Cost-effectiveness analyses and utility weights

Published cost-effectiveness analyses and health-state utility weights that a model of this product could cite, adapt, or compare against, grouped by how each article relates to the product and its indication. Every value is quoted from the article it cites, and section 3 lists the searches that found the articles.

12 cost-effectiveness analyses and 6 studies reporting utility weights · 12 sources

Overview

Item	Detail
Literature searched	Tufts Medical Center Cost-Effectiveness Analysis Registry, searched October 21, 2026; the queries are listed in section 3.
Cost-effectiveness analyses	12
Studies reporting utility weights	6

Cost-effectiveness analyses

Analyses of Relutrigine

No evidence found.

Treatments for the same indication

No evidence found.

The same disease in other populations or settings

Genetically guided therapy versus usual care in drug-resistant epilepsy, Australia: Gordon et al. (2022)

Relevance: Evaluates treatment guided by a pharmacogenomics panel against usual care for drug-resistant epilepsy in Australia.

“Our study assessed the cost-effectiveness of a pharmacogenomics panel for patients with drug-resistant epilepsy, compared with usual care.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A cost-utility analysis was employed using a discrete event simulation model.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The microsimulation model aggregated the costs and benefits of genetically guided treatment versus usual care for 5000 simulated patients.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The mean cost of the genetically guided treatment option was AU$98 199 compared with AU$95 386 for usual care.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Corresponding mean QALYs were 4.67 compared with 4.28 for genetically guided and usual care strategies, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental cost per QALY gained was AU$7381.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In probabilistic sensitivity analyses, the incremental cost per QALY gained was AU$6321 (95% uncertainty interval = AU$3604-AU$9621), with a 100% likelihood of being cost-effective in the Australian health care system.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Ketogenic diet, vagus nerve stimulation, and care as usual in children with intractable epilepsy, Netherlands: de Kinderen et al. (2015)

Relevance: Evaluates the ketogenic diet and vagus nerve stimulation against care as usual in children with intractable epilepsy in the Netherlands from a health care perspective, therapies section 2 lists for SCN8A-related epilepsy.

“A Markov decision analytical model was constructed to estimate the incremental costs, quality-adjusted life years (QALYs) and successfully treated patient (i.e. 50% or more seizure reduction) of the treatment strategies KD, VNS and CAU, from a health care perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The base case considered children with intractable epilepsy (i.e. two or more antiepileptic drugs had failed) aged between 1 and 18 years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The objective of this study was to estimate the expected cost-utility and cost-effectiveness of the ketogenic diet (KD), vague nerve stimulation (VNS) and care as usual (CAU), using a decision analytic model with a 5-year time horizon.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Our results suggest that KD is more effective and less costly, and thus cost-effective compared with VNS, after 12 months.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“However, compared to CAU, neither KD nor VNS are cost-effective options, they are both more effective but also more expensive (€346,899 and €641,068 per QALY, respectively).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At 5 years, VNS is cost-effective compared with KD and CAU (€11,378 and €68,489 per QALY, respectively) and has a 51% probability of being cost-effective at a ceiling ratio of €80,000 per QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Ketogenic diet versus care as usual in children and adolescents with intractable epilepsy, Netherlands: Wijnen et al. (2017)

Relevance: Evaluates the ketogenic diet against care as usual in children and adolescents with intractable epilepsy in the Netherlands, a dietary therapy section 2 lists for SCN8A-related epilepsy.

“To examine long-term retention rate, clinical outcomes, cost-utility and cost-effectiveness of the Ketogenic Diet (KD) compared with care as usual (CAU) in children and adolescents with intractable epilepsy from a societal perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Participants were randomized into a KD or CAU group.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Seizure frequency, quality adjusted life years (QALYs), side-effects, seizure severity, health care costs, production losses, patient and family costs were assessed at baseline and during 16-months of follow-up.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“48 children were included in the analyses of this study (26 from KD group).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Mean costs per patient in the CAU group were €53,367 (extrapolated) compared to €61,019 per patient in the KD group, resulting in an ICER of €46,564 per responder.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Cost per QALY rose well above any acceptable ceiling ratio.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“However, as only a minimal difference in QALYs was found between the KD group and the CAU group, the resulting cost per QALY ratios were inconclusive.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Vagus nerve stimulation after implantation versus the period before implantation in pediatric drug-resistant epilepsy, United States: Helmers et al. (2012)

Relevance: Reports outcomes and costs of vagus nerve stimulation in children with drug-resistant epilepsy in United States Medicaid data, a therapy section 2 lists for SCN8A-related epilepsy when standard antiseizure medications are ineffective.

