2016, not based on real patient data when this data is being recommended for use in pharmacoeconomic studies and therefore sometimes does not accurately and fully reflect the actual treatment of patients. On the other hand, the author's study did not have a treatment group when estimating, as well as the simulation model only stopped at one year without assessing the stable state of the patient after treatment; therefore, it is impossible to determine the cost of BCMDT patients using IM or NL for treatment.
Through data collection of chronic stage CKD patients using IM drugs and CKD patients resistant to or intolerant to IM using NL drugs, the average age of patients was about 47, lower than the average age of disease in European countries and the US; but similar to other studies in Asia.
[49] as well as studies in Vietnam [2], [27], [36]. Similar results were also noted for the male:female ratio, with a higher incidence in men than in women [2], [27], [36], [42], [48]. The difference in the incidence of BCMDT by gender has been noted early in many studies, not only in BCMDT but also in other cancers with the majority of cases being higher in men than in women and the survival prognosis/average survival time of men being lower than that of women [53]. This implies that, although men have fewer adverse risks than women, they have a lower survival rate, and therefore need to be taken into account during treatment [53]. Other relevant sociodemographic characteristics including education level, residence and occupation were also examined in this study. The results showed that the majority of patients had an education level of high school or lower (over 70%), lived in other provinces (over 70%), and about 30% belonged to low-income occupations (farmers, housewives, unemployed/unable to work). This result is similar to epidemiological studies of BCMDT patients in Indonesia (68% had high school education or lower, 27% belonged to low-income occupations) [123] and Brazil (76% had high school education or lower, 28% had low income and most patients lived far from the hospital) [122]. Thus, based on the income of the patients, when the drug aid program ends, it can be predicted that at least 30% of patients will stop treatment or switch to HU treatment with low treatment efficacy and high side effects. In addition
In addition, a study in Brazil also noted that educational level is a demographic factor that has a great influence on the effectiveness of drug treatment and patient mortality [122]. This is explained by the author as higher education helps patients better understand the nature of the disease, persevere and be motivated to comply with long-term, regular treatment methods and come for follow-up visits on time [122]. Thereby, it can be predicted that the rate of patients who will stop treatment or switch to HU treatment will be even higher. This may not only increase the burden of disease but also increase the economic burden on families and society, when patients may progress to a more severe stage with higher treatment costs.
Compared to the study by author Luu Thi Phuong Anh [2] conducted in 2019 estimating the cost of treating BCMDT patients using TKI, the estimated cost of the project is about 3 times lower. This is explained by the change in the price of IM and NL drugs after the GIPAP/VPAP Drug Aid Program Summary Conference and the implementation of solutions after the aid program ended in February 2020 (IM drug price decreased from 404,250 VND to 116,000 VND, a decrease of 3.5 times and NL drug price decreased from 707,435 VND to 241,000 VND, a decrease of 2.9 times) [10].
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Although the cost of IM and NL drugs has decreased significantly and is covered by the Health Insurance Fund at 80% instead of 50% according to Circular No. 01/2020/TT-BYT [6], the cost of treatment is still a major economic burden not only for BCMDT patients who do not participate in health insurance but also for patients with health insurance with low health insurance payment rates. In the hypothetical case of BCMDT patients with a health insurance payment rate of 80%, the total cost of one treatment for BCMDT patients using TKI is about 3.5 - 5.9 million VND. This cost is higher than the average monthly income per capita in 2018 (VND 3.8 million or VND 3.9 million after converting to 2019 based on the CPI index) and many times higher than the average medical expenditure per person with outpatient treatment in 1 month in 2016 (VND 98,242 or VND 108,238 after converting to 2019 based on the CPI index) [41].
High drug costs and low health insurance payments for drugs are major barriers that make patients reluctant to participate in treatment, reduce treatment compliance, leading to serious consequences, wasting drugs and being costly for patients.
including drug resistance, disease progression, need for stem cell transplantation and possible death [67], [69], [71]. Typically, in studies conducted in the US such as by Dusetzina SB et al. (2014) [69], it was shown that patients having to pay a high co-payment limit reduced their ability to comply with treatment by 42% compared to patients with a low co-payment limit; or in the study by Doshi JA et al. (2016)
[67] showed that the group of patients not eligible for payment support had to pay a co-payment of 25% to 33% for TKI drugs in the early stages, which made patients reluctant to participate in treatment, thereby reducing the ability to receive benefits from insurance in later stages and reducing the effectiveness of treatment. Therefore, in developing countries like Vietnam, diversifying payment methods besides payment from health insurance sources or increasing health insurance payments for patients, maintaining drug aid programs or negotiating drug price reductions is necessary and humane. In addition, there is strong evidence that national health insurance supplemented by private health insurance is successful in offsetting large payments for patients [51].
