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OBJECTIVE To evaluate the cost-utility of as-needed inhaled budesonide/formoterol versus budesonide maintenance therapy combined with as-needed inhaled terbutaline （hereinafter referred to as budesonide maintenance therapy） in patients with mild asthma from the perspective of the Chinese health service system. METHODS A Markov model of mild asthma was established based on an international multicenter randomized controlled clinical study （SYGMA 2 study）； the model cycle was one week， and the model had a whole horizon of 60 years. The cost only included direct medical cost， and utility value was derived from the data of EuroQol 5-Dimension 5-Level in the SYGMA 2 study and published literature. The total cost and total output of the above two inhalation therapies for patients with mild asthma were calculated， with discount rate of 5%. The stability of the model was evaluated by sensitivity analysis. RESULTS The total cost of as-needed inhaled budesonide/formoterol and budesonide maintenance therapy were 25 884 yuan and 45 822 yuan， respectively， and the effectiveness were 30.51 quality- adjusted life years （QALYs） and 30.50 QALYs， respectively. The former scheme was an absolute advantage. One-way sensitivity analyses showed that the price of drug （terbutaline and budesonide/formoterol） and average number of inhalations per day were the main influencing parameters， but they had little influence on the results of basic analysis. Probabilistic sensitivity analysis showed that the probability of as-needed budesonide/formoterol being cost-effective was 100%. CONCLUSIONS Compared with budesonide maintenance therapy， as-needed inhalation of budesonide/formoterol in mild asthma patients is more cost-effective.
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OBJECTIVE To evaluate the effectiveness， safety and economics of Aidi injection combined with first-line chemotherapy for non-small cell lung cancer （NSCLC）， and to provide evidence-based reference for clinical drug use and decision. METHODS Retrieved from PubMed， Embase， the Cochrane Library， CNKI， Wanfang databases， VIP and Health Technology Assessment （HTA） related websites， HTA reports， systematic evaluation/meta-analysis and economic evaluations about Aidi injection combined with first-line chemotherapy for NSCLC were collected from the inception to Aug. 2022. After data extraction and quality evaluation， descriptive analysis was performed for the results of included studies. RESULTS A total of 16 pieces of literature were included， involving 1 piece of systematic review， 13 pieces of meta-analysis， 2 pieces of pharmacoeconomic studies. Compared with first-line chemotherapy， Aidi injection combined with first-line chemotherapy could improve response rate and disease control rate， prolonged survival time， improved survival quality， reduced the incidence of nausea and vomiting， leukocytopenia and thrombocytopenia， and improved immune function， but had controversial effects on liver and kidney function. With the payment threshold set by 3 times the national per capita GDP in 2019， Aidi injection combined with first-line chemotherapy had a more economical probability. CONCLUSIONS Aidi injection combined with first-line chemotherapy for NSCLC has good efficacy and safety， and has certain economic benefits. However， given the limited pharmacoeconomic studies included， the economic conclusions obtained need to be carefully interpreted.
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OBJECTIVE To evaluate the cost-effectiveness of pembrolizumab combined with chemotherapy versus placebo combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma （BTC） from the perspective of China’s health system. METHODS A partitioned survival model was constructed based on the KEYNOTE-966 study data. The simulation period was 21 days， and the simulation time was the patient’s whole life. Using quality-adjusted life year （QALY） as the output indicator， the cost-utility analysis method was used to evaluate the cost-effectiveness of the two schemes mentioned above. Univariate and probabilistic sensitivity analyses were performed to verify the results of the basic analysis， and to explore the cost-effectiveness under the scenario of drug donation scheme. RESULTS The basic analysis showed that both the cost and effectiveness of the pembrolizumab group were higher than those of the placebo group， and the incremental cost-effectiveness ratio （ICER） was 3 909 359.78 yuan/QALY， which was higher than the willingness-to-pay （WTP） threshold of 3 times 2022 gross domestic product （GDP） per capita （257 094 yuan）， indicating no cost-effectiveness. The results of univariate sensitivity analysis showed that the utility discount rate， the utility value of progression-free survival （PFS） status， the cost discount rate， and the cost of pembrolizumab had a great influence on ICER. Probabilistic sensitivity analysis verified the robustness of the results of basic analysis， and concluded that when the WTP threshold was greater than 1 500 000 yuan/QALY， the pembrolizumab group became cost-effective. The results of the scenario analysis showed that considering the drug donation scheme of pembrolizumab for low-income people， although its treatment cost was significantly reduced， it was still not cost-effective. CONCLUSIONS At the WTP threshold of 3 times China’s GDP per capita in 2022， pembrolizumab combined with chemotherapy is not cost-effective compared with placebo combined with chemotherapy for advanced or unresectable BTC.
