Cannabidiol for Adjuvant Treatment of Seizures Associated with Lennox–Gastaut Syndrome and Dravet Syndrome: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

@article{Wijnen2020CannabidiolFA,
  title={Cannabidiol for Adjuvant Treatment of Seizures Associated with Lennox–Gastaut Syndrome and Dravet Syndrome: An Evidence Review Group Perspective of a NICE Single Technology Appraisal},
  author={Ben F. M. Wijnen and Nigel Armstrong and Bram L. T. Ramaekers and Willem J. A. Witlox and Marie E. Westwood and Debra Fayter and Steve Ryder and Titas Buksnys and Gill Worthy and Kate Misso and Sabine E. Grimm and Jos Kleijnen and Manuela A. Joore},
  journal={PharmacoEconomics},
  year={2020},
  volume={38},
  pages={1043 - 1053}
}
GW Research Ltd. provided two separate, but similar, submissions to the National Institute for Health and Care Excellence (NICE) on the clinical and cost-effectiveness of cannabidiol (CBD) 10 mg/kg/day, trade name Epidyolex®, for the adjunctive treatment of seizures associated with Lennox–Gastaut syndrome (LGS) and Dravet syndrome (DS). This paper highlights important methodological issues related to the company submissions, the Evidence Review Group (ERG) reports, and the subsequent… 

Comment on “Cost-Effectiveness of Cannabidiol Adjunct Therapy Versus Usual Care for the Treatment of Seizures in Lennox-Gastaut Syndrome”

Given the high seizure frequency associated with LGS and DS, model structures based on absolute seizure frequency and seizure-free days may be more appropriate to reflect the heterogeneity of the patient population and capture the characteristics of the disease.

Advances in the design and discovery of novel small molecule drugs for the treatment of Dravet Syndrome

The authors review the current treatment strategies as well as potential drugs in the initial stages of clinical evaluation and review drugs with protective activity in mice models of Dravet syndrome.

The Continued Need for Transparent Evidence on the Cost Effectiveness of Drugs for Rare Diseases

In this study, the cost effectiveness of cannabinoid oil was assessed, which is fundamentally different from the intervention (Epidiolex®) in the other two studies cited by the authors, and was independently funded and has been subject to peer review as part of the publication process.

Different Cannabis sativa Extraction Methods Result in Different Biological Activities against a Colon Cancer Cell Line and Healthy Colon Cells

The data suggest that Cannabis extracts, when properly prepared, can significantly decrease cancer cell viability while protecting healthy cells from cytotoxic effects, and post-processing of extracts poses a significant limitation in predicting therapeutic response based on the composition of the crude extract.

References

SHOWING 1-10 OF 28 REFERENCES

Cannabidiol with clobazam for treating seizures associated with Lennox–Gastaut syndrome

  • Medicine, Psychology
  • 2019
People having treatment outside this recommendation may continue without change to the funding arrangements in place before this guidance was published, until they and their NHS clinicians consider it appropriate to stop.

Definition and natural history of Lennox‐Gastaut syndrome

Lennox‐Gastaut syndrome (LGS) is a rare epileptic encephalopathy with a peak age of onset of 3–5 years of age. Reported prevalence rates for LGS vary widely from 1–10% of all childhood epilepsies.

Management of Seizures in Lennox-Gastaut Syndrome

The difficulty early in the course of Lennox-Gastaut syndrome is distinguishing this diagnosis from severe myoclonic epilepsy of infancy (Dravet syndrome) or fromMyoclonic-astatic epilepsy (Doose syndrome), as the seizure patterns in these three syndromes may overlap at the onset.

Dravet syndrome: Seizure control and gait in adults with different SCN1A mutations

Different types of SCN1A mutations and seizure control, occurrence of convulsive status epilepticus (cSE), and the presence of crouch gait in adult patients are correlated.

National Institute for Health and Care Excellence.

  • M. Cowie
  • Medicine
    European heart journal
  • 2015
In this procedure, an endoscope and a carbon dioxide laser are inserted through the mouth and used to cut through the muscle, to relieve obstruction and improve swallowing.

Unit Costs of Health and Social Care 2004

This volume includes a considerable expansion of information about the costs of child social care services, based largely on the results of a series of studies commissioned under the Department of Health's Costs and Effectiveness of Services for Children in Need research initiative.

Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices--Modeling Studies.

  • M. WeinsteinB. O'brien B. Luce
  • Political Science
    Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
  • 2003
It is the responsibility of model developers to conduct modeling studies according to the best practicable standards of quality and to communicate results with adequate disclosure of assumptions and with the caveat that conclusions are conditional upon the assumptions and data on which the model is built.

Cannabidiol for adjuvant treatment of seizures associated with Lennox-Gastaut syndrome [ID1308] Document B

  • Company evidence submission: Submission to National Institute of Health and Care Excellence. Single technology appraisal (STA). GW Research Ltd. 2019. https ://www.nice.org.uk/guida nce/ta615 /evide nce/appra isal-consu ltati on-commi ttee-paper s-pdf70176 27422 . Accessed 24 Jan
  • 2019

GWP42003-P: interim synoptic report 2

  • Full version for regulatory submission: an open label extension study to investigate the safety of cannabidiol (GWP42003-P; CBD) in children and adults with inadequately controlled Dravet or Lennox–Gastaut syndromes (Protocol No. GWEP1415) [PDF provided with the company’s submission]. GW Research Lt
  • 2019