Budesonide with multi-matrix technology as second-line treatment for ulcerative colitis: evaluation of long-term cost-effectiveness in the Netherlands

Abstract

Aims: Budesonide with multi-matrix technology (MMX) is an oral corticosteroid, shown to have high topical activity against ulcerative colitis (UC) while maintaining low systemic bioavailability with few adverse events. The aim of this study was to evaluate the cost-effectiveness of budesonide MMX versus commonly used corticosteroids, in the second-line treatment of active mild-to-moderate UC in the Netherlands.

Materials and methods: An eight-state Markov model with an 8 week cycle length captured remission, four distinct therapy stages, hospitalization, possible colectomy and mortality. Remission probability for budesonide MMX was based on the CORE-II study. Population characteristics were derived from the Dutch Inflammatory Bowel Disease South Limburg cohort (n = 598) and included patients with proctitis (39%), left-sided (42%) and extensive disease (19%). Comparators (topical budesonide foam and enema, oral budesonide and prednisolone) were selected based on current Dutch clinical practice. Treatment effects were evaluated by network meta-analysis using a Bayesian framework. Cost-effectiveness analysis was performed over a 5 year time horizon from a societal perspective, with costs, health-state and adverse event utilities derived from published sources. Outcomes were weighted by disease extent distribution and corresponding comparators.

Results: Budesonide MMX was associated with comparable quality-adjusted life year (QALY) gain versus foam and oral formulations (+0.01 QALYs) in the total UC population, whilst being cost-saving (EUR 366 per patient). Probabilistic sensitivity analysis evaluated an 86.6% probability of budesonide MMX being dominant (cost-saving with QALY gain) versus these comparators. Exploratory analysis showed similar findings versus prednisolone.

Limitations: Differing definitions of trial end-points and remission across trials meant indirect comparison was not ideal. However, in the absence of head-to-head clinical data, these comparisons are reasonable alternatives and currently offer the only comparison of second-line UC treatments.

Conclusions: In the present analysis, budesonide MMX was shown to be cost-effective versus comparators in the total UC population, for the second-line treatment of active mild-to-moderate UC in the Netherlands.

Keywords:

• Cost-effectiveness
• ulcerative colitis
• corticosteroid
• budesonide
• prednisolone

JEL classification codes:

• C51
• I19

Transparency

Declaration of funding

HEVA-HEOR received funding from Ferring Pharmaceuticals for analyzing the cost-effectiveness of budesonide MMX in the Netherlands, including the production of a technical report. Ossian Health Economics and Communications received funding from HEVA-HEOR for the writing of the present manuscript. Employees from HEVA-HEOR, Ossian Health Economics and Communications, and Ferring Pharmaceuticals were involved in reviewing and revising aspects of this manuscript.

Author contributions: A.G. and S.R. were involved in conception and design of the cost-effectiveness analysis, and drafting a technical report that was used as the basis of the final manuscript. O.G., Y.L.Y.S. and J.K. provided feedback on several drafts of the manuscript.

Declaration of financial/other relationships

A.G. and S.R. have disclosed that they are employees of HEVA-HEOR. O.G. and Y.L.Y.S. have disclosed that they are employees of Ferring Pharmaceuticals. J.K. has disclosed that he has no significant relationships with or financial interests in any commercial companies related to this study or article.

JME peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgements

Audrey Petitjean (HEVA-HEOR) was involved in the review of the technical report of the analysis, on which the manuscript is based. Michel Cucherat (University Lyon I) was involved in conducting the NMA used in the analysis, on which the manuscript is based. Sam Malkin and William Valentine (Ossian Health Economics and Communications) were involved in writing of the present manuscript.