Factors influencing the reimbursement of cancer drugs in Europe: A scoping review.

RATIONALE: Reimbursement process of oncology drugs in Europe occurs within a complex decision-making process that varies between Member States. Distinctions between the States trigger societal debates since it is necessary to balance access to medicines and health systems sustainability. AIMS AND OBJECTIVES: We aimed to review the evidence concerning factors associated with the reimbursement decision or Health Technology Agency recommendation of oncology drugs in Europe. METHODS: A systematic literature search was performed in two databases from inception to august 2023. Screening and data extraction were performed by pairs. RESULTS: Thirteen articles were included and encompassed data from 11 nations. Seven articles showed that cost-effective (C-E) drugs and lower Incremental Cost-Effectiveness Ratios (ICERs) had higher likelihood of reimbursement. Disease severity might influence the reimbursement decision with financial agreements. Improvement in clinical outcomes, substantial clinical benefit (p < 0.01) or overall survival gains (p < 0.05) were positively associated. Orphan drug designation impact varies between countries but positive decisions are usually achieved under specific conditions. Clinical and C-E uncertainty frequently led to reimbursement with financial agreements or outcomes-based conditions. Sociodemographic factors as: social health insurance system, higher Gross Domestic Product and larger elderly population were positively associated with reimbursement (p < 0.01). CONCLUSION: There is a need for further research into key determinants of reimbursement decisions in Europe and the development of drug access models that can effectively address and overcome costs and effectiveness uncertainties.

Registry cost description of carpal tunnel release in Finland in 2011-2015.

OBJECTIVES: In this study, we evaluated the amount of public funds spent on the operative treatment of carpal tunnel syndrome (CTS) in Finland in 2011-2015. DESIGN: A registry-based cost burden study. SETTING: The data were collected in primary and secondary care in both private and public hospitals, covering the whole population of Finland. PARTICIPANTS: We collected the total number of patients with new CTS diagnoses and the total number of patients undergoing surgery from the Care Register for Health Care, Finland's national register. INTERVENTIONS: Open carpal tunnel release (OCTR). OUTCOME MEASURES: We collected the costs of the OCTR procedure from diagnosis-related group prices. The Social Insurance Institution of Finland provided the total amount of euros reimbursed for sick leaves. We then combined the average amount of reimbursed sick leave with our estimated cost of the treatment chain to approximate the average cost per patient. RESULTS: The average amount of public funds used for diagnosing and surgically treating new CTS in 2011-2015 in Finland, including reimbursements for sick leaves, was €2759 per patient in 2015 currency. The average direct procedure cost was €1020. We found no clear trend in total cost per patient, but the proportion of surgically treated patients rose from 63.14% to 73.09%. The total annual cost of these treatments was between €18 128 420 and €22 569 973. CONCLUSIONS: The average amount of public funds used to surgically treat one patient with new CTS in 2011-2015 in Finland was €2759, making the total annual burden €20.7 million.

Early vocational rehabilitation and psychological support for trauma patients to improve return to work (the ROWTATE trial): study protocol for an individually randomised controlled multicentre pragmatic trial.

BACKGROUND: Moderately severe or major trauma (injury severity score (ISS) > 8) is common, often resulting in physical and psychological problems and leading to difficulties in returning to work. Vocational rehabilitation (VR) can improve return to work/education in some injuries (e.g. traumatic brain and spinal cord injury), but evidence is lacking for other moderately severe or major trauma. METHODS: ROWTATE is an individually randomised controlled multicentre pragmatic trial of early VR and psychological support in trauma patients. It includes an internal pilot, economic evaluation, a process evaluation and an implementation study. Participants will be screened for eligibility and recruited within 12 weeks of admission to eight major trauma centres in England. A total of 722 participants with ISS > 8 will be randomised 1:1 to VR and psychological support (where needed, following psychological screening) plus usual care or to usual care alone. The ROWTATE VR intervention will be provided within 2 weeks of study recruitment by occupational therapists and where needed, by clinical psychologists. It will be individually tailored and provided for ≤ 12 months, dependent on participant need. Baseline assessment will collect data on demographics, injury details, work/education status, cognitive impairment, anxiety, depression, post-traumatic distress, disability, recovery expectations, financial stress and health-related quality of life. Participants will be followed up by postal/telephone/online questionnaires at 3, 6 and 12 months post-randomisation. The primary objective is to establish whether the ROWTATE VR intervention plus usual care is more effective than usual care alone for improving participants' self-reported return to work/education for at least 80% of pre-injury hours at 12 months post-randomisation. Secondary outcomes include other work outcomes (e.g. hours of work/education, time to return to work/education, sickness absence), depression, anxiety, post-traumatic distress, work self-efficacy, financial stress, purpose in life, health-related quality of life and healthcare/personal resource use. The process evaluation and implementation study will be described elsewhere. DISCUSSION: This trial will provide robust evidence regarding a VR intervention for a major trauma population. Evidence of a clinically and cost-effective VR intervention will be important for commissioners and providers to enable adoption of VR services for this large and important group of patients within the NHS. TRIAL REGISTRATION: ISRCTN: 43115471. Registered 27/07/2021.

