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Updated cost-effectiveness analysis of adebrelimab plus chemotherapy for extensive-stage small cell lung cancer in China

Por: Long · Y. · Wang · H. · Xie · X. · Li · J. · Xu · Y. · Zhou · Y.
Objective

The CAPSTONE-1 trial demonstrated that adebrelimab-based immunotherapy yielded a favourable survival benefit compared with chemotherapy for patients with extensive-stage small cell lung cancer (ES-SCLC). This study aims to evaluate the cost-effectiveness of this immunotherapy in the treatment of ES-SCLC from a healthcare system perspective in China.

Design

The TreeAge Pro software was used to establish a three-state partitioned survival model. Survival data came from the CAPSTONE-1 trial (NCT03711305), and only direct medical costs were included. Utility values were obtained from the published literature. Sensitivity analysis was performed to explore the robustness of the model. The cost-effectiveness of immunotherapy was investigated through scenario and exploratory analyses in various settings.

Outcome measures

Total costs, incremental costs, life years, quality-adjusted life-years (QALYs), incremental QALYs and incremental cost-effectiveness ratio (ICER).

Results

The basic analysis revealed that the adebrelimab group achieved a total of 1.1 QALYs at a cost of US$65 385, while the placebo group attained 0.78 QALYs at a cost of US$12 741. ICER was US$163 893/QALY. Sensitivity analysis confirmed that the model was robust. Results from scenario and exploratory analyses indicated that the combination of adebrelimab and chemotherapy did not demonstrate cost-effectiveness in any scenario.

Conclusions

From the perspective of the Chinese healthcare system, adebrelimab in combination with chemotherapy for the treatment of ES-SCLC was not economical compared with chemotherapy.

Cost-effectiveness of behavioural counselling intervention compared with non-intervention for adult patients with metabolic syndrome to prevent cardiovascular diseases and type 2 diabetes in Japan: a microsimulation modelling study

Por: Akune · Y. · Anezaki · H. · Nakao · Y. M. · Goto · R.
Objectives

Nationwide lifestyle intervention—specific health guidance (SHG) in Japan—employs counselling and education to change unhealthy behaviours that contribute to metabolic syndrome, especially obesity or abdominal obesity. We aimed to perform a model-based economic evaluation of SHG in a low participation rate setting.

Design

A hypothetical population, comprised 50 000 Japanese aged 40 years who met the criteria of the SHG, used a microsimulation using the Markov model to evaluate SHG’s cost-effectiveness compared with non-SHG. This hypothetical population was simulated over a 35-year time horizon.

Setting

SHG is conducted annually by all Japanese insurers.

Outcome measures

Model parameters, such as costs and health outcomes (including quality-adjusted life-years, QALYs), were based on existing literature. Incremental cost-effectiveness ratios were estimated from the healthcare payer’s perspective. Deterministic and probabilistic sensitivity analyses (PSA) were conducted to evaluate the uncertainty around the model input parameters.

Results

The simulation revealed that the total costs per person in the SHG group decreased by JPY53 014 (US$480) compared with that in the non-SHG group, and the QALYs increased by 0.044, wherein SHG was considered the dominant strategy despite the low participation rates. PSA indicated that the credibility intervals (2.5th–97.5th percentile) of the incremental costs and the incremental QALYs with the SHG group compared with the non-SHG group were –JPY687 376 to JPY85 197 (–US$6226 to US$772) and –0.009 to 0.350 QALYs, respectively. Each scenario analysis indicated that programmes for improving both blood pressure and blood glucose levels among other risk factors for metabolic syndrome are essential for improving cost-effectiveness.

Conclusions

This study suggests that even small effects of counselling and education on behavioural modification may lead to the prevention of acute life-threatening events and chronic diseases, in addition to the reduction of medication resulting from metabolic syndrome, which results in cost savings.

Regional differences in health resource allocation: a longitudinal study in the Chengdu-Chongqing economic circle, China

Por: Zhou · M.
Objective

To analyse regional differences in health resource allocation in the Chengdu-Chongqing economic circle.

Design

A longitudinal analysis that collected data on health resource allocation from 2017 to 2021.

Setting

The number of beds, health technicians, licensed (assistant) physicians, registered nurses and financial allocations per 1000 population in the 42 regions of Chengdu-Chongqing economic circle were used for the analysis.

Methods

The entropy weight technique for order preference by similarity to an ideal solution (TOPSIS) method and the rank sum ratio (RSR) method were used to evaluate the health resource allocation.

