Medicine is a very important health resource in China. Although numerous efforts are paid to pre-marketed medicines, little is done to address practical problems in marketed medicines. The rational use and allocation of marketed medicines remain a major concern for decision-makers in China. It has been recognized that economic evaluation is an efficient tool for prioritizing the choice, and optimizing the use of medicines. This paper has explored the methods and principles for conducting economic evaluation of marketed medicines. Different strategies will be adopted for economic evidence for marketed medicines in terms of adequacy and sufficiency.However, a standard study pathway should be applied in economic evaluation of marketed medicines. Besides, the aspects for developing economic framework and the methods for reviewing existing economic evidence are also introduced in this paper, particularly, for marketed medicines within the same therapeutic group.
ObjectiveTo evaluate the efficacy and cost-effectiveness of thymalfasin (Tα1) as an adjuvant therapy for hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC) after surgery.MethodsPatients with HBV-related HCC who underwent hepatectomy from February 2007 to December 2015 in West China Hospital of Sichuan University, Chengdu Military General Hospital, or the Third Affiliated Hospital of Sun Yat-sen University were retrospectively collected and divided into the Tα1 group and the observation group. Log-rank test and Kaplan-Meier curve were used to assess the overall survival, recurrence-free survival, and safety of patients. A Markov model was used to calculate the quality-adjusted life years (QALYs) and incremental cost-effectiveness ratio (ICER) of the Tα1 group compared with the observation group. Cost data was from the hospital information system of the three hospitals. Utility scores mainly came from published data. Sensitivity analyses were applied to explore the impact of essential variables.ResultsA total of 208 patients with HCC after liver resection were enrolled, among them 48 received Tα1 treatment (the Tα1 group) and 160 were only followed up (the observation group). There was no significant difference between the two groups in the baseline characteristics. The median overall survival of the Tα1 group and the observation group was 102.0 months [95% confidence interval (CI) (74.8, 129.2) months] and 81.6 months [95%CI (65.7, 97.6) months], respectively, and the difference was statisitically significant (P=0.047); the median recurrence-free survival was 66.7 months [95%CI (17.3, 116.1) months] and 37.4 months [95%CI (28.7, 46.2) months], respectively, and the difference was statistically significant (P=0.044). There were no grade Ⅲ-Ⅳ adverse events and no treatment-related death occurred. The ICER of Tα1 group was ￥108 050.02/QALY, which was less than the willingness to pay (￥177 785.25/QALY).ConclusionsTα1, as an adjuvant therapy for HBV-related HCC patients, can improve the prognosis of the patients, and the cost is within the acceptable level in our country, so this strategy is likely to be a cost-effective option compared with the observation group.
ObjectiveTo systematically review the economics evaluation studies on the early screening or diagnosis of primary immunodeficiency diseases (PID). MethodsWeb of Science, CRD, PubMed, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect the economics evaluation studies on the early screening or diagnosis of PID from inception to July 1st, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies, then, a descriptive systematic review was performed. ResultsA total of 10 studies focusing on SCID were included. The results showed that under a relatively high threshold, the early screening and diagnosis of SCID were cost-effective, which can reduce severe infections in patients and treatment costs while improving patient’s survival. ConclusionCurrent evidence shows that early diagnosis of PID can reduce costs and improve benefits. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusion.