Similar QALY gain in primary and revision knee arthroplasty: A cost analysis and Markov model.

PURPOSE: The aim of this study is to investigate the cost-effectiveness of revision total knee arthroplasty compared to primary total knee arthroplasty in terms of cost-per-quality-adjusted life year (QALY). METHODS: Data were retrieved for all primary and revision total knee replacement (TKA) procedures performed at a tertiary Swiss hospital between 2006 and 2019. A Markov model was created to evaluate revision risk and we calculated lifetime QALY gain and lifetime procedure costs through individual EuroQol 5 dimension (EQ-5D) scores, hospital costs, national life expectancy tables and standard discounting processes. Cost-per-QALY gain was calculated for primary and revision procedures. RESULTS: EQ-5D data were available for 1343 primary and 103 revision procedures. Significant QALY gains were seen following surgery in all cases. Similar, but significantly more QALYs were gained following primary TKA (PTKA) (5.67 ± 3.98) than following revision TKA (RTKA) (4.67 ± 4.20). Cost-per-QALY was €4686 for PTKA and €10,364 for RTKA. The highest average cost-per-QALY was seen in two-stage RTKA (€12,292), followed by one-stage RTKA (€8982). CONCLUSION: RTKA results in a similar QALY gain as PTKA. The costs of achieving health gain are two to three times higher in RTKA, but both procedures are highly cost-effective. LEVEL OF EVIDENCE: Economic level II.

Oral nutritional supplements improve clinical outcomes and are cost-effective for hospitalized patients in China.

OBJECTIVE: This study assessed the therapeutic benefits and modeled the cost-effectiveness of oral nutritional supplements (ONS) in China. METHODS: Data were collected from 27 152 adult inpatients between January 1, 2018, and December 31, 2020. Propensity score matching was used for balancing the baseline characteristics between the ONS group and non-ONS group. A decision-tree model was developed to assess the cost-effectiveness of ONS for patients with nutritional risk, and the incremental cost-effectiveness ratio was the metric to determine the most cost-effective strategy. One-way sensitivity and probabilistic sensitivity analyses were conducted to assess the model's stability. In addition, subgroup analysis was conducted based on clinical characteristics. Differences in clinical outcomes between the groups were compared using Student's t test, Mann-Whitney U test, or chi-square test. RESULTS: The ONS group displayed significantly lower levels of prealbumin, albumin, hemoglobin, and BMI than the non-ONS group at admission. The incidence of malignant tumors, intestinal obstruction, and inflammatory bowel disease was significantly higher in the ONS group than the non-ONS group. The ONS group had a significantly higher effective rate than the non-ONS group (51.7% versus 50.3%, P < 0.05). Analysis of the decision-tree model revealed that the ONS group experienced an increase in cost of 19 850.96 yuan but achieved an additional 1.3406 effectiveness rate, resulting in an incremental cost-effectiveness ratio of 14 807.51, which fell below China's 2020 per capita gross domestic product of 71 965 yuan. Sensitivity analysis further confirmed the robustness of the model. CONCLUSIONS: ONS are demonstrated a high rate of efficacy, although patients currently using ONS are typically in a severe disease state. In addition, ONS is cost-effective. We suggest that the reimbursement coverage of ONS be expanded to include in-hospital patients who are at high nutritional risk.

The effect of community diagnostic centres on volume and waiting time for diagnostic procedures in the UK.