Results

The number of licensed (assistant) physicians per 1000 population in the Chengdu-Chongqing economic circle (3.01) was lower than the average in China (3.04) in 2021. According to the entropy weight–TOPSIS method, Yuzhong in Chongqing had the largest C-value and the highest ranking. Jiangbei in Chongqing and Chengdu and Ya’an in Sichuan Province had higher C-values and were ranked in the top 10. Jiangjin, Hechuan, Tongnan and Zhongxian in Chongqing and Guang’an in Sichuan Province had lower C-values and were all ranked after the 30th place. According to the RSR method, the 42 regions were divided into three grades of good, medium and poor. The health resource allocations of Yuzhong, Jiangbei, Nanchuan, Jiulongpo and Shapingba in Chongqing and Chengdu and Ya’an in Sichuan Province were of good grade, those of Tongnan, Jiangjin, Yubei and Dazu in Chongqing and Guang’an and Dazhou in Sichuan Province were of poor grade, and the rest of the regions were of medium grade.

Conclusion

The regional differences in health resource allocation in the Chengdu-Chongqing economic circle were more obvious, the health resource allocation in Chongqing was more polarised and the health resource allocation in Sichuan Province was more balanced, but the advantaged regions were not prominent enough.

Cross-sectional analysis of use of real-world data in single technology appraisals of oncological medicine by the National Institute for Health and Care Excellence in 2011-2021

Por: Kang · J. · Cairns · J.
Objectives

This study aims to identify how real-world data (RWD) have been used in single technology appraisals (STAs) of cancer drugs by the National Institute for Health and Care Excellence (NICE).

Design

Cross-sectional study of NICE technology appraisals of cancer drugs for which guidance was issued between January 2011 and December 2021 (n=229). The appraisals were reviewed following a published protocol to extract the data about the use of RWD. The use of RWD was analysed by reviewing the specific ways in which RWD were used and by identifying different patterns of use.

Primary outcome measure

The number of appraisals where RWD are used in the economic modelling.

Results

Most appraisals used RWD in their economic models. The parametric use of RWD was commonly made in the economic models (76% of the included appraisals), whereas non-parametric use was less common (41%). Despite widespread use of RWD, there was no dominant pattern of use. Three sources of RWD (registries, administrative data, chart reviews) were found across the three important parts of the economic model (choice of comparators, overall survival and volume of treatment).

Conclusions

NICE has had a long-standing interest in the use of RWD in STAs. A systematic review of oncology appraisals suggests that RWD have been widely used in diverse parts of the economic models. Between 2011 and 2021, parametric use was more commonly found in economic models than non-parametric use. Nonetheless, there was no clear pattern in the way these data were used. As each appraisal involves a different decision problem and the ability of RWD to provide the information required for the economic modelling varies, appraisals will continue to differ with respect to their use of RWD.

Cost-effectiveness of the top 100 drugs by public spending in Canada, 2015-2021: a repeated cross-sectional study

Por: Gaudette · E. · Rizzardo · S. · Zhang · Y. · Pothier · K. R. · Tadrous · M.
Objectives

To assess the distribution and spending by cost-effectiveness category among those drugs with the highest public spending levels in Canada.

Design

Repeated cross-sectional study.

Setting

The Canadian provinces of Manitoba, Ontario, New Brunswick, Nova Scotia, Prince Edward Island and Newfoundland.

Main outcomes and measures

Cost-effectiveness assessments by the Canadian Agency for Drugs and Technologies in Health (CADTH) for top-100 brand-name outpatient drugs by gross public plan spending in any year between 2015 and 2021 in Canada Institute for Health Information’s National Prescription Drug Utilization Information System data. Gross public plan spending by cost-effectiveness category.

Results

From 2015 to 2021, 152 brand-name drugs occupied a top-100 rank and were included in the analysis. Of those, 117 had been assessed by CADTH. During the 7-year period, there was an increase in both top-100 drugs with cost-effective (from 18 to 24) and cost-ineffective (from 29 to 41) assessments, while drugs not assessed or with an unclear assessment declined (from 31 to 19 and from 22 to 16, respectively). As a share of spending on top-100 drugs with an assessment, spending on cost-effective drugs was mostly stable at 40%–46% from 2015 to 2021, while spending on cost-ineffective drugs increased from 30% to 45%.

Conclusion

A large and growing share of public drug spending has been allocated to cost-ineffective drugs in Canada. Dedicating large budgets to such treatments prevents spending with greater health impact elsewhere in the healthcare system and could restrain the capacity to pay for groundbreaking pharmaceutical innovation in the future.

Estimating the economic burden of stroke in China: a cost-of-illness study

Por: Ma · J. · Tian · Z. · Chai · P. · Wan · Q. · Zhai · T. · Guo · F. · Li · Y.
Objectives

Stroke imposes a heavy economic burden and loss of productivity on individuals and society. This study assessed a range of crucial factors, including direct costs and indirect costs, to gauge the economic implications of stroke in China. These outcomes were evaluated with specific reference to the year 2018, using the Chinese yuan (¥) as the unit of measurement and providing the corresponding purchasing power parity dollar ($PPP) currency value.

