Quantitative RT-PCR (RQ-PCR) is an essential test for BCR-ABL transcripts monitoring in patients with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKI), to guide therapy and for monitoring after a discontinuation attempt in patients with deep molecular response. RT-PCR (RQ-PCR) is currently not reimbursed by the public health system in Brazil. Aims: To assess the proportion of CML patients treated with first-line imatinib eligible for discontinuation, and to calculate the financial impact resulting from IM discontinuation. Methods: Between January 2010 and December 2011, 151 consecutive cases of chronic phase myeloid leukemia treated with Glivec first-line therapy were evaluated. Between June and December 2013 there was a switch in treatment from Glivec to generic IM. Cases that exhibited stable MR4.5 for 2 years with first-line IM were selected for the study. Cases which switched treatment to second-generation inhibitors and patients older than 75 years of age were excluded from the study. The methodology used was a pharmacoeconomic cost-utility analysis. Glivec monthly cost has been estimated at U$ 3,257.54 and the generic IM U$ 365.48, while the unitary value for the PCR test was U$ 117.49. In order to calculate the period of IM consumption, the median age of the patients and the life expectancy data released in 2015 by the Brazilian Institute of Geography and Statistics (IBGE) of 75 years was considered. In the first analysis, the life expectancy for the sample group, and the total cost of treatment (cost of Glivec, generic IM and four annual RQ-PCR tests for each patient) were calculated. The second analysis consisted of a hypothetical calculation of costs under the scenario where the study group is therapy-free (estimating that the survival rate under discontinued therapy was similar to data available in the literature, with discontinuation success of 50% in this group) with molecular monitoring by PCR monthly in the first year, bimestrial in the second year and every three months from the third year on. Results: One hundred fifty-one cases were analyzed, with a median age at diagnosis of 45. From those, 56 (37%) patients achieved stable MR4.5 with a median time to achieve MR4.5 of 71 months. The median duration of follow-up was 8 (0-10) years. In the last follow-up, 108 patients were still in treatment, 10% (11/108) with Glivec, 90% (97/108) on generic IM. Patients excluded from the analysis: 4 cases aged more than 75 years; 13 that switched therapy to another TKI and one during the bone marrow transplant period. Finally, 38/56 (25%) patients who obtained MR4.5 with IM were eligible for analysis. Analysis 1: Total treatment cost for the 38 eligible cases, if the individuals sustained continuous use of IM, considering the life expectancy of 75 years. The calculations resulted in an average of 29 years of treatment, with an estimated cost of U$ 9,363,866.00. Analysis 2: The cost after discontinuation of generic IM, estimating that 50% of patients would resume the treatment. Nineteen cases were analyzed. The costs related to the monthly PCR exam in year 1, bimestrial exam in year 2 and trimester in year 3, until the patient reaches 75 years of age, have been calculated, with a total cost of U$ 7,823.515. Conclusions: The economy resulting from the discontinuation of treatment (US$1.540.340,00) by 19 patients could support 219 patients tests over 29 years, or 12.110 tests each year. This data is relevant, providing that RQ-PCR is essential for the appropriate management of CML and to allow safe discontinuation of the therapy in eligible patients. Such results may help to change the current health policies concerning RQ-PCR tests reimbursement for CML management and future attempting of TFR in Brazil. Disclosures Centrone: Novartis: Honoraria; Janssen: Honoraria. Magalhaes:Novartis: Honoraria. Pagnano:Pint Pharma: Consultancy; Abbvie: Consultancy; Sandoz: Consultancy.
INTRODUCTION In Brazil, Chronic Myeloid Leukemia patients receive almost exclusively first-line imatinib ;since 2013, only generic drugs and copies of imatinib mesylate have been used in first-line treatments throughout the whole Public Health System (Sistema Único de Saúde - SUS) and, in several institutions within the private system.. However, in the public system, which corresponds to 75% of the health assistance in the country, there is no funding for such exams, which are performed exclusively by the donation of vouchers from pharmaceutical industries. Within the private healthcare system, there is an average coverage of 04 PCR exams per annum, which allows for adequate monitoring, but is not sufficient for the implementation of treatment discontinuation in eligible patients. AIMS To analyse real world data related to 140 patients from a Brazilian private institution and determine, using epidemiological and treatment characteristics, the cost of branded imatinib mesylate, generic imatinib mesylate and nilotinib chloride first-line treatment, under the context where the discontinuation of treatment is unfeasible, and, as a secondary aim, to determine the financial impact from an eventual discontinuation of patients that achieved MR 4.0 METHODS Retrospective data from 140 patients with Chronic Myeloid Leukemia in chronic phase treated solely with first-line imatinib mesylate were analysed, including 95% of patients treated with generic imatinib and 5% with branded imatinib mesylate . Median treatment : 07 years. Median age of diagnosis of the studied population was 47 years .Life expectancy is 76.8 years (IBGE-2018). In order to facilitate , 14 patients that had overcome life expectancy of 76.8 years of age were excluded (Source: IBGE), resulting in a total of 126 patients. The cost of treatment was estimated for the following first-line treatments: branded imatinib mesylate, generic imatinib mesylate, nilotinib chloride, in a scenario with or without discontinuation, including the cost of all PCR exams necessary in order to achieve adequate monitoring.It was evidenced, within that group, 26 patients with a criterial stable response of MR4.0 (80%) and MR4.5 (20%), confirmed by 04 PCR exams performed from a two years minimum period of ongoing treatment. Concerning discontinuation, the calculation included: 12 exams within treatment year 1, 06 exams in year 2, trimestral exams from year 3 to year 5, and semestral from year 6 on, even for those who resumed treatment. Projection of the financial impact from treatment with or without discontinuation in the studied population - 26 patients including follow-up PCR exams post-discontinuation for 29 years RESULTS STUDY OF THE REAL COST OF TREATMENTS Without descontinuation : Branded IMATINIB - total cost : US$ 173.773.130 / cost per patient: US$ 1.241.237 GENERIC IMATINIB US$ 113.297.014 / cost per patient: US$ 809,264 NILOTINIB US$ 168.231.166 / cost per patient: US$ 1.201.651 PROJECTION OF BUDGET IMPACT OF DISCONTINUATION BRANDED IMATINIB AND GENERIC IMATINIB, it was considered a rate of 20% eligible patients for discontinuation, and that 50% of such patients needed to resume the treatment within 12 months. BRANDED IMATINIB : US$ 17.206.022 total economy / US$682.779 per patient . GENERIC IMATINIB US$ 11.141.59 total economy and US$ 442.127 per patient. Regarding the drug NILOTINIB, the analysis used 45% of eligible patients for discontinuation and considered 50% of patients resuming the treatment, resulting in an economy of US$ 37.463.140. DISCUSSION We present, based on Brasíndice data, real cost projection for the available lines of treatment for first line and, lastly, that the financial impact from treatment discontinuation of 15% of the total study population would fund : Branded imatinib :146.422 exams ;Imatinib copies- 94.827 ; Nilotinib- 318.853 . The data shows that the non coverage of such exams deprives patients of the possibility to maintain stable molecular response in the absence of treatment and makes both the discontinuation practice and the chance to significantly reduce costs to the health system, whether public or private, unfeasable. CONCLUSION The analysis and projections performed in the study suggest economically viable solution to conduct systematic molecular tests in order to monitor patients therapeutic response, under a treatment discontinuation scenario for eligible patients. Disclosures Centrone: Janssen: Honoraria; Novartis: Honoraria. Silva:Janssen: Honoraria. Aranha:Janssen: Honoraria.
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