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 TA
ID number 1403

Provisional Schedule

Draft guidance 27 February 2024 - 19 March 2024
Committee meeting: 4 04 April 2024

Project Team

Project lead Kate Moore

Email enquiries

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

Stakeholders

Companies sponsors Pfizer (voxelotor)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups AOFAC Foundation
  Blood Cancer UK
  Cianna’s Smile
  Essenelle Foundation
  Sickle Cell Society
Professional groups UK Forum on Haemoglobin Disorders
  National Haemoglobinopathy Panel
  Royal College of Pathologists
  Royal College of Physicians
Comparator companies AAH Pharmaceuticals (hydroxycarbamide) - not participating
  Alliance Healthcare (hydroxycarbamide) - not participating
  Bristol-Myers Squibb (hydroxycarbamide) - not participating
  Masters Pharmaceuticals (hydroxycarbamide) - not participating
  Medac GmbH (hydroxycarbamide) - not participating
  Medihealth (hydroxycarbamide) - not participating
  Nova Laboratories (hydroxycarbamide) - not participating
  Phoenix Healthcare Distribution (hydroxycarbamide) - 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 Health Specialised Services Committee

Timeline

Key events during the development of the guidance:

Date Update
08 February 2024 Committee meeting: 3
03 January 2024 The third appraisal discussion will be held in on 8 February 2024, following the appeal hearing in September 2023. The appeal outcome was discussed by NICE’s Guidance Executive who have asked for this to go back to committee for further discussion around identifying a plausible ICER or ICER range. This discussion will be based on the existing evidence and, because of the confidential commercial arrangements of treatments used in the economic modelling, will be conducted wholly in private. Draft guidance will be released for public consultation after the meeting and we will contact stakeholders with an anticipated release date for draft guidance after the meeting.
05 December 2023 Appeal decision
14 September 2023 Appeal
28 July 2023 - 11 August 2023 Final draft guidance
14 June 2023 Committee meeting: 2
28 February 2023 The company, Pfizer UK, will be submitting new evidence to address the issues raised in the draft guidance that was issued for consultation. To allow sufficient time for this, the second committee discussion scheduled has been rearranged to 14 June 2023.
03 February 2023 The company, Pfizer UK, has asked to submit new evidence to address the issues raised in the draft guidance that was issued for consultation. NICE has confirmed that it will accept the additional analyses and that this will result in a change to the timelines for the appraisal. Therefore, the second committee meeting discussion currently scheduled for 16 March 2023 has been cancelled. The new date for the committee meeting discussion will be shared in due course.
11 January 2023 - 01 February 2023 Draft guidance
07 December 2022 Committee meeting: 1
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