ArticlePharmacoEconomics2020
Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria.
Article in PharmacoEconomics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed, 24 citations in OpenAlex.
- Shifting From Systemic to Precision-Targeted Complement Therapies: Opportunities and Hurdles.European journal of immunology · 2026Review
- Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations.Cost effectiveness and resource allocation : C/E · 2025Review
- Article
- Two-decade battle with myasthenia gravis: A breakthrough case report on the long-term success with eculizumab and ravulizumab treatment.Clinical case reports · 2024Article
- Population pharmacokinetic, pharmacodynamic and efficacy modeling of SB12 (proposed eculizumab biosimilar) and reference eculizumab.European journal of clinical pharmacology · 2024Article
- Dosing Patterns of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab in the United States: A Retrospective Claims-Based Analysis.Advances in therapy · 2024Article
- Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands.The European journal of health economics : HEPAC : health economics in prevention and care · 2023Article
- The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.Journal of comparative effectiveness research · 2023Article
- Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab and Eculizumab in Complement Inhibitor-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria: A Matching-Adjusted Indirect Comparison.Advances in therapy · 2023Article
- Composite endpoint to evaluate complement inhibition therapy in patients with paroxysmal nocturnal hemoglobinuria.European journal of haematology · 2022Article
- First Report on Successful Conversion of Long-Term Treatment of Recurrent Atypical Hemolytic Uremic Syndrome With Eculizumab to Ravulizumab in a Renal Transplant Patient.Transplant international : official journal of the European Society for Organ Transplantation · 2022Article
- Case Report: Atypical HUS Presenting With Acute Rhabdomyolysis Highlights the Need for Individualized Eculizumab Dosing.Frontiers in pediatrics · 2022Article
- Current Opinions on the Clinical Utility of Ravulizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria.Therapeutics and clinical risk management · 2021Review
- A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?Therapeutic advances in rare diseaseReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundParoxysmal nocturnal hemoglobinuria, characterized by intravascular hemolysis and venous thrombosis, can be managed with eculizumab, an inhibitor of the complement system; however, patients may periodically experience breakthrough hemolysis. Ravulizumab is a newly approved treatment for paroxysmal nocturnal hemoglobinuria that may reduce breakthrough hemolysis risk, thus improving health-related quality of life and reducing treatment costs.
objectiveThe objective of this study was to compare the costs and benefit of treatment with ravulizumab vs eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria, from a US payer perspective.
methodsA cost-utility analysis was conducted using a semi-Markov model, informed by clinical experts. Lifetime costs and benefit (quality-adjusted life-years) (both discounted at 3% per annum) and incremental cost-effectiveness ratios were estimated, over a lifetime horizon. Results are reported for an entire treated population and subgroups of eculizumab treatment history. Scenario analyses were characterized by assumptions of non-inferiority between treatments, in terms of breakthrough hemolysis incidence and blood transfusion requirements, and of variations in eculizumab dosing adjustments used in response to breakthrough hemolysis.
resultsIn the base-case analysis for the overall population, there was a positive impact on health-related quality of life (quality-adjusted life-year gain of 1.67) and costs were lower (- $1,673,465), for ravulizumab vs eculizumab. This led to a negative incremental cost-effectiveness ratio (- $1,000,818, indicating cost savings per quality-adjusted life-year gained). Health-related quality-of-life improvement and cost savings were also observed in all cohorts and scenario analyses.
conclusionsIn adults with paroxysmal nocturnal hemoglobinuria, ravulizumab is associated with improved health-related quality of life and provides a large cost saving from the perspective of a US payer, when compared with eculizumab.
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Registered trials
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