Suggested remit: To appraise the clinical and cost effectiveness of pegzilarginase within its marketing authorisation as an add-on treatment for arginase-1 deficiency.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Further information The topic routing was discussed at the Topic Selection Oversight Panel (TSOP) in April 2023. The panel concluded that the topic was suitable for Highly Specialised Technologies. Please see project documents for further details. The evaluation is expected to begin with an invitation to participate in September 2023, with a first committee meeting in May 2024.
Process HST Standard
ID number 4029

Project Team

Project lead Emily Richards

Email enquiries


Companies sponsors • Immedica
Others Birmingham Children’s Hospital NHS Foundation Trust
  Department of Health and Social Care
  Great Ormond Street Hospital
  NHS England
  Queen Elizabeth Hospitals Birmingham NHS Foundation Trust
  Royal Manchester Children’s Hospital
  Salford Royal Hospital
  Sheffield Children’s Hospital
  University College London Hospital NHS Foundation Trust
Patient carer groups Beacon
  Brain and Spine Foundation (UK)
  Brain Charity
  British Liver Trust
  Children’s Liver Disease Foundation
  Gene People
  Genetic Alliance UK
  Metabolic Support UK
  Neurological Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  British Association for the Study of the Liver
  British Association of Endocrine and Thyroid Surgeons
  British Geriatrics Society
  British Inherited Metabolic Disease Group
  British Neuropathological Society
  British Paediatric Neurology Association
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  Institute of Neurology
  National Metabolic Biochemistry Network
  National Neuroscience Advisory Group
  Neonatal and Paediatric Pharmacists Group
  Primary Care and Community 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
  Society for Endocrinology
  UK Clinical Pharmacy Association
Associated guideline groups NICE - National Guideline Alliance
  NICE - National Guideline Centre
Associated public health groups National Institute for Health and Care Excellence
  Public Health Wales
  UK Health Security Agency (Formerly Public Health England)
Comparator companies Eurocept International
Evidence review group National Institute for Health Research Health Technology Assessment Programme (NETSCC)
  School of Health and Related Research (ScHARR)
General commentators All Wales Inherited Metabolic Disease Service
  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 Services, Cardiff and Vale UHB
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division - Commissioning for Scotland's Health
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Brain Research UK
  Cochrane Cystic Fibrosis and Genetic Disorders Group
  Cochrane Hepato-Biliary Group
  Cochrane Metabolic and Endocrine Disorders Group
  Cochrane UK
  Foundation for Liver Research
  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
17 January 2024 Invitation to participate
21 September 2023 Please note that following on from a request received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin in early-January 2024. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission.
07 September 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
07 September 2023 Topic selection
07 September 2023 Topic selection
07 September 2023 In progress
17 March 2023 (10:00) Scoping workshop
17 January 2023 - 14 February 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
17 January 2023 In progress

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