Suggested remit: To appraise the clinical and cost effectiveness of vamorolone within its marketing authorisation for the treatment of inflammation associated with Duchenne muscular dystrophy.
 
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
Process STA Standard
ID number 4024

Provisional Schedule

Committee meeting 05 March 2024
Expected publication 22 May 2024

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter

Stakeholders

Companies sponsors Santhera (vamorolone)
Others Department of Health and Social Care
  NHS England
Patient carer groups Action Duchenne
  Duchenne UK
  Gene People
  Muscular Dystrophy UK
Professional groups Association of British Neurologists
  British Paediatric Respiratory Society
  Neonatal and Paediatric Pharmacists Group
  Royal College of Physicians
Comparator companies AAH Pharmaceuticals Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Accord-UK Ltd (prednisone; prednisolone) (confidentiality agreement not signed, not participating)
  Advanz Pharma (dexamethasone, prednisone; prednisolone) (confidentiality agreement not signed, not participating)
  Aerona Clinical Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Almus Pharmaceuticals Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Alliance Healthcare (Distribution) Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Aspen (dexamethasone) (confidentiality agreement not signed, not participating)
  Aspire Pharma Ltd (dexamethasone) (confidentiality agreement not signed, not participating)
  Bristol Laboratories Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Cheplapharm (deflazacort) (confidentiality agreement not signed, not participating)
  Focus Pharmaceuticals Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Genesis Pharmaceuticals Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Logixx Pharma Solutions Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Medihealth (Northern) Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Phoenix Labs (prednisone) (confidentiality agreement not signed, not participating)
  Relonchem Ltd (prednisolone) (confidentiality agreement not signed, not participating)
  Sigma Pharmaceuticals PLC (prednisolone) (confidentiality agreement not signed, not participating)
  Wockhardt UK Ltd (prednisone; prednisolone) (confidentiality agreement not signed, not participating)
  Zentiva (prednisone) (confidentiality agreement not signed, not 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
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee

Timeline

Key events during the development of the guidance:

Date Update
01 August 2023 Invitation to participate
01 August 2023 The topic routing was considered by the Topic Selection Oversight Panel (TSOP) in April 2023. The panel concluded that the topic should be routed to the Technology Appraisal (TA) work programme. Please see project documents for further details.
31 October 2022 (14:00) Scoping workshop
08 September 2022 - 06 October 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
08 September 2022 In progress. Scoping commenced
30 September 2021 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

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