February 2013 Vol 6, No 1, Special Issue - Health Economics
Wayne Kuznar

Beneficial treatment options for patients with myelofibrosis are significantly lacking, although 1 drug, ruxolitinib (Jakafi), has proved effective in clinical trials. Compared with best available therapy, ruxolitinib proved to be a cost-effective option for patients with myelofibrosis, according to the findings from a cost-effectiveness study that was presented by Khalid El Ouagari, PhD, of Novartis Pharmaceuticals Canada, and colleagues.

Ruxolitinib is an orally available, se­­lective inhibitor of Janus kinase (JAK)1 and JAK2 of the JAK-STAT signaling pathway. In the clinical trial COMFORT-II, ruxolitinib was proved to be clinically efficacious and was superior to best available therapy at diminishing splenomegaly and its condition-associated symptoms, while also enhancing patient quality of life.

The latest trial was designed to determine the cost value of adding ruxolitinib to other myelofibrosis drug treatment options and the cost-effectiveness of ruxolitinib compared with best available therapy for the treatment of myelofibrosis.

A Markov model was used to determine the cost, health outcomes, and the cost-effectiveness of ruxolitinib versus best available therapy, consisting of a treatment or a combination treatment that is selected based on the COMFORT-II trial guidance.

All patients started the model having myelofibrosis with splenomegaly. The model tracked the progression of 4 health states—responder, nonresponder, leukemic transformation, and death—for 12 weeks during the patients’ lifetime.

The rate by which patients navigated through the 4 health states was influenced by risk status at the study’s start, as well as by the success in or the inability of achieving responder state (≥35% reduced spleen volume and/or a lack of symptoms, such as weight loss, fever, or night sweats).

Using a 1-way sensitivity analysis and a probabilistic sensitivity analysis, the outcomes measured were cost over time, life-years, quality of life-years, time when the patient did not have leukemic transformation, and the time as a treatment responder.

All patients had intermediate-2 risk (51%) or high-risk (49%) myelofibrosis, according to the International Working Group for Myelofibrosis Research and Treatment risk categorization.

Treatment Cost, QALY: Ruxolitinib versus Best Available Therapy
The average treatment cost of a patient receiving ruxolitinib was $494,859, of which $205,484 was attributed to the drug cost.

By comparison, the cost of managing a patient with best available therapy was $421,755, of which $59,289 was drug cost.

Patients who were receiving ruxolitinib had less resource-use cost, mainly because patients with reduced spleens usually had less physician visits and less hospital admissions, according to the model assumptions, resulting in similar overall cost between the 2 approaches.

The ruxolitinib treatment arm produced a higher financial burden for leukemic transformation as a result of enhanced overall survival. Consequently, more adverse events and more transfusion-dependency costs were also seen in the ruxolitinib arm.

Quality-adjusted life-years (QALYs) were 4.01 in the ruxolitinib arm and 2.82 in the best available therapy arm. The overall incremental cost-­effectiveness ratio of ruxolitinib was $61,444 per QALY.

Because there is a better survival outcome for intermediate-2 risk patients than for high-risk patients, the ruxolitinib group ended up with greater QALYs and subsequent additional costs over the patients’ lifetimes. In addition, although there may be no survival advantage with the removal of constitutional symptoms, such as weight loss or fever, because patients with less symptomatic issues sustain treatment with ruxolitinib for a longer duration, there is more drug-related cost.

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