Narcolepsy and Idiopathic Hypersomnia Real-World Treatment Patterns and Changes in Alerting Agent Use After Low-Sodium Oxybate Initiation: A Retrospective Claims Analysis

Various medications are prescribed to treat narcolepsy and idiopathic hypersomnia (IH), including low-sodium oxybate (LXB) and alerting agents (AAs; i.e., stimulants and wake-promoting agents). This study assessed treatment patterns in individuals with narcolepsy/IH, as well as changes in AA utilization following LXB initiation. Optum® Market Clarity data were used to examine individuals aged ≥ 7 years with narcolepsy and individuals aged ≥ 18 years with IH. Treatment patterns were assessed following index until the end of the study period (07/2007–09/2023) and in the post-LXB approval period (narcolepsy: 07/2020–09/2023; IH: 08/2021–09/2023). Changes in AA treatment in the 180 days following LXB initiation were evaluated. Data were analyzed descriptively. The narcolepsy cohort included 43,252 individuals (mean [SD] age 42.8 [17.2] years; 62.9% female). In the total and post-LXB (n = 10,220) follow-up periods, 30.9% and 35.3%, respectively, did not receive any treatment of interest. The most frequent first-line treatments were AAs (total follow-up: 52.9%; 76.5% among those treated; post-LXB period: 49.3%; 76.1% among those treated). Among 788 individuals with AA treatment prior to LXB initiation, 42.9% experienced AA discontinuation or reduction following LXB initiation. The IH cohort included 24,528 individuals (mean [SD] age 45.8 [15.7] years; 61.7% female). In the total and post-LXB (n = 2971) follow-up periods, 54.1% and 50.3%, respectively, did not receive any treatment of interest. The most frequent first-line treatments were AAs (total follow-up: 31.2%; 67.9% among those treated; post-LXB period: 36.2%; 72.9% among those treated). Among 96 individuals with AA treatment prior to LXB initiation, 53.1% experienced AA discontinuation or reduction following LXB initiation. Numerous treatment trajectories were observed after narcolepsy/IH diagnosis, highlighting the complexity in treating these conditions. Many individuals did not receive any treatment over the total follow-up period, which may indicate a treatment gap. AA discontinuation or reduction was common following LXB initiation. Many medications are approved by the US Food and Drug Administration to treat narcolepsy; only one treatment, low-sodium oxybate, is approved to treat idiopathic hypersomnia, but doctors often prescribe other treatments “off-label.” We tracked the medications that people received after being diagnosed with narcolepsy or idiopathic hypersomnia, as well as how their treatments changed over time. We also evaluated people who had been prescribed alerting agents (stimulant medications or wake-promoting agents) and whether those treatments changed after they were prescribed low-sodium oxybate. We found that people received many different treatments and often had changes to their prescriptions. However, almost a third of people with narcolepsy (30.9%) and about half of people with idiopathic hypersomnia (54.1%) did not receive any treatment at all. Most people were first prescribed alerting agents. Over a third of people with narcolepsy (42.9%) and half of people with idiopathic hypersomnia (53.1%) who had previously taken alerting agents reduced their alerting agent dosage or discontinued alerting agents after starting to take low-sodium oxybate. This study suggests that treating narcolepsy and idiopathic hypersomnia is complicated and likely shaped by individual treatment histories, symptoms, and needs. Some people who take low-sodium oxybate may be able to reduce other treatments for narcolepsy and idiopathic hypersomnia. Future research should try to understand why some people with narcolepsy or idiopathic hypersomnia do not receive treatment and if there are barriers to treatment that need to be addressed.