Many health care systems are looking to implement policies to improve productivity and accessibility of health care. In this paper we use data from the English National Health Service to evaluate the effect of introducing new "Community Diagnostic Centres" in 2021 which aim to increase volume, reduce waiting times, and increase accessibility to diagnostic procedures. Our results show an increase in volume of diagnostic procedures associated with the introduction of CDCs at local NHS organisations. We find some evidence the increase is driven by an increase in MRI scans in particular, and this result is larger for CDCs located in more deprived local areas. We find no effect on waiting times which may indicate some demand response to increased availability of tests.

Complex management and descriptive cost analysis of kidney transplant candidates: a descriptive cross-sectional study.

BACKGROUND: The organisational care needs involved in accessing kidney transplant have not been described in the literature and therefore a detailed analysis thereof could help to establish a framework (including appropriate timing, investment, and costs) for the management of this population. The main objective of this study is to analyse the profile and care needs of kidney transplant candidates in a tertiary hospital and the direct costs of studying them. METHODS: A descriptive, cross-sectional study was conducted using data on a range of variables (sociodemographic and clinical characteristics, study duration, and investment in visits and supplementary tests) from 489 kidney transplant candidates evaluated in 2020. RESULTS: The comorbidity index was high (> 4 in 64.3%), with a mean of 5.6 ± 2.4. Part of the study population had certain characteristics that could hinder their access a kidney transplant: physical dependence (9.4%), emotional distress (33.5%), non-adherent behaviours (25.2%), or language barriers (9.4%). The median study duration was 6.6[3.4;14] months. The ratio of required visits to patients was 5.97:1, meaning an investment of €237.10 per patient, and the ratio of supplementary tests to patients was 3.5:1, meaning an investment of €402.96 per patient. CONCLUSIONS: The study population can be characterised as complex due to their profile and their investment in terms of time, visits, supplementary tests, and direct costs. Management based on our results involves designing work-adaptation strategies to the needs of the study population, which can lead to increased patient satisfaction, shorter waiting times, and reduced costs.

Multidisciplinary full-mouth rehabilitation using minimally invasive dental implant therapy: a report on three different clinical scenarios.

Conventional operative dental procedures are being revisited with an approach that seeks to preserve the original tissue as much as possible. Implant dentistry has also seen the advent of various techniques which make minimal alteration to natural tissue. The flapless technique involves implant placement either through a freshly extracted socket or through a tansmucosal punch hole, without elevating mucoperiosteal flap. This paper presents a report on three cases of patient centric, conventional dental implant-based full-mouth rehabilitation, which was successfully carried out using the flapless technique, under varied clinical situations. Each case showed a favourable outcome in terms of restoration of the form and function of the patient's dentition.

Impella versus VA-ECMO for the treatment of patients with cardiogenic shock: the Impella Network Project - observational study protocol for cost-effectiveness and budget impact analyses.

INTRODUCTION: The treatment of patients with cardiogenic shock (CS) encompasses several health technologies including Impella pumps and venoarterial extracorporeal membrane oxygenation (VA-ECMO). However, while they are widely used in clinical practice, information on resource use and quality of life (QoL) associated with these devices is scarce. The aim of this study is, therefore, to collect and comparatively assess clinical and socioeconomic data of Impella versus VA-ECMO for the treatment of patients with severe CS, to ultimately conduct both a cost-effectiveness (CEA) and budget impact (BIA) analyses. METHODS AND ANALYSIS: This is a prospective plus retrospective, multicentre study conducted under the scientific coordination of the Center for Research on Health and Social Care Management of SDA Bocconi School of Management and clinical coordination of Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute in Milan. The Impella Network stemmed for the purposes of this study and comprises 17 Italian clinical centres from Northern to Southern Regions in Italy. The Italian network qualifies as a subgroup of the international Impella Cardiac Surgery Registry. Patients with CS treated with Impella pumps (CP, 5.0 or 5.5) will be prospectively recruited, and information on clinical outcomes, resource use and QoL collected. Economic data will be retrospectively matched with data from comparable patients treated with VA-ECMO. Both CEA and BIA will be conducted adopting the societal perspective in Italy. This study will contribute to generate new socioeconomic evidence to inform future coverage decisions. ETHICS AND DISSEMINATION: As of May 2024, most of the clinical centres submitted the documentation to their ethical committee (N=13; 76%), six centres received ethical approval and two centres started to enrol patients. Study results will be published in peer-reviewed publications and disseminated through conference presentations.

