Suggested remit: To appraise the clinical and cost effectiveness of idebenone within its marketing authorisation for treating visual impairment in Leber’s hereditary optic neuropathy (LHON) in people aged 12 years and over.
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 was re-considered for guidance development by the Topic Selection Oversight Panel in January 2023. The panel concluded that Idebenone meets the selection and eligibility criteria for medicines and has been selected for evaluation. A scoping consultation will take place in May 2023, to develop the draft scope and determine the final routing.
Process STA Standard
ID number 547

Project Team

Project lead Kate Moore

Email enquiries

External Assessment Group BMJ Technology Assessment Group (BMJ-TAG), BMJ


Companies sponsors Chiesi
Others Department of Health and Social Care
  NHS England
Patient carer groups LHON Society
Professional groups Royal College of Ophthalmologists
  Royal College of Physicians
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health - 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
24 April 2024 - 16 May 2024 Draft guidance: 1
08 May 2024 Following the committee meeting on 3 April 2024, we issued draft guidance for consultation. The company, Chiesi Limited, has asked to provide additional evidence to address the committee’s requests as part of their response to the consultation. To allow time for the additional evidence to be submitted by the company and to be critiqued by the External Academic Group, this will no longer be discussed at the committee meeting on 4 June 2024. The second committee meeting discussion will be rearranged, and we will update all stakeholders with the new date in due course.
03 April 2024 Committee meeting: 1
07 September 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
07 September 2023 Topic selection
07 September 2023 In progress
23 August 2023 Invitation to participate
23 August 2023 The topic routing was considered by the Topic Selection Oversight Panel (TSOP) in July 2023. The panel concluded that the topic should be routed to the Technology Appraisal (TA) work programme. Please see project documents for further details
22 August 2023 Topic selection
17 May 2023 - 15 June 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
17 May 2023 In progress. Scoping commenced
20 February 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

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