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 28 November 2019
Expected publication 03 June 2020

Project Team

Project lead Jo Ekeledo

Email enquiries

Consultees

Companies sponsors AveXis
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Muscular Dystrophy UK
  Spinal Muscular Atrophy UK (SMA UK)
  The Annabelle Rose Foundation for Spinal Muscular Atrophy
  TreatSMA
Professional groups Association of British Neurologists
  British Society for Children's Orthopaedic Surgery
  Royal College of Pathologists
  Royal College of Physicians

Commentators

Comparator companies Biogen
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups None

Timeline

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