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OBJECTIVE To evaluate the cost-effectiveness of clopidogrel versus aspirin monotherapy regimens for secondary prevention of ischemic stroke and to provide economic evidence and reference for clinical medication and decision-making. METHODS Based on the CAPRIE trial， a Markov model was constructed； the probabilities of risk events， health utility values， and costs of risk event management were obtained from relevant literature. The cycle length was 6 months， and the time horizon was 10 years. A discount rate of 5% per year was applied. The primary outcomes were total costs， quality-adjusted life-years （QALYs）， and incremental cost-effectiveness ratio （ICER）. Cost-utility analysis was performed for above 2 regimens by using TreeAge Pro software. The one-way sensitivity analysis， probabilistic sensitivity analysis and scenario analysis were conducted to validate the robustness of the analyses. RESULTS Compared with the aspirin regimen （325 mg/d of CAPRIE trial dose）， the ICER values of clopidogrel regimen for secondary stroke prevention for 10 years， 20 years and 30 years were 4 284.06， 4 201.20 and 3 986.78 yuan/QALY， respectively， which were E-mail：liuxiaoyanrj@sjtu.edu.cn all less than the willing-to-pay （WTP） threshold of one time 。 China’s per capita gross domestic product （GDP） in 2021. E-mail：scilwsjtu-wb@yahoo.com Compared with the aspirin regimen （clinically recommended dose in China， 100 mg/d）， the ICER values of clopidogrel regimen for stroke secondary prevention for 10 years， 20 years and 30 years were 58 238.27， 42 164.72 and 36 164.77 yuan/QALY， respectively， which were all less than WTP threshold. When comparing with aspirin regimen of 325 mg/d， results of one-way sensitivity analysis showed that the cost of clopidogrel and aspirin， probability of the first recurrence of ischemic stroke were sensitive factors of model. Results of probabilistic sensitivity analysis showed that when WTP was set at one time GDP per capita in China in 2021， clopidogrel had a probability of being cost- effective of about 66.5%. Results of scenario analysis showed that neither changing the time horizon to 10， 20 or 30 years nor using different doses of aspirin （50， 100， 150， 200 or 250 mg/d） would not alter any conclusions. CONCLUSIONS Compared with aspirin monotherapy， clopidogrel monotherapy is more cost-effective for secondary prevention of ischemic stroke.
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Objetivos: contribuir para a geração de dados de avaliação econômica de estratégias de empoderamento farmacoterapêutico para pacientes com Diabetes Mellitus tipo 2 (DM tipo 2). Métodos: este estudo farmacoeconômico é aninhado a um ensaio clínico com controle não randomizado que incluiu pacientes ≥18 anos de idade, cadastrados no HIPERDIA. Os pacientes foram alocados em um modelo de Markov conforme valores de hemoglobina glicada do acompanhamento. As probabilidades do surgimento de complicações relativas ao DM, incluindo-se óbito, foram estimadas por dez anos. Cada complicação do DM tipo 2 teve seu custo estabelecido para determinação do custo anual. Resultados: entre os participantes da intervenção, não ocorrem óbitos ocasionados por DM tipo 2, e a progressão de complicações mantém-se estável durante os anos simulados, enquanto, no grupo controle, 60% dos pacientes podem evoluir para óbito nos dez anos, e a probabilidade de serem acometidos por complicações relacionadas ao DM tipo 2 é crescente. Com relação aos custos, ao final de dez anos, os pacientes que participaram da Estratégia Individual de Empoderamento Farmacoterapêutico (EIEF) tiveram um custo médio de UU$134,45 poupando a vida de 100% dos pacientes, e os pacientes do atendimento convencional um custo médio de UU$237,12 e 40% dos pacientes acompanhados chegariam ao final do ciclo com vida. Conclusão: a EIEF parece ser uma alternativa economicamente viável em longo prazo, bem como para a promoção do controle glicêmico.