Methods

A cost-of-illness methodology was used to ascertain the economic implications of stroke in 2018. Within the constraints of this approach, economic costs were defined as ‘direct costs’ or ‘indirect costs’. We estimated direct costs from sample data, the National Health Service Survey and the National Health Account and Health Statistical Yearbook. A human capital method was used to conservatively estimate indirect costs.

Results

In 2018, of the economic burden of stroke in China, the direct costs were ¥247.8 billion ($PPP 58.6 billion) and indirect costs were ¥704.4 billion ($PPP 166.5 billion). The curative care expenditure for stroke was ¥193.1 billion ($PPP 45.7 billion), consuming nearly 5.5% of curative expenditure. The cost of stroke treatment relied heavily on public financing, with 58% from social health insurance and 14% from government sources.

Conclusions

A significant economic burden is imposed by stroke on China’s economy, and there is a risk of underestimating this burden if indirect costs are not comprehensively considered. The importance of implementing effective preventive measures and screening strategies for stroke, with a particular focus on high-risk populations, is underscored by this study’s findings. Such investments in public health have the potential to yield substantial benefits.

A socially interdependent choice framework for social influences in healthcare decision-making: a study protocol

Por: Nouwens · S. P. H. · Veldwijk · J. · Pilli · L. · Swait · J. D. · Coast · J. · de Bekker-Grob · E. W.
Objectives

Current choice models in healthcare (and beyond) can provide suboptimal predictions of healthcare users’ decisions. One reason for such inaccuracy is that standard microeconomic theory assumes that decisions of healthcare users are made in a social vacuum. Healthcare choices, however, can in fact be (entirely) socially determined. To achieve more accurate choice predictions within healthcare and therefore better policy decisions, the social influences that affect healthcare user decision-making need to be identified and explicitly integrated into choice models. The purpose of this study is to develop a socially interdependent choice framework of healthcare user decision-making.

Design

A mixed-methods approach will be used. A systematic literature review will be conducted that identifies the social influences on healthcare user decision-making. Based on the outcomes of a systematic literature review, an interview guide will be developed that assesses which, and how, social influences affect healthcare user decision-making in four different medical fields. This guide will be used during two exploratory focus groups to assess the engagement of participants and clarity of questions and probes. The refined interview guide will be used to conduct the semistructured interviews with healthcare professionals and users. These interviews will explore in detail which, and how, social influences affect healthcare user decision-making. Focus group and interview transcripts will be analysed iteratively using a constant comparative approach based on a mix of inductive and deductive coding. Based on the outcomes, a social influence independent choice framework for healthcare user decision-making will be drafted. Finally, the Delphi technique will be employed to achieve consensus about the final version of this choice framework.

Ethics and dissemination

This study was approved by the Erasmus School of Health Policy and Management Research Ethics Review Committee (ESHPM, Rotterdam, The Netherlands; reference ETH2122-0666).

Modifiable prognostic factors of high societal costs among people on sick leave due to musculoskeletal disorders: findings from an occupational cohort study

Por: Killingmo · R. M. · Tveter · A. T. · Pripp · A. H. · Tingulstad · A. · Maas · E. · Rysstad · T. · Grotle · M.
Objectives

The objective was to identify modifiable prognostic factors of high societal costs among people on sick leave due to musculoskeletal disorders, and to identify modifiable prognostic factors of high costs related to separately healthcare utilisation and productivity loss.

Design

A prospective cohort study with a 1-year follow-up.

Participants and setting

A total of 549 participants (aged 18–67 years) on sick leave (≥ 4 weeks) due to musculoskeletal disorders in Norway were included.

Outcome measures and method

The primary outcome was societal costs aggregated for 1 year of follow-up and dichotomised as high or low, defined by the top 25th percentile. Secondary outcomes were high costs related to separately healthcare utilisation and productivity loss aggregated for 1 year of follow-up. Healthcare utilisation was collected from public records and included primary, secondary and tertiary healthcare use. Productivity loss was collected from public records and included absenteeism, work assessment allowance and disability pension. Nine modifiable prognostic factors were selected based on previous literature. Univariable and multivariable binary logistic regression analyses were performed to identify associations (crude and adjusted for selected covariates) between each modifiable prognostic factor and having high costs.

Results

Adjusted for selected covariates, six modifiable prognostic factors associated with high societal costs were identified: pain severity, disability, self-perceived health, sleep quality, return to work expectation and long-lasting disorder expectation. Depressive symptoms, work satisfaction and health literacy showed no prognostic value. More or less similar results were observed when high costs were related to separately healthcare utilisation and productivity loss.

Conclusion

Factors identified in this study are potential target areas for interventions which could reduce high societal costs among people on sick leave due to musculoskeletal disorders. However, future research aimed at replicating these findings is warranted.

