Pharmacoeconomics of ruxolitinib therapy in patients with myelofibrosis

Abstract

Background:

Overall survival (OS) and other important clinical trial end-points seem increasingly more elusive in supporting rapid and efficient incorporation of innovative cancer drugs in clinical practice. This study proposes a clinical trial based pharmacoeconomic framework to assess the therapeutic and economic value of ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis.

Methods:

Individual patient level 144 week follow-up data from the COMFORT-II trial was used to account for the crossover effect on overall survival. Lifetime treatment benefits and costs were estimated considering detailed patterns of both ruxolitinib dose adjustments and blood transfusion needs.

Results:

The authors estimate a 3.3 years increment in life expectancy (HR = 0.30; 95% CI = 0.17–0.55; p-value <0.001) and an incremental cost-effectiveness ratio of €40,000 per life year gained with the use of ruxolitinib.

Conclusion:

This study also demonstrates how valuable information from clinical trials can be used to support informed decisions about the early incorporation of innovative drugs.

Keywords::

• Ruxolitinib
• Myelofibrosis
• Cost-effectiveness
• Individual level data

Transparency

Declaration of funding

This research project was funded by Novartis.

Declaration of financial/other relationships

Novartis contracted with Exigo for the development of the model. BV, VA, JA, and JF are employed by Exigo Consultores. Exigo Consultores provided support in the form of salaries for the authors, but did not have any additional role in the study design, data collection and analysis, decision to publish, or preparation of the manuscript. JME peer reviewers on this manuscript have no relevant financial or other relationships to disclose.