Objectives: contribute to the data generation for the economic evaluation of pharmacotherapeutic empowerment strategies for type 2 diabetes mellitus patients (type 2 DM). Method: This pharmacoeconomic study is nested in a clinical trial with non-randomized control that included patients ≥18 years old, registered in HIPERDIA. The patients were allocated to a Markov model according to the follow-up glycated hemoglobin values. The probabilities of the appearance of complications related to DM, including death, have been estimated for ten years. Each complication of type 2 DM had its cost established to determine the annual cost. Results: Among the participants in the intervention, there are no deaths caused by type 2 DM, and the progression of complications remains stable during the simulated years, whereas in the control group, 60% of the patients can progress to death in ten years and the probability of being affected by complications related to type 2 DM is increasing. Regarding costs, at the end of ten years, patients who participated in Individual Pharmacotherapeutic Empowerment Strategy (IPES) had an average cost of US$ 134.45, saving 100% of patient's lives, and conventional care patients cost an average of US$ 237.12 and 40% of the patients followed would reach the end of the life cycle. Conclusion: The IPES seems like an economically viable and long-term economic alternative and promotes glycemic control.
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Diabetes Mellitus , Cost-Benefit Analysis , Costs and Cost Analysis , Empowerment , Glycemic ControlABSTRACT
OBJECT IVE To evaluate the efficacy ，safety and cost-effectiveness of Qili qiangxin capsule in the treatment of chronic heart failure ，and provide reference for drug selection and evaluation in relevant institutions. METHODS Meta-analysis was performed to investigate clinical efficacy and safety of Qili qiangxin capsule combined with routine treatment （combined treatment group）versus routine treatment （routine treatment group ）in the treatment of chronic heart failure. From the perspective of Chinese health care system ，a decision tree model was constructed. The time horizon of the model was 1 year. The effective rate obtained by meta-analysis was taken as the effect parameter ，and the total cost was calculated by drug cost and hospitalization cost ，to evaluate the cost-effectiveness of combined treatment versus routine treatment in the treatment of chronic heart failure. Subgroup analysis was carried out according to the course of treatment and literature quality ，and one-way sensitivity analysis and probability sensitivity analysis were adopted to check the robustness of basic analysis results. RESULTS Total of 72 literatures involving 9 575 patients were included in meta-analysis. Results of meta-analysis showed that effective rate ，left ventricular ejection fraction ， N-terminal fragment of the prohomone brain-type natriuretic peptide and 6 minute walking distance in combined treatment group were all better than those of routine treatment group ， while its safety was similar to routine therapy. The results of cost-effectiveness analysis showed that the cost of combined therapy was 1 867 yuan higher than that of routine therapy ，patients could get 0.016 QALYs more ，and the incremental cost-effectiveness ratio （ICER）was 117 861 yuan/QALY. If only high-quality literature were included for meta-analysis and the effectiveness parameters were obtained ，the ICERs of the combined therapy versus routine therapy were 102 162 yuan/QALY（based on all high-quality literature ）and 72 354 yuan/QALY（based on high-quality literature with treatment course of 24 weeks）. The results of the probability sensitivity analysis showed that taking twice China ’s per capita gross domestic product in 2020 as the willingness to pay threshold ，the probability of cost-effectiveness for Qili qiangxin capsule combined with routine therapy was 67.1％ . CONCLUSIONS Compared with routine therapy ，Qili qiangxin capsule combined with routine therapy has better clinical efficacy ，equivalent safety and cost-effective.
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OBJECTIVE To systematically evaluate the economical efficiency of marketed a naplastic lymphoma kinase （ALK）-tyrosine kinase inhibitors （TKI）for the treatment of ALK-positive non-small cell lung cancer （NSCLC）in China ，and to provide a reference for the selection of China ’s medical insurance list and drug pricing. METHODS Computer searches of databases such as CNKI ，Wanfang database ，VIP，PubMed，Embase and the Cochrane Library were conducted to collect pharmacoeconomic evaluation studies of four marketed ALK-TKI （crizotinib，seretinib，aletinib and ensatinib ）in the treatment of ALK-positive NSCLC in China during the inception to July 2021. The qualities of the included literature were evaluated using CHEERS checklist ，and analyzed systematically in terms of both methodological and economic outcomes. RESULTS A total of 6 literatures were included ，with a compliance rate of 71％ to 83％ for the CHEERS list criteria ，and the overall quality of the literature was high. In terms of methodological analysis ，the pharmacoeconomic evaluation methods included in the study were mainly model-based （Markov or partitioned survival models ） and real-world data-based cost-utility analysis. Most research perspectives were health insurance payer and health system perspectives ；all cost types were direct medical costs. In terms of economical efficiency analysis ，compared with chemotherapy plan ，2 studies confirmed that ALK-TKI （crizotinib，seretinib）were not economic ，1 study confirmed ALK-TKI （crizotinib） showed economical efficiency. Seretinib showed relatively higher economical efficiency when compared to other ALK-TKIs. High drug prices were the main factor why ALK-TKI was not economically viable for treating ALK-positive NSCLC. CONCLUSIONS The second-generation ALK-TKI （seretinib， 163.com alectinib） have better economical efficiency than the first-generation ALK-TKI （crizotinib）. The economical efficiency of seretinib is the best among the second-generation · ALK-TKI. The economical efficiency of chemotherapy regimen is better than that of the second-generation ALK-TKI （seretinib）. Economic comparison between chemotherapy and first-generation ALK-TKI （crizotinib）remains controversial.
