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

Project Team

Project lead Jeremy Powell

Email enquiries

Stakeholders

Companies sponsors Orion Pharma (ganaxolone)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups CDKL5 UK
Professional groups Association of British Neurologists
  Epilepsy Nurses Association
  Royal College of Physicians
  UK Clinical Pharmacy Association
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
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
31 October 2023 The company has identified an error in the evidence for this appraisal, which they consider may affect the committee’s considerations. NICE is investigating this issue. While we investigate, the final draft guidance is withdrawn, and the appeal period is suspended.
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02 August 2023 - 23 August 2023 Draft guidance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