Suggested Remit: To appraise the clinical and cost effectiveness of voxelotor within its marketing authorisation for treating sickle cell disease .
 
Status In progress
Technology type Medicine
Decision Selected
Process STA 2018
ID number 1403

Provisional Schedule

Committee meeting: 1 07 December 2022
Expected publication 01 March 2023

Project Team

Project lead Kate Moore

Email enquiries

External Assessment Group Liverpool Reviews and Implementation Group, University of Liverpool

Stakeholders

Companies sponsors Global Blood Therapeutics (voxelotor)
Others Department of Health and Social Care
  NHS East Staffordshire CCG
  NHS England
  NHS Greenwich CCG
  Welsh Government
Patient carer groups Action for Sick Children
  AOFAC Foundation
  African Caribbean Leukaemia Trust
  Blood UK
  Black Health Agency for Equality
  Cianna’s Smile
  Contact a Family
  Equalities National Council
  Gene People
  Genetic Alliance UK
  National Children’s Bureau
  Pain UK
  Sickle Cell and Young Stroke Survivors
  Sickle Cell Society
  SickleKan
  South Asian Health Foundation
  Specialised Healthcare Alliance
  UK Thalassaemia Society
Professional groups Association of Genetic Nurses & Counsellors
  British Blood Transfusion Society
  British Committee for Standards in Haematology
  British Geriatrics Society
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Haematology
  British Society for Human Genetics
  Immunodeficiency UK
  Neonatal and Paediatric Pharmacists Group
  NHS Blood and Transplant
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics and Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Forum on Haemoglobin Disorders
  UK Genetic Testing Network
  United Kingdom National Screening Committee
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies AAH Pharmaceuticals (hydroxycarbamide)
  Alliance Healthcare (hydroxycarbamide)
  Bristol-Myers Squibb (hydroxycarbamide)
  Masters Pharmaceuticals (hydroxycarbamide)
  Medac GmbH (hydroxycarbamide)
  Medihealth (hydroxycarbamide)
  Nova Laboratories (hydroxycarbamide)
  Phoenix Healthcare Distribution (hydroxycarbamide)
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Hospital Information Services – Jehovah’s Witnesses
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane Haematological Malignancies Group
  Cochrane UK
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
05 April 2022 Invitation to participate
05 April 2022 In progress. Invitation to participate
16 March 2022 As you will be aware, the Department for Health and Social Care has asked NICE to conduct an appraisal of Voxelotor for treating haemolytic anaemia in people with sickle cell disease. Please note that following on from a request received from the company, the timelines for this evaluation have been revised and it is now anticipated to begin in early April 2022. These timings are based on a request from the company to reschedule the evaluation, in order to analyse additional data.
28 September 2021 Please note that following on from a request received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin in early February 2022. The additional time will allow the company to gather and review further data to enhance the development of a robust dossier of evidence.
23 April 2021 - 24 May 2021 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
19 February 2021 The Department for Health and Social Care has asked NICE to conduct an appraisal of Voxelotor for treating sickle cell disease. Please note that following on from information received from the company, the timelines for this appraisal have now been scheduled and the appraisal is anticipated to begin in mid-September 2021 when we will write to you about how you can get involved.
24 June 2019 The National Institute for Health and Care Excellence (NICE) has been asked to consider an appraisal of voxelotor for treating sickle cell disease. We have recently invited stakeholders to respond to a written consultation on the draft scope. On receiving updated regulatory timing information from the company that is developing voxelotor, NICE has agreed that holding a scoping exercise at this time would not be appropriate. Consequently, the consultation on the draft scope will be closed. The scoping exercise will be rescheduled to take place in line with anticipated regulatory approval timings. We apologise for any inconvenience this may cause. If you have any comments or concerns please contact the project manager for this proposed appraisal Emily Richards on (44 (0)161 413 4070 or via email on emily.richards@nice.org.uk)
23 May 2019 - 21 June 2019 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

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