Long-term use of pitolisant to treat patients with narcolepsy: Harmony III Study

Dauvilliers, Yves; Arnulf, Isabelle; Szakács, Zsolt; Leu‐Semenescu, Smaranda; Lecomte, Isabelle; Scart-Grès, Catherine; Lecomte, Jeanne‐Marie; Schwartz, Jean‐Charles; Bastuji, Hélène; Vieccherini, Marie Françoise; Pépin, Jean; Salva, M. A. Quera; Stoll, Anne Thibault

doi:10.1093/sleep/zsz174

Cited by 224 publications

(46 citation statements)

References 19 publications

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“…In this study, we examined the cost‐effectiveness of narcolepsy treatment with pitolisant as compared to standard treatment. Standard treatment corresponds to the established clinical practice in Scandinavia for the treatment of narcolepsy type I: modafinil or, lately also, methylphenidate combined with a selective serotonin and noradrenaline reuptake inhibitor . In our base‐case calculations, we estimated the incremental cost per additional quality‐adjusted life year at SEK 491 128 (monotherapy) and SEK 356 337 (add‐on).…”

Section: Discussionmentioning

confidence: 99%

“…in Scandinavia for the treatment of narcolepsy type I: modafinil or, lately also, methylphenidate combined with a selective serotonin and noradrenaline reuptake inhibitor. 35 In our base-case calculations, we estimated the incremental cost per additional quality-adjusted life year at SEK 491 128 (monotherapy) and SEK 356 337 (add-on).…”

Section: Standard Treatment Corresponds To the Established Clinical Pmentioning

confidence: 99%

“…The NMB is defined as the number of gained QALYs multiplied with the willingness-to-pay for an additional QALY, minus incremental costs (NMB = λ•∆E − ΔC, where λ is the willingness-to-pay threshold). 35 The empirical analysis of the cost-effectiveness of one treatment alternative compared with a competing alternative requires valid empirical measures of the variables and parameters that enter the cost-effectiveness measure. In most cases, secondary data are utilized, and the resulting cost-effectiveness measure is accompanied with an assessment of its sensitivity to changes in the chosen empirical measures.…”

Section: Kristianmentioning

confidence: 99%

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The cost utility of pitolisant as narcolepsy treatment

Bolin

Niska²,

Pirhonen

et al. 2020

Acta Neurol Scand

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Objectives The cost‐effectiveness of available pharmacological treatments for narcolepsy is largely unknown. Available pharmacological treatments are associated with tolerability, abuse, and adherence issues. Pitolisant is the first inverse agonist of the histamine H3 receptor to be prescribed for the treatment of narcolepsy with and without cataplexy. Studies suggest that pitolisant is both as effective as previously introduced drugs and is associated with fewer adverse effects. The objective in this study was to estimate the cost‐effectiveness of pitolisant as monotherapy, and pitolisant as an adjunctive treatment to modafinil, compared with standard treatment. Materials & Methods Calculations were performed using a Markov model with a 50‐year time horizon. Healthcare utilization and quality‐adjusted life years (QALYs) for each treatment alternative were calculated assuming no treatment effect on survival. Probabilistic sensitivity analyses were performed for treatment effectiveness and healthcare cost parameters. Results The cost per additional quality‐adjusted life year was estimated at SEK 356 337 (10 SEK ≈ 1 Euro) for pitolisant monotherapy, and at SEK 491 128 for pitolisant as an adjunctive treatment, as compared to standard treatment. The cost‐effectiveness measure was demonstrated to be particularly sensitive to the assumptions made concerning indirect effects on total healthcare utilization and the pitolisant treatment cost. Conclusions The incremental cost‐effectiveness ratios were below the unofficial willingness‐to‐pay threshold at SEK 500 000. The estimated costs per additional QALY obtained here are likely to overestimate the true cost‐effectiveness ratio since significant potential indirect effects—pertaining both to labor‐market and household‐related productivity—of treatment are not taken into account.