“To evaluate clinical outcomes, quality-adjusted life years (QALY), and costs associated with VNS in pediatric patients with drug-resistant epilepsy in a real-world setting.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A retrospective analysis was conducted using Medicaid data (USA).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Incidence rate ratios (IRR) and costs ($2010) were estimated.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“QALYs were estimated using number of seizure-related events.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Average total healthcare costs were lower Post-VNS vs. Pre-VNS ($18,437 vs. $18,839 quarterly [adjusted p = 0.052]).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Average total healthcare costs were lower Post-VNS vs. Pre-VNS period ($14,546 vs. $19,695 quarterly [adjusted p = 0.002]).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Lifetime QALY gain after VNS was 5.96 (patients 1-11 years) and 4.82 years (patients 12-17 years).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Related diseases and analogous treatments

Fenfluramine added to standard of care versus standard of care alone in Dravet syndrome, Sweden: Malmberg et al. (2024)

Relevance: Evaluates fenfluramine added to standard of care against standard of care alone in Dravet syndrome in Sweden over a lifetime horizon, with quality of life estimates for people with the disease and their caregivers.

“Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000 SEK.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000 SEK.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Cannabidiol with clobazam versus usual care in Dravet syndrome, England: NICE (2019)

Relevance: Reports the NICE appraisal of cannabidiol with clobazam against usual care for seizures in Dravet syndrome in England, a cannabinoid therapy of the kind section 2 lists for SCN8A-related epilepsy.

“The company presented a Markov state-transition cohort model to estimate the cost effectiveness of cannabidiol.” (opens the source at this quote in a new tab)
“The model had a time horizon of 90 years and a cycle length of 3 months.” (opens the source at this quote in a new tab)
“It had 4 health states, based on the number of convulsive seizures a patient had each month, to capture the costs and health effects.” (opens the source at this quote in a new tab)
“It concluded that the subgroup of patients taking clobazam was most relevant to this appraisal, and that it would not consider the overall trial population further.” (opens the source at this quote in a new tab)
“This resulted in an incremental cost-effectiveness ratio (ICER) of £32,471 per QALY gained.” (opens the source at this quote in a new tab)
“The committee concluded that the cumulative effect of addressing these uncertainties was likely to have increased the ICER.” (opens the source at this quote in a new tab)
“The committee concluded that, despite these uncertainties (see section 3.20), when it considered the uncaptured benefits, cannabidiol represents an effective treatment and a good use of NHS resources.” (opens the source at this quote in a new tab)

Adjunctive cannabidiol versus usual care in Lennox-Gastaut syndrome, United States: Neuberger et al. (2020)

Relevance: Evaluates adjunctive cannabidiol against usual care alone in Lennox-Gastaut syndrome from a United States payer perspective, with health states defined by percentage reduction in drop seizure frequency.

“This analysis assessed the cost effectiveness of CBD adjunct therapy compared with usual care alone in LGS from the US payer perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“We developed a lifetime horizon Markov decision analytic model.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Fifteen-year-old patients entering the model transitioned to states representing a percentage reduction in drop seizure frequency from baseline, where they remained until reverting back to baseline drop seizure frequency, or death.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and outcomes were discounted at 3%.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Compared with usual care alone, CBD yielded 0.7 additional quality-adjusted life-years (QALYs) and $314,900 additional healthcare expenditure, resulting in $451,800 per QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Results from the 5000-simulation probabilistic sensitivity analysis indicated a 0% chance of CBD being cost effective at a $150,000 per QALY willingness-to-pay threshold, with a 95% credible range for the incremental cost-effectiveness ratio of $325,300-$690,000 per QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Adjunctive stiripentol versus clobazam and valproate alone in children with Dravet syndrome, Canada: Elliott et al. (2018)

Relevance: Evaluates adjunctive stiripentol added to clobazam and valproate in Dravet syndrome in Canada.

“The aim of this study was to evaluate the cost effectiveness of stiripentol as an adjunctive treatment to clobazam and valproate for treatment of Dravet syndrome from the perspective of the Canadian public healthcare payer.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Probabilistic analyses were conducted using a Markov model with health states related to seizure frequency.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A 10-year horizon was used.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“All costs are expressed in 2017 Canadian dollars ($Can).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Compared with clobazam and valproate alone, the adjunctive use of stiripentol is associated with an ICER of $Can151,310.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At a willingness-to-pay threshold of $Can50,000, the probability that stiripentol was the optimal treatment was 5.2%.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The cost of stiripentol would need to be reduced by 61.4% for stiripentol to be cost effective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Cannabinoid oil and stiripentol added to clobazam and valproate versus clobazam and valproate alone in children with Dravet syndrome, Canada: Elliott et al. (2020)

Relevance: Evaluates cannabinoid oil and stiripentol added to clobazam and valproate in Dravet syndrome in Canada.