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OBJECTIVE To eva luate the economy of loratinib versus crizo tinib in the first-line treatment of anaplastic lymphoma kinase （ALK）-positive advanced non-small cell lung cancer （NSCLC）from the perspective of China ’s health system ， and to provide reference for the product pricing and related medical decisions of the drug in other regions of China except for Hong Kong. METHODS Markov model and partition survival model both constructed based on the CROWN data （the simulation time limit was 10 years and the cycle period was 4 weeks）；the quality adjusted life year （QALY）was used as the outcome index to calculate the incremental cost-effectiveness ratio （ICER）. One-way sensitivity analysis ，probability sensitivity analysis and scenario analysis were used to verify the robustness of the results. RESULTS The basic analysis results based on Markov model showed that compared with crizotinib group ，the per capita cost of loratinib group increased by 17 867 588.63 yuan，the per capita utility increased by 1.76 QALYs，and the ICER was 10 152 038.99 yuan/QALY. The basic analysis results based on the partition survival model showed that compared with the crizotinib group ，the Δ 基金项目：江苏省博士后科研资助计划项目（No.2021K496C）； per capita cost of loratinib group increased by 18 009 592.54 2020年度高校哲学社会科学研究一般项目（No.2020SJA0070） yuan，the per capita utility increased by 1.74 QALYs，and the ＊硕士研究生 。研究方向 ：药物经济学 、卫生经济与政策 。 E-mail：sunlei_cpu@163.com ICER was 10 350 340.54 yuan/QALY. The results of one-way # 通信作者：教授，博士生导师。研究方向：药物经济学、卫生经 sensitivity analysis of the two models both showed that 济与政策。E-mail：ma86128@sina.com progression-free survival （PFS）state utility v alue，progression- ·1102· China Pharmacy 2022Vol. 33 No. 9 中国药房 2022年第33卷第9期 disease（PD）state utility value and loratinib cost had great influence on the results. The results of probability sensitivity analysis showed that when 1-3 times of China ’s per capita GDP in 2020 was taken as the willingness to pay threshold ，the probability of loratinib being economical was 0. The recommended unit price of loratinib per 100 mg was 657.10-815.60 yuan. CONCLUSIONS For patients with ALK-positive advanced NSCLC ，loratinib is more effective than crizotinib in the first-line treatment ，but it is not economical under the current price ；reasonably lowering the price of loratinib can increase the probability of its economy.
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To evaluate the pharmacoeconomic value of Qidong Yixin Oral Liquid in the treatment of viral myocarditis(Qi-Yin deficiency syndrome) by supplementing Qi, nourishing the heart, calming the mind, and relieving palpitation, the present study performed the Meta-analysis based on the published papers on Qidong Yixin Oral Liquid by AMSTAR and carried out pharmacoeconomic evaluation using TreeAge Pro by the cost-effectiveness analysis. The results showed that the quality of the included papers was good. After four weeks of treatment, Qidong Yixin Oral Liquid combined with the conventional treatment regimen was superior to the conventional treatment in improving creatine kinase isoenzyme, and the difference was statistically significant. Furthermore, the treatment cost was also higher than that of conventional treatment, with an incremental cost-effectiveness ratio of CNY 95.89, accounting for 0.30% of per capita disposable income. The results of sensitivity analysis showed that the research results were robust. Therefore, based on the assumption that the per capita disposable income in 2020 was the threshold of patients' willingness to pay, it is more economical for patients with viral myocarditis to use Qidong Yixin Oral Liquid combined with conventional secondary prevention regimen than conventio-nal secondary prevention regimen alone. The economic evaluation of Qidong Yixin Oral Liquid in the treatment of viral myocarditis will help physicians and patients choose optimal treatment options, improve rational clinical medication, and provide references for the efficient allocation and utilization of medical resources in China.