Trial registration number

NCT04196634, 12 December 2019.

Economic burden of adverse perinatal outcomes from births to age 5 years in high-income settings: a protocol for a systematic review

Por: Haile · T. G. · Pereira · G. · Norman · R. · Tessema · G. A.
Background

Adverse perinatal outcomes such as preterm, small for gestational age, low birth weight, congenital anomalies, stillbirth and neonatal death have devastating impacts on individuals, families and societies, with significant lifelong health implications. Despite extensive knowledge of the significant and lifelong health implications of adverse perinatal outcomes, information on the economic burden is limited. Estimating this burden will be crucial for designing cost-effective interventions to reduce perinatal morbidity and mortality. Thus, we will quantify the economic burden of adverse perinatal outcomes from births to age 5 years in high-income countries.

Methods and analysis

A systematic review of all primary studies published in English in peer-reviewed journals on the economic burden for at least one of the adverse perinatal outcomes in high-income countries from 2010 will be searched in databases—MEDLINE (Ovid), EconLit, CINAHL (EBSCO), Embase (Ovid) and Global Health (Ovid). We will also search using Google Scholar and snowballing of the references list of included articles. The search terms will include three main concepts—costs, adverse perinatal outcome(s) and settings. We will use the Consolidated Health Economics Evaluation Reporting Standards 2022 and 17 criteria from the critical appraisal of cost-of-illness studies to assess the quality of each study. We will report the findings based on the Preferred Reporting Items for Systematic Reviews and Meta-analyses 2020 statement. Costs will be converted into a common currency (US dollar), and we will estimate the pooled cost and subgroup analysis will be done. The reference lists of included papers will be reviewed.

Ethics and dissemination

This systematic review will not involve human participants and requires no ethical approval. The results of this review will be published in a peer-reviewed journal.

PROSPERO registration number

CRD42023400215.

Use of removable support boot versus cast for early mobilisation after ankle fracture surgery: cost-effectiveness analysis and qualitative findings of the Ankle Recovery Trial (ART)

Por: Baji · P. · Barbosa · E. C. · Heaslip · V. · Sangar · B. · Tbaily · L. · Martin · R. · Docherty · S. · Allen · H. · Hayward · C. · Marques · E. M. R.
Objectives

To estimate the cost-effectiveness of using a removable boot versus a cast following ankle fracture from the National Health Service and Personal Social Services (NHS+PSS) payer and societal perspectives and explore the impact of both treatments on participants’ activities of daily living.

Design

Cost-effectiveness analyses and qualitative interviews performed alongside a pragmatic multicentre randomised controlled trial.

Setting

Eight UK NHS secondary care trusts.

Participants

243 participants (60.5% female, on average 48.2 years of age (SD 16.4)) with ankle fracture. Qualitative interviews with 16 participants. Interventions removable air boot versus plaster cast 2 weeks after surgery weight bearing as able with group-specific exercises.

Primary and secondary outcome measures

Quality-adjusted life years (QALYs) estimated from the EQ-5D-5L questionnaire, costs and incremental net monetary benefit statistics measured 12 weeks after surgery, for a society willing-to-pay £20 000 per QALY.

Results

Care in the boot group cost, on average, £88 (95% CI £22 to £155) per patient more than in the plaster group from the NHS+PSS perspective. When including all societal costs, the boot saved, on average, £676 per patient (95% CI –£337 to £1689). Although there was no evidence of a QALY difference between the groups (–0.0020 (95% CI –0.0067 to 0.0026)), the qualitative findings suggest participants felt the boot enhanced their quality of life. Patients in the boot felt more independent and empowered to take on family responsibilities and social activities.

Conclusions

While the removable boot is slightly more expensive than plaster cast for the NHS+PSS payer at 12 weeks after surgery, it reduces productivity losses and the need for informal care while empowering patients. Given that differences in QALYs and costs to the NHS are small, the decision to use a boot or plaster following ankle surgery could be left to patients’ and clinicians’ preferences.

Trial registration number

ISRCTN15497399, South Central—Hampshire A Research Ethics Committee (reference 14/SC/1409).

Primary care system-level training and support programme for the secondary prevention of domestic violence and abuse: a cost-effectiveness feasibility model

Por: Cochrane · M. · Szilassy · E. · Coope · C. · Emsley · E. · Johnson · M. · Feder · G. · Barbosa · E. C.
Objectives

This study aimed to evaluate the prospective cost-effectiveness of the Identification and Referral to Improve Safety plus (IRIS+) intervention compared with usual care using feasibility data derived from seven UK general practice sites.

