Status In progress
Decision Selected
Process HST
ID number 1642

Provisional Schedule

Committee meeting 08 September 2022
Expected publication 25 January 2023

Project Team

Project lead Gavin Kenny

Email enquiries

External Assessment Group Warwick Evidence, Warwick Medical School, University of Warwick


Companies sponsors PTC Therapeutics (ataluren)
Others • Department of Health
  • National Commissioning Group for Rare Neuromuscular Disorders
  • NHS England
  • MRC Centre for Neuromuscular Diseases
  • Queen Square Centre for Neuromuscular Diseases UCL
  • Great Ormond Street Hospital
  • Cardiff and Vale University Health Board
  • Abertawe Bro Morgannwg University Health Board
Patient carer groups • Action Duchenne
  • Action for Sick Children
  • Alex’s Wish
  • Arthritis & Musculoskeletal Alliance
  • Black Health Agency
  • Children’s Society
  • Contact
  • Disability Rights UK
  • DMD Pathfinders
  • Duchenne Family Support Group
  • Duchenne UK
  • Findacure
  • Gene People
  • Genetic Alliance UK
  • Harrison’s Fund
  • Joining Jack
  • Muscular Dystrophy UK
  • South Asian Health Foundation
  • Specialised Healthcare Alliance
  • Together for Short Lives
Professional groups • Association of Anaesthetists
  • Association of Genetic Nurses & Counsellors
  • Association of Respiratory Nurses
  • Association of Surgeons of Great Britain and Ireland
  • British Dietetic Association
  • 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 for 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
  • UK Clinical Pharmacy Association
  • UK Genetic Testing Network
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
  • 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
  • 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
  • John Walton Centre for Muscular Dystrophy Research (Newcastle University)
  • MRC Centre for Neuromuscular Diseases
  • MRC Clinical Trials Unit
  • North Star Clinical Network
  • National Institute for Health Research


Key events during the development of the guidance:

Date Update
10 January 2022 Invitation to participate
17 November 2021 - 15 December 2021 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators
13 May 2021 NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) have reached an agreement that will extend the Managed Access Agreement (MAA) for Translarna (ataluren) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene. This agreement extends the MAA, that was originally scheduled to end in July 2021, to January 2023. This will enable PTC Therapeutics to access more complete data in preparation for their resubmission to NICE and the re-evaluation of ataluren before the end of the MAA, the outcome of which will determine how ataluren will be commissioned in the long term.
09 April 2021 The Managed Access Agreement (MAA) for ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene is scheduled to end in July 2021. NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) are exploring options to extend the MAA to account for the new timeline for the NICE re-evaluation, which was paused as part of NICE’s COVID-19 workplan prioritisation framework. Discussions are continuing and an update will be issued as soon as the outcome is known.
06 April 2020 In progress. Topic is in progress
06 April 2020 Topic update: this evaluation has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.

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