Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria

Jeremiah Donoghue, Matthew Youngs, Alex Reeve, Krishna Vydyula, Natalia Kunst, Roochi Trikha and Daniel Gallacher ()
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Jeremiah Donoghue: University of Birmingham
Matthew Youngs: University of Warwick
Alex Reeve: University of Warwick
Krishna Vydyula: University of Warwick
Natalia Kunst: University of York
Roochi Trikha: King’s College Hospital
Daniel Gallacher: University of Birmingham

PharmacoEconomics, 2025, vol. 43, issue 5, No 4, 499-508

Abstract: Abstract In 2024, the National Institute for Health and Care Excellence (NICE) recommended two new health technologies for paroxysmal nocturnal haemoglobinuria. This review systematically compares the clinical and cost-effectiveness evidence considered within the NICE single technology appraisals of iptacopan, danicopan and pegcetacoplan, examines the consistency of the clinical evidence and economic modelling, and considers whether single technology appraisals are a suitable apparatus for consistent decision making. The studies used different follow-up lengths and used different definitions for reporting breakthrough haemolysis (BTH), but otherwise reported similar outcomes and found a significant benefit for their interventions. A lack of direct evidence and unreliable indirect comparisons meant that naïve comparisons across trials were carried into the economic modelling despite differences in their control arms. Approaches to modelling BTH and associated dose escalation differed across appraisals, despite information for pegcetacoplan coming from the same source in each appraisal, which had a large impact on the economic results. This review raises the question of whether NICE should implement multiple technology appraisals more frequently to reduce these inconsistences. Additionally, we recommend the development of a framework for revisiting positive recommendations when the implementation of health technologies deviates from assumptions made in the economic modelling to ensure cost-effective healthcare is preserved.

Date: 2025
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DOI: 10.1007/s40273-025-01472-5

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