Evidence map›Paper›PMID 38441854›Full record

ArticlePharmacoEconomics - open2024

A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

Colin Burke, Catriona Crossan, Emma Tyas, Matthew Hemstock, Dawn Lee, Sally Bowditch

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in PharmacoEconomics - open, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02544763 (A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.7field-weighted citation impact, top 33% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02544763 phase3completednot on this map

A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients With Tuberous Sclerosis Complex Who Experience Inadequately-controlled Seizures

TypeinterventionalSponsorJazz PharmaceuticalsRan2016 to 2019Enrolled224ConditionsTuberous Sclerosis Complex, SeizuresArmsGWP42003-P, Placebo
3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 2 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 1 institution in 1 country.

Colin BurkeLumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK. Colin.Burke@lumanity.com.ORCID http://orcid.org/0009-0001-4671-7600
Catriona CrossanLumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK.
Emma TyasLumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK.ORCID http://orcid.org/0009-0002-6984-1425
Matthew HemstockLumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK.ORCID http://orcid.org/0000-0002-8585-0586
Dawn LeeLumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK.ORCID http://orcid.org/0000-0003-4027-8456
Sally BowditchJazz Pharmaceuticals, Inc., 1 Cavendish Place, Marylebone, London, W1G 0QF, UK.ORCID http://orcid.org/0000-0002-6918-2936
University of Suffolk · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesThe aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex

methodsA cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature.

resultsIn 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. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. 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. Results were robust to sensitivity and scenario analyses.

conclusionsAt 12 mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥ 2 years who are refractory to current treatment.

Identifiers

PMID38441854
PMCPMC11252107
OpenAlexW4392462470

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.