Status In progress
Decision Selected
Process HST
ID number 800

Project Team

Project lead Daniel Davies

Email enquiries


Companies sponsors Chiesi Ltd
Others Addenbrooke's Lysosomal Disorders Unit
  Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit
  Department of Health
  National Hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit
  NHS England
  Royal Free Lysosomal Storage Disorders Unit
  Willink Unit, Genetic Medicine, Central Manchester Foundation Trust
Patient carer groups MPS Society
Professional groups Royal College of Physicians
  Royal College of Paediatrics and Child Health
Comparator companies None
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
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
18 January 2023 Committee meeting: 5
21 October 2022 - 25 November 2022 Evaluation consultation: 2 re-consultation
08 July 2022 - 16 August 2022 Evaluation consultation: 2
08 June 2022 Committee meeting: 4
06 June 2022 Updated scope reissued
06 June 2022 Draft scope documents
26 June 2020 Velmanase alfa for treating alpha-mannosidosis will be scheduled back into the work programme. Timelines for the continuation of this evaluation are to be determined. A further update in due course will be shared in due course. The new timelines are subject to staff capacity and the ongoing management of the COVID-19 situation and we will continue to review our plans and update our stakeholders accordingly
29 August 2019 Committee meeting: 3
08 July 2019 The company (Chiesi) are to submit further information to NICE for the HST Committee consideration. Therefore a further committee discussion of this HST evaluation has been scheduled to take place on Thursday 29 August 2019.
02 October 2018 This HST evaluation has been put on hold. The company which markets this technology (Chiesi) are currently developing an offering for submission to NHS England. Once confirmation of the submission has been received NICE will be in a position to provide a further update on timelines in order to progress the HST evaluation. We will update you on the next steps in due course.
28 June 2018 Committee meeting: 2
23 May 2018 - 14 June 2018 Evaluation consultation: 1
25 April 2018 Committee meeting: 1
08 November 2017 Invitation to participate
20 March 2017 Referral
20 March 2017 In progress. Topic referred.
22 September 2016 - 21 October 2016 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