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

Rebecca Bresnahan (), Rachel Houten (), Janette Greenhalgh (), Sarah Nevitt (), James Mahon (), Sophie Beale (), Angela Boland (), Devarshi Bhattacharyya (), Yenal Dundar (), Joanne McEntee (), Shreyans Gandhi (), Nigel Fleeman () and Marty Chaplin ()
Additional contact information
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

PharmacoEconomics - Open, 2023, vol. 7, issue 4, No 2, 525-536

Abstract: 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

Date: 2023
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DOI: 10.1007/s41669-023-00408-z

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