OBJECTIVES: To evaluate the impact of clinical and economic evidence on health technology assessment (HTA) decisions in narcolepsy.

METHODS: HTA reimbursement decisions between July 2016 and April 2021 from SMC (Scotland), HAS (France), G-BA (Germany), TLV (Sweden), CADTH (Canada), and PBAC (Australia) for wakefulness-promoting agents used in the treatment of narcolepsy were included in the data set. HTAs with deferred or no decisions were excluded from the analysis. Clinical and economic from each review was identified and categorized as positive, negative, or neutral. Observations were made and results were reported.

RESULTS: A total of 23 HTA reviews covering sodium oxybate, armodafinil, methylphenidate, modafinil, pitolisant, and solriamfetol were included in the analysis of which, 61% (n = 14) were recommended and 39% (n = 9) were not recommended. HAS recommended the most drugs (5) followed by G-BA (2). Methylphenidate and modafinil were the most recommended drugs and sodium oxybate followed by pitolisant were the least recommended. Sixty-four percent of recommended reviews included positive clinical evidence compared with only 33% of reviews that were not recommended. For reviews that included economic evidence (n = 9), 100% of recommended reviews included positive economic evidence compared with none of the reviews that were not recommended. Eighty-eight percent of reviews that were not recommended included negative economic evidence. Only one recommended review that showed both positive clinical and economic evidence was identified from PBAC.

CONCLUSIONS: The majority of reviewed drugs were recommended by HTA agencies for use in narcolepsy. Drugs with positive clinical and economic evidence are more likely to be recommended, while those with negative evidence were not recommended. As narcolepsy can lead to potential disability, it deserves further attention and awareness from health care providers and researchers.