Access: Not open access

Not quoted: The results are reported in the full text, which is closed access.

“We performed a probabilistic cost-utility analysis from the perspective of the Canadian public health care system, comparing cannabinoid oil and stiripentol (both on a background of clobazam and valproate) with clobazam and valproate alone.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and quality-adjusted life-years (QALYs) were estimated using a Markov model that followed a cohort of children aged from 5 to 18 years through model states related to seizure frequency.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The cost effectiveness of adjunctive cannabinoid oil, adjunctive stiripentol, and clobazam/valproate alone was assessed through sequential analysis.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“All costs are expressed in 2019 Canadian dollars, and costs and QALYs were discounted at a rate of 1.5% per year.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental cost per QALY gained with the use of adjunctive cannabinoid oil, from the health care system perspective, was $32,399 compared with clobazam and valproate.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Stiripentol was dominated by cannabinoid oil, producing fewer QALYs at higher costs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At a willingness-to-pay threshold of $50,000, cannabinoid oil was the optimal treatment in 76% of replications.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“From a societal perspective, cannabinoid oil dominated stiripentol and clobazam/valproate.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Add-on cannabidiol versus usual care alone in treatment-refractory seizures associated with tuberous sclerosis complex, England and Wales: Burke et al. (2024)

Relevance: Evaluates add-on cannabidiol against usual care alone for treatment-refractory seizures associated with tuberous sclerosis complex from a National Health Service perspective in England and Wales, with health states based on seizure frequency and seizure-free days.

“A cohort-based model was developed using a National Health Service perspective and lifetime horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and quality-adjusted life-years (QALYs) were discounted at a rate of 3.5%, in line with the NICE reference case” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In the base case, compared with usual care alone, 12 mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The results of the PSA showed that compared with usual care alone, cannabidiol plus usual care was associated with an ICER of £27,761.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

Ketogenic diet, vagus nerve stimulation, and resective surgery versus a third antiseizure drug in children with drug-resistant epilepsy and tuberous sclerosis complex, United States: Fallah et al. (2016)

Relevance: Compares vagus nerve stimulation, the ketogenic diet, resective surgery, and a further antiseizure medication for drug-resistant epilepsy in children with tuberous sclerosis complex in the United States.

Access: Not open access

Not quoted: The results are reported in the full text, which is closed access.

“A cost-utility analysis, from a third-party payer perspective, was performed for children with drug-resistant epilepsy that had failed to improve with 2 antiseizure drugs (ASDs) and that was amenable to resective epilepsy surgery, across a time-horizon of 5years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Four strategies were included: (1) resective epilepsy surgery, (2) vagus nerve stimulator (VNS) implantation, (3) ketogenic diet, and (4) addition of a third ASD (specifically, carbamazepine).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Given a willingness-to-pay (WTP) of $100,000 per QALY, the addition of a third ASD ($6600 for a gain of 4.14 QALYs) was the most cost-effective treatment strategy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In a secondary analysis, if the child whose epilepsy had failed to improve with 3 ASDs, ketogenic diet, addition of a fourth ASD, and resective epilepsy surgery were incrementally cost-effective treatment strategies.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Vagus nerve stimulator implantation was more expensive yet less effective than alternative strategies and should not be prioritized.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Rufinamide versus topiramate and lamotrigine as adjunctive treatment in children with Lennox-Gastaut syndrome, United Kingdom: Verdian and Yi (2010)

Relevance: Evaluates rufinamide against topiramate and lamotrigine in children with Lennox-Gastaut syndrome in the United Kingdom.

“To estimate the cost-effectiveness of rufinamide relative to topiramate and lamotrigine as adjunctive treatment for children with Lennox-Gastaut Syndrome (LGS).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A Markov decision analytic model was developed to estimate the incremental cost-effectiveness ratio over a three-year time horizon in patients with LGS uncontrolled by up to three antiepileptic drugs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Efficacy and safety estimates were made using indirect/mixed-treatment comparisons of data obtained from published literature.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Rufinamide resulted in an incremental QALY gain of 0.079 relative to topiramate and 0.021 relative to lamotrigine.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental costs of rufinamide were pound1632 and pound3209, relative to topiramate and lamotrigine, resulting in an incremental cost per QALY gained of pound20,538 and pound154,831, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Utility weights

Utility weights from analyses of Relutrigine

No evidence found.

Utility weights for the same indication

No evidence found.

Utility weights for the same disease in other populations or settings

Utility weights for seizure freedom, improvement, no improvement, and withdrawal, intractable childhood epilepsy: de Kinderen et al. (2015)

Relevance: Utilities for seizure freedom, improvement, no improvement, and withdrawal in children with intractable epilepsy treated with the ketogenic diet or vagus nerve stimulation.

Access: Not open access