Suggested remit: To appraise the clinical and cost effectiveness of nusinersen and risdiplam within their marketing authorisations for treating spinal muscular atrophy.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Further information Scoping commenced.
Process MTA Standard
ID number 6195

Provisional Schedule

Committee meeting 02 October 2024
Expected publication 04 December 2024

Project Team

Project lead Louise Jafferally

Email enquiries

External Assessment Group Warwick Evidence, Warwick Medical School, University of Warwick


Companies sponsors Biogen (Nusinersen)
  Roche (Risdiplam)
Others Department of Health and Social Care
  NHS England
Patient carer groups Gene People
  Muscular Dystrophy UK
  Spinal Muscular Atrophy UK
Professional groups Adult SMA Reach
  Association of British Neurologists
  British Paediatric Respiratory Society
  British Society of Physical and Rehabilitation Medicine
  Royal College of Physicians
  SMA Reach UK
  UK National Screening Committee
Comparator companies Novartis Gene Therapies, Inc. (onasemnogene abeparvovec) (confidentiality agreement signed, participating)
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
  Welsh Government
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
05 January 2024 Invitation to participate
28 July 2023 - 25 August 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
28 July 2023 In progress. Scoping commenced.

For further information on our processes and methods, please see our CHTE processes and methods manual