Method

A cost–utility analysis was conducted to assess the potential cost-effectiveness of IRIS+, an enhanced model of the UK’s usual care. IRIS+ assisted primary care staff in identifying, documenting and referring not only women, but also men and children who may have experienced domestic violence/abuse as victims, perpetrators or both. A perpetrator group programme was not part of the intervention per se but was linked to the IRIS+ intervention via a referral pathway and signposting. A Markov model was constructed from a societal perspective to estimate mean incremental costs and quality-adjusted life years (QALYs) of IRIS+ compared with to usual care over a 10-year time horizon.

Results

The IRIS+ intervention saved £92 per patient and produced QALY gains of 0.003. The incremental net monetary benefit was positive (£145) and the IRIS+ intervention was cost-effective in 55% of simulations at a cost-effectiveness threshold of £20 000 per QALY.

Conclusion

The IRIS+ intervention could be cost-effective or even cost saving from a societal perspective in the UK, though there are large uncertainties, reflected in the confidence intervals and simulation results.

Intended changes in smoking behaviour of Dutch young adults after an increase in excise tax: a cross-sectional survey

Por: Visscher · K. · Everaars · B. · Suijkerbuijk · A. W. · Lambooij · M. · de Wit · G. A.
Objectives

Increasing the price of tobacco is one of the most effective measures to reduce the prevalence of smoking. In the Netherlands, the excise tax on tobacco increased by 1.14 in 2020, raising the price of a standard package of cigarettes to 8.00. This study investigates how young adults intend to change their smoking behaviour in the case of hypothetical price increases of a pack of cigarettes, and which background characteristics are associated with intended behaviour change.

Design

A cross-sectional online survey was carried out between September and November 2020. Smokers indicated how they would react to several hypothetical increases in price. Four behavioural options were investigated: smoking less, quitting smoking, switching to another/cheaper product and buying cheaper cigarettes cross-border.

Participants

Data were obtained from 776 Dutch smokers between 15 and 25 years.

Results

At a hypothetical price of 10 per package, most respondents reported an intention to smoke less (67%), followed by switching to another/cheaper product (61%), quitting smoking (49%) and shopping for cigarettes cross-border (47%). Prior quit attempts, agreeing with the increase in excise tax and the intention to quit smoking in the future increased the odds of changing behaviour. Higher self-efficacy decreased the odds of behavioural change.

Conclusion

Many young adults intend to change their smoking behaviour in the event of increased prices. Although intended behaviour can deviate significantly from actual behaviour, an increase in excise tax may result in a significant amount of quit attempts and reduced smoking among young adults.

Analysis of maternal and child health spillover effects in PEPFAR countries

Por: Gaumer · G. · Crown · W. H. · Kates · J. · Luan · Y. · Hariharan · D. · Jordan · M. · Hurley · C. L. · Nandakumar · A.
Objectives

This study examined whether the US President’s Emergency Plan for AIDS Relief (PEPFAR) funding had effects beyond HIV, specifically on several measures of maternal and child health in low-income and middle-income countries (LMICs). The results of previous research on the question of PEPFAR health spillovers have been inconsistent. This study, using a large, multicountry panel data set of 157 LMICs including 90 recipient countries, adds to the literature.

Design

Seven indicators including child and maternal mortality, several child vaccination rates and anaemia among childbearing-age women are important population health indicators. Panel data and difference-in-differences estimators (DID) were used to estimate the impact of the PEPFAR programme from inception in 2004 to 2018 using a comparison group of 67 LMICs. Several different models of baseline (2004) covariates were used to help balance the comparison and treatment groups. Staggered DID was used to estimate impacts since all countries did not start receiving aid at PEPFAR’s inception.

Setting

All 157 LMICs from 1990 to 2018.

Participants

90 LMICs receiving PEPFAR aid and cohorts of those countries, including those required to submit annual country operational plans (COP), other recipient countries (non-COP), and three groupings of countries based on cumulative amount of per capita aid received (high, medium, low).

Interventions

PEPFAR aid to combat the HIV epidemic.

Primary outcome measures

Maternal mortality and child mortality rates, vaccination rates to protect children for diphtheria, whooping cough and tetanus, measles, HepB3, and tetanus, and prevalence of anaemia in women of childbearing age.

Results

Across PEPFAR recipient countries, large, favourable PEPFAR health effects were found for rates of childhood immunisation, child mortality and maternal mortality. These beneficial health effects were large and significant in all segments of PEPFAR recipient countries studied. We also found significant and favourable programme effects on the prevalence of anaemia in women of childbearing age in PEPFAR recipient countries receiving the most intensive financial support from the PEPFAR programme. Other recipient countries did not demonstrate significant effects on anaemia.

Conclusions

This study demonstrated that important health indicators, beyond HIV, have been consistently and favourably influenced by PEPFAR presence. Child and maternal mortality have been substantially reduced, and childhood immunisation rates increased. We also found no evidence of ‘crowding out’ or negative spillovers in these resource-poor countries. These findings add to the body of evidence that PEPFAR has had favourable health effects beyond HIV. The implications of these findings are that foreign aid for health in one area may have favourable health effects in other areas in recipient countries. More research is needed on the influence of the mechanisms at work that create these spillover health effects of PEPFAR.