Productivity costs due to human papillomavirus-related cancer mortality in the United Kingdom.

BACKGROUND: Human papillomavirus (HPV) causes several cancers such as cervical cancer and some head and neck (oral cavity, pharynx, and larynx), vulval, vaginal, anal, and penile cancers. As HPV vaccination is available, there is potential to prevent these cancers attributed to HPV and consequently the burden associated with them. The aim of this analysis was to estimate the number of HPV-related cancer deaths and the productivity costs due to years of life lost (YLL) in the United Kingdom (UK). METHOD: A model was developed utilizing UK 2019 mortality data sourced from country-specific databases for England, Scotland, Wales, and Northern Ireland for the following HPV-related cancers: head and neck (ICD-10 C00-14 and C32), cervix uteri (C53), vaginal (C51), vulval (C52), anal (C21), and penile (C60). The proportion of deaths and years of life lost (YLL) due to HPV were estimated using HPV attributable fractions for each anatomic location from the published literature. Labor force participation, retirement ages, and mean annual earnings, discounted at 3.5% annually, were applied to YLL to calculate the present value of future lost productivity (PVFLP). RESULTS: A total of 1817 deaths due to HPV-related cancers were reported in the UK in 2019 resulting in 31,804 YLL. Restricting to only YLL that occurred prior to retirement age yielded a total YPLL of 11,765 and a total PVFLP of £187,764,978. CONCLUSIONS: There is a high disease burden in the UK for HPV-related cancers, with a large economic impact on the wider economy due to productivity losses. Implementing and reinforcing public health measures to maintain high HPV vaccination coverage in both males and females may further facilitate reduction of this burden.

Clinical drivers of hospitalisation in patients with mitochondrial diseases.

Background: Mitochondrial diseases in adults are generally chronic conditions with a wide spectrum of severity contributing to disease burden and healthcare resource utilisation. Data on healthcare resource utilisation in mitochondrial diseases are limited. Objectives: We performed a retrospective longitudinal study to investigate the clinical drivers of hospitalisation in adult patients with mitochondrial diseases to better understand healthcare resource utilisation. Methods: We recruited participants from our specialised Mitochondrial Disease Clinic in Sydney, Australia between September 2018 and December 2021. We performed a retrospective chart review for the period 2013-2022 considering emergency department (ED) and/or hospital admission notes, as well as discharge summaries. We used multiple linear regression models to examine the association between the type of presenting symptom(s) and duration of hospital stay and frequency of admissions, while adjusting for relevant covariates. Results: Of the 99 patients considered, the duration of hospitalisation ranged from 0 to 116 days per participant and the number of admissions ranged from 0 to 21 per participant. Participants with one or more mitochondrial disease-associated admissions constituted 52% of the study cohort. 13% of the participants presented to the ED without requiring an admission and 35% never attended the ED or required a hospital admission during this period. Neurological (p<0.0001), gastroenterological (p=0.01) and symptoms categorised as 'other' (p<0.0001) were the main presentations driving the total number of days admitted to hospital. A statistically significant association was evident for the number of admissions and all types of presenting symptoms (p<0.0001). Conclusion: There are variable reasons for hospitalisation in adults with mitochondrial diseases, with neurological and gastroenterological presentations being associated with prolonged and complex hospitalisation. A better understanding of clinical drivers such as these allows for better informed and well-coordinated management aimed at optimising healthcare resource utilisation.

Risk-Adapted Breast Screening for Women at Low Predicted Risk of Breast Cancer: An Online Discrete Choice Experiment.