Based on the results of the systematic review, 2/10 similar studies in the world estimate indirect and direct costs outside of medical care, which in some cases can account for a high proportion, causing a large economic burden for patients. The results of the analysis of the characteristics of chronic stage BCMDT patients using TKI of the topic show that over 80% of patients live in other provinces and cities for treatment and up to 20% of patients have relatives accompanying them when they return for a check-up, so estimating these costs in the topic is practical and important, helping managers have a clearer and more comprehensive view of the economic burden not only of treatment but also of other costs of patients and their families. Compared with studies analyzing the cost of treating chronic CKD with estimates of indirect and direct non-medical costs, the estimated results of the study were about 2 times lower than the study in Thailand [98] and much lower than the study conducted in Switzerland [ 77]. This difference can be explained by differences in the estimated components and differences in unit prices and income levels; specifically, the study in Thailand included estimates of indirect costs due to lost productivity due to CKD.
but did not estimate the indirect costs of companions and workers , the study in Switzerland estimated the public consumption cost and Switzerland has a per capita income 10.5 times higher than Vietnam. This shows that cost estimation needs to be conducted separately in each country because it truly reflects the economic burden of disease in each country. Analysis of cost components shows that, if considering the total cost of treatment for patients, direct and indirect non-medical costs account for a very small proportion (about 5%), however, if viewed from another perspective, when not considering the cost of TKI drugs in the total cost of treatment, direct and indirect non-medical costs account for up to 35-40% of the total cost of treatment. These costs are mainly travel and food costs when waiting for the patient's results and will be a major barrier when it can exceed the affordability of many people because most patients are from other provinces and have low incomes. Therefore, developing programs to support non-medical costs or building a policy to allocate drugs to specialized or provincial general hospitals for outpatients when they have reached a stable disease state to reduce travel costs and waiting time is necessary for patients to be able to maintain treatment and achieve treatment success [117].
Side effects of TKIs are a matter of concern. The most common side effects include hematological toxicities (thrombocytopenia and neutropenia) and non-hematological toxicities (edema, weight gain, musculoskeletal pain, and gastrointestinal disorders) [19], [23]. Each patient is affected by the side effects of TKIs to varying degrees, most of which are tolerable, and nearly 95% of side effects occur in the first year and increase by no more than 5% in the second year [23], [127]. Although the study did not estimate the cost of treatment for each type of side effect in the total cost of treatment, according to clinical experts and previous studies, these costs account for a very small proportion of the total cost of treatment and therefore do not affect the study's conclusions [101].
In addition to the cost of treating chronic stage patients, the cost of bone marrow transplantation and the cost of treating acute leukemia are fully and specifically estimated in the topic. Although
The estimated results of the thesis do not distinguish between the cost of bone marrow transplantation due to the cause of CKD or the cost of treating advanced CKD, nor do they consider the impact of comorbidities on the total cost of treatment because: (1) the difficulties in collecting data when the number of patients undergoing bone marrow transplantation is very small due to the high cost of bone marrow transplantation and therefore the rate of CKD patients undergoing bone marrow transplantation is extremely low; this is similar to CKD patients in the chronic stage and when the patient has progressed, the patient's life expectancy is very short; (2) according to the opinion of clinical experts, bone marrow transplantation or treatment for advanced CKD patients is performed similarly to that of other disease groups, the only difference being that the treatment effectiveness of the group of patients transitioning from CKD is lower than that of the remaining group of patients. Therefore, the estimation of the cost of bone marrow transplantation treatment for all disease groups and the estimation of the cost of treatment for acute leukemia instead of the acute leukemia patients of the topic will not have a big difference; on the other hand, the implementation of one-way sensitivity analysis and probabilistic sensitivity analysis performed in the following sections has strengthened the reliability of the study results. Overall, this is the first pharmacoeconomic study to evaluate the cost of treatment in these two patient groups and this can be a reference for other economic evaluation studies using models including bone marrow transplantation or acute leukemia disease status.
Although the cost of medical services is taken from the list of outpatient and inpatient medical examination and treatment costs of each patient, these costs only include 5/7 components of a full cost, the remaining 2 components (house depreciation costs and training - scientific research costs) will be calculated correctly and fully according to the roadmap [16], [33]. Thus, the treatment cost results in the thesis are reflecting lower than the actual cost, or it can be said that the cost-effectiveness analysis results of the topic are lower than the reality. However, for chronic patients using TKI, the cost of treatment drugs accounts for up to 95% of the total direct medical costs, so the results will not be affected too much even when the price of medical services changes. In addition, by one-way sensitivity analysis, it is again shown that even when
The ICER index is not much affected by the change in health care costs within the range of value fluctuations.
