Suggested remit - To appraise the clinical and cost effectiveness of onasemnogene abeparvovec within its marketing authorisation for treating spinal muscular atrophy type 1.
Status In progress
Process HST
ID number 1473

Provisional Schedule

Committee meeting: 1 31 October 2019
Expected publication 22 April 2020

Project Team

Project lead Jo Ekeledo

Email enquiries


Companies sponsors AveXis
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups ACT for SMA
  Action for Sick Children
  Arthritis and Musculoskeletal Alliance
  Brain and Spine Foundation
  Brain Charity
  Contact a Family
  Disability Rights UK
  Genetic Alliance UK
  Leonard Cheshire Disability
  Muscular Dystrophy UK
  Muslim Council of Britain
  National Children’s Bureau
  Neurological Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Spinal Muscular Atrophy UK (SMA UK)
  The Annabelle Rose Foundation for Spinal Muscular Atrophy
  Together for Short Lives
Professional groups Association of Anaesthetists
  Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  Association of Paediatric Anaesthetists of Great Britain and Ireland
  Association of Surgeons of Great Britain and Ireland
  British Dietetic Association
  British Institute of Musculoskeletal Medicine
  British Myology Society
  British Neuropathological Society
  British Orthopaedic Association
  British Paediatric Neurology Association
  British Society for Children's Orthopaedic Surgery
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  British Society of Rehabilitation Medicine
  Chartered Society for Physiotherapy
  Institute of Neurology
  Neonatal & Paediatric Pharmacists Group
  Primary Care Neurology Society
  Royal College of Anaesthetists
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal College of Surgeons
  Royal Pharmaceutical Society
  Royal Society of Medicine
  SMA Reach
  UK Clinical Pharmacy Association
  UK Genetic Testing Network


Comparator companies Biogen
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
  National Services Division
  Neurological Alliance of Scotland
  NHS Alliance
  NHS Confederation
  NHS Commercial Medicines Unit
  Scottish Medicines Consortium
  Wales Neurological Alliance
  Welsh Health Specialised Services Committee
Relevant research groups John Walton Muscular Dystrophy Research Centre


Key events during the development of the guidance:

Date Update
06 June 2019 Invitation to participate
03 June 2019 In progress, To evaluate the benefits and costs of onasemnogene abeparvovec within its marketing authorisation for treating spinal muscular atrophy type 1 for national commissioning by NHS England
06 November 2018 (10:00) Scoping workshop (Manchester)
19 September 2018 - 17 October 2018 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance