Suggested remit: To evaluate the benefits and costs of OTL-200 within its marketing authorisation for treating metachromatic leukodystrophy for national commissioning by NHS England.
Status In progress
Process HST
ID number 1666

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


Companies sponsors Orchard Therapeutics (OTL-200)
Others Department of Health and Social Care
  NHS England
Patient carer groups Alex, The Leukodystrophy Charity
  ArchAngel MLD Trust
  MLD Support Association UK
  MPS Society
Professional groups British Paediatric Neurology Association
  Royal College of Physicians


Associated public health groups 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 Health Specialised Services Committee
Relevant research groups None


Key events during the development of the guidance:

Date Update
10 June 2021 Committee meeting: 2
15 April 2021 Committee meeting: 1
23 September 2020 Invitation to participate
23 September 2020 In progress. In progress
06 April 2020 Topic update: this evaluation has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.
27 January 2020 (14:00) Scoping workshop (London)
14 November 2019 - 12 December 2019 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