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:
- Prioritised
- Process:
- HST
- ID number:
- 1284
Email enquiries
If you have any queries please email scheduling@nice.org.uk
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