Suggested remit: To appraise the clinical and cost effectiveness of risdiplam within its marketing authorisation for treating spinal muscular atrophy in children and adults.
Status In progress
Process STA 2018
ID number 1631

Project Team

Project lead Louise Jafferally

Email enquiries

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


Companies sponsors Roche Products (risdiplam)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Muscular Dystrophy UK
  Spinal Muscular Atrophy UK (SMA UK)
  Treat SMA
Professional groups Association of British Neurologists
  Neonatal & Paediatric Pharmacists Group
  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
  Medicines and Healthcare Products Regulatory Agency
  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
13 July 2021 Committee meeting: 2
02 June 2021 - 23 June 2021 Appraisal consultation
11 May 2021 Committee meeting: 1
27 August 2020 Invitation to participate
27 August 2020 In progress. As the Invitation to Participate has now been sent out, this appraisal is now in progress.
28 July 2020 The Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of risdiplam within its marketing authorisation for treating spinal muscular atrophy in children and adults. I also felt it helpful to write and advise you of the draft timelines we have planned for your information. The appraisal is expected to start in approximately late August 2020 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately early November 2020. For information, these timings are based on a delay for this topic due to COVID-19 and on a mutual agreement between the company and NICE, following a request from the company, in order to facilitate a suitably comprehensive and robust evidence submission.
27 January 2020 - 24 February 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

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