Cost-Effectiveness and Value of Information Analyses of Caplacizumab for the Treatment of Thrombotic Thrombocytopenic Purpura in Brazil

Julia Simões Correa Galendi (), Hannes Rasch, Carlos Antonio Caramori, Rafael Dezen Gaiolla, Dirk Müller and Vania Santos Nunes Nogueira
Additional contact information
Julia Simões Correa Galendi: São Paulo State University/UNESP, Medical School
Hannes Rasch: University Hospital of Cologne
Carlos Antonio Caramori: São Paulo State University/UNESP, Medical School
Rafael Dezen Gaiolla: São Paulo State University/UNESP, Medical School
Dirk Müller: University Hospital of Cologne
Vania Santos Nunes Nogueira: São Paulo State University/UNESP, Medical School

Applied Health Economics and Health Policy, 2025, vol. 23, issue 4, No 8, 649-660

Abstract: Abstract Objectives The objective was to evaluate the cost effectiveness of caplacizumab for the treatment of patients with acquired immune thrombocytopenic thrombotic purpura (iTTP) compared to standard of care from the perspective of the Brazilian Unified Health System (SUS). Methods A decision tree followed by a Markov model with a lifetime horizon was developed. Patients entered the model with an acute iTTP event. All patients were assumed to be admitted to hospital where they either respond to treatment or die. The model offered three health states: remission, relapse or death. Input data were obtained from literature searches with the data on efficacy of caplacizumab based on the HERCULES trial. The incremental cost-effectiveness ratio (ICER) was compared to the willingness-to-pay threshold for rare diseases of Brazilian reais (R$)120,000/quality-adjusted life years (QALYs). In addition to various sensitivity analyses, a value of information (VOI) analysis was conducted. Results In the base case, caplacizumab resulted in 0.70 QALYs gained, and cost R$1,333,601 more, with an ICER of R$1,901,729/QALY. The cost of the caplacizumab vial was the most influential parameter. Probabilistic analysis showed that caplacizumab was not cost effective in any iterations for the threshold of the rare disease. The expected value of perfect information per year is R$0. Conclusion Although caplacizumab results in incremental QALYs, based on the proposed cost, caplacizumab is not cost effective from the SUS perspective, and VOI results indicate that further research would not be worthwhile.

Date: 2025
References: Add references at CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s40258-025-00968-7 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:aphecp:v:23:y:2025:i:4:d:10.1007_s40258-025-00968-7

Ordering information: This journal article can be ordered from
http://www.springer.com/economics/journal/40258

DOI: 10.1007/s40258-025-00968-7

Access Statistics for this article

Applied Health Economics and Health Policy is currently edited by Timothy Wrightson

More articles in Applied Health Economics and Health Policy from Springer
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().