribing practices were reflected by the variability of physicians’ reporting of the percentage of their patients being prescribed once-nightly, twice-nightly, and thrice-nightly dosing regimens. Most physicians (68%) reported discussing adjusting individualized treatment to accommodate occasional changes to patients’ routines; the most common reasons were consuming contraindicated beverages (alcohol; 65%) and travel (59%). Adjusting total nightly dose (68%) and dose timing (68%) were preferred adjustment methods. Most physicians (88%) felt the ability to individualize oxybate dosing was important and had a positive impact on ability to provide care. For each representative scenario, physicians provided several dose-adjustment recommendations, and physician responses encouraged patient participation in treatment decision-making.

Conclusion

Physicians provided guidance supportive of oxybate dose adjustments to accommodate occasional changes in patients’ routines, and perceived individualized dosing as important in providing care.

Plain Language Summary

Why was the research needed?

Narcolepsy is an uncommon condition that causes individuals to feel sleepy throughout the day. Other symptoms may include sudden muscle weakness. There is no cure for narcolepsy, but there are several treatments, including medicines known as oxybates. This study focused on how doctors use oxybate to treat patients with narcolepsy.

How was the research done?

This study was an online survey of doctors who were treating 2 or more patients with narcolepsy. Patients had been taking oxybate for at least 6 months. The survey asked doctors about their patients’ normal oxybate usage, how often and why they discussed oxybate treatment, and which ways oxybate treatment was usually adjusted.

What are the results?

Twenty-five doctors took part in this study. Most doctors said they discuss personalized treatment to help with occasional changes in their patients’ daily lives. The most common reasons for making treatment changes were consuming alcohol and travel. The most common changes were total nightly amount and timing of oxybate. Most doctors said the ability to personalize oxybate treatment is important and has a positive impact.

What does the research mean?

This study provides valuable knowledge on real-world oxybate treatment patterns and the conditions when doctors make clinical decisions about treating patients with narcolepsy.

Graphical Abstract

Keywords:

• sodium oxybate
• low-sodium oxybate
• calcium
• magnesium
• potassium
• sodium oxybates
• prescribing patterns
• personalized dosing

Abbreviations

BMI, body mass index; EDS, excessive daytime sleepiness; FDA, Food and Drug Administration; FD-SXB, fixed-dose, high-sodium oxybate; LXB, low-sodium oxybate (calcium, magnesium, potassium, and sodium oxybates); SD, standard deviation; SXB, sodium oxybate; US, United States.

Data Sharing Statement

All relevant data are provided within the article. Jazz has established a process to review requests from qualified external researchers for data from Jazz-sponsored clinical trials in a responsible manner that includes protecting patient privacy, assurance of data security and integrity, and furthering scientific and medical innovation. Additional details on Jazz Pharmaceuticals data sharing criteria and process for requesting access can be found at: https://www.jazzpharma.com/science/clinical-trial-data-sharing/.

Ethics Approval and Informed Consent

Centralized institutional review board approval for this study was obtained by the New England Independent Review Board (Needham, MA). All participants provided electronic informed consent to participate in the study. Participants received an honorarium for their participation in this study.

Acknowledgments

The authors would like to thank the physicians for their participation in this study. The authors also gratefully acknowledge the contributions of Diane Menno of Jazz Pharmaceuticals for her review and approval of the statistical analysis plan and assistance in interpretation of the results, as well as Jessica Vu, Jeremy Franz, Beth Baldys, and Fabio Gratton from inVibe for their work in recruitment and execution of the study and their assistance in analyzing the qualitative data from the study. Under the direction of the authors, Sean Anderson, PhD, and Michael Theisen, PhD, of Peloton Advantage, LLC, an OPEN Health company, provided medical writing and editorial support, which was funded by Jazz Pharmaceuticals.

Author Contributions

All authors have substantially revised or critically reviewed the article; have agreed on the journal to which the article will be submitted; reviewed and agreed on all versions of the article before submission, during revision, the final version accepted for publication, and any significant changes introduced at the proofing stage; and agreed to take responsibility and be accountable for the contents of the article.

A Roy: Conceptualization (equal); Methodology (equal); Investigation (equal); Writing – review and editing (equal).

D Ito: Conceptualization (equal); Methodology (equal); Formal analysis (equal); Investigation (equal); Writing – review and editing (equal).

S Morris: Conceptualization (equal); Methodology (equal); Investigation (equal); Writing – review and editing (equal).

S Candler: Writing – review and editing (equal).

J Profant: Conceptualization (equal); Methodology (equal); Formal analysis (equal); Investigation (equal); Writing – review and editing (equal).

C Bae: Conceptualization (equal); Methodology (equal); Investigation (equal); Writing – review and editing (equal).

Disclosure

AR has received consultancy fees from Jazz Pharmaceuticals and Harmony Biosciences. DI is an employee of Stratevi, a consulting firm that has received research funding from Jazz Pharmaceuticals to conduct this study. SM and JP are former employees of Jazz Pharmaceuticals who, in the course of their employment, received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc. SC is a current employee of Jazz Pharmaceuticals who, in the course of his employment, has received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc. CB has received consultancy fees from Jazz Pharmaceuticals. The authors report no other conflicts of interest in this work.

Additional information

Funding

This study was sponsored by Jazz Pharmaceuticals.