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.
 
Status In progress
Process HST
ID number 1284

Provisional Schedule

Committee meeting: 1 13 January 2022
Expected publication 27 June 2022

Project Team

Project lead Jo Ekeledo

Email enquiries

Evidence Review Group / Assessment Group Centre for Reviews and Dissemination and Centre for Health Economics, University of York

Consultees

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

Commentators

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
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 how we develop guidance, please see our page about NICE highly specialised technologies guidance