BACKGROUND: A risk-stratified breast screening program could offer low-risk women less screening than is currently offered by the National Health Service. The acceptability of this approach may be enhanced if it corresponds to UK women's screening preferences and values. OBJECTIVES: To elicit and quantify preferences for low-risk screening options. METHODS: Women aged 40 to 70 y with no history of breast cancer took part in an online discrete choice experiment. We generated 32 hypothetical low-risk screening programs defined by 5 attributes (start age, end age, screening interval, risk of dying from breast cancer, and risk of overdiagnosis), the levels of which were systematically varied between the programs. Respondents were presented with 8 choice sets and asked to choose between 2 screening alternatives or no screening. Preference data were analyzed using conditional logit regression models. The relative importance of attributes and the mean predicted probability of choosing each program were estimated. RESULTS: Participants (N = 502) preferred all screening programs over no screening. An older starting age of screening, younger end age of screening, longer intervals between screening, and increased risk of dying had a negative impact on support for screening programs (P < 0.01). Although the risk of overdiagnosis was of low relative importance, a decreased risk of this harm had a small positive impact on screening choices. The mean predicted probabilities that risk-adapted screening programs would be supported relative to current guidelines were low (range, 0.18 to 0.52). CONCLUSIONS: A deintensified screening pathway for women at low risk of breast cancer, especially one that recommends a later screening start age, would run counter to women's breast screening preferences. Further research is needed to enhance the acceptability of offering less screening to those at low risk of breast cancer. HIGHLIGHTS: Risk-based breast screening may involve the deintensification of screening for women at low risk of breast cancer.Low-risk screening pathways run counter to women's screening preferences and values.Longer screening intervals may be preferable to a later start age.Work is needed to enhance the acceptability of a low-risk screening pathway.

Cost-effectiveness of posterior versus anterior surgery for cervical radiculopathy: results from a multicentre randomised non-inferiority trial (FACET).

PURPOSE: For cervical nerve root compression, anterior cervical discectomy with fusion (anterior surgery) or posterior foraminotomy (posterior surgery) are safe and effective options. Posterior surgery might have a more beneficial economic profile compared to anterior surgery. The purpose of this study was to analyse if posterior surgery is cost-effective compared to anterior surgery. METHODS: An economic evaluation was performed as part of a multicentre, noninferiority randomised clinical trial (Foraminotomy ACDF Cost-effectiveness Trial) with a follow-up of 2 years. Primary outcomes were cost-effectiveness based on arm pain (Visual Analogue Scale (VAS; 0-100)) and cost-utility (quality adjusted life years (QALYs)). Missing values were estimated with multiple imputations and bootstrap simulations were used to obtain confidence intervals (CIs). RESULTS: In total, 265 patients were randomised and 243 included in the analyses. The pooled mean decrease in VAS arm at 2-year follow-up was 44.2 in the posterior and 40.0 in the anterior group (mean difference, 4.2; 95% CI, - 4.7 to 12.9). Pooled mean QALYs were 1.58 (posterior) and 1.56 (anterior) (mean difference, 0.02; 95% CI, - 0.05 to 0.08). Societal costs were €28,046 for posterior and €30,086 for the anterior group, with lower health care costs for posterior (€12,248) versus anterior (€16,055). Bootstrapped results demonstrated similar effectiveness between groups with in general lower costs associated with posterior surgery. CONCLUSION: In patients with cervical radiculopathy, arm pain and QALYs were similar between posterior and anterior surgery. Posterior surgery was associated with lower costs and is therefore likely to be cost-effective compared with anterior surgery.

Barriers to the introduction of novel advanced targeted treatments for Australian dermatology patients: Are skin diseases symptomatic of a systemic healthcare problem?

The aim of this article is to provide education to clinicians about certain barriers restricting the use of advanced targeted treatments in Australian health care. For illustrative purposes, the article focuses on dermatological conditions, but the content is relevant to all specialties that treat inflammatory and chronic diseases. Barriers to care discussed result in a lower than necessary standard of care for patients in Australia despite important advancements in medicine.

Geographical patterns of implementing a government subsidy program: implications for health outcomes and nutrient intake in Iran.

Introduction: The lack of access to a diverse and nutritious diet has significant health consequences worldwide. Governments have employed various policy mechanisms to ensure access, but their success varies. Method: In this study, the impact of changes in food assistance policy on food prices and nutrient security in different provinces of Iran, a sanctioned country, was investigated using statistical and econometric models. Results: Both the old and new policies were broad in scope, providing subsidized food or cash payments to the entire population. However, the implementation of these policies led to an increase in the market price of food items, resulting in a decline in the intake of essential nutrients. Particularly, the policy that shifted food assistance from commodity subsidies to direct cash payments reduced the price sensitivity of consumers. Consequently, the intake of key nutrients such as Vitamin C and Vitamin A, which are often constrained by their high prices, decreased. To improve the diets of marginalized populations, it is more effective to target subsidies towards specific nutrient groups and disadvantaged populations, with a particular focus on food groups that provide essential nutrients like Vitamin A and Vitamin C in rural areas of Iran. Discussion: More targeted food assistance policies, tailored to the specific context of each province and income level, are more likely to yield positive nutritional outcomes with minimal impact on food prices.