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Humans , Cost-Benefit Analysis , Drugs, Chinese Herbal/therapeutic use , Economics, Pharmaceutical , Myocarditis/drug therapy , Qi , Yin Deficiency/drug therapyABSTRACT
OBJECTIVE：To evaluate the economy performance of dexamethasone （DXM）combined with rituximab （RTX） for the first-line treatment of chronic primary immune thrombocytopenia （ITP）in adults. METHODS ：From the perspective of China ’s medical and health system ，Markov model for eight states was constructed with a period of 4 weeks and a time limit of 20 years， using DXM regimen as control. The cost-utility of DXM+RTX regimen for the treatment of chronic ITP in adults were evaluated. The parameters of clinical efficacy and utility value were derived from own published literature ；cost parameters were from the MENET website and the official websites of local health committees and medical insurance bureaus ；one-way sensitivity analysis ， probability sensitivity analysis and scenario analysis were performed to observe the uncertainty of model and data source. RESULTS：The average cost of DXM+RTX regimen was 51 064 dollars and that of DXM regimen was 50 455 dollars. Compared with DXM regimen ，DXM+RTX regimen yielded an additional 0.14 QALYs for each patient ；the incremental cost-effectiveness ratio（ICER）was 4 356 dollars/QALY，and was lower than the willingness-to-pay threshold of China ’s per capita gross domestic product（GDP）in 2020. In the one-way sensitivity analysis ，the cost of drugs was the main driver in the model. Probability sensitivity analysis demonstrated that DXM+RTX regimen had 57.5％-61.0％ probability of being cost-effective at a willingness- to-pay threshold of 1-3 times per capita GDP in 2020. The results of scenario analysis showed that DXM+RTX regimen would have obvious long-term benefits ，and the utility value had little impact on the conclusion. CONCLUSIONS ：DXM + RTX is more economical than DXM in the treatment of chronic ITP in adults ，but the results have the uncertainty.
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OBJECTIVE：To review the method and results of pharmacoeconomic evaluation of aspirin for cardiovascular disease prevention ，and to provide reference for economic evaluation of aspirin and clinical medication decision. METHODS ： Using“cardiovascular disease ”“cost-effectiveness”“cost-utility”“cost-benefit”“cost effectiveness ”“cost utility ”as the Chinese search terms ，using“cost-effectiveness”“cost-utility”“cost-benefit”“economic analysis ”“pharmacoeconomics”as English search terms，relevant literatures about pharmacoeconomic evaluation of aspirin for cardiovascular disease prevention published during January 1，2000 to January 17，2021 were retrieved from CNKI ，Wanfang database ，VIP，PubMed，Web of Science ，the Cochrane Library. After screening literatures according to inclusion and exclusion criteria ，extracting relevant data ，the quality of included literatures was evaluated with CHEERS scale. The method and results of pharmacoeconomic evaluation of aspirin in the prevention of cardiovascular diseases were analyzed statistically in terms of basic information ，literature quality ，model structure and elements ，health status and utility value ，cost items and sources ，health output ，economic evaluation and sensitivity analysis. RESULTS & CONCLUSIONS ：Nine literatures were included ，and the total coincidence rates of the literatures were all above 80.00％. The pharmacoeconomic evaluation of aspirin in the prevention of cardiovascular disease mainly adopted Markov model ， and the model structure was relatively mature. The cost mainly considered the direct cost ，and the data mainly came from the medical insurance database ；utility was calculated according to the utility value of health state ，which mostly came from the existing literatures. The sensitivity analysis adopted deterministic sensitivity analysis and probabilistic sensitivity analysis ，and the main influential factor was cost. It was economical to use aspirin for cardiovascular disease prevention in most cases ，and aspirin was more economical for primary prevention of cardiovascular disease. It is suggested that domestic scholars can refer to China ’s pharmacoeconomic guidelines to carry out relvant pharmacoeconomic evaluation research more standardized.
