Health Decision

A cross-sectional study of national drug procurement policy knowledge, attitudes, and practices among medical personnel in Yunnan Province

Fri, 12 Jul 2024 00:00:00 +0800

Objective: This study assesses the knowledge, attitudes, and practices (KAP) concerning the National Drug Procurement

Policy among medical personnel in Yunnan Province, focusing on their understanding, acceptance, and application of the policy.

Methods: A comprehensive survey involving 919 medical professionals across diverse healthcare settings in Yunnan was

conducted using a structured, self-administered questionnaire. It encompassed demographic information, knowledge of the

drug procurement policy, attitudes towards its implementation, and related practices. Analytical methods included descriptive

statistics, ANOVA, regression analysis, principal component analysis (PCA), and KMeans clustering to discern patterns and key

influencers of policy perception and application.

Results: The survey demonstrated a significant gender imbalance (79.54% female) and a younger and middleaged majority

(35.58% aged 18-30, 54.84% aged 31-50). Most participants held higher educational qualifications, with 62.13% possessing a

Bachelor’s degree. Professional qualifications were found to significantly impact policy understanding. Variations in perceptions

of the policy’s implementation were significant across different professional roles, as indicated by ANOVA. Correlation analysis

revealed substantial links between policy understanding and aspects like ‘Agreement on Drug Consistency’ (r = 0.62) and

‘Reducing Medication Burden’ (r = 0.45). Regression analysis identified ‘Technical Qualification’ as a key determinant of policy

comprehension. Factor analysis unveiled critical dimensions influencing perceptions, including Policy Understanding & Education,

Operational Impact, Economic Implications, Drug Selection & Usage, and Industry Dynamics. PCA and KMeans clustering

exposed a wide spectrum of understanding and opinions among the medical staff.

Conclusion: The findings highlight notable gaps in understanding and implementing the National Drug Procurement Policy

among Yunnan’s medical personnel. This underlines the necessity for targeted educational and communication strategies,

advocating policy refinement to address the diverse needs of the healthcare workforce, thereby enhancing policy efficacy and

healthcare outcomes.

Key words: drug procurement policy, medical personnel, Yunnan province, healthcare policy, professional qualifications,

prescription practices, healthcare management, policy implementation

Cost-effectiveness analysis of mirogabalin in treatment of diabetic peripheral neuropathic pain and post-herpetic neuralgia in China

Yao Wu, Libo Tao, Chang Liu, Fangxu Wang, Shuang Sun — Fri, 12 Jul 2024 00:00:00 +0800

Objective: To evaluate the cost-effectiveness of mirogabalin in treatment of Chinese patients with DPNP or PHN, and to discuss

its reasonable pricing strategy.

Methods: A cost-effectiveness analysis was performed by constructing a three-state Markov model (mild, moderate, and

severe pain) to compare mirogabalin with placebo and pregabalin for DPNP or PHN patients from Chinese healthcare system

perspective. The efficacy parameters were derived from randomized clinical trials (RCTs) comparing mirogabalin with placebo in

the treatment of PHN or DPNP, as well as from network meta-analyses comparing mirogabalin/pregabalin with placebo RCTs.

The health utility values were obtained from published literatures. Cost parameters contained cost of pregabalin, supportive

care, and other medical resource utilization, were sourced from both published literature and public price database. Sensitivity

analysis was conducted on key parameters to verify the stability of the results.

Results: With one-year time horizon, for PHN, the incremental quality-adjusted life years (ΔQALYs) of mirogabalin compared with

placebo and pregabalin were 0.048 and 0.025 respectively; For DPNP, the ΔQALYs of mirogabalin compared with placebo and

pregabalin were 0.021 and 0.017 respectively. Using the national volume-based procurement price of pregabalin as a reference,

with 1 and 3 times the per capita GDP in China (￥89,358, 2023) as thresholds, the daily cost of mirogabalin was found to be

cost-effectiveness when it was within 1.64 times (DPNP) or 2.07 times (PHN), 2.47 times (DPNP) or 3.46 times (PHN) of the

daily cost of pregabalin. One-way sensitivity showed that, utility values of mild and moderate pain, costs of mirogabalin had the

biggest influence on incremental cost-effectiveness ratio.

