Status In progress
Process STA pre-2018
ID number 1069

Provisional Schedule

Final appraisal document 03 June 2019 - 17 June 2019
Expected publication 26 June 2019

Project Team

Project lead Joanne Ekeledo

Email enquiries

Evidence Review Group / Assessment Group School of Health and Related Research (ScHARR)


Companies sponsors Biogen
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Muscular Dystrophy UK
  Spinal Muscular Atrophy UK
Professional groups Association of British Neurologists
  Association of Paediatric Anaesthetists of Great Britain and Ireland
  British Paediatric Neurology Association
  British Society for Children's Orthopaedic Surgery
  Royal College of Physicians
  SMA Reach


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 John Walton Muscular Dystrophy Research Centre


Key events during the development of the guidance:

Date Update
30 April 2019 Following the meeting on 6 March 2019, the appraisal committee asked NHS England and Biogen to enter into commercial discussions to see whether a managed access arrangement could be agreed for nusinersen (Spinraza) consistent with NICE’s framework for determining cost effectiveness. The outcome of these discussions and the consequences for guidance development for this topic will be considered by the committee on Wednesday 8 May. Given the nature of the discussion, this will be held in private.
06 March 2019 Committee meeting: 3
01 February 2019 Following recent discussions Biogen have now submitted to NICE a revised submission for the committee to consider. As a result NICE will be holding a committee meeting to review nusinersen on 6 March 2019.
12 November 2018 Following the 2nd appraisal committee meeting for nusinersen on Tuesday 23 October, we advised registered stakeholders that NICE and Biogen were continuing to progress discussions and that we are not in a position to provide the outcome of the committee’s deliberations. We wish to reiterate to all stakeholders and the wider SMA community that these discussions remain ongoing. We recognise that this continuing uncertainty will be concerning for the community but please be assured that NICE are committed to swiftly progressing discussions with Biogen and NHS England and we will keep all those involved up to date with progress as soon as there is something to report.
23 October 2018 Committee meeting: 2
14 August 2018 - 05 September 2018 Appraisal consultation: 1
19 July 2018 To allow time for consultation on the provisional recommendation (once released), the second committee discussion of nusinersen for treating spinal muscular atrophy has had to be rescheduled and will now be considered by committee on Tuesday 23 October 2018.
27 June 2018 Committee meeting: 1
18 January 2018 Invitation to participate
04 January 2018 In progress, In progress

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance