Abstract
In 2018, venetoclax in combination with rituximab (VenR), received a marketing authorization from the European Medicines Agency for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (R/R CLL). Currently Ibrutinib (Ibr) is the preferential treatment in this population in Portugal. As suggested by the literature, VenR has similar effectiveness to Ibr. Therefore, this analysis aims to compare VenR vs. Ibr costs in CLL R/R patients from the payers’ perspective. A three-state partitioned survival model was used assuming hazard ratios of 1 for progression free survival (PFS) and overall survival (OS) for VenR versus Ibr. PFS and OS data from the MURANO randomized clinical trial (May 2018 data cut) were used to estimate parametric curves that allowed to extrapolate over a lifetime horizon. Resource utilization and unit cost for the Portuguese setting were based on clinical experts’ panel, Portuguese Diagnosis Related Group data, national tariffs and national catalog for the price of medicines. One-way sensitivity analysis was conducted to assess uncertainty in model assumptions and inputs. Treatment with VenR instead of Ibr allows cost savings of €103,440 per patient. Regarding del(17p)/TP53 status, the average savings were €84,486 per patient with these abnormalities and €117,977 per patient without them. This result is a consequence of treatment with VenR having a maximum of 24 months while Ibr is used until progression, without effectiveness gains. In sensitivity analysis, cost-savings ranged between €30,021 and €261,629, being PFS extrapolation the most important source of uncertainty. 24-month fixed duration treatment with VenR is a cost-saving option versus Ibr in all scenarios for R/R CLL patients, with savings of €103,440 per patient in base-case. Sensitivity analyses demonstrated the robustness of the results.
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