Rapid paper
Key considerations for modelling the long-term costs and benefits of treatments for ANCA-associated vasculitis
V. Berdunov1, A. Ramirez De Arellano2, T. Li3, H. Vintderdag4, G. Baxter5
- Putnam, London, UK. vlad.berdunov@putassoc.com
- CSL Vifor, Glattbrugg, Switzerland.
- Putnam, London, UK.
- CSL Vifor, Glattbrugg, Switzerland.
- CSL Vifor, Staines-upon-Thames, UK.
CER17496
2024 Vol.42, N°4
PI 0782, PF 0785
Rapid paper
Free to view
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PMID: 38526008 [PubMed]
Received: 19/01/2024
Accepted : 12/02/2024
In Press: 25/03/2024
Published: 29/04/2024
Abstract
OBJECTIVES:
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of severe and chronic autoimmune diseases. Patients undergo two treatment phases: inducing remission and maintaining remission to prevent organ damage. Immunosuppressants, including glucocorticoids (GCs) are used as first-line treatment, but long-term GC use is associated with toxic effects. Novel treatments reduce or replace the need for long-term GC, and therefore can reduce GC-related toxicity. The evolving treatment landscape has presented new challenges for health technology assessment (HTA) of new treatments in AAV and long-term modelling of costs and outcomes in this disease.
METHODS:
Using the appraisal of avacopan in England (NICE) as a case study, this paper aims to identify the key challenges involved in the economic evaluation of new treatments for AAV, with a particular focus on the long-term modelling of the treatment costs and benefits for the purpose of HTA. The outcome of this study is a set of recommendations for modelling the cost-effectiveness of new treatments for AAV from the HTA perspective.
RESULTS:
The discussion focuses on the appropriate model structure, approach to modelling end-stage renal disease (ESRD) as a key determinant of costeffectiveness, capturing the impact of GC-related adverse events, and estimation of short and long-term costs of AAV.
CONCLUSIONS:
Economic evaluation of new treatments for AAV needs to capture all relevant downstream effects. ESRD is a key driver of cost-effectiveness but is associated with major uncertainty. Future observational studies need to offer sufficient detail to allow for differentiation in event rates across treatment options.