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OBJECTIVE：To evaluate the economy of pe rospirone in the treatment of schizophrenia ，to provide guidance for clinically proper use of medications more cost-effectively ，and related health decision-making . METHODS ：A short-term decision tree model was constructed from the perspective of medical insurance payer to calculate the cost and health outcomes of different treatment plans considering major adverse events including extrapyramidal reaction ，weight gain ，diabetes，hyperlipidemia. The cost-utility of perospirone were compared with quetiapine ，aripiprazole and olanzapine respectively ，using QALYs as the measure of health outcomes ，3 times GDP per capita as the willingness-to-pay threshold ；probability sensitivity analysis was performed. RESULTS：The results of base-case analysis showed that the cost of perospirone （6 688.25 yuan）was lower than those of quetiapine （9 887.45 yuan），aripiprazole（13 284.65 yuan）and olanzapine （15 332.80 yuan）. The utility of perospirone （0.79 QALYs）was better than those of quetiapine （0.76 QALYs），aripiprazole（0.77 QALYs）and olanzapine （0.75 QALYs）. Compared with quetiapine ， aripiprazole and olanzapine ，peropirone had lower cost and higher health outcome ，which indicated that strong dominance favors perospirone over the other 3 drugs. The results of sensitivity analysis were consistent with those of base-case analysis. CONCLUSIONS：Perospirone has economic advantages in treating schizophrenia patients compared to other commonly used atypical antipsychotic drugs.
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OBJECTIVE：To provid e reference for safe ，effective and economical medication scheme for type 2 diabetes mellitus（T2DM）. METHODS ：Markov model was established for rosiglitazone sodium and metformin in the treatment of T 2DM. According to the development characteristics of T 2DM，the development of T 2DM was simulated by the dynamic changes of event-free，complications and deaths of T 2DM. The long-term cost and effect of rosiglitazone sodium and metformin in the treatment of T 2DM were obtained by regression analysis and queue simulation analysis. QALYs was used as a health output indicator，and t he superiority and inferiority of different schemes were judged by the ICER value ，and in our study ICER value was WTP（12 000 yuan per year ）of diabetics. The sensitivity of cost ，utility and discount was analyzed to check the stability of the analysis results. RESULTS ：Cost-effectiveness analysis of Markov model showed that the cumulative cost and health effectiveness of rosiglitazone sodium therapy were 25 164.00 yuan and 7.50 QALYs，while 17 773.36 yuan and 7.36 QALYs for metformin ； ICER of rosiglitazone sodium relative to metformin was 50 983.08 yuan/QALYs，which was greater than WTP ，so the metformin treatment was an advantageous scheme. Sensitivity analysis showed that health utility value and discount rate of diabetes mellitus greatly influenced analysis results of the model ，but advantage plan had not changed within the sensiitivity analysis range seted in this study. CONCLUSIONS ：For T 2DM，metformin is more cost-effective than rosiglitazone sodium.
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OBJECTIVE：To evaluate the economics of first-line therapy drug for metastatic renal cell carcinoma （mRCC）as sunitinib，sorafenib and pazopanib ，and to provide reference for the adjustment of medical insurance list and clinical medication decision. METHODS ：Using“metastatic renal cell carcinoma ”“mRCC”“sunitinib”“sorafenib”“pazopanib”“cost-effectiveness” “cost-utility”“cost-benefit”“economic analysis ”as the Chinese and English retrieval words ，relevant literatures published during Jan. 1st，2006 to Jul. 15th，2019 were retrieved from PubMed ，Web of Science ，the Cochrane Library ，CNKI，Wanfang database ， VIP. The literatures were screened according to inclusion and exclusion criteria . The quality of the included literatures was evaluated with CHEERS scale. The effectiveness and economy of sunitinib ，sorafenib and pezoparib in the treatment of mRCC were compared qualitatively after the relevant data were extracted. RESULTS ：A total of 10 literatures were included ，and the total coincidence rates of 7 literatures over 75.00％. Among the 4 literature studies of sulatinib vs. sorafenib ，3 literature studies pointed out that sulatinib was the absolute advantage scheme ，and 1 literature study pointed out that sorafenib was more economical ； among the 6 literature studies of sunitinib vs. pezoparib ，4 literature studies indicated that pezoparib was the absolute advantage scheme，and 2 literature studies indicated that sunitinib was more economical. CONCLUSIONS ：In most cases ，the efficacy and economy of pezoparib in the treatment of mRCC is better than sunitinib and sorafenib ，but real world data shows that sunitinib is more economical.
