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 08 October 2020
Expected publication 25 March 2021

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 None
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
26 June 2020 Onasemnogene abeparvovec for treating type 1 spinal muscular atrophy has now been rescheduled into the work programme and is due to be discussed at the HST committee on 8 October 2020. Timelines and the expected publication date will be updated in due course. The new timelines are subject to staff capacity and the ongoing management of the COVID-19 situation and we will continue to review our plans and update our stakeholders accordingly.
03 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 HST evaluation will progress as planned with company and Evidence Review Group input into the next phase of the timeline. NICE will provide further updates on the progress of the evaluation when known.
07 February 2020 The company which markets this technology have advised NICE of an extension to their regulatory timings. Therefore the first committee discussion scheduled to take place on Thursday 26 March 2020 will be rescheduled. A further update will be shared in due course.
17 October 2019 The company which markets this technology have advised NICE of an extension to their regulatory timings. Therefore the first committee discussion scheduled to take place on Thursday 28 November 2019 has been rescheduled and will now take place on Thursday 26 March 2020 at NICE Manchester
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