Following on from information provided to NICE by the company in May 2018 the appraisal of Eteplirsen for treating Duchenne muscular dystrophy [ID1003] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
- Status:
- Discontinued
- Technology type:
- Medicine
- Decision:
- Prioritised
- Process:
- HST
- ID number:
- 1003
Email enquiries
If you have any queries please email scheduling@nice.org.uk
Stakeholders
- Companies sponsors
- Sarepta Therapeutics
- Others
- Department of Health and Social Care
- NHS England
- Patient carer groups
- Action Duchenne
- Action for Sick Children
- Alex’s Wish
- Arthritis & Musculoskeletal Alliance
- Children’s Society
- Contact a Family
- Disability Rights UK
- DMD Pathfinders
- Duchenne Family Support Group
- Duchenne Now
- Duchenne UK
- Findacure
- Genetic Alliance
- Harrison’s Fund
- Information Advice and Support Service Network
- Joining Jack
- Leonard Cheshire Disability
- Muscular Dystrophy UK
- Muslim Council of Britain
- National Children’s Bureau
- South Asian Health Foundation
- Specialised Healthcare Alliance
- Together for Short Lives
- Professional groups
- Association of Anaesthetists
- Association of British Neurologists
- Association of Genetic Nurses & Counsellors
- Association of Respiratory Nurse Specialists
- Association of Surgeons of Great Britain and Ireland
- British Cardiovascular Society
- British Dietetic Association
- British Heart Rhythm Society
- British Institute of Musculoskeletal Medicine
- British Orthopaedic Association
- British Paediatric Neurology Association
- British Paediatric Respiratory Society
- British Society for Genetic Medicine
- British Society of Rehabilitation Medicine
- British Thoracic Society
- Chartered Society of Physiotherapy
- Physiotherapy Pain Association
- Primary Care Respiratory Society
- Royal College of Anaesthetists
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Paediatrics and Child Health
- Royal College of Pathologists
- Royal College of Physicians
- Royal College of Surgeons
- Royal Pharmaceutical Society
- Royal Society of Medicine
- The National Congenital Anomaly and Rare Disease Registration Service
- UK Genetic Testing Network
- UK Clinical Pharmacy Association
- Associated public health groups
- Public Health England
- Public Health Wales
- 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
- Department of Health, Social Services and Public Safety for Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- National Association of Primary Care
- National Pharmacy Association
- NHS Alliance
- NHS Commercial Medicines Unit
- NHS Confederation
- NHS National Services Scotland
- Scottish Medicines Consortium
- Welsh Government
- Welsh Health Specialised Services Committee
- Relevant research groups
- British Myology Society
- Cochrane Cystic Fibrosis and Genetic Disorders Group
- Cochrane Musculoskeletal Group
- Duchenne Research Fund
- MRC Centre for Neuromuscular Diseases
- MRC Clinical Trials Unit
- Newcastle University John Walton Centre for Muscular Dystrophy Research
- North Star Clinical Network
- National Institute for Health Research
- TREAT-NMD
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 15 November 2022 | Discontinued. Following on from information provided to NICE by the company in May 2018 the appraisal of Eteplirsen for treating Duchenne muscular dystrophy [ID1003] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued. |
| 04 July 2018 | Suspended. Suspended |
| 04 July 2018 | Note added to the project documents |
| 11 May 2018 | Note added to the project documents |
| 16 March 2018 | Invitation to participate |
| 13 March 2018 | In progress. Topic referred |
| 01 March 2017 | Referral |
| 04 August 2016 - 25 August 2016 | Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators |
For further information on our processes and methods, please see our CHTE processes and methods manual