The application of economic tools to evaluate the cost and health benefits and screen out more cost-effective drugs and technologies is an important measure to improve efficiency of medical resource allocation in China. Given the inherent differences between strict clinical trials and clinical routine practice, using trial-based economic evaluations to guide relevant medical decisions may lead to a certain risk of value deviation. Recent development of real-world data provides opportunities to assess the cost-effectiveness of drugs under the practical utilization, and has gradually become a new research hotspot. However, the complexity of the actual clinical environment also puts higher demands on researchers and decision makers to construct, understand and apply real-world evidence. In order to further prompt the normalization of economic evaluation based on real-world data and promote the scientific application of real-world evidence in medical and health decision-making, this project aims at the crucial issues including scope, research design and quality evaluation, to clarify the key considerations on the using of real-world evidence in medical decision-making. Combined with the international guidelines, the latest advancement of relevant research areas and the advice and opinions from multidisciplinary experts, we aim to provide technical references and guidance for researchers and decision makers, and to strengthen the evidence base of management policies.
Objective This study analyzed the medical expenditure and its influential factors, and compared the clinical effectiveness and medical expenditure of three major drugs. Methods We designed the cohort study to compare the difference of medical and pharmaceutical expenditures between patients with and without underlying diseases. Multi-linear regression was applied to analyze the influential factors. Incremental expenditure-effectiveness ratio was applied to study three clinically important drugs. Results The curing rate of non-critical patients was statistically significant than critical patients (73.68%, 99.38%, P=0.000) .The curing rate of non-critical patients without underlying diseases was statistically significant than those with underlying diseases in the cohort (96%, 99.66%, P=0.001 6). No significance was identified in the critical patients cohort. The medical expenditure of non-critical patients with and without underlying diseases were 7 879.22 and 7 172.23 RMB per capita, respectively. Accordingly, the medical expenditure in critical patients was 24 912.89 and 26 433.53 RMB per capita. No significance was identified in the two cohorts. Medical expenditure was positively correlated with age and disease severity, with its equation y=4585.71+79.04X1+17188.87X2 (X1: age, X2: disease severity). Regarding the clinical effectiveness and medical expenditure, no significance was identified in critical patients who administered small and medium dose of Methylprednisolone. The expenditure-effectiveness ratios of Ribavirin that was administered by non-critical patients without underlying dissuades were 6 107 and 4 225 RMB, respectively. Accordingly, the expenditure-effectiveness ratios of Thymosin were 11 651 and 6 107 RMB. Conclusions The curing rate of non-critical patients without underlying diseases was higher than the counterpart in the cohort. No influence of underlying diseases was found in the critical patient cohort. Medical expenditure was positively correlated with age and disease severity. Small-and-medium dose of Methylprednisolone might not influence the curing rate and medical expenditure in critical patients. The effectiveness of Thymosin for non-critical patients with and without underlying diseases was not significantly different. However, additional 5 877 RMB occurred if Thymosin was administrated. Likewise, the effectiveness of Ribavirin for non-critical patients remains the same. However, additional 1 082 RMB was consumed in Ribavirin-administrated patient.
Objective To investigate the clinical effectiveness of the total Flavones of Hippophae Rhamnoides L. (TFH) and compare its cost-effectiveness ratio with enalapril in patients with essential hypertension. Methods Among 3 971 staff members in two universities, 155 eligible patients with blood pressure (BP) ≥160/100 mmHg were screened and included in this study. By using a random number table, the patients were randomized to the two groups: TFH was given to the treatment group while enalapril in the control group. At the end of the six week, BP, total cost and cost-effectiveness ratio were measured in both TFH and enalapril groups. Results After six weeks, blood pressures in the TFH and enalapril groups decreased by 12.7±11.6/8.1±5.1 mmHg and 15.2±9.7/10.1±7.3 mmHg respectively, while the improvement rates of BP were 73.24% (52/71) and 74.65% (53/71) respectively. There were no differences between TFH and enalapril groups in lowering BP amplitude and total effectiveness rate. The incidence of side effect in TFH group was 11.27% (8/71), which was significantly lower than that of enalapril 29.6% (21/71). The total cost of TFH group was 9 294.6 RMB with the cost effectiveness ratio of 732/1 147 RMB per mmHg and 179 RMB per case. In the enalapril group, the total cost added up to 13 236 RMB with cost effectiveness ratio of 870/1 310 per mmHg and 250 RMB per case. Sensitivity analysis indicated that TFH was better than enalapril with respect to clinical economic value even when enalapril price dropped to 1.8 RMB for tablet (5 mg). Conclusion Compared with enalapril, TFH is an effective and economic drug in treating patients with hypertension.