Objective To evaluate the ambulatory surgery mode by using health economical mothods and provide reference for optimization and decision of surgical operation mode. Methods The patients who underwent unilateral flexible ureteroscopic holmium laser lithotriphy for ureteral calculi in Xiangya Hospital, Central South University between January 1st to December 31th, 2015 were selected in this study, including 59 with ambulatory surgery mode (the ambulatory group) and 65 with special in-hospital surgery mode (the special group). The differences in average bed occupancy time, cost, therapeutic effect, and satisfaction between the two operation modes were compared. Results The average bed occupancy time in the ambulatory group and the special group was (1.03±0.18) and (6.35±0.74) days, respectively, and the difference was statistically significant (P<0.05). The patients in both groups were followed up for one month after the operation, and the incidence of complications was 6.8% (4/59) in the ambulatory group and 6.2% (4/65) in the special group, without significant difference (P>0.05). The satisfaction score in the ambulatory group and the special group was 96.48±0.23vs. 96.53±0.18 without significant difference (P>0.05). The differences in direct medical cost [(17 738.28±1 027.85)vs. (21 307.67±554.41) yuan], direct non-medical cost [(103.39±18.25) vs. (630.76±78.90) yuan], indirect cost[ (266.93±47.12) vs. (1 640.44±190.55) yuan], and total cost [(18 128.10±1 037.76) vs. (23 558.29±619.20) yuan] between the ambulatory group and the special group were all statistically significant (P<0.05). The treatment effect index in the ambulatory group and the special group was 0.96 and 1.05, respectively; the cost-effect ratio was 18 883.44 and 22 436.47, respectively. Sensitivity analysis showed that the adjusted cost-effect ratio in the ambulatory group (16 629.64) was still lower than that in the special group (20 534.91). Conclusions The cost-effect ratio of ambulatory surgery mode is superior than that of special in-hospital surgery mode, and there is no obvious difference in patients satisfaction between the two modes. Ambulatory surgery mode can be recommended to patients who meet the indications of day surgery.
Objective To investigate cost-effectiveness of no indwelling urinary catheter in pulmonary lobectomy patients and to confirm the advances of no indwelling urinary catheter. Methods We recruited 148 lung cancer patients who were scheduled for pulmonary lobectomy under general anesthesia in West China Hospital from July through December 2015. These patients were divided into two groups including an indwelling urinary catheter group (74 patients, 45 males and 29 females, at age of 52.55±19.87 years) and a no indwelling urinary catheter group (74 patients, 42 males and 32 females, at age of 54.03±16.66 years). Indexes of cost-effectiveness of the two groups were compared. Results There was no statistical difference between the two groups in duration of indwelling catheter (1.56±0.0.65 d versus 1.68±0.91 d, P=0.077). Material expense(4 811.48 yuan versus 296.74 yuan, P=0.045), cost of nursing care (7 413.32 yuan versus 457.32 yuan, P=0.013), and total expense (12 224.8 yuan versus 754.06 yuan, P=0.000) in the indwelling catheter group were higher than those in the no indwelling catheter group. Material expense per patient (65.02±5.62 yuan/patient-time versus 4.01±0.00 yuan/patient-time, P=0.000), cost of nursing care per patient (100.18±7.19 yuan/patient-time versus 6.18±1.22 yuan/patient-time, P=0.000), and total cost per patient (165.20±12.81 yuan/patienttime versus 10.19±1.22 yuan/patient-time, P=0.000) in the indwelling catheter group were higher than those in the no indwelling catheter group. Conclusion Both costs and labor of nurse can be cut down for appropriate lung cancer patients undergoing lobectomy without routine indwelling urinary catheter.
ObjectiveTo compare the safety and comfort of patients with or without postoperative gastric tube placement after esophageal cancer surgery, and analyze the cost and nursing time of gastric tube placement. Methods The patients with esophageal cancer undergoing minimally invasive surgery in West China Hospital of Sichuan University in 2021 were enrolled. The patients were divided into a gastric tube indwelling group and a non gastric tube indwelling group according to whether the gastric tube was indwelled after the operation. The safety and comfort indicators of the two groups were compared. Results A total of 130 patients were enrolled. There were 66 patients in the gastric tube indwelling group, including 53 males and 13 females, aged 61.80±9.05 years and 64 patients in the non gastric tube indwelling group, including 55 males and 9 females, aged 64.47±8.00 years. Six patients in the non gastric tube indwelling group needed to place gastric tube 1 to 3 days after the operation due to their condition. There was no statistical difference in the incidence of postoperative complications between the two groups (P>0.05). The subjective comfort of patients in the gastric tube indwelling group was significantly lower than that in the non gastric tube indwelling group (P<0.001), and the incidence of foreign body sensation in the throat of patients in the gastric tube indwelling group was higher than that in the non gastric tube indwelling group (P<0.001). The average nursing time in the gastric tube indwelling group was about 59.58 minutes, and the average cost of gastric tube materials and nursing was 378.24 yuan per patient. Conclusion No gastric tube used after operation for appropriate esophageal cancer patients will not increase the incidence of postoperative complications (pulmonary infection, anastomotic leakage, chylothorax), but can increase the comfort of patients, save cost and reduce nursing workload, which is safe, feasible and economical.
