Suggested Remit: To evaluate the benefits and costs of Lenti-D within its marketing authorisation for treating cerebral adrenoleukodystrophy (CALD) for national commissioning by NHS England.
Following on from information provided to NICE by the company in December 2021, the appraisal of Elivaldogene autotemcel for treating cerebral adrenoleukodystrophy [ID1284] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
 
Status Discontinued
Technology type Medicine
Decision Selected
Process HST
ID number 1284

Email enquiries

Stakeholders

Companies sponsors Bluebird Bio (Elivaldogene autotemcel)
Others Department of Health and Social Care
  Great Ormond Street Hospital Metabolic Unit
  NHS England
  Royal Manchester Children’s Hospital
  Royal Liverpool Children's Hospital, Alder Hey
  University College London Hospital Charles Dent Metabolic Unit
Patient carer groups African Caribbean Leukaemia Trust
  Anthony Nolan
  Alex – The Leukodystrophy Charity
  Brain and Spine Foundation
  Contact
  DKMS
  Findacure
  Genetic Alliance
  Metabolic Support UK
  Neurological Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  The Brain Charity
Professional groups Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  British Association of Endocrine and Thyroid Surgeons
  British Neuropathological Society
  British Paediatric Neurology Association
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  British Society for Paediatric Endocrinology and Diabetes
  Institute of Neurology
  Primary Care Neurology Society
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics and 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 England
  Public Health Wales
Comparator companies None
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
  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
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Wales Neurological Alliance
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Brain Research UK
  Cochrane Metabolic and Endocrine Disorders Group
  Genomics England
  MRC Clinical Trials Unit
  National Hospital for Neurology and Neurosurgery
  National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
10 February 2023 Discontinued. Following on from information provided to NICE by the company in December 2021, the appraisal of Elivaldogene autotemcel for treating cerebral adrenoleukodystrophy [ID1284] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
02 December 2021 Suspended. As you will be aware, the Department for Health and Social Care has asked NICE to carry out a Highly Specialised Technology evaluation of Elivaldogene autotemcel for treating cerebral adrenoleukodystrophy [ID1284]. For information the company have advised that they will no longer have a commercial presence in Europe in the near future and will not therefore be making a submission, or making this gene therapy available in the UK. Therefore, in collaboration with the company, NICE has suspended this evaluation from its current work programme. As this evaluation has been referred NICE will continue to monitor any development and will update interested parties if the situation changes.
04 August 2021 Invitation to participate
26 July 2021 In progress. In progress
21 January 2021 (14:00) Scoping workshop
24 November 2020 - 21 December 2020 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

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