The Cost Effectiveness of Rufinamide in the Treatment of Lennox-Gastaut Syndrome in the UK

Benedict, Ágnes; Verdian, L; Maclaine, Grant

doi:10.2165/11313640-000000000-00000

Cited by 22 publications

(48 citation statements)

References 32 publications

(63 reference statements)

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“…14 Available information about the economic utility of these AEDs in LGS, however, is very limited, although recent analyses have reported the cost-effectiveness of rufinamide, based on reduced frequency of total seizures and drop attacks 15 and lower incremental costs per quality-adjusted life-years. 16 With the recent U.S. approval of clobazam as an adjunctive treatment in LGS, the current analysis was conducted to estimate the budget impact of including clobazam as a treatment option horizon was chosen to reflect the budget cycles of U.S. commercial payers.…”

Section: Model Structure and Inputsmentioning

confidence: 99%

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Skornicki¹,

Clements²,

O’Sullivan³

2014

JMCP

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BACKGROUND: In October 2011, clobazam was FDA-approved for adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS), a debilitating childhood epilepsy characterized by drop attacks, for patients 2 years and older.OBJECTIVE: To assess the budget impact of adding clobazam to an antiepileptic drug (AED) portfolio containing topiramate, lamotrigine, and rufinamide in a hypothetical, 100,000-member commercially insured health plan.METHODS: Patient characteristics and AED efficacy (decrease in dropseizure frequency) were modeled with clinical data. Medical costs were derived from administrative claims data from a large U.S. managed health plan, with the assumption that 2.3% of drop seizures required medical care. Two-year budget impact was measured. Results were expressed as the overall difference in costs (medical and pharmacy) to a health plan and cost per member per month (PMPM) after addition of clobazam. Analyses of alternative scenarios were performed.RESULTS: With the assumption that 0.04% of the plan population had LGS, adding clobazam to the formulary resulted in cost savings of $98,059 in year 1 and $131,690 in year 2 (savings of $0.08 and $0.11 PMPM, respectively). Analyses of alternative scenarios with lower seizure rates upon discontinuation or greater long-term efficacy for lamotrigine and topiramate did not substantially alter conclusions. The assumption that fewer drop seizures required medical care resulted in a savings of approximately $5,000 per year with clobazam, which suggested that medically attended drop seizures drive costs.CONCLUSIONS: Medically attended drop seizures are a major cost driver for LGS patients. Adding clobazam to a health plan formulary can have a positive overall budget impact through decreased medical costs associated with drop seizures.

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Section: Model Structure and Inputsmentioning

confidence: 99%

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Skornicki¹,

Clements²,

O’Sullivan³

2014

JMCP

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“…Overall, due to inferior efficacy in controlling DS compared to clobazam, the other medications were all associated with higher overall medical costs. This remained true when the estimated number of DS requiring medical attention was set at 2.3% using data from a survey of physicians in the United Kingdom in conjunction with a claims database from the United States 50. At a lower threshold of 0.76%, topiramate would become the most cost-effective medication in the group.…”

Section: Cost-effectivenessmentioning

confidence: 99%

Comprehensive overview: efficacy, tolerability, and cost-effectiveness of clobazam in Lennox–Gastaut syndrome

Faulkner¹

2015

TCRM

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Clobazam is the newest medication approved by the US Food and Drug Administration (FDA) for the treatment of Lennox–Gastaut syndrome (LGS) in patients at least 2 years of age, although the medication has been available in countries around the world to treat epilepsy and anxiety disorders for many years. Though classified as a benzodiazepine, the drug differs structurally from other drugs in the class as it possesses nitrogen atoms at the 1 and 5 positions within the heterocyclic ring rather than at the 1 and 4 positions. This difference and the classification of clobazam as a partial agonist are believed to be responsible for the decreased incidence of sedative effects compared to other benzodiazepines. Adverse events associated with clobazam use in clinical trials have generally been mild to moderate in nature. Data from an open-label extension trial have confirmed that clobazam is efficacious for the treatment of seizures associated with LGS, particularly atonic seizures (drop seizures), over the long term. Tolerance to the drug’s antiepileptic effects does not seem to be a common occurrence. The drug has proven to be a cost-effective option for therapy, particularly due to its ability to decrease the number of seizures that require medical treatment. Clobazam represents a welcome addition to the treatment options for LGS.