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Section: Discussionmentioning

confidence: 99%

Section: Standard Treatment Corresponds To the Established Clinical Pmentioning

confidence: 99%

Section: Kristianmentioning

confidence: 99%

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The cost utility of pitolisant as narcolepsy treatment

Bolin

Niska²,

Pirhonen

et al. 2020

Acta Neurol Scand

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“…Modulation of γ-aminobutyric acid (GABA) B (GABA B ) receptors (sodium oxybate, baclofen) or histamine H 3 receptors (H3Rs) has effects on EDS, cataplexy and other REM dissociative symptoms (e.g. hypnagogic and hypnopompic hallucinations); in addition, GABA B receptor modulation affects symptoms of sleep disruption [1,2,8,9]. Medications historically used for treatment of EDS (modafinil, armodafinil, stimulants, sodium oxybate) and cataplexy (sodium oxybate, venlafaxine) have demonstrated efficacy in managing these symptoms.…”

Section: Introductionmentioning

confidence: 99%

Recently Approved and Upcoming Treatments for Narcolepsy

Thorpy

2020

CNS Drugs

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Narcolepsy is a chronic, disabling neurologic disorder characterised by excessive daytime sleepiness (EDS) and, in up to 60% of patients, cataplexy. Treatments for narcolepsy are aimed at improving wakefulness (e.g. modafinil, armodafinil, stimulants), reducing cataplexy attacks (e.g. sodium oxybate, venlafaxine), and treating the symptoms of disturbed nocturnal sleep, sleep paralysis and sleep-related hallucinations (e.g. sodium oxybate). In general, medications that increase the release, or inhibit the reuptake, of norepinephrine or dopamine have wake-promoting effects and are useful in managing EDS, whereas medications that inhibit serotonin or norepinephrine reuptake have anticataplectic effects. Modulation of γ-aminobutyric acid B (GABA B) receptors or histamine H 3 receptors (H3Rs) has effects on both EDS and cataplexy. Pitolisant, an H3R antagonist, and solriamfetol, a dopamine and norepinephrine reuptake inhibitor, are the most recently approved treatments for EDS associated with narcolepsy in the European Union (pitolisant) and the USA (pitolisant and solriamfetol). Several new agents are being developed and tested as potential treatments for EDS and cataplexy associated with narcolepsy; these agents include novel oxybate formulations (once-nightly [FT218]; low sodium [JZP-258]), a selective norepinephrine reuptake inhibitor (AXS-12), and a product combining modafinil and an astroglial connexin inhibitor (THN102). This review summarises the mechanisms of action, pharmacokinetics, efficacy, and safety/tolerability of recently approved and emerging treatments for narcolepsy. Key Points Excessive daytime sleepiness and cataplexy are common and disabling symptoms associated with narcolepsy. Emerging treatments, including two recently approved medications (pitolisant and solriamfetol) and several medications still in development (FT218, JZP-258, AXS-12, THN102, SUVN-G3031, TAK-925), provide new options for the treatment of narcolepsy.

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“…Again, ESS scores and cataplexy severity decreased. More notable was that 20.2% of the individuals dropped out of the trial due to lack of efficacy of pitolisant 26. In another open-label trial presented as an abstract at the same meeting, pitolisant decreased ESS scores.…”

Section: Resultsmentioning

confidence: 99%

Update on the treatment of narcolepsy: clinical efficacy of pitolisant

Calik

2017

NSS

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Narcolepsy is a neurological disease that affects 1 in 2,000 individuals and is characterized by excessive daytime sleepiness (EDS). In 60–70% of individuals with narcolepsy, it is also characterized by cataplexy or a sudden loss of muscle tone that is triggered by positive or negative emotions. Narcolepsy decreases the quality of life of the afflicted individuals. Currently used drugs treat EDS alone (modafinil/armodafinil, methylphenidate, and amphetamine), cataplexy alone (“off-label” use of antidepressants), or both EDS and cataplexy (sodium oxybate). These drugs have abuse, tolerability, and adherence issues. A greater diversity of drug options is needed to treat narcolepsy. The small molecule drug, pitolisant, acts as an inverse agonist/antagonist at the H3 receptor, thus increasing histaminergic tone in the wake promoting system of the brain. Pitolisant has been studied in animal models of narcolepsy and used in clinical trials as a treatment for narcolepsy. A comprehensive search of online databases (eg, Medline, PubMed, EMBASE, the Cochrane Library Database, Ovid MEDLINE, Europe PubMed Central, EBSCOhost CINAHL, ProQuest Research Library, Google Scholar, and ClinicalTrials.gov) was performed. Nonrandomized and randomized studies were included. This review focuses on the outcomes of four clinical trials of pitolisant to treat narcolepsy. These four trials show that pitolisant is an effective drug to treat EDS and cataplexy in narcolepsy.

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Long-term use of pitolisant to treat patients with narcolepsy: Harmony III Study

Cited by 224 publications

References 19 publications

The cost utility of pitolisant as narcolepsy treatment

The cost utility of pitolisant as narcolepsy treatment

Recently Approved and Upcoming Treatments for Narcolepsy

Update on the treatment of narcolepsy: clinical efficacy of pitolisant

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