Suggested remit: To appraise the clinical and cost effectiveness of ravulizumab within its marketing authorisation for treating paroxysmal nocturnal haemoglobinuria.
 
Status In progress
Process STA 2018
ID number 1457

Provisional Schedule

Expected publication 19 May 2021

Project Team

Project lead Gavin Kenny

Email enquiries

Consultees

Companies sponsors Alexion Pharma UK (ravulizumab)
  Department of Health and Social Care
  NHS England
Patient carer groups PNH Support
  The Aplastic Anaemia Trust
Professional groups Royal College of Physicians

Commentators

Comparator companies Alexion Pharma UK (eculizumab) (confidentiality agreement not signed, not participating)
General commentators British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee

Timeline

Key events during the development of the guidance:

Date Update
15 April 2021 - 29 April 2021 Final appraisal document
06 July 2020 Invitation to participate
17 June 2020 - 01 July 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
15 June 2020 In progress. This has now been rescheduled into the work programme and is due to be discussed at committee on 10 March 2021.
15 June 2020 This topic has been rescheduled into the work programme as a Single Technology Appraisal within the Technology Appraisal (TA) process, following feedback from the company and stakeholders that eculizumab – which has been available via NHS England’s commissioning statement – should be included as a comparator for this assessment. The company and stakeholders requested NICE reconsider its decision on the comparator as they believe best supportive care (BSC) alone is not the appropriate comparator for this assessment. If NICE acknowledged eculizumab as the comparator for ravulizumab, the only viable approach would be to consider ravulizumab through the TA programme. As such the referral for ravulizumab has been revised from a Highly Specialised Technology (HST) to a TA.
21 April 2020 Schedule affected by COVID-19. Topic update: this evaluation has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.
07 April 2020 Invitation to participate
26 March 2020 In progress. DHSC referral received as a HST
20 February 2020 - 19 March 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
02 November 2018 (10:00) Scoping workshop (London)
19 September 2018 - 17 October 2018 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