A Systematic Review of Cost-Effectiveness of Treating Out of Hospital Cardiac Arrest: Implications for Resource-limited Health Systems.

Background: Out-of-hospital cardiac arrest (OHCA) is a prevalent condition with high mortality and poor outcomes even in settings where extensive emergency care resources are available. Interventions to address OHCA have had limited success, with survival rates below 10% in national samples of high-income countries. In resource-limited settings, where scarcity requires careful priority setting, more data is needed to determine the optimal allocation of resources. Objective: To establish the cost-effectiveness of OHCA care and assess the affordability of interventions across income settings. Methods: The authors conducted a systematic review of economic evaluations on interventions to address OHCA. Included studies were (1) economic evaluations (beyond a simple costing exercise); and (2) assessed an intervention in the chain of survival for OHCA. Article quality was assessed using the CHEERs checklist and data summarised. Findings were reported by major themes identified by the reviewers. Based upon the results of the cost-effectiveness analyses we then conduct an analysis for the progressive realization of the OHCA chain of survival from the perspective of decision-makers facing resource constraints. Results: 468 unique articles were screened, and 46 articles were included for final data abstraction. Studies predominantly used a healthcare sector perspective, modeled for all patients experiencing non-traumatic cardiac OHCA, were based in the US, and presented results in US Dollars. No studies reported results or used model inputs from low-income settings. Progressive realization of the chain of survival could likely begin with investments in TOR protocols, professional prehospital defibrillator use, and CPR training followed by distribution of AEDs in high-density public locations. Finally, other interventions such as indiscriminate defibrillator placement or adrenaline use, would be the lowest priority for early investment. Conclusion: Our review found no high-quality evidence on the cost-effectiveness of treating OHCA in low-resource settings. Existing evidence can be utilized to develop a roadmap for the development of a cost-effective approach to OHCA care, however further economic evaluations using context-specific data are crucial to accurately inform prioritization of scarce resources within emergency care in these settings.

Cost-effectiveness of craniotomy versus decompressive craniectomy for UK patients with traumatic acute subdural haematoma.

OBJECTIVE: To estimate the cost-effectiveness of craniotomy, compared with decompressive craniectomy (DC) in UK patients undergoing evacuation of acute subdural haematoma (ASDH). DESIGN: Economic evaluation undertaken using health resource use and outcome data from the 12-month multicentre, pragmatic, parallel-group, randomised, Randomised Evaluation of Surgery with Craniectomy for Patients Undergoing Evacuation-ASDH trial. SETTING: UK secondary care. PARTICIPANTS: 248 UK patients undergoing surgery for traumatic ASDH were randomised to craniotomy (N=126) or DC (N=122). INTERVENTIONS: Surgical evacuation via craniotomy (bone flap replaced) or DC (bone flap left out with a view to replace later: cranioplasty surgery). MAIN OUTCOME MEASURES: In the base-case analysis, costs were estimated from a National Health Service and Personal Social Services perspective. Outcomes were assessed via the quality-adjusted life-years (QALY) derived from the EuroQoL 5-Dimension 5-Level questionnaire (cost-utility analysis) and the Extended Glasgow Outcome Scale (GOSE) (cost-effectiveness analysis). Multiple imputation and regression analyses were conducted to estimate the mean incremental cost and effect of craniotomy compared with DC. The most cost-effective option was selected, irrespective of the level of statistical significance as is argued by economists. RESULTS: In the cost-utility analysis, the mean incremental cost of craniotomy compared with DC was estimated to be -£5520 (95% CI -£18 060 to £7020) with a mean QALY gain of 0.093 (95% CI 0.029 to 0.156). In the cost-effectiveness analysis, the mean incremental cost was estimated to be -£4536 (95% CI -£17 374 to £8301) with an OR of 1.682 (95% CI 0.995 to 2.842) for a favourable outcome on the GOSE. CONCLUSIONS: In a UK population with traumatic ASDH, craniotomy was estimated to be cost-effective compared with DC: craniotomy was estimated to have a lower mean cost, higher mean QALY gain and higher probability of a more favourable outcome on the GOSE (though not all estimated differences between the two approaches were statistically significant). ETHICS: Ethical approval for the trial was obtained from the North West-Haydock Research Ethics Committee in the UK on 17 July 2014 (14/NW/1076). TRIAL REGISTRATION NUMBER: ISRCTN87370545.

