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 Awaiting development
Process STA Standard
ID number 6145

Provisional Schedule

Committee meeting 12 June 2024
Expected publication 23 October 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Centre for Reviews and Dissemination and Centre for Health Economics, University of York


Companies sponsors BioMarin (cerliponase alfa)
Others Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit
  Bristol Royal Hospital for Children
  Department of Health and Social Care
  Newcastle upon Tyne Hospitals NHS Foundation Trust, Great North Children’s Hospital
  NHS England
Patient carer groups Batten Disease Family Association
Professional groups Royal College of Physicians
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 Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research
  Rare Disease Research Partners


Key events during the development of the guidance:

Date Update
31 October 2023 Invitation to participate
14 September 2023 - 12 October 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
14 September 2023 In progress. Draft scope issued for consultation.
11 August 2023 Awaiting development

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