Conclusion: As a novel calcium channel modulator, the cost-effectiveness of mirogabalin is greatly influenced by the reference

drug. When pricing in China, it is essential to select appropriate comparator drugs and their prices as references.

Key words: cost-effectiveness, mirogabalin, diabetic peripheral neuropathic pain, postherpetic neuralgia

Long-term cost-effectiveness analysis of onceweekly Semaglutide versus Dulaglutide for the treatment of type 2 diabetes after the renewal of the national drug reimbursement list in China

Yang Shen, Xianwen Chen, Hao Zhang, Hao Hu — Fri, 12 Jul 2024 00:00:00 +0800

Objective: Once-weekly semaglutide was considered a dominant therapy for type 2 diabetes versus dulaglutide in China in a

previous study. However, after the renewal of the national drug reimbursement list in the last two years, the prices of both drugs

were changed. This study aims to reassess the long-term cost-effectiveness of once-weekly semaglutide versus dulaglutide

for the treatment of type 2 diabetes uncontrolled on metformin in China under new prices.

Methods: Long-term health and economic outcomes of once-weekly semaglutide 0.5 mg, 1.0 mg, and dulaglutide 1.5 mg

were assessed over 40 years, using the Institute of Health Economics Diabetes Cohort Model from a Chinese healthcare

system perspective, discounted at 5% annually. Baseline cohort characteristics and treatment effects were derived from the

head-to-head clinical trial SUSTAIN 7. Costs included direct medical costs related to anti-hyperglycemic and complications

treatment. National median bidding prices of drugs in January 2024 were used. The robustness of the results was assessed

through one-way and probabilistic sensitivity analyses.

Results: Once-weekly semaglutide 0.5 mg and 1.0 mg were estimated to improve discounted life expectancy by 0.04 and 0.09

years, and discounted quality-adjusted life expectancy by 0.08 and 0.19 quality-adjusted life years (QALYs) versus dulaglutide

1.5 mg, respectively. At the new price, total costs were estimated to be 5476 Chinese yuan (CNY) lower with once-weekly

semaglutide 0.5 mg versus dulaglutide 1.5 mg, among which CNY 4117 reduction came from avoidance of macrovascular and

microvascular complications. Total costs were projected to be CNY 6711 lower with semaglutide 1.0 mg versus dulaglutide 1.5

The higher drug cost of semaglutide 1.0 mg was fully offset by the CNY 10711 reduction in the cost of treating macrovascular

and microvascular complications. Sensitivity analyses verified the robustness of the results.

Conclusion: Once-weekly semaglutide was still considered dominant (more effective and less costly) versus dulaglutide for

patients with type 2 diabetes in China.

Key words: China, cost-effectiveness, diabetes, Dulaglutide, Semaglutide

Possible factors influencing the development of the number of pharmacists in China: A time series VAR model analysis from the perspective of supply and demand theory

Fri, 12 Jul 2024 00:00:00 +0800

Objective: The supply and demand theory is used as a perspective to analyze the impact and prediction of factors such as

the number of consultations in medical institutions on the number of hospital pharmacists in China.

Methods: A total of 21 years of time-series data on the number of pharmacists, doctors and nurses in China’s hospitals, the

number of consultations, the per capita health cost and the per capita GDP were collected from 2002 to 2022, and a vector

auto-regressive model influencing the number of pharmacists in China was established to analyze the dynamic process of the

influence of the number of consultations, the per capita health cost and the per capita GDP on the number of pharmacists and

the direction of the influence in China.

Results: There is a unidirectional Granger causality and long-term cointegration relationship between the number of consultations

and the number of pharmacists, and the number of consultations will have a greater impact on the number of pharmacists in the

long run and will be more stable in the later period; there is also a unidirectional Granger causality and long-term cointegration

relationship between the per capita health cost and the number of pharmacists, and the per capita health cost will have a greater

impact on the number of pharmacists in the short run.

Conclusion: The rise in the number of pharmacists will occur along with increases in the number of visits, health costs per

capita, and GDP per capita, with health costs per capita having a greater impact on the number of pharmacists in the short

term, and visits and GDP per capita having a greater impact on the number of pharmacists in the long term and being more

stable in the later years.

Key words: number of pharmacists, vector auto-regressive model, number of consultations, per capita health costs, GDP

per capita

A systematic literature review of quality of life among people living with HIV/AIDS in China

Liya Fan, Zhiliu Tang, Ian Jacob, Yi Chen, Huanyu Li, Yu Zhang — Fri, 12 Jul 2024 00:00:00 +0800

Objective: This study aims to systematically characterize the health-related quality of life (HRQoL) among people living with

HIV/AIDS (PLWHA) in China.

Methods: The systematic literature searching was conducted from database establishment until June 28 2023 (PubMed,

Embase, ISPOR, CNKI, Wanfang, and VIP Database), and health utility values were estimated using mapping algorithms.

Results: 11,290 literatures were screened, of which 290 met the eligibility criteria without PLWHA restriction and covered 18

different scales. Inclusion criterion: (1) Studies investigating Chinese PLWHA, (2) Studies that utilize multi-dimensional HRQoL

scales, (3) Sample size ≥100 participants, and exclusion criterion: (1) Studies not targeting on Chinese PLWHA, (2) Studies utilizing

unspecified or undefined scales, (3) Studies in which the scale scores for different domains were not reported, (4) Non-research

materials (editorials, correspondences, theses, etc., (5) Duplicate studies. Analysis was based on 179 studies with the most

frequently used top 3 scales, including the 36-item short form survey (SF-36) in 64 (22%) studies, World Health Organization

Quality of Life Questionnaire for HIV Brief Version (WHOQOL-HIV-BREF) in 58 (20%) studies, and medical outcomes study HIV

health survey (MOS-HIV) in 57 (20%) studies.

The 179 studies were published between 2007 and 2023. 13 studies started since 2020 after COVID-19 outbreak. 122

were non-interventional studies and 57 were prospective interventional controlled trials. 137 out of 159 studies where gender

information was available reported a higher proportion of male participants. Only 3 studies published before 2020 specified the

treatment regimens, all included in the National Free Antiretroviral Treatment Program (NFATP) and none was targeting integrase

strand transfer inhibitor (INSTI).

Across these 3 scales, non-interventional studies consistently indicated lower HRQoL in PLWHA compared to the general

population, while in prospective trials it indicated HRQoL improvements through interventions like nursing care, management

strategies and highly active antiretroviral therapy.

Median utility values estimated from the MOS-HIV were 0.81 and 0.78, respectively, based on two mapping algorithms.

Subgroup analyses indicated lower utility in AIDS patients compared to HIV-infected individuals, with differences ranging from

0.01 to 0.09 across various studies using different mapping algorithms. Both groups were associated with lower utility than

that in general population in China.

Conclusion: The overall HRQoL among Chinese PLWHA is worse than the that in general population, emphasizing the urgent

need for effective treatment strategies to improve the HRQoL for PLWHA.

Key words: people living with HIV/AIDS, quality of Life, China, systematic literature review

Clinical comprehensive evaluation and drug utilization pattern study of treatment drugs for depression

Fri, 12 Jul 2024 00:00:00 +0800

Objective: Based on comprehensive analysis of real-world drug usage data, representative drugs are selected for clinical

comprehensive value assessment, exploring factors influencing the clinical efficacy of antidepressant medications.

Methods: This study extracted complete medical information of all depressive disorder patients in a hospital in Sichuan

Province. By using the Apriori algorithm to analyze the association rules of drugs in the sorted clinical pathways, Paroxetine

Hydrochloride, Venlafaxine Hydrochloride, and Agomelatine were ultimately selected. These three medications were studied

in terms of safety, efficacy, cost-effectiveness, innovation, suitability, and accessibility using a research method that combines

literature data with real-world data.

Results: Literature research indicated that Agomelatine outperformed the other two medications in terms of efficacy and cost

effectiveness, real-world research results differed. In terms of efficacy, Agomelatine had an effectiveness rate of 21.1%, while

Paroxetine and Venlafaxine had rates of 68.9% and 61.9%, respectively. Regarding cost-effectiveness, Agomelatine presented

an absolute cost-effectiveness disadvantage. In terms of safety, literature and drug instructions have shown that agomelatine

is the safest. In real-world studies, the reported adverse events of Venlafaxine Hydrochloride at SOC level are similar to those

of Paroxetine Hydrochloride. Agomelatine has not been marketed in the United States. Regarding accessibility, according to

the WHO/HAI method, the affordability of all three medications was less than 1, indicating good affordability for all. In terms

of innovation, Agomelatine possesses a more innovative mechanism of action and is currently the only antidepressant on the

market that specifically stimulates melatonin receptors. Regarding suitability, Agomelatine has a longer storage shelf life than

the other two medications. However, there was no significant difference among the three drugs in other aspects.

Conclusion: Through analysis, we found that the current treatment pathway for depressive disorders has not yet formed a

complete system. Each of the three drugs have their advantages in various dimensions.

Key words: clinical comprehensive evaluation, drug utilization pattern, depression, real-world data, literature data

Cost-effectiveness analysis of Sintilimab vs. Docetaxel as the second-line therapy of squamous non-small cell lung cancer in China

Fri, 12 Jul 2024 00:00:00 +0800

Objective: There is a scarcity of sufficient pharmacoeconomic information in China regarding utilizing sintilimab as a second

line therapy for squamous non-small cell lung cancer (NSCLC). This study aimed to estimate sintilimab’s cost-effectiveness

compared to docetaxel for treating squamous NSCLC in China.

Methods: A comparative analysis was conducted using a partitioned survival model to contrast the cost and patients’ quality

adjusted life years (QALYs) associated with sintilimab and docetaxel. Clinical data for long-term survival projection and adverse

event (AE) probabilities were drawn from the phase III ORIENT-3 clinical trial. Utility data and cost were gathered from relevant

literature and local public databases. Sensitivity analyses were executed to ensure our findings’ reliability.

Results: Base case analysis unveiled that sintilimab led to 0.32 more QALY and $2546.34 more cost compared to docetaxel

for squamous NSCLC in China. This translated to an incremental cost-effectiveness ratio (ICER) of $8115.74/QALY gained,

which falls below China’s per capita GDP in 2022. Our findings remained consistent and robust in both one-way deterministic

and probabilistic sensitivity analyses, regardless of the willingness-to-pay setting.

Conclusion: Sintilimab as a second-line therapy for squamous NSCLC patients is cost-effective in the perspective of Chinese

healthcare system.

Key words: squamous non-small cell lung cancer, cost-effectiveness, sintilimab, docetaxel, second-line therapy

Cost-effectiveness analysis of the combination of low-dose nivolumab with triple metronomic chemotherapy for advanced head and neck squamous cell carcinoma in China

Fri, 12 Jul 2024 00:00:00 +0800

Objective: The combination of low-dose nivolumab with triple metronomic chemotherapy (TMC-I) proposes a novel approach,

potentially enhancing patient prognosis while mitigating financial barriers. The purpose of this study was to compare the cost

effectiveness of TMC-I compared to triple metronomic chemotherapy (TMC) in the treatment of advanced head and neck

squamous cell carcinoma (HNSCC) patients in China, the largest developing country.

Methods: A partitioned survival model (PSM) was developed based on a randomized clinical trial from the perspective of Chinese

health care system. Costs and utility were derived from open-access databases and literatures. A 5% annual discount rate was

applied to both costs and outcomes. The primary outcome was incremental cost-effectiveness ratio (ICER). A willingness-to-pay

(WTP) threshold of ￥44,679/QALY based on supply-side and ￥134,037/QALY based on demand-side were set. Sensitivity

analyses including one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analysis were conducted to test

the model stability; subgroup analyses were also included.

Results: TMC-I yielded an additional 0.41 quality-adjusted life years (QALYs) while increasing costs by ￥47,346.98 relative

to TMC, leading an ICER of ￥116,374.22/QALY. Sensitivity analysis showed that utility of progression-free survival (PFS) had

the greatest impact on results. In scenario analysis which the utilities calculated by the time-to-death (TTD) were adopted, the

results showed that the ICER was ￥114,795.25/QALY. In the probabilistic sensitivity analysis, the probabilities that TMC-I was

cost-effective at thresholds of ￥134,037/QALY, ￥44,679/QALY gained were 60.9%, 9.4%, respectively. Subgroup analysis

results indicated TMC-I was dominated vs. TMC for patients with no previous taxane and PD-L1 score > 50.

Conclusion: For Patients with recurrent or newly diagnosed advanced head and neck squamous cell carcinoma, TMC-I is

cost-effective at a WTP thresholds of ￥134,037/QALY and is not cost-effective when the WTP thresholds was ￥44,679/QALY

compared with TMC.

Key words: cost-effectiveness, low-dose nivolumab, triple metronomic chemotherapy, head and neck squamous cell carcinoma

Cost-effectiveness analysis of Trastuzumab Deruxtecan as second-line treatment for HER2- positive advanced breast cancer

Feiyi Xiao, Wudong Xiao, Xue Li, Rui Li, Kun Zhao — Fri, 12 Jul 2024 00:00:00 +0800

Objective: To analyze the cost effectiveness of trastuzumab detrastuzumab (T-Dxd) or trastuzumab emtezumab (T-DM1) in

patients with HER2-positive advanced breast cancer who were previously treated with trastuzumab and taxanes.

Methods: Based on the perspective of China’s health system, a partitioned survival model is constructed to simulate the direct

medical costs, life years and quality-adjusted life years of patients throughout their life cycle. The basic scenario set by the model

is: based on the data of the Asian-Pacific population in the DESTINY-Breast03 (DB03) phase III clinical trial, the experimental

group and the control group were given intravenous infusion of T-Dxd and T-DM1 at a frequency of every 3 weeks respectively

until the patients’ disease progressed. Clinical effect data and health utility values come from individual patient-level data of the

Asia-Pacific population in the DB03 Phase III clinical trial. Direct medical costs include drug costs, follow-up treatment costs,

adverse event treatment costs, medical service costs, examination and testing costs, and hospice care costs.

Results: In the basic scenario, T-Dxd can reduce the risk of disease progression in patients, and compared with T-DM1, patients’

life years and quality-adjusted life years are improved. Calculated based on China’s per capita GDP in 2022 (85,698 yuan),

the incremental cost-effectiveness ratio (ICER) in the case of donated drugs is within the threshold of 2 times per capita GDP.

Conclusion: Trastuzumab Deruxtecan (T-Dxd) can extend the life years and improve the quality of life of patients with indications.

Under the condition of drug donation, the use of T-Dxd has a comparative advantage of cost-effectiveness.

Key words: cost-effectiveness analysis, trastuzumab deruxtecan, breast cancer, antibody-drug conjugate, economy

Current practices of model validation in economic evaluation: A systematic review in neoplasms

Fri, 12 Jul 2024 00:00:00 +0800

Objective: Model validation is crucial for ensuring confidence in economic models, and guidelines emphasize the need for

researchers to validate the pharmacoeconomic models they construct. This systematic review summarizes current practices

and challenges in model validation for neoplasm economic evaluations, providing recommendations for improvement.

Methods: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, we

searched PubMed, MEDLINE, Embase, and ScienceDirect for model-based economic evaluations published between 2021

and 2023. The studies were screened and extracted by two researchers independently. The frequency of each model validation

type was assessed, along with descriptions of specific practices, validation outcomes, and adjustments made based on the

validation results.

Results: Among the final 362 articles, 139 (38%) conducted model validation. External validation was the most commonly used

validation method (47%), calibrating the model and comparing simulated outcomes with real-world evidence. Face validation

(45%) relied on insights from clinical experts and economists. Internal validation (19%) employed tools such as the incremental

mixture importance sampling (IMIS) algorithm and TreeAge. Cross validation (9%) compared data from similar events, while

predictive validation (4%) used long-term follow-up data. Of the 39 studies (28%) that reported validation results, none of them

made any adjustments based on the validation outcomes. The Assessment of the Validation Status of Health-Economic decision

models (AdViSHE), a validation-assessment tool, was utilized in three studies for model validation. Additionally, 10% of economic

evaluations lacked clear validity, while 96% had one to three validity dimensions, and only 4% had four to five dimensions.

Conclusion: Most studies had limited and brief model validation practices without comprehensive descriptions. Researchers

are encouraged to employ multiple validation methods and provide detailed descriptions of their validation practices, results,

and adjustments. Active development and utilization of model evaluation tools should be promoted among scholars.

Key words: model validation, economic evaluation, current practices, systematic review

Disease burden and treatment patterns in adult patients with severe asthma: A real-world study based on claims data from China

Ke Zhang, Xiaoning He, Jing Wu — Fri, 12 Jul 2024 00:00:00 +0800

Objective: To assess the treatment pattern and disease burden in adults with severe asthma in China.

Methods: This retrospective, observational, cohort study with one-year follow-up was conducted using a healthcare claims

database spanning 2018 through 2020, from Tianjin, China. Patients diagnosed with asthma, who had ≥2 prescriptions of

medium-to-high dose of inhaled corticosteroids (ICS) along with additional controllers, were identified to represent severe

asthma population in China. Patients were categorized as controlled, sub-optimally controlled , and uncontrolled by the times

of burst systemic corticosteroids, short-acting β2-agonist (SABA) and hospitalization. Medication use, asthma exacerbations,

and related costs in the follow-up period were also reported.

Results: 2,418 patients with severe asthma were included, with an average age of 56.15 years, male 55.62%. 93.51% of

patients used inhaled corticosteroid/long-acting β2 (ICS/LABA). 10.75% of patients used oral corticosteroids (OCS) (6.13%,

23.83% and 33.96% in controlled, sub-optimally controlled and uncontrolled group, respectively). For patients with OCS,

the average duration of OCS was 82.73 days (53.35, 102.27 and 111.21 days in three groups, respectively). 10.67% of

patients experienced ≥ 1 exacerbation and 3.93% experienced ≥ 2 exacerbations (1.73%,1.68% and 26.89% in three groups,

respectively). The average frequency of annual exacerbations was 0.21 (0.09, 0.26 and 1.31 in three groups, respectively). The

average cost for per exacerbation was ¥1441.83 (¥293.69, ¥550.86 and ¥2366.86 in three groups respectively). The average

asthma-related annual cost was ¥2846.08, of which medication costs exceeded 90% (¥2105.70, ¥3559.35 and ¥8506.83 in

three groups, respectively).

Conclusion: This study shows that ICS/LABA is widely used in patients with severe asthma, and OCS is more used in patients

with uncontrolled asthma. Worse asthma control is associated with more frequent and severe asthma attack and higher medical

cost, which reveals the necessity of effective management and treatment to improve the asthma control.

Key words: severe asthma, control level, treatment pattern, disease burden, real-world study

Establishment of a predictive model for antidepressant efficacy based on machine learning

Fri, 12 Jul 2024 00:00:00 +0800

Objective: Establishing a model can accurately predict the depressive patient’s response to different drugs, thereby providing

personalized treatment plans to improve treatment outcomes.

Methods: Depressive patients who have taken Paroxetine hydrochloride or Venlafaxine hydrochloride or Agomelatine at a

hospital in Sichuan Province are the subjects. By analyzing their medical records, medication histories, and basic information,

an predictive model is constructed to predict the efficacy of antidepressant medications based on eXtreme Gradient Boosting.