ObjectiveTo evaluate the economics of duplizumab in combination with optimal supportive care versus optimal supportive care for moderate-to-severe atopic dermatitis in adults from the perspective of our health system. MethodsA Markov model embedded in a decision tree was constructed to compare the long-term cost-utility of dupilumab combined with optimal supportive care versus optimal supportive care, and a sensitivity analysis was performed on the results. ResultsThe results of the basic analysis showed that compared with the optimal supportive care, dupliyuzumab in combination with optimal supportive care resulted in 3.82 more QALYs, while its cost was 125 549.42 yuan more. The ICER was 32 854.83 yuan/QALY, which was less than one times China's per capita GDP in 2022, and was economical. Univariate sensitivity analysis showed that factors such as Dupilumab-16-week post - no response utility value, Dupilumab-52-week post response rate and Dupilumab-52-week adherence had a greater impact on the cost changes. The results of the probabilistic sensitivity analysis showed a stable model structure and good robustness. ConclusionIn adult patients with moderate-to-severe atopic dermatitis, dupliyuzumab in combination with an optimal supportive care regimen is more cost-effective compared to an optimal supportive care regimen.
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.
Objective To compare the economic effectiveness of universal screening, high-risk population screening, and no screening strategies for thyroid disease prevention and control among pregnant women in China through cost-effectiveness analysis, providing evidence-based support for optimizing health policy decisions on prenatal thyroid disease screening. Methods Based on the characteristics of thyroid disorders during pregnancy, a combined decision tree and Markov model was developed to conduct a lifetime cost-effectiveness analysis across three strategies: no screening, high-risk population screening, and universal screening. Sensitivity analyses were performed on key parameters. Results Base-case analysis demonstrated that universal screening was the most cost-effective strategy when the World Health Organization (WHO)-recommended payment threshold of 1×gross domestic product (GDP) per capita was used, with an incremental cost-effectiveness ratio (ICER) of 20636.18 yuan per quality-adjusted life year (QALY) compared to no screening, followed by high-risk population screening (ICER=21071.71 yuan/QALY). The results of the sensitivity analysis showed a strong stability of the model. Conclusions Of the 3 screening programs for thyroid disease in pregnancy, universal screening is the most cost-effective when the WHO-recommended payment threshold of 1×GDP per capita is used.
Objective To systematically review the cost-effectiveness of gefitinib for advanced non-small cell lung cancer (NSCLC), in order to provide the economics values of gefitinib for clinical application. Method We electronically searched databases including PubMed, Ovid, Embase, Cochrane Library, Medline, China National Knowledge Internet, VIP, and Wanfang database for articles about the cost-effectiveness of gefitinib for advanced NSCLC patients from January 1946 to October 2017, and then performed a systematic literature review of economic evaluations of geftinib. Results A total of 20 independent studies were included in the present systematic review, in which 8 were the first-line treatment, 9 were the second-line treatment, 1 was the third-line treatment, and 2 were maintenance treatment. The most common comparison was gefitinib vs. chemotherapy (n=7), and other comparisons were gefitinib vs. erlotinib (n=4), gefitinib vs. docetaxel (n=3), gefitinib vs. placebo (n=2), gefitinib vs. icotinib (n=2), gefitinib vs. afatinib (n=1), and gefitinib vs. other treatments (n=1). For the advanced NSCLC patients, the first- or second-line treatment with gefitinib compared to chemotherapy was considered to be more cost-effective, especially in patients with mutated epidermal growth factor receptor gene. As the second-line treatment, gefitinib was considered to be more economical than erlotinib and docetaxel. Conclusion Gefitinib is considered to be a cost-effective strategy for the advanced NSCLC patients as the first- or second-line therapy.