Cost-effectiveness of granulocyte colony-stimulating factors (G-CSFs) for the prevention of febrile neutropenia (FN) in patients with cancer.

PURPOSE: Clinical practice guidelines recommend the use of all approved granulocyte colony-stimulating factors (G-CSFs), including filgrastim and pegfilgrastim, as primary febrile neutropenia (FN) prophylaxis in patients receiving high- or intermediate-risk regimens (in those with additional patient risk factors). Previous studies have examined G-CSF cost-effectiveness by cancer type in patients with a high baseline risk of FN. This study evaluated patients with breast cancer (BC), non-small cell lung cancer (NSCLC), or non-Hodgkin's lymphoma (NHL) receiving therapy who were at intermediate risk for FN and compared primary prophylaxis (PP) and secondary prophylaxis (SP) using biosimilar filgrastim or biosimilar pegfilgrastim in Austria, France, and Germany. METHODS: A Markov cycle tree-based model was constructed to evaluate PP versus SP in patients with BC, NSCLC, or NHL receiving therapy over a lifetime horizon. Cost-effectiveness was evaluated over a range of willingness-to-pay (WTP) thresholds for incremental cost per quality-adjusted life year (QALY) gained. Sensitivity analyses evaluated uncertainty. RESULTS: Results demonstrated that using biosimilar filgrastim as PP compared to SP resulted in incremental cost-effectiveness ratios (ICERs) well below the most commonly accepted WTP threshold of €30,000. Across all three countries, PP in NSCLC had the lowest cost per QALY, and in France, PP was both cheaper and more effective than SP. Similar results were found using biosimilar pegfilgrastim, with ICERs generally higher than those for filgrastim. CONCLUSIONS: Biosimilar filgrastim and pegfilgrastim as primary prophylaxis are cost-effective approaches to avoid FN events in patients with BC, NSCLC, or NHL at intermediate risk for FN in Austria, France, and Germany.

Impact of health system specialty pharmacies on total cost of care in cancer treatment, a multisite review.

INTRODUCTION: Integrated delivery networks can use medically integrated dispensing of oral oncolytics on site through health system specialty pharmacies. There is little published research examining cost savings. Our objective was to demonstrate the financial value of health system specialty pharmacies among patients receiving oral oncolytics dispensed through fully, partially, and non-integrated dispensing strategies. METHODS: This was a retrospective cohort study of adult patients from Symphony Health's Integrated Dataverse® repository who filled a prescription for an agent of interest from 7/1/16-6/30/20 that was written within 25 US health systems. Outcomes included costs, healthcare resource utilization, and duration of therapy. RESULTS: In total, 36,816 patients were included; 986 patients (2.7%) integrated, 1,822 (4.9%) partially integrated, and 34,008 (92.4%) non-integrated. Mean 6-month medical charge and oncolytic prescription costs were lower for the integrated group ($36,831; $55,786) than the partially integrated ($46,304, p = 0.053; $63,295, p = 0.071) and non-integrated groups ($54,261, p < 0.001; $65,005, p = 0.004). In most healthcare resource utilization categories, the integrated group had the lowest patient percentage utilizing medical care. Duration of therapy was lower on average by ∼3 months in the integrated vs non-integrated group, which may represent closer monitoring of patient medical records and need for fills vs autoship practices. CONCLUSIONS: Patients receiving oral oncolytics through medically integrated dispensing at health system specialty pharmacies may have lower medical and pharmacy costs and decreased healthcare resource utilization. This study adds to the growing body of literature supporting integrated delivery networks and integrated dispensing. Further research is needed to demonstrate the value of medically integrated dispensing through health system specialty pharmacies in the delivery of treatment to patients with cancer and other high-cost diseases.

Economic Impact of Transformation to Acute Myeloid Leukemia Among Actively Managed Patients with Higher-Risk Myelodysplastic Syndromes in the United States.

INTRODUCTION: Transformation of higher-risk myelodysplastic syndromes (MDS) to acute myeloid leukemia (AML) may be associated with increased healthcare resource utilization (HCRU) and costs. To describe this economic impact, HCRU and costs were compared between US patients who experienced transformation to AML and those who did not. METHODS: Using the Optum administrative claims data, this retrospective matched cohort study identified patients (≥ 18 years old) with higher-risk MDS who initiated first-line therapy between January 1, 2008, and June 30, 2019. Patients whose disease transformed to AML were matched 1:1 to patients whose disease did not transform, based on the duration of follow-up. The follow-up period was divided into two periods: pre- (before transformation to AML) and post-AML (after transformation to AML). For patients who did not transform to AML, pre- and post-AML periods were determined using the transformation date of their matched pair. HCRU and total adjusted costs (2019 US dollars, $) were compared between patients who transformed to AML and those who did not. RESULTS: A total of 118 matched patient pairs were included in the study. The hospitalization rate was significantly higher in patients who transformed than in those who did not during the entire follow-up (58.8% vs. 44.1%; P = 0.0295) and post-AML (47.5% vs. 28.0%; P = 0.0028) periods. Across all periods, supportive care use was significantly higher among patients who transformed to AML vs. patients who did not transform. Adjusted mean monthly costs for patients with higher-risk MDS who transformed to AML were higher than those who did not transform ($25,964 vs. $19,150; P < 0.0001). The observed total cost difference was more notable in the post-AML period ($36,424 vs. $14,860; P < 0.0001). CONCLUSIONS: Patients with higher-risk MDS whose disease transformed to AML incurred significantly higher healthcare costs compared to those whose disease did not transform, highlighting the important need for treatments that prevent or delay transformation.

Duration of frontline therapy and impact on clinical outcomes in newly diagnosed multiple myeloma patients not receiving frontline stem cell transplant.