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OBJECTIVE：To know about the research status of health utility value obtained by mapping method in pharmacoeconomic evaluation ，and to provide reference for bibliometric study in pharmacoeconomic evaluation . METHODS ： Using“Mapping method ”“Health utility value ”“Cost-utility”“Utility point system ”as Chinese and English keywords ，retrieved from CNKI ，Wanfang database ，PubMed，Medline，Ebsco，Ovid and Wiley database ，empirical journal documents published from the inception to Dec. 31st，2018 about using mapping method to obtain health utility value were collected. The bibliometrics was used to statistically analyze basic information of included literature ，the construction and test of the model ，the type of the best model and so on. RESULTS ：The 124 included documents were all published in English journal. In the construction and testing of the mapping model ，the most frequently used econometric methods ，performance evaluation indicators and model testing methods were ordinary least squares （OLS），mean absolute error （MAE）and residual normality test ，application frequency of which were 97 times（31.60％），89 times（24.93％）and 62 times（21.09％）. There are 117 articles that define the best mapping model ，of which 101 articles（86.32％）have the best direct mapping effect. Most of the non-utility measurement scales adopted specific scales （92 articles，77.97％），and a few literatures adopted the universal scale （26 articles，22.03％）. The most utility measurement scales were 3-level European 5-dimensional health scale （79 articles，66.95％）. CONCLUSIONS ：The domestic empirical researches that use the mapping method to obtain health utility values need to be developed yet. A series of mapping models successfully developed by foreign scholars not only provide the feasibility of using non-utility measurement scales for cost-utility analysis ，but also provide more ideas for China to choose the corresponding econometric methods ，evaluation indicators and mapping methods in the empirical research of the mapping method in the future.
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Background: Diabetes Mellitus is a worldwide growing problem causing threat to patient's health because of its association with various complications and comorbidities. It is a chronic disease requiring lifelong medication which further adds to the economic burden. The objective of this study was to evaluate the prescribing pattern and to do pharmacoeconomic analysis of prescribed antidiabetic drugs.Methods: This observational cross sectional study was conducted for 12 months duration in Outpatient Pharmacy of tertiary care hospital. Prescriptions with antidiabetic drugs were captured and evaluation of prescribing pattern along with pharmacoeconomic analysis of antidiabetic drugs was done.Results: A total of 611 prescriptions with antidiabetic drugs were analyzed. There were total 4034 drugs in all prescriptions with a mean of 6.6 drugs per prescription. 4.28% of drugs were prescribed by generic name and 58.9% of prescribed drugs were from essential drug list. Dual drug therapy was prescribed in maximum number of patients (42.2%) followed by monotherapy (28.8%). More commonly prescribed class of antidiabetic drugs was biguanides as monotherapy (n=119) and its combination with sulfonylureas was prescribed maximally among dual drug therapy (n=158). Cost of monthly therapy for antidiabetic drugs prescribed as monotherapy was least with Biguanides (? 98.89/ month) whereas combination of biguanides and thiazolidinediones was least expensive among dual drug therapy (? 216/ month).Conclusions: Biguanides was the most common prescribed class of antidiabetic drugs among monotherapy and its combination with sulfonylureas was most prescribed as dual drug therapy and both of these therapies were economical.
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Background & objectives: Tyrosine kinase inhibitors (TKIs) targeting the epidermal growth factor receptor (EGFR) have been evaluated in patients with advanced non-small cell lung cancer (NSCLC). Erlotinib and gefitinib are the first-generation EGFR-TKIs for patients with NSCLC. However, there is a paucity of studies comparing the effectiveness of these two drugs. Hence, this study was aimed to compare the effectiveness and safety of erlotinib and gefitinib in NSCLC patients. Methods: This study included 71 NSCLC patients who received EGFR-TKIs between 2013 and 2016. Adverse drug reaction of both erlotinib (n=37) and gefitinib (n=34) was determined and graded according to Common Terminology Criteria for Adverse Events grading system. Effectiveness was measured using response evaluation criteria in solid tumours and progression-free survival (PFS). Pharmacoeconomic analysis was performed by cost-effective analysis. Results: When comparing safety profile, both the drugs had similar adverse events except for dermal side effects such as acneiform eruption (51.4%), rash (54.05%) and mucositis (59.5%) for erlotinib and 20.6, 26.5 and 29.4 per cent for gefitinib, respectively. The PFS of the two drugs was compared to differentiate the effectiveness of erlotinib and gefitinib. There was no significant difference between the effectiveness of the two drugs. The pharmacoeconomic analysis showed that gefitinib was more cost-effective than erlotinib. Interpretation & conclusions: This study showed that erlotinib and gefitinib had similar effectiveness but gefitinib had a better safety profile compared to erlotinib. Therefore, gefitinib could be considered a better option for NSCLC patients compared to erlotinib. However, further studies need to be done with a large sample to confirm these findings.