Although CML is not a common disease and the number of patients undergoing bone marrow transplantation and acute leukemia patients receiving chemotherapy is limited, the sample size of the study is still one of the strengths and demonstrates the scale of the topic that needs to be discussed, with a total of 440 chronic phase CML patients using TKI drugs, 96 bone marrow transplant patients and 145 acute leukemia patients collected at two leading hospitals in the North and the South in the treatment of blood diseases and bone marrow transplantation with a large number of patients. On the other hand, the random sampling method in these two hospitals shows that the research results of the topic are highly representative of the patient population studied in Vietnam. Although there is a difference in the study sample size between disease status groups and this may cause confusion or disagreement for clinical researchers when the sample size in RCT studies or intervention studies requires uniformity between disease groups. However, the specificity of pharmaceutical economic research is to build simulation models of different disease states and the disease states in the model are separate; therefore, depending on the epidemiological characteristics of that disease state, there will be a corresponding sample size.
Another strength and detail of the study is the analysis of costs from both the payer and societal perspectives. Thus, the results of the study not only provide a comprehensive and comprehensive view of the economic burden of the disease but also provide evidence and data for payers and policy makers in developing and promulgating treatment policies and payment policies related to drugs.
4.1.2.4. Effectiveness of each disease state
For cancer therapies, QALYs are an important measure of effectiveness because they not only measure the life expectancy of patients but also the QoL of those years. As more patients are treated with TKIs, meaning that their lives are expected to be longer, the quality of life gained from those years will play a role.
central in evaluating the effectiveness of therapies [124]. Therefore, the QALY index chosen as the output result for the topic is an advantage, because this index helps the study determine whether similar results are repeated, reducing bias and error for the study [127].
Among the utility indices in disease states, a total of 3/9 utility indices were recorded from Vietnamese people, of which 2 important utility indices, the utility indices of BCMDT patients using IM and NL, were collected and analyzed in the study.
CLCS of BCMDT patients was previously performed by author Tran TA
[136] in 2016. Despite the similarity in research objectives, the study by author Tran TA used the specialized EORTC-QLQ C30 questionnaire instead of the EQ-5D-5L toolkit as in the topic. Comparing the two toolkits, the EQ-5D-5L toolkit has more advantages in cost-effectiveness analysis studies because: (1) the questionnaire has a Vietnamese version that has been evaluated for accuracy and reliability, the number of questions is short (5 questions) and quite simple, so it is convenient for interviews;
(2) The collected questionnaire can be easily converted to a utility index based on the CLCS conversion scale, so it is often used in pharmacoeconomic analysis studies while the EORTC-QLQ C30 tool has not been able to do this [106]; (3) the CLCS conversion scale for the current EQ-5D-5L questionnaire was developed specifically for Vietnamese people in 2017-2018 under the supervision of international experts [103]; therefore, it is a great advantage in research when reflecting the actual treatment effectiveness at the local level as well as being comparable with other studies in the world; especially when CCMDT is a genetic disease and the treatment effectiveness of TKI drugs can vary depending on the characteristics of different populations. In addition, the study by author Tran TA [136] did not classify CLCS into groups (new or drug-resistant patients; IM, NL or chemotherapy). In addition, the results of the survey conducted at 2 leading hospitals in treatment with a sample size of up to 460 patients compared to only surveying at one hospital as in the study by author Tran TA [136], thereby helping to fully reflect and represent the CLCS of BCMDT patients.
The results of the estimated QOL index of patients with CKD using TKI of the project compared with the results of other studies in the world are similar. Specifically, the study evaluating QOL using the EQ-5D tool on CKD patients using IM by author O'Brien et al. gave QOL scores after 3, 6, 9, 12 months of treatment ranging from 0.840-0.855 [115] or by author Teng-Chou Chen et al. gave an average QOL score of 0.80 ± 0.08 [61]. Compared with the results of studies on other chronic diseases, the QOL assessment of the project gave similar results; specifically, the QOL score of acquired immunodeficiency disease ranged from 0.78-0.95, chronic obstructive pulmonary disease ranged from 0.43-0.62 and breast cancer was 0.72-0.81 [65].
Comparing the CLCS of patients using NL and IM showed that patients using NL had lower CLCS than those in the IM group (0.875 vs. 0.889), however the difference between the two groups was not statistically significant (p=0.2837) even when NL was only used after the patient was resistant or intolerant to IM. This suggests that early use of NL will bring great benefits to patients [128]. However, this needs to be verified clinically.
The lack of a step 1 NL utility index for BCMDT patients and the assumption that this utility index is equal to that of patients using IM drugs step 1 is a major drawback of the study because this may lead to a lower total effectiveness over the entire life of the patient, or in other words, may lead to a higher ICER index than in reality and this may cause bias in the study conclusions. Although this assumption is based on studies evaluating CLCS in the world as well as similar studies when analyzing the cost-effectiveness of NL in step 1 treatment of BCMDT, the development of treatment guidelines allowing the use of NL drugs in step 1 treatment for BCMDT patients is necessary to help update the results of the topic, open up many future research directions and provide many treatment options.
For the utility indices in the remaining disease states, using a systematic review method based on the Cochrane guidelines and newly conducted in March 2020 with the priority criteria of selecting the most recently updated systematic reviews or studies conducted in Asia with large sample sizes and multinational scale, the utility indices in the topic were selected from a total of 1,248 articles.