Suggested remit: To appraise the clinical and cost effectiveness of givinostat within its marketing authorisation for treating Duchenne muscular dystrophy in people 6 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
Process STA Standard
ID number 6323

Provisional Schedule

Committee meeting 13 May 2025

Project Team

Project lead Leena Issa

Email enquiries

Stakeholders

Companies sponsors ITF Pharma UK (givinostat)
Others Alder Hey Children’s Hospital NHS Foundation Trust, Liverpool
  Bristol Royal Hospital for Children, Bristol
  Department of Health and Social Care
  Dubowitz Neuromuscular Centre (DNC)
  MD UK Oxford Neuromuscular Centre, Oxford
  MRC Centre for Neuromuscular Diseases
  NHS England
  Queen Square Centre for Neuromuscular Diseases UCL
  Robert Jones and Agnes Hunt Orthopaedic Hospital, Oswestry
  Royal Manchester Children’s Hospital NHS Foundation Trust, Manchester
  Ryegate Centre, Sheffield Children’s NHS Foundation Trust, Sheffield
  The Addenbrooke’s Neuromuscular Service, Cambridge
  The John Walton Muscular Dystrophy Research Centre, Newcastle
  The National Hospital for Neurology and Neurosurgery, London
  The Walton Centre, Liverpool
  Wessex Neurological Centre, Southampton General Hospital, Southampton
Patient carer groups Action Duchenne
  Alex's Wish
  Arthritis & Musculoskeletal Alliance
  Beacon
  Duchenne Family Support Group
  Duchenne Research Fund
  Duchenne UK
  Gene People
  Genetic Alliance UK
  Harrison’s Fund
  Joining Jack
  Muscular Dystrophy UK
  Pathfinders Neuromuscular Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Together for Short Lives
Professional groups Association of Anaesthetists of Great Britain & Ireland
  Association of British Neurologists
  Association of Chartered Physiotherapists in Respiratory Care
  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 Geriatrics Society
  British Institute of Musculoskeletal Medicine
  British Myology Society
  British Orthopaedic Association
  British Paediatric and Adolescent Bone Group
  British Paediatric Neurology Association
  British Paediatric Respiratory Society
  British Society for Children's Orthopaedic Surgery
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Paediatric Endocrinology and Diabetes
  British Society of Physical and Rehabilitation
  British Thoracic Society
  Chartered Society of Physiotherapy
  Neonatal and Paediatric Pharmacists Group
  Primary Care Respiratory Society UK
  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
  Society for Endocrinology
  The National Congenital Anomaly and Rare Disease Registration Service
  The North Star Clinical Network
  UK Clinical Pharmacy Association
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies PTC Therapeutics (ataluren)
General commentators All Wales Inherited Metabolic Disease Service
  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
  National Services Division
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Bone Research Society
  Cochrane Cystic Fibrosis and Genetic Disorders Group
  Cochrane Musculoskeletal Group
  Cochrane UK
  Genomics England
  MRC Centre for Neuromuscular Diseases
  MRC Clinical Trials Unit
  National Institute for Health Research
  Orthopaedic Research UK
  TREAT-NMD

Timeline

Key events during the development of the guidance:

Date Update
07 August 2024 Topic routing was discussed at the NICE Prioritisation Board in June 2024. The Board concluded that the topic was suitable for a Technology Appraisal. Please see project documents for further details.
05 August 2024 Please note that following on from a request received from the company, the timelines for this appraisal have been revised. The deadline for submissions is expected in approximately early December 2024. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission.
13 June 2024 Invitation to participate
09 April 2024 - 08 May 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6323
09 April 2024 In progress. Scoping commenced.
13 July 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