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Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

Author

Listed:
  • Rebecca Bresnahan

    (University of Liverpool)

  • Rachel Houten

    (University of Liverpool)

  • Janette Greenhalgh

    (University of Liverpool)

  • Sarah Nevitt

    (University of Liverpool)

  • James Mahon

    (Coldingham Analytical Services)

  • Sophie Beale

    (Hare Research)

  • Angela Boland

    (University of Liverpool)

  • Devarshi Bhattacharyya

    (University of Liverpool)

  • Yenal Dundar

    (University of Liverpool)

  • Joanne McEntee

    (North West Medicines Information Centre)

  • Shreyans Gandhi

    (Kings College Hospital NHS Foundation Trust)

  • Nigel Fleeman

    (University of Liverpool)

  • Marty Chaplin

    (University of Liverpool)

Abstract

As part of the Single Technology Appraisal (STA) process, the UK National Institute for Health and Care Excellence (NICE) invited Apellis Pharmaceuticals/Sobi to submit evidence for the clinical and cost effectiveness of pegcetacoplan versus eculizumab and pegcetacoplan versus ravulizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults whose anaemia is uncontrolled after treatment with a C5 inhibitor. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned as the Evidence Review Group (ERG). The company pursued a low incremental cost-effectiveness ratio (ICER) Fast Track Appraisal (FTA). This was a form of STA processed in a shorter time frame and designed for technologies with company base-case ICER

Suggested Citation

  • Rebecca Bresnahan & Rachel Houten & Janette Greenhalgh & Sarah Nevitt & James Mahon & Sophie Beale & Angela Boland & Devarshi Bhattacharyya & Yenal Dundar & Joanne McEntee & Shreyans Gandhi & Nigel Fl, 2023. "Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal," PharmacoEconomics - Open, Springer, vol. 7(4), pages 525-536, July.
  • Handle: RePEc:spr:pharmo:v:7:y:2023:i:4:d:10.1007_s41669-023-00408-z
    DOI: 10.1007/s41669-023-00408-z
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