Cost-effectiveness of camrelizumab combined with chemotherapy in the first-line treatment of recurrent or metastatic nasopharyngeal carcinoma in China

Por: Zhao · Z. · Chen · T. · Zhou · Z. · Guo · R. · Liu · Q.
Objective

This study aimed to investigate the cost-effectiveness of adding Chinese-developed anti-PD-1 antibody camrelizumab to first-line platinum-doublet chemotherapy in patients with recurrent or metastatic nasopharyngeal carcinoma (L/M NPC) from the perspective of Chinese healthcare system.

Design

A Markov model consisting of four health states, progression-free survival, first progression survival, second progression survival and death, was built to simulate 3-week patient transitions over a 20-year horizon. A direct comparison between first-line camrelizumab in combination with gemcitabine plus cisplatin and gemcitabine plus cisplatin was performed by calculating transition probabilities from the CAPTAIN-1st trial. Costs and utilities were collected from the local public database and literature. One-way and probabilistic sensitivity analyses were employed to evaluate the robustness of the model.

Setting

The Chinese healthcare system perspective.

Participants

A hypothetical cohort of Chinese patients with pathologically diagnosed L/M NPC who had an Eastern Cooperative Oncology Group performance status of 0 or 1.

Interventions

First-line camrelizumab in combination with camrelizumab and gemcitabine plus cisplatin (CGP) versus gemcitabine plus cisplatin (GP).

Primary outcome measure

Cost, quality-adjusted life-years (QALYs), incremental cost-effectiveness ratio (ICER).

Results

The baseline analysis demonstrated that, compared with first-line GP, first-line CGP yields an effectiveness increase of 0.26 QALY, accompanied by an increment of US$6137.59 in healthcare cost. This results in an ICER of US$23 482.32/QALY. With the willingness-to-pay (WTP) threshold for a QALY set at US$37 654.50, first-line CGP proves to be cost-effective in 97.20% of the iterations. Deterministic sensitivity analyses indicated that the uncertainty in model parameters had no substantial effect on our results. Probability sensitivity analysis indicated that CGP was cost-effective at the assumed WTP threshold.

Conclusion

For Chinese patients with L/M NPC, adding Chinese-developed anti-PD-1 antibody camrelizumab to the first-line GP chemotherapy may be cost-effective.

First-line treatment with durvalumab plus chemotherapy versus chemotherapy alone for metastatic non-small-cell lung cancer in the USA: a cost-effectiveness analysis

Por: Zheng · Z. · Fang · L. · Cai · H.
Objective

The objective of this study was to evaluate the cost-effectiveness of durvalumab in combination with chemotherapy compared with chemotherapy alone as first-line therapy for metastatic non-small-cell lung cancer (NSCLC) from the perspective of the US payer.

Methods

Based on the POSEIDON clinical trial, a partition survival model was developed to compare the cost-effectiveness of durvalumab in combination with chemotherapy versus chemotherapy alone for the first-line treatment of metastatic NSCLC. The model’s primary outcomes were costs, life years (LYs), quality-adjusted LYs (QALYs) and the incremental cost-effectiveness ratio (ICER). The analysis only considered direct medical costs, and health utility value was determined using published literature. The robustness of the model was tested by probabilistic sensitivity analyses.

Results

The combination therapy of durvalumab and chemotherapy improved survival by 0.713 QALYs at an incremental cost of $64 104.638 compared with chemotherapy alone, resulting in an ICER of $89 908.328 per QALY gained from the US payer perspective. The combination therapy had a 92.3% probability of being cost-effective at a willingness-to-pay threshold of $150 000 per QALY based on incremental net health benefits. Sensitivity analyses confirmed the model’s consistency, and none of the parameters significantly influenced the findings.

Conclusion

Durvalumab in combination with chemotherapy represents a more cost-effective strategy for first-line therapy in patients with metastatic NSCLC in the USA compared with chemotherapy alone.

mHealth application for improving treatment outcomes for patients with multidrug-resistant tuberculosis in Vietnam: an economic evaluation protocol for the V-SMART trial

Por: Cheng · Q. · Dang · T. · Nguyen · T.-A. · Velen · K. · Nguyen · V. N. · Nguyen · B. H. · Vu · D. H. · Long · C. H. · Do · T. T. · Vu · T.-M. · Marks · G. B. · Yapa · M. · Fox · G. J. · Wiseman · V.
Introduction

The Strengthen the Management of Multidrug-Resistant Tuberculosis in Vietnam (V-SMART) trial is a randomised controlled trial of using mobile health (mHealth) technologies to improve adherence to medications and management of adverse events (AEs) in people with multidrug-resistant tuberculosis (MDR-TB) undergoing treatment in Vietnam. This economic evaluation seeks to quantify the cost-effectiveness of this mHealth intervention from a healthcare provider and societal perspective.

