COST- EFFECTIVENESS OF DEEP MOLECULAR RESPONSE WITH FIRST- LINE NILOTINIB VS. IMATINIB IN PH+ CHRONIC MYELOID LEUKAEMIA MEASURED BY EFFECTIVELY TREATED PATIENT- YEARS IN BULGARIA

Borissov B, Grudeva-Popova Zh, Balatzenko G, Spassov B

Prescriptia Ltd, Sofia, Bulgaria

Medical University, Medical Faculty – Plovdiv, Department of Clinical Oncology; University Hospital "Sv. Georgi" – Department of Clinical hematology, Plovdiv, Bulgaria

Laboratory of Cytogenetics & Molecular Biology, National Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria

Clinic of Clinical Hematology, National Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria

OBJECTIVES: This analysis reviews the available evidence for the cost-effectiveness of nilotinib vs. imatinib for the first-line treatment of Philadelphia chromosome-positive Chronic myeloid leukaemia (Ph+ CML) measured by Effectively Treated Patient-years (ETPY) per major molecular response (MMR) and complete cytogenetic response (CCyR). METHODS: A standard de novo cost-effectiveness model compares nilotinib vs. imatinib for first-line treatment in Ph+ CML patients. “Effective treatment” was defined as CCyR or MMR achieved. Cost and utilities are discounted at 3.5%. No administration, treatment of side effects and indirect costs are included. Willingness to pay /λ/ was set at the level of 20.000€ ($24.103). RESULTS: First- line nilotinib gains 3.65 added ETPY for MMR compared to imatinib (1.19) and 7.12 for CСyR vs. 4.55. The incremental cost per ETPY is favorable for nilotinib – 13.242€ (15.956$) per MMR and 2.697€ (4.377$) per CCyR. Sensitivity analyses confirm the robustness of the model results. CONCLUSIONS: ETPYs is a clinically meaningful measure of patient benefit, combining length of life with the likelihood of attaining internationally acknowledged best-practice recommendations on efficacy. Uncertainties and well- known shortcomings in the cost-effectiveness analysis based on cost per QALY would be substantially reduced with the use of ETPY. The analysis demonstrates that first-line nilotinib is more cost-effective than first-line imatinib.