BACKGROUND: Extended first-line therapy (1LT) has improved clinical outcomes in newly diagnosed multiple myeloma (NDMM). This retrospective study of NDMM patients evaluated the relationship between dose-attenuation of 1LT and duration of therapy (DOT) and DOT on outcomes. METHODS: Adults with NDMM not undergoing stem cell transplant (SCT) from January 1, 2012 toMarch 31, 2018 from the Integrated Oncology Network were included; 300 were randomly selected for chart review. 1LT DOT, time to next treatment (TTNT), progression-free survival (PFS), and overall survival (OS) were estimated using Kaplan-Meier analysis. Marginal structural models evaluated relationships between DOT and TTNT, PFS, and OS at 2 years accounting for confounders and survival bias from the time-dependent nature of DOT. RESULTS: Of 300 chart-reviewed patients, 93 were excluded for incomplete data or meeting exclusion criteria. Among 207 NDMM patients, median age was 74 years; 146 (70.5%) did not receive dose-attenuation during 1LT. Patients with short DOT were older, frailer, with a higher comorbidity burden, and a significantly lower proportion had an Eastern Cooperative Oncology Group PS = 0. As DOT increased, more patients underwent dose-attenuation (p < 0.0001). The median 1LT DOT was 20.9 (95% confidence interval [CI]: 13.9, 26.4) versus 4.2 months (95% CI: 3.2, 4.9) for patients receiving versus not receiving dose-attenuation, respectively (p < 0.0001). After accounting for survival bias, confounder-adjusted TTNT was prolonged with each additional month of 1LT (odds ratio [OR]: 0.76 [95% CI: 0.75, 0.78]); likelihoods of risks of disease progression (OR: 0.87 [95% CI: 0.86, 0.88]) and death at 2 years (OR: 0.72 [95% CI: 0.70, 0.74]) were reduced with each month of 1LT (p < 0.0001 for all outcomes). CONCLUSIONS: Dose-attenuated 1LT was associated with longer DOT among patients with non-SCT NDMM. Each additional month of 1LT was associated with a reduced adjusted likelihood of disease progression and death at 2 years. Dose-attenuation of 1LT can extend DOT; longer DOT may improve clinical outcomes.

Clinical impact of transformation to acute myeloid leukemia in patients with higher-risk myelodysplastic syndromes.

Aim: Forty percent of patients with higher-risk myelodysplastic syndromes (HR-MDS) transform to acute myeloid leukemia (AML). Materials & methods: This retrospective study assessed the impact of HR-MDS transformation to AML on OS in a 6-month landmark analysis and the results were validated using a time-varying analysis. Results: The rate of AML transformation was 26.9% at 1 year. Patients who transformed to AML had a higher risk of death than patients who did not in the 6-month landmark analysis (HR: 1.82; p: 0.0072) and time-varying analysis at 1 year (HR: 2.85; p < 0.0001). Patients treated with azacitidine and decitabine in first-line therapy had similar results. Conclusion: HR-MDS transformation to AML is associated with inferior OS in patients with HR-MDS initiating first-line therapy.

Up to 40% of patients with higher-risk myelodysplastic syndromes (HR-MDS) could experience deterioration or progression to acute myeloid leukemia (AML), a type of blood cancer. We conducted a study to assess whether HR-MDS progression to AML had an unfavorable impact on patient life expectancy after initiating treatment for HR-MDS. Our study concluded that patients who experienced progression to AML had a higher chance of dying immediately after their progression to AML than patients who did not. Patients who received azacitidine and decitabine as their first treatment for HR-MDS had similar results.

The Impact of Baseline Risk Factors on the Incidence of Febrile Neutropenia in Breast Cancer Patients Receiving Chemotherapy with Pegfilgrastim Prophylaxis: A Real-World Data Analysis.

Background: There are sparse data addressing whether standard risk factors for febrile neutropenia (FN) are relevant in patients receiving myelosuppressive chemotherapy and primary prophylaxis for FN, which would have implications for variables to consider during real-world comparative analyses of FN incidence. Objective: To assess the impact of baseline patient-specific risk factors and regimen risk on the incidence of FN in patients receiving pegfilgrastim primary prophylaxis. Methods: This was a retrospective observational study in patients with breast cancer (BC) who received myelosuppressive chemotherapy and prophylactic pegfilgrastim identified January 1, 2017-May 31, 2018 from MarketScan® research databases. The outcomes were defined as incidence of FN in the first cycle and among all cycles of chemotherapy using three different definitions for FN. Logistic regression and generalized estimating equations models were used to compare outcomes among patients with and without patient-specific risk factors and among those receiving regimens categorized as high-, intermediate-, or other-risk for FN (low-risk or undefinable by clinical practice guidelines). Results: A total of 4460 patients were identified. In the first cycle of therapy, patients receiving intermediate-risk regimens were at up to 2 times higher risk for FN across all definitions than those receiving high-risk regimens (P<0.01). The odds ratio for main FN among patients with ≥4 versus 0 risk factors was 15.8 (95% confidence interval [CI]: 1.5, 169.4; P<0.01). Patients with ≥3 FN risk factors had significantly greater risks for FN across all cycles of treatment than those with no risk factors; this was true for all FN definitions. Discussion: The choice of FN definition significantly changed the impact of risk factors on the FN outcomes in our study, demonstrating the importance of evaluating all proxies for true FN events in a database study. This is particularly important during real-world study planning where potential missteps may lead to bias or confounding effects that render a study meaningless. Conclusions: In patients with BC receiving chemotherapy with pegfilgrastim prophylaxis, patient-specific risk factors and regimen risk levels are determinants of FN risk. In real-world studies evaluating FN incidence, it is imperative to consider and control for these risk factors when conducting comparative analyses.