Methods and analysis

The V-SMART trial will recruit 902 patients treated for MDR-TB across seven participating provinces in Vietnam. Participants in both intervention and control groups will receive standard community-based therapy for MDR-TB. Participants in the intervention group will also have a purpose-designed App installed on their smartphones to report AEs to health workers and to facilitate timely management of AEs. This economic evaluation will compare the costs and health outcomes between the intervention group (mHealth) and the control group (standard of care). Costs associated with delivering the intervention and health service utilisation will be recorded, as well as patient out-of-pocket costs. The health-related quality of life (HRQoL) of study participants will be captured using the 36-Item Short Form Survey (SF-36) questionnaire and used to calculate quality-adjusted life-years (QALYs). Incremental cost-effectiveness ratios (ICERs) will be based on the primary outcome (proportion of patients with treatment success after 24 months) and QALYs gained. Sensitivity analysis will be conducted to test the robustness of the ICERs. A budget impact analysis will be conducted from a payer perspective to provide an estimate of the total budget required to scale-up delivery of the intervention.

Ethics and dissemination

Ethical approval for the study was granted by the University of Sydney Human Research Ethics Committee (2019/676), the Scientific Committee of the Ministry of Science and Technology, Vietnam (08/QD-HDQL-NAFOSTED) and the Institutional Review Board of the National Lung Hospital, Vietnam (13/19/CT-HDDD). Study findings will be published in peer-reviewed journals and conference proceedings.

Trial registration number

ACTRN12620000681954.

Cost-effectiveness of Spironolactone for Adult Female Acne (SAFA): economic evaluation alongside a randomised controlled trial

Por: Pyne · S. · Sach · T. H. · Lawrence · M. · Renz · S. · Eminton · Z. · Stuart · B. · Thomas · K. S. · Francis · N. · Soulsby · I. · Thomas · K. · Permyakova · N. V. · Ridd · M. J. · Little · P. · Muller · I. · Nuttall · J. · Griffiths · G. · Layton · A. M. · Santer · M.
Objective

This study aims to estimate the cost-effectiveness of oral spironolactone plus routine topical treatment compared with routine topical treatment alone for persistent acne in adult women from a British NHS perspective over 24 weeks.

Design

Economic evaluation undertaken alongside a pragmatic, parallel, double-blind, randomised trial.

Setting

Primary and secondary healthcare, community and social media advertising.

Participants

Women ≥18 years with persistent facial acne judged to warrant oral antibiotic treatment.

Interventions

Participants were randomised 1:1 to 50 mg/day spironolactone (increasing to 100 mg/day after 6 weeks) or matched placebo until week 24. Participants in both groups could continue topical treatment.

Main outcome measures

Cost-utility analysis assessed incremental cost per quality-adjusted life year (QALY) using the EQ-5D-5L. Cost-effectiveness analysis estimated incremental cost per unit change on the Acne-QoL symptom subscale. Adjusted analysis included randomisation stratification variables (centre, baseline severity (investigator’s global assessment, IGA

Results

Spironolactone did not appear cost-effective in the complete case analysis (n=126 spironolactone, n=109 control), compared with no active systemic treatment (adjusted incremental cost per QALY £67 191; unadjusted £34 770). Incremental cost per QALY was £27 879 (adjusted), just below the upper National Institute for Health and Care Excellence’s threshold value of £30 000, where multiple imputation took account of missing data. Incremental cost per QALY for other sensitivity analyses varied around the base-case, highlighting the degree of uncertainty. The adjusted incremental cost per point change on the Acne-QoL symptom subscale for spironolactone compared with no active systemic treatment was £38.21 (complete case analysis).

Conclusions

The results demonstrate a high level of uncertainty, particularly with respect to estimates of incremental QALYs. Compared with no active systemic treatment, spironolactone was estimated to be marginally cost-effective where multiple imputation was performed but was not cost-effective in complete case analysis.

Trial registration number

ISRCTN registry (ISRCTN12892056).

Measuring the success of programmes of care for people living with dementia: a protocol for consensus building with consumers to develop a set of Core Outcome Measures for Improving Care (COM-IC)

Por: Comans · T. · Nguyen · K. · Gray · L. · Flicker · L. · Williamson · P. · Dodd · S. · Kearney · A. · Cunningham · C. · Morris · T. · Nunn · J. · Trepel · D. · Almeida · O. P. · Kenny · D. · Welch · A. · Lowthian · J. A. · Quinn · J. · Petrie · G. · Dao-Tran · T.-H. · Manchha · A. · Kurrle · S.
Introduction

The Core Outcome Measures for Improving Care (COM-IC) project aims to deliver practical recommendations on the selection and implementation of a suite of core outcomes to measure the effectiveness of interventions for dementia care.

