Suggested remit: To appraise the clinical and cost effectiveness of ganaxolone within its marketing authorisation for treating seizures caused by CDKL5 deficiency disorder (CDD) in people aged 2 years and over.
Status In progress
Technology type Medicine
Decision Awaiting decision
Process TA
ID number 3988

Provisional Schedule

Expected publication 22 November 2023

Project Team

Project lead Jeremy Powell

Email enquiries


Companies sponsors Orion Pharma (ganaxolone)
Others Central Manchester Foundation Trust, Willink Unit, Genetic Medicine
  Department of Health and Social Care
  Manchester centre for genomic medicine
  NHS England
  Rare Disease Collaborative Network (RDCN) for CDKL5 Bristol Royal Hospital for Children
  Welsh Government
Patient carer groups Brain and Spine Foundation
  Brain Charity
  Epilepsy Action
  Epilepsy Society
  Gene People
  Genetic Alliance UK
  Neurological Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Young Epilepsy
Professional groups Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  British Association of Neurologists
  British Geriatrics Society
  British Neuropathological Society
  British Paediatric Epilepsy Group
  British Paediatric Neurology Association
  British Society for Cell and Gene Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  Epilepsy Nurses Association
  Epilepsy Society
  Institute of Neurology
  International League Against Epilepsy UK
  Neonatal and Paediatric Pharmacists Group
  Neuromodulation Society of UK and Ireland
  Primary Care and Community Neurology Society
  Primary Care Neurology Society
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Genetic Testing Network
Associated public health groups Public Health Wales
  UK Health Security Agency
Evidence review group PenTAG Health Technology Assessment
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Inherited Metabolic and Lysosomal Disease Service, Cardiff and Vale UHB
  Medicines and Healthcare Products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division
  Neurological Alliance of Scotland
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Wales Neurological Alliance
  Welsh Health Specialised Services Committee
Relevant research groups Brain Research UK
  British Neurological Research Trust
  Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane Epilepsy Group
  Cochrane UK
  Epilepsy Research UK
  Genomics England
  MRC Clinical Trials Unit
  National Hospital for Neurology and Neurosurgery
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
06 September 2023 Committee meeting: 2
02 August 2023 - 23 August 2023 Draft guidance
06 July 2023 Committee meeting: 1
14 November 2022 Topic selection
25 August 2022 Invitation to participate
28 March 2022 (14:00) Scoping workshop
31 January 2022 - 28 February 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
24 November 2021 In progress

For further information on our processes and methods, please see our CHTE processes and methods manual