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Background: Cancer is one of the most expensive and lethal noncommunicable diseases globally. Availability and affordability of anticancer drugs are the most important factors on which management of cancer depends. The objective of the study was to evaluate the variation of cost among different brands of anti-cancer drugs available in the Indian market.Methods: 揅urrent Index of Medical Specialties� July-October 2018 and 揇rug Update� Sept - 2018 were used to obtain cost in INR* (Indian National Rupees) of anticancer drugs manufactured by different pharmaceutical companies in India, in the same strength and dosage form. Percentage cost variations were calculated by minimum and maximum costs of anticancer drug of different brands.Results: Percentage variation in cost was analyzed for 41 different formulations of 27 anticancer drugs. Highest cost variability seen with Alkylating agent Carboplatin 150 mg injection (1100%) and lowest with Antimetabolite anticancer agent Cytarabine 500 mg injection (6.56%). Three formulations showed more than 500% cost variation, largest with Carboplatin 150 mg injection (1100%) followed by Anastrozole 1 mg tablet (870%) and Letrozole 1 mg tablet (508.42%).Conclusions: Present study finding showed significant cost variation in different brand of many anticancer drugs in India. These results indicated that greater price transparencies required. This price variation issue requires a much more in-depth analysis of the health care system.
ABSTRACT
Rare diseases are usually defined as diseases with a low incidence in the population. According to re-ports, there were 6084 rare diseases found worldwide in 2016, involving 3715 related genes. 80% of rare diseases are caused by heredity, dominant or recessive inheritance or mutations. The definition of rare diseases by”incidence”in the population varies from country to country, like the EU <1/2000 and the United States <1/2500. A drug used to prevent, treat, or diagnose a rare disease is called an orphan drug. Due to the difficulty of research and development in this small target population, the high investment risk and the high R&D cost, the problem associated with high price of orphan drugs is very prominent. In the cost-effectiveness evaluation, the resulting incremental cost-effectiveness ratio(ICER)is likely to exceed the willingness to pay threshold adopted by most countries and regions, resulting in multiple countries raising its willingness to pay threshold reference standard(3-25 times GDP per capital)for orphan drugs, or simply ex-empting health technology assessment(HTA)assessment for orphan drugs and only evaluating the impact on medical in-surance fund. This paper systematically reviewed the various internationally accepted orphan drug HTA assessment meth-odologies. Based on the characteristics of rare diseases, the paper discusses the establishment of a model for the evalua-tion of orphan drugs and the multi-dimensional value assessment framework for HTA. At the same time, according to the internationally accepted norm, the paper also explores the way to determine the willingness to pay threshold in HTA as-sessment for orphan drugs in China.
ABSTRACT
Gastrointestinal stromal tumor (GIST) is a relatively rare type of gastrointestinal tract tumors. Thus, it is difficult to perform randomized controlled trials (RCTs) of GIST in a single center, which are often plagued by a small number of participants. Real world study (RWS) is a complement to the evidence derived from traditional RCT. Emerging sources of real world data offer enormous opportunity for deeper understanding of why treatments work (or not) and for whom. Evidence generated from RWD can help clarify best use of treatments for individuals and populations, and care value. Thus, RWS of GIST has attracted much attention. RWS helps us better understand the diagnosis, treatment, prognosis and outcome prediction of GIST in clinical practice. GIST is often misdiagnosed as other tumor. A diagnostic test provides evidence on how well a test correctly identifies or rules out GIST. Therapeutic research aims to evaluate the effectiveness and/or safety of a therapy for GIST. Prognostic research aims to forecast the likely outcome of GIST, explore the factors affecting the outcome, and analyze quality of life. Predictive research aims to quantify the probability of identification or health outcome of GIST based on a set of predictors. Pharmacoeconomic data in real world can evaluate the cost-effectiveness of medicinal products for GISTs and serve as a guidance tool for optimal healthcare resource allocation. Attaching importance to data sourse and data quality, strenthening communicate with a qualified statistician, and the implementation of a standardized process are necessary for performing a high-quality RWS.