Methods and analysis

COM-IC embeds a participatory action approach to using the Alignment–Harmonisation–Results framework for measuring dementia care in Australia. Using this framework, suitable core outcome measures will be identified, analysed, implemented and audited. The methods for analysing each stage will be codesigned with stakeholders, through the conduit of a Stakeholder Reference Group including people living with dementia, formal and informal carers, aged care industry representatives, researchers, clinicians and policy actors. The codesigned evaluation methods consider two key factors: feasibility and acceptability. These considerations will be tested during a 6-month feasibility study embedded in aged care industry partner organisations.

Ethics and dissemination

COM-IC has received ethical approval from The University of Queensland (HREC 2021/HE001932). Results will be disseminated through networks established over the project, and in accordance with both the publication schedule and requests from the Stakeholder Reference Group. Full access to publications and reports will be made available through UQ eSpace (https://espace.library.uq.edu.au/), an open access repository hosted by The University of Queensland.

Cost-effectiveness analysis of probiotic peanut oral immunotherapy (PPOIT) versus placebo in Australian children with peanut allergy alongside a randomised trial

Por: Huang · L. · Dalziel · K. · Lloyd · M. · Loke · P. · Lozinsky · A. C. · Tang · M.
Objective

To compared the cost-effectiveness of coadministration of a probiotic adjuvant with peanut oral immunotherapy (PPOIT) with placebo (no treatment) in children with peanut allergy.

Design

Prospectively planned cost-effectiveness analysis alongside a randomised control trial.

Setting

The Royal Children’s Hospital, Melbourne, Australia.

Participants

56 children with peanut allergy aged 1–10 years at recruitment.

Intervention

A daily dose of probiotic Lactobacillus rhamnosus CGMCC 1.3724 (NCC4007) and peanut oral immunotherapy administered for 1.5 years.

Main outcomes measures

Costs were considered from a healthcare system perspective and included costs of treatment delivery and adverse events. Effectiveness outcomes included rate of sustained unresponsiveness (SU) and quality-adjusted life years (QALYs). The cost-effectiveness of PPOIT versus placebo was analysed using patient-level data. Time horizon was 10 years from commencement of PPOIT treatment, comprising 1.5 years of treatment (actual data), 4 years of post-treatment follow-up (actual data), and 4.5 years of extrapolation thereafter (modelling).

Results

Healthcare cost per patient over 10 years was higher for PPOIT compared with placebo ($A9355 vs $A1031, p

Conclusions

Cost per QALY gained using PPOIT compared with no treatment is approximately $A20 000 (£10 000) and is well below the conventional value judgement threshold of $A50 000 (£25 000) per QALY gained, thus deemed good value for money ($A1= £0.5 approximately).

Trial registration number

Australian New Zealand Clinical Trials Registry ACTRN12608000594325; Post-results.

Economic evaluation of serplulimab plus chemotherapy as the first-line treatment of oesophageal squamous cell carcinoma in China

Por: Chen · F. · Long · Y. · Yang · J. · Zhong · K. · Liu · B.
Objective

The ASTRUM-007 study confirmed the significant efficacy and safety of serplulimab plus chemotherapy for patients with locally advanced/metastatic, programmed cell death-ligand 1 positive oesophageal squamous cell carcinoma (OSCC). The economics of this regimen, however, is unclear. Therefore, this study aimed to evaluate the cost-effectiveness of adding serplulimab to chemotherapy for the treatment of advanced OSCC from the perspective of the Chinese healthcare system.

Design

A partitioned survival model was established to simulate the costs and outcomes of chemotherapy versus serplulimab plus chemotherapy. The survival data came from the ASTRUM-007 study. Only direct medical costs were considered, and utility values were referred to the literature. Sensitivity analysis was performed to assess the effect of parameter uncertainty on the model.

Outcome measures

Total costs, incremental costs, life years, quality-adjusted life years (QALYs), incremental QALYs and incremental cost-effectiveness ratio (ICER).

Results

The base case analysis showed that the cost of serplulimab plus chemotherapy (US$69 356) was US$41 607 higher than that of chemotherapy (US$27 749), but it also gained 0.38 QALYs more (1.38 vs 1 QALYs), with an ICER of US$110 744.36/QALY, which was higher than the willingness to pay. The factors that most influenced the ICER were the price of serplulimab, weight and utility value of the progression-free survival stage. The subgroup analysis and scenario analysis also demonstrated that serplulimab plus chemotherapy was not economical.

Conclusions

Compared with chemotherapy, serplulimab coupled with chemotherapy was not cost-effective for the treatment of advanced OSCC in China.

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