Effectiveness and cost-effectiveness of combination therapy versus monotherapy in malignant melanoma
Dilay Özdemir1, Melanie Büssgen2
1University of Hamburg, Hamburg, Germany.
Background:
Until 2010, stage III or IV malignant melanoma (MM) had a poor prognosis. The discovery of immune checkpoint inhibitors (ICIs) in 2011 changed the treatment landscape. Promising results in patient survival with a checkpoint inhibitor prompted research into combination therapies. In 2016, the first combination therapy has been approved as first-line therapy for advanced MM.
Objective:
The aim of this work is to investigate to what extent combination therapy is (cost-)effective compared to monotherapy in stage III or IV MM.
Methods:
A systematic literature search was performed (Web of Science, PubMed, PubPharm, EconLit, and Cochrane Library); searching for publications published over the past decade that examine the cost-effectiveness in terms of cost/QALY and the effectiveness in terms of survival and response of combination therapy in comparison to monotherapy in stage III or IV MM patients.
Results:
A total of 11 randomized controlled trials (RCTs) and five cost-utility analyses met our inclusion criteria. Nine clinical trials demonstrated superiority of combination therapy over monotherapy. The combination of B-rapidly accelerated fibrosarcoma (BRAF) protein and mitogen-activated kinase (MEK) protein inhibitors is not cost-effective in any country. Three analyses demonstrate the cost-effectiveness of combination therapy with ICI compared to monotherapy.
Conclusion:
Combination therapy is more effective compared to monotherapy. While combined ICIs are cost-effective compared to monotherapy, this is not the case for the combination of BRAF and MEK inhibitors.
Insights
Combination therapy improves survival for malignant melanoma (MM) patients. Immune checkpoint inhibitor (ICI) combinations are cost-effective, but BRAF/MEK inhibitor combinations are not.
Area of Science:
- Oncology
- Health Economics
- Pharmacoeconomics
Background:
- Advanced malignant melanoma (MM) historically had a poor prognosis before 2010.
- Immune checkpoint inhibitors (ICIs) revolutionized MM treatment starting in 2011.
- Combination therapies emerged as a promising strategy for advanced MM, with the first approved in 2016.
Purpose of the Study:
- To evaluate the cost-effectiveness of combination therapy versus monotherapy for stage III or IV MM.
- To compare the effectiveness (survival, response rates) of combination therapies against monotherapies.
Main Methods:
- Systematic literature search across major databases (Web of Science, PubMed, etc.).
- Inclusion of randomized controlled trials (RCTs) and cost-utility analyses from the past decade.
- Focus on cost per Quality-Adjusted Life Year (QALY) and clinical effectiveness metrics.
Main Results:
- Eleven RCTs and five cost-utility analyses met inclusion criteria.
- Nine clinical trials showed combination therapy superior to monotherapy.
- BRAF/MEK inhibitor combination therapy was not cost-effective; ICI combinations were cost-effective.
Conclusions:
- Combination therapy offers superior effectiveness compared to monotherapy for advanced MM.
- Combined immune checkpoint inhibitors (ICIs) demonstrate cost-effectiveness.
- Combination therapy with BRAF and MEK inhibitors is not a cost-effective strategy.
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