ABSTRACT
BACKGROUND: Medical and regulatory communities are increasingly interested in the utility of real-world evidence (RWE) for answering questions pertaining to drug safety and effectiveness but concerns about validity remain. A principled approach to conducting RWE studies may alleviate concerns and increase confidence in findings. This study sought to predict the findings from the PRONOUNCE trial using a principled approach to generating RWE. METHODS: This propensity-score (PS) matched observational cohort study utilized 3 claims databases to compare the occurrence of major adverse cardiovascular events (MACE) among initiators of degarelix vs. leuprolide. Patients were included if they had history of prostate cancer and atherosclerotic cardiovascular disease. Subjects were excluded if they didn't have continuous database enrollment in the year prior to treatment initiation, were exposed to androgen deprivation therapy or experienced an acute cardiovascular event within 30 days prior to treatment initiation, or had a history or risk factors of QT prolongation. RESULTS: There were 12,448 leuprolide and 1,969 degarelix study-eligible patients before matching, with 1,887 in each arm after PS-matching. The results for MACE comparing degarelix to leuprolide in the observational analysis (hazard ratio= 1.35; 95% confidence interval = 0.94-1.93) was consistent with the subsequently released PRONOUNCE result (hazard ratio = 1.28; 95% confidence interval = 0.59-2.79). CONCLUSIONS: This study successfully predicted the result of a comparative cardiovascular safety trial in the oncology setting. Although the findings are encouraging, limitations of measuring cancer stage and tumor progression are representative of challenges in attempting to generalize whether claims-based RWE can be used as actionable evidence.
ABSTRACT
OBJECTIVES: A peer-support program called Resilience In Stressful Events (RISE) was designed to help hospital staff cope with stressful patient-related events. The aim of this study was to evaluate the impact of the RISE program by conducting an economic evaluation of its cost benefit. METHODS: A Markov model with a 1-year time horizon was developed to compare the cost benefit with and without the RISE program from a provider (hospital) perspective. Nursing staff who used the RISE program between 2015 and 2016 at a 1000-bed, private hospital in the United States were included in the analysis. The cost of running the RISE program, nurse turnover, and nurse time off were modeled. Data on costs were obtained from literature review and hospital data. Probabilities of quitting or taking time off with or without the RISE program were estimated using survey data. Net monetary benefit (NMB) and budget impact of having the RISE program were computed to determine cost benefit to the hospital. RESULTS: Expected model results of the RISE program found a net monetary benefit savings of US $22,576.05 per nurse who initiated a RISE call. These savings were determined to be 99.9% consistent on the basis of a probabilistic sensitivity analysis. The budget impact analysis revealed that a hospital could save US $1.81 million each year because of the RISE program. CONCLUSIONS: The RISE program resulted in substantial cost savings to the hospital. Hospitals should be encouraged by these findings to implement institution-wide support programs for medical staff, based on a high demand for this type of service and the potential for cost savings.
