Bortezomib (VELCADE), a new proteasome inhibitor, was recently licensed by the FDA for treating patients with relapsed and refractory multiple myeloma (MM), and has now also been approved for similar use in Europe. A model was developed to evaluate and compare the costs and health benefits of bortezomib, as a third-line treatment for patients with relapsed and refractory MM, relative to best supportive care in the UK. A two-part mathematical model of survival was built and calibrated using individual patient data from the SUMMIT trial (n=202 ;
) , a multi-center, pivotal phase II trial of bortezomib in 202 patients with relapsed and refractory MM. Patients in SUMMIT were 18 years of age or older, with a life expectancy of more than three months and a Karnofsky score >=60. In the first part of the survival model, the time to disease progression for patients on bortezomib (n=202) or best supportive care (n=38) was estimated. In the second part, the time from disease progression till death was calculated, based on the sub-sample of patients for whom relevant data were available (n=79). Health-related utility was assessed for the effects of the drug and also the natural course of the disease on quality of life, yielding different utility weights pre- and post-disease progression. Those values were derived by mapping individual patient reports of quality of life in SUMMIT (EORTC-QLQ30 and MY24, FACIT-Fatigue and FACT/GOG-Neurotoxicity subscales) onto the 5 scales of the EQ-5D instrument. Resource use data from SUMMIT were used to estimate costs from the perspective of the UK NHS for bortezomib administration, hospital care, medications and diagnostic tests and surgical procedures on an individual patient basis. Results: By delaying the rate at which disease progresses, bortezomib produced survival gains relative to best supportive care that range between 7.75 to 12.09 months of life depending on the method of survival estimation. Additional total direct costs associated with the novel agent were £17290 or £24121 if the additional costs incurred during the extended period of survival are included. The incremental cost-effectiveness ratio for bortezomib was in the range £17161- £33539 per life year gained and £26714 - £51,666 per quality-adjusted life year gained. Conclusions: These results suggest that bortezmib is a cost-effective third-line treatment option for patients with RRMM in the UK. This study provides a methodological framework for undertaking preliminary economic evaluations of new drugs for the treatment of cancer with the aim of informing purchasing decisions based on limited outcome data.
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