Suggested remit: To appraise the clinical and cost effectiveness of Beremagene geperpavec within its marketing authorisation for treating skin wounds assocatied with dystrophic epidermolysis bullosa.

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 routing was re-considered by the Topic Selection Oversight Panel (TSOP) in September 2023. The panel concluded that the topic should remain routed to the Technology Appraisal (TA) work programme. Please see project documents for further details. The timelines for the evaluation are to be confirmed.
Process:
STA Standard
ID number:
3959

Provisional Schedule

Committee meeting: 1:
03 September 2025
Expected publication:
29 January 2026

Project Team

Project lead
Jeremy Powell

Email enquiries

If you have any queries please email [email protected]


External Assessment Group:
School of Health and Related Research (ScHARR), University of Sheffield

Stakeholders

Companies sponsors
Krystal Biotech Inc (beremagene geperpavec)
Others
Department of Health and Social Care
 
NHS England
Patient carer groups
Anthony Nolan
 
Beacon
 
Changing Faces
 
Cure EB
 
DEBRA UK
 
Gene People
 
Genetic Alliance UK
 
Let’s Face It
 
South Asian Health Foundation
 
Specialised Healthcare Alliance
Professional groups
Counsellors
 
Association of Paediatric Emergency Medicines
 
British Association of Dermatologists
 
British Dermatological Nursing Group
 
British Geriatrics Society
 
British Society for Cutaneous Allergy
 
British Society for Gene and Cell Therapy
 
British Society for Genetic Medicine
 
British Society for Paediatric and Adolescent Dermatology
 
British Trauma Society
 
National Diagnostic Epidermolysis Bullosa Laboratory
 
Neonatal and Paediatric Pharmacists Group
 
Primary Care Dermatology Society
 
Royal College of Emergency Medicine
 
Royal College of General Practitioners
 
Royal College of Nursing
 
Royal College of Paediatrics & Child Health
 
Royal College of Pathologists
 
Royal College of Physicians
 
Royal Pharmaceutical Society
 
Royal Society of Medicine
 
Society of Tissue Viability
 
St John's Institute of Dermatology
 
Surgical Dressing Manufacturers Association
 
UK Clinical Pharmacy Association
Associated public health groups
Public Health Wales
 
UK Health Security Agency
Comparator companies
Chiesi (Birch bark extract)
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- Northern Ireland
 
Healthcare Improvement Scotland
 
Medicines and Healthcare products Regulatory Agency
 
National Association of Primary Care
 
National Pharmacy Association
 
NHS Confederation
 
Scottish Medicines Consortium
 
Welsh Government
 
Welsh Health Specialised Services Committee
Relevant research groups
University Hospitals Birmingham, Adult Epidermolysis Bullosa Service
 
Birmingham Women’s and Children’s Hospital, Epidermolysis Bullosa Service
 
British Skin Foundation
 
Centre of Evidence-based Dermatology, University of Nottingham
 
Cochrane Skin Group
 
Dermatrust
 
Genomics England
 
Great Ormond Street Children’s Hospital, London, Dermatology
 
Guy’s and St Thomas’ Hospital, London, Dermatology
 
Health Services Research Unit, Department of Public Health, University of Oxford
 
Skin Inflammation Genetics Group, King’s College London
 
MRC Clinical Trials Unit
 
National Institute for Health Research
 
Wounds UK

Timeline

Key events during the development of the guidance:

Date Update
10 February 2025 Invitation to participate
02 December 2024 - 16 December 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
02 December 2024 In progress. We consulted on the draft scope for this appraisal in 2022. The appraisal was paused but is now restarting. To ensure that the scope is up to date we are now holding a short second consultation.
15 January 2024 The topic routing was re-considered by the Topic Selection Oversight Panel (TSOP) in September 2023. The panel concluded that the topic should remain routed to the Technology Appraisal (TA) work programme. Please see project documents for further details. The timelines for the evaluation are to be confirmed.
11 January 2024 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
11 January 2024 Topic selection
28 July 2023 Following a routing challenge raised by the company, regarding the Topic Selection Oversight Panel (TSOP) decision to route Beremagene Geperpavec as a Technology Appraisal, the start of this evaluation is delayed. The timelines for the evaluation will be confirmed once TSOP have reviewed the challenge.
14 June 2023 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
14 June 2023 Topic selection
14 June 2023 Topic selection. Routing update
19 January 2023 (14:00) Scoping workshop
09 November 2022 - 07 December 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
20 May 2022 In progress. Scoping commencing

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