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“…This section provides an overview of the efficacy and tolerability of rufinamide in the adjunctive treatment of LGS according to the results of the pivotal randomized controlled trial [7] and its openlabel, long-term (up to 3 years) extension, [8] which were used as the sources of the input data for the cost-utility [9] and cost-effectiveness [10] analyses discussed in section 4.…”

Section: Clinical Profile Of Rufinamidementioning

confidence: 99%

“…Two fully published pharmacoeconomic analyses have assessed the cost effectiveness [10] or cost utility [9] of rufinamide in the treatment of LGS. Both analyses were performed from the perspective of the UK NHS and included direct medical and social care costs only.…”

Section: Pharmacoeconomic Analysesmentioning

confidence: 99%

Rufinamide

McCormack¹

2012

PharmacoEconomics

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Rufinamide (Inovelon®), a triazole derivative, is an oral antiepileptic drug approved in the EU as adjunctive therapy in the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients aged ≥4 years. The efficacy of oral rufinamide as adjunctive therapy in patients with LGS uncontrolled on one to three concomitant antiepileptic drugs was demonstrated in a pivotal, 12-week, randomized, double-blind trial. Rufinamide significantly reduced the 28-day frequency of both drop attacks and total seizures compared with placebo, and significantly increased the proportions of patients experiencing a ≥50% reduction in each seizure frequency. A significantly higher proportion of rufinamide than placebo recipients recorded an improvement in seizure severity at the end of treatment. Reductions in the frequency of drop attacks and total seizures were maintained in a long-term (up to 3 years), open-label extension study. Oral rufinamide was generally well tolerated in patients with LGS. Somnolence and vomiting were the most common adverse events occurring more frequently with rufinamide than with placebo. Two pharmacoeconomic analyses, using decision-analysis models with 3-month cycles over a time horizon of 3 years, assessed the cost effectiveness and cost utility, respectively, of rufinamide compared with topiramate and lamotrigine as adjunctive therapy in patients with LGS from the perspective of the UK NHS. The cost-effectiveness analysis suggested that rufinamide would be associated with incremental costs of £62 (drop attacks) or £2151 (total seizures) per 1% increase in the number of patients achieving a >50% reduction in seizure frequency over 3 years. The cost-utility analysis predicted that the incremental cost per QALY gained for rufinamide compared with the next less-costly and undominated therapy would be more than 5-fold higher than the commonly accepted willingness-to-pay threshold range in the UK. In conclusion, the available pharmacoeconomic data indicate that rufinamide is more effective, but more expensive, than alternative adjunctive therapies approved for use in patients with LGS in the UK. Rufinamide would appear to be a cost-effective alternative to topiramate. Although rufinamide exceeds conventional cost-effectiveness thresholds when compared with lamotrigine, it may still be considered a valuable treatment option for a devastating orphan disease such as LGS.

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The Cost Effectiveness of Rufinamide in the Treatment of Lennox-Gastaut Syndrome in the UK

Cited by 22 publications

References 32 publications

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Comprehensive overview: efficacy, tolerability, and cost-effectiveness of clobazam in Lennox–Gastaut syndrome

Rufinamide

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The Cost Effectiveness of Rufinamide in the Treatment of Lennox-Gastaut Syndrome in the UK

Cited by 22 publications

References 32 publications

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Budget Impact Analysis of Antiepileptic Drugs for Lennox-Gastaut Syndrome

Comprehensive overview: efficacy, tolerability, and cost-effectiveness of clobazam in Lennox&ndash;Gastaut syndrome

Rufinamide

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Product

Resources

About

Comprehensive overview: efficacy, tolerability, and cost-effectiveness of clobazam in Lennox–Gastaut syndrome