Results: For the prediction model of Paroxetine hydrochloride, 52 variables selected by the model were used to construct an

efficient predictive model. In the training set, the model achieved an AUC value of 0.6354 and a K-S value of 0.1944, indicating

good performance in correctly classifying positive and negative samples and distinguishing different predictive probability

thresholds. In the validation set, the AUC was 0.6065, and K-S was 0.1847, confirming the model’s effectiveness on new data.

Compared to actual clinical data, the efficacy of sertraline hydrochloride was approximately 61.9%. The model’s predictions

aligned well with real-world data, reinforcing its reliability and practicality. As for venlafaxine hydrochloride, the model achieved

an AUC of 0.5745 and K-S of 0.149 on the training set, while the validation set showed an AUC of 0.5298 and K-S of 0.0597.

These results suggest that the model’s performance in predicting venlafaxine hydrochloride is mediocre. Comparing with

clinical data, the efficacy of venlafaxine hydrochloride was approximately 68.9%, indicating a discrepancy between the model’s

predictions and actual outcomes, possibly due to the uneven distribution of the training set samples. As for agomelatine, due

to the insufficient number of agomelatine samples collected at the sample hospital (less than 200), an effective predictive model

could not be established.

Conclusion: In clinical practice, doctors can make more informed treatment decisions and achieve personalized antidepressant

therapy with the assistance of this model.

Key words: predictive model, machine learning, antidepressant efficacy, depressive patients, clinical practice

Exploring the application of health economics in clinical practice guidelines: A case study of breast cancer

Fri, 12 Jul 2024 00:00:00 +0800

Objective: Besides safety and efficacy evidence, economic evidence is a crucial consideration in forming recommendations

for clinical practice guidelines (CPGs). However, the extent to which economic evidence is utilized in Chinese CPGs remains

unclear. Our study aims to systematically analyze the application of economic evidence in CPGs, providing insights for future

guideline formulation and updates.

Methods: A systematic search was conducted for relevant breast cancer CPGs published from 2017 to 2023, along with breast

cancer pharmacoeconomic evaluation literature from 2015 to 2017 in China. Using the Appraisal of Guidelines for Research &

Evaluation Instrument II (AGREE II) to select the guideline with the highest quality score and analyze the alignment between the

recommendations in the guideline and the published pharmacoeconomic evaluation results.

Results: 30 breast cancer CPGs and 59 breast cancer pharmacoeconomic evaluation studies were included. The CSCO

guidelines received the highest quality score. Among the included pharmacoeconomic evaluation studies, 25 were mentioned

in the guideline recommendations, but economic evidence from 15 studies did not align with the guideline recommendations.

Conclusion: Currently, economic evidence is seldom considered in published breast cancer CPGs in China. In future guideline

formulation and updates, the involvement of health economists should be prioritized to enhance and guide the recommendations

in the guidelines.

Key words: clinical practice guidelines, health economic, cost-effectiveness, breast cancer

Optimizing covariate selection and results inference in anchored matched adjusted indirect comparison method

Mingjun Rui, Hongchao Li, Yingcheng Wang — Fri, 12 Jul 2024 00:00:00 +0800

Indirect comparison methods become particularly prominent in pharmacoeconomic evaluations. This study delves into the

anchored matched adjusted indirect comparison (MAIC) method, spotlighting the challenges of selecting appropriate covariates

and distinguishing between predictive and prognostic factors. In addition, our research bridges the gap of MAIC results application

inference, enhancing the methodological rigor and applicability of MAIC analyses. Through theoretical exploration and a detailed

case study of toripalimab and pembrolizumab in the neoadjuvant treatment of NSCLC, we demonstrate the significant impact of

covariate selection on the outcomes of pharmacoeconomic evaluations. Analyzing the individual patient data by using statistical

methods alone is insufficient to identify all potential prognostic factors. Instead, a combination of previously published related

research and expert consultations is necessary. The individual patient data network meta-analysis should be employed if the

shared effect modifier assumption is not met to make the MAIC results be inferred for the real-world decision-making population.

