Suggested remit: To appraise the clinical and cost effectiveness of sirolimus within its marketing authorisation for treating angiofibroma from tuberous sclerosis complex in people 3 years and older.
The company has informed NICE that it will be reviewing and revising its evidence submission for this appraisal. Therefore, we are suspending the appraisal while we consider the next steps.
Status Suspended
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
Process STA Standard
ID number 3990

Provisional Schedule

Committee meeting: 1 11 July 2024
Expected publication 18 September 2024

Project Team

Project lead Jeremy Powell

Email enquiries

External Assessment Group Bristol Technology Assessment Group, University of Bristol


Companies sponsors Plusultra (sirolimus)
Others Department of Health and Social Care
  NHS England
Patient carer groups Changing Faces
  Genetic Alliance UK
  Gene People
  Lets Face It
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Tuberous Sclerosis Association
Professional groups Association of Genetic Nurses & Counsellors
  British Association of Dermatologists
  British Dermatological Nursing Group
  British Geriatrics Society
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  General Medical Council
  Neonatal and Paediatric Pharmacists Group
  Primary Care Dermatology Society
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Genetic Testing Network
  UK Kidney Association
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies none
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Great Ormond Street Hospital
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  NHS Confederation
  Paediatric Neurodisability TSC Clinic, Alder Hey Children’s Hospital
  Paediatric Neurology TSC Clinic, University Hospitals Bristol
  Scottish Medicines Consortium
  TSC Team, Children’s Centre, Royal United Hospital Bath
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups British Skin Foundation
  Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane Skin Group
  Cochrane UK
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
09 May 2024 Suspended. The company has informed NICE that it will be reviewing and revising its evidence submission for this appraisal. Therefore, we are suspending the appraisal while we consider the next steps.
15 December 2023 Invitation to participate
15 December 2023 At the company's request, the appraisal has restarted.
18 October 2023 Please note that following on from information received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin during late November 2023 when we will write to you about how you can get involved.
06 June 2023 Please note that following on from advice received from the company, further information regarding the timelines for this appraisal will be available in due course. The planned Committee meeting will not take place. In the meantime, NICE will continue to monitor the situation and will provide an update as and when the situation changes.
24 October 2022 (14:00) Scoping workshop
16 August 2022 - 14 September 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
16 August 2022 In progress. Scoping commenced
28 January 2022 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