Health economics analysis has become increasingly important in recent years. It is essential to master the use of relevant software to conduct research in health economics. TreeAge Pro software is widely used in the healthcare decision analysis. It can carry out decision analysis, cost-effectiveness analysis, and Monte Carlo simulation. With powerful functionlity and outstanding visualization, it can build Markov disease transition models to analyze Markov processes according to disease models and accomplish decision analysis with decision trees and influence diagrams. This paper introduces cost-effectiveness analysis based on Markov model with examples and explains the main graphs.
Objective To evaluate the cost-effectiveness of three LTBI screening strategies: the tuberculin skin test (TST), the T-SPOT.TB and the combination of TST and T-SPOT (TST+T.SPOT), to provide economic evidence for T.SPOT application in China. Methods A decision analysis model evaluated three strategies among a cohort of 1000 tuberculosis (TB) close contacts, using incremental cost-effectiveness of prevention a active TB patient (1 year post contact). Meta analyses were conducted to calculate the key parameters of T.SPOT and TST. The official data or literature was searched and the unaccessible data was to specify other parameters, such as cost, LTBI prevalence, etc. The one-way sensitivity analysis was performed, varying key parameters over a wide range of reasonable values to evaluate the impact of data uncertainties and to determine the robustness of our overall conclusion. Results a) As for the total cost, the TST+T.SPOT strategy (?212 213.81 per 1 000 contacts) cost the least, while the single T.SPOT strategy cost the most; b) Subsequently, the TST+T.SPOT strategy required less contacts to be treated to prevent an active case of TB (8.31) than the single TST strategy (25.67); c) the TST+T.SPOT strategy shared the most cost-effectiveness (?3 063.50 per active TB case prevented) than the single TST or T.SPOT strategy; and d) The results of one-way sensitivity analyses showed that cost-effectiveness values were sensitive to changes in LTBI prevalence (gt;60%), Sen and Spn of TST test (gt;70%), with the single TST being superior to the single T.SPOT. Conclusion The Single T.SPOT strategy enjoys the most cases prevented from active TB, while the TST+S.SPOT strategy is the most cost-effective. The conclusion is sensitive to a few parameters, such as LTBI prevalence, but the TST+T.SPOT strategy is always the best.