Key words: matched adjusted indirect comparison, covariate selection, results inference

Pharmacoeconomic studies on biologic agents for ankylosing spondylitis: A systematic review

Jiaqi Shi, Ziqi Zhao, Wenxin Zhou, Ming Hu — Fri, 12 Jul 2024 00:00:00 +0800

Objective: This study aims to systematically review and assess the quality of published pharmacoeconomic studies on biologic

agents for ankylosing spondylitis conducted both domestically and internationally.

Methods: PubMed, Embase, and the Cochrane Library databases were searched using English keywords such as “ankylosing

spondylitis” and “pharmacoeconomics.” Chinese databases, including China National Knowledge Infrastructure (CNKI) and

Wanfang Database, were searched using Chinese keywords such as “ankylosing spondylitis”, “cost” and “effectiveness”. The

search was conducted from the inception of the databases until February 2023. The quality of the included studies was assessed

using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS 2022) and the Quality of Health Economics

Studies Instrument (QHES). Two researchers extracted data from the included studies, and descriptive analysis was performed

to summarize the characteristics of the included studies.

Results: A total of 24 English-language studies were included in the review. The included studies were published between 2004

and 2020, with 19 studies classified as high-quality and 5 studies as “general quality studies.” Furthermore, 18 studies (75%) were

model-based pharmacoeconomic evaluations, including Markov models, mathematical models, and discrete event simulation

models. In terms of economic evaluations, Tumour necrosis factor (TNF) inhibitors and IL-17 inhibitors were found to be more

cost-effective compared to traditional nonsteroidal anti-inflammatory drug (NSAIDs) treatment. Factors such as the BASDAI 50

response rate and disease-related costs had varying degrees of impact on the incremental cost-effectiveness analysis results.

Conclusion: The overall results indicate that, in the short-term treatment scenario, biologic agents are not cost-effective

compared to traditional treatment options, but in the long-term treatment scenario, biologic agent treatment becomes more

cost-effective. The most cost-effective intervention measures varied among different studies comparing different biologic agents.

Key words: ankylosing spondylitis, biologicals, pharmacoeconomic evaluation, systematic review, TNFis inhibitors, IL-17 inhibitor

The parameter variation of one-way sensitivity analysis in economic evaluation: Global systematic review

Fri, 12 Jul 2024 00:00:00 +0800

Objective: One-way sensitivity analysis (OSA) plays a crucial role in economic evaluations (EEs), yet knowledge about parameter

range and research practices is limited. Understanding these patterns is essential for reliable healthcare decision-making. This

study aims to describe OSA patterns in EEs, providing insights into parameter ranges and good research practices.

Methods: Systematic searches on PubMed, MEDLINE, Embase, and ScienceDirect were conducted for model-based EEs

published in English (2021-2023). Neoplasm-related articles were prioritized due to their abundance. Screening, data extraction,

and quality assessment were performed by two independent reviewers. Study characteristics, OSA methods, and OSA result

diagrams were collected. Study quality was assessed using the Criteria for Health Economic Quality Evaluation (CHEQUE) tool.

Results: Among 7,885 records, 362 articles were extracted. Economic parameters such as price per unit, packaging cost,

and number of healthcare utilization were commonly varied in OSA (94%). Efficacy parameters (84%), utility parameters (79%),

and safety parameters related to adverse event and complication (54%) were also frequently varied. Only 67% of the articles

described parameter variation methods, 73% listed parameter change ranges, and only 34% explicitly stated the rationale

behind parameter variations. A taxonomy of OSA range types was developed, including practices such as literature review,

confidence intervals, practical context, clinical opinion, extreme values, percentage/distance variations, guideline provisions,

author assumptions, unknown ranges/types, uncertain variations, and others. Good practices for OSA were recommended.

Tornado diagrams were the most common OSA result plots (88%), accompanied by table, curve, and line chart. Method quality

scored 13 out of 18 points, while reporting quality scored 12 out of 17 points.

Conclusion: This systematic review revealed unsatisfactory quality in the methods and reporting of OSA in neoplasm EEs.

Recommendations are provided for parameter variation methods, ranges, references, and chart presentation for future OSA

studies.

Key words: parameter variation, one-way sensitivity, systematic review, economic evaluation