Suggested remit: To evaluate the benefits and costs of olipudase alfa within its marketing authorisation for treating acid sphingomyelinase deficiency (Niemann Pick disease type B and AB) for national commissioning by NHS England.
Status In progress
Technology type Medicine
Decision Selected
Process HST Standard
ID number 3913

Project Team

Project lead Vonda Murray

Email enquiries

External Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter


Companies sponsors Sanofi (olipudase alfa)
Others Department of Health and Social Care
  Health Technology Wales (HTW)
  NHS England
Patient carer groups Niemann-Pick UK
Professional groups British Inherited Metabolic Disease Group
  Royal College of Physicians
Associated public health groups None
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
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
03 April 2024 Appeal
05 March 2024 - 19 March 2024 Final draft guidance
07 December 2023 Committee meeting: 2
26 October 2023 - 16 November 2023 Draft guidance
05 October 2023 Committee meeting: 1
21 July 2023 In progress. In progress
21 July 2023 In progress. website update
21 July 2023 In progress
14 June 2022 Invitation to participate
17 March 2022 (10:00) Scoping workshop
19 January 2022 - 16 February 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

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