Objective To evaluate the cost-effectiveness of chemotherapy in children with newly diagnosed Hodgkin lymphoma at low-, intermediate-, and high-risk. Methods From the perspective of health system, a decision-tree model was designed for cost-effectiveness analysis. The chemotherapy regimens of low-risk group included OEPA (vincristine, etoposide, prednisone, doxorubicin), AV-PC (doxorubicin, vincristine, prednisone, cyclophosphamide), and ABVD (doxorubicin, bleomycin, vincristine, dacarbazine); intermediate-risk group included OEPA, ABVE-PC (doxorubicin, bleomycin, vincristine, etoposide, prednisone, cyclophosphamide) and ABVD; high-risk group included OEPA, ABVE-PC, ABVD and BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, prednisone). The effectiveness and cost parameters were derived from the event-free survival rate reported in the literature, the drug linked reference price in Sichuan province, and treatment price of medical institutions. Univariate and probabilistic sensitivity analysis were performed to explore the impact of uncertainty. Results In the low-risk group, compared with AV-PC, the incremental cost-effectiveness ratios (ICER) of OEPA and ABVD were 80 700 yuan and 108 799 yuan, respectively. In the intermediate-risk group, compared with OEPA, the ICER of ABVE-PC and ABVD were ?17 737 yuan and ?4 701 yuan respectively. In the high-risk group, compared with ABVE-PC, the ICER of OEPA, ABVD and BEACOPP were 149 262, 472 090 and 64 652 yuan, respectively. Univariate sensitivity analysis showed that in low-risk group, the most influential factors were cost of OEPA and cost of ABVD; in moderate-risk group were cost of ABVE-PC and cost of OEPA; in the high-risk group were cost of OEPA, cost of ABVD, and cost of BEACOPP, respectively. The results of probabilistic sensitivity analysis are basically consistent with those of the main analysis. Conclusion If China's per capita gross domestic product in 2023 (89 358 yuan) was used as the willingness-to-pay (WTP) threshold, OEPA in the low-risk group, OEPA in the intermediate-risk group and BEACOPP in the high-risk group are cost-effective.
ObjectivesThis study aimed to study the economic effect of five kinds of detection systems for nucleic acid, which were based on five kinds of working electrodes: gold electrode, glassy carbon electrode, carbon paste electrode, screen printing electrode, and indium-tin-oxide (ITO) glass electrode.MethodsThe cost of completing a single test was taken as the cost of economic analysis. The Youden index was used to represent the effect of cost-effectiveness analysis (CEA). Meanwhile, the cost-utility analysis (CUA) and incremental cost-effectiveness ratio (ICER) were used for the economic analysis of the corresponding system.ResultsThe cost of five detection systems based on gold electrode, glass carbon electrode, carbon paste electrode, screen printing electrode, and ITO glass electrode was 3.70 yuan/unit, 4.20 yuan/unit, 5.25 yuan/unit, 33.98 yuan/unit and 5.01 yuan/unit, respectively. The Youden indexes of all five systems were 1. The cost effectiveness (C/E) were 3.70, 4.20, 5.25, 33.98, and 5.01, respectively. The cost utility (C/U) were 6.61, 6.89, 9.91, 62.93, and 9.45, respectively. The C'/E and C'/U of the gold electrode detection system were the minimum (2.96 and 5.29). Compared with the system applying the gold electrode, the system using the glassy carbon electrode had ΔC >0 and ?E0 >0; When carbon paste electrode, screen printing electrode, and ITO glass electrode system were used, ?C was >0 and ?E0 was <0.ConclusionsFrom the perspective of CEA and CUA, the system using the gold electrode has the best economic effect. The sensitivity analysis proved the reliability of CEA and CUA results. According to the ICER, gold electrode or glassy carbon electrode can be used in clinical practice with the choice depending on the user.
ObjectiveTo compare the cost-effectiveness of etanercept combined with methotrexate to methotrexate plus placebo in the treatment of rheumatoid arthritis and to provide references for clinical practice.MethodsDecision tree model was developed to estimate the cost-effectiveness from the perspective of the health care system by TreeAge Pro 2016 software. The cost-effectiveness of the two treatments were compared by incremental analysis, and the robustness of the results were analyzed by sensitivity analysis.ResultsThe cost of etanercept combined methotrexate group in one year duration was ￥212 692, the effective rate (ACR50) was 66.4%; the cost of methotrexate combined with placebo group in one year duration was ￥572, the effective rate (ACR50) was 40.6%. The incremental cost-effectiveness ratio of two groups was ￥818 000/person, and the sensitivity analysis showed that the results were robust.ConclusionEtanercept combined methotrexate is significant more effective than methotrexat. But the cost of etanercept combined methotrexate is too high to afford and is not economical compared to methotrexate.