Health economic analysis of polygenic risk score use in primary prevention of coronary artery disease - A system dynamics model.

Background: Primary prevention programs utilising traditional risk scores fail to identify all individuals who suffer acute cardiovascular events. We aimed to model the impact and cost effectiveness of incorporating a Polygenic risk scores (PRS) into the cardiovascular disease CVD primary prevention program in Australia, using a whole-of-system model. Methods: System dynamics models, encompassing acute and chronic CVD care in the Australian healthcare setting, assessing the cost-effectiveness of incorporating a CAD-PRS in the primary prevention setting. The time horizon was 10-years. Results: Pragmatically incorporating a CAD-PRS in the Australian primary prevention setting in middle-aged individuals already attending a Heart Health Check (HHC) who are determined to be at low or moderate risk based on the 5-year Framingham risk score (FRS), with conservative assumptions regarding uptake of PRS, could have prevented 2, 052 deaths over 10-years, and resulted in 24, 085 QALYs gained at a cost of $19, 945 per QALY with a net benefit of $724 million. If all Australians overs the age of 35 years old had their FRS and PRS performed, and acted upon, 12, 374 deaths and 60, 284 acute coronary events would be prevented, with 183, 682 QALYs gained at a cost of $18, 531 per QALY, with a net benefit of $5, 780 million. Conclusions: Incorporating a CAD-PRS in a contemporary primary prevention setting in Australia would result in substantial health and societal benefits and is cost-effective. The broader the uptake of CAD-PRS in the primary prevention setting in middle-aged Australians, the greater the impact and the more cost-effective the strategy.

Economics of primary healthcare: cost estimation of clinical services at primary care facilities in the six countries of the Gulf Cooperation Council.

OBJECTIVE: While the Gulf Cooperation Council (GCC) countries have demonstrated a strong commitment to strengthening primary healthcare (PHC), the costs of delivering these services in this region remain relatively unexplored. Understanding the costs of PHC delivery is essential for effective resource allocation and health system efficiency. DESIGN: We used an ingredient-based method to estimate the cost of delivering a selection of services at PHC facilities in the six GCC countries in 2019. Services were categorised into eight programmes: immunisation; non-communicable diseases (NCDs); oral and dental care; child health; nutrition; mental health; reproductive, maternal, neonatal and child health and general practice. The cost estimation focused on two key ingredients: the costs of drugs and supplies and the healthcare workforce cost. The coverage rates of specific types of health services, including screening and mental health services, were also estimated. Data for the analysis were obtained from ministries of health, health statistics reports, online databases, national surveys and scientific literature. RESULTS: The estimated costs of delivering the selected services at public PHC facilities in the six GCC countries totalled US$5.7 billion in 2019, representing 0.34% of the combined 2019 GDP. The per capita costs varied from US$69 to US$272. General practice and NCD programmes constituted 79% of the total costs modelled while mental health ranged between 0.0% and 0.3%. Over 8 million individuals did not receive NCD screening services, and over 30 million did not receive needed mental health services in public PHC facilities across the region. CONCLUSIONS: To our knowledge, this is the first study to estimate the costs of services delivered at PHC facilities in the GCC countries. Identifying the main cost drivers and the services which individuals did not receive can be used to help strengthen PHC to improve efficiency and scale up needed services for better health outcomes.

Cost-utility analysis of second-line axicabtagene ciloleucel versus standard of care in Japan based on the ZUMA-7 trial.

Aim: To perform a cost-effectiveness analysis comparing axicabtagene ciloleucel (axi-cel) with standard of care (SoC; salvage chemoimmunotherapy, followed by high-dose therapy with autologous stem cell rescue for responders) for second-line (2L) treatment of adults with relapsed or refractory large B-cell lymphoma (r/r LBCL) in the pivotal ZUMA-7 trial data from a Japanese payer perspective. Materials & methods: A three-state partitioned survival model was utilized using population and clinical inputs from the ZUMA-7 trial data over a lifetime horizon. Results: Axi-cel was associated with greater incremental quality-adjusted life-years (2.06) and higher incremental total costs ($48,685.59/¥6.9 million) leading to an incremental cost-effectiveness ratio of $23,590.34/¥3.3 million per quality-adjusted life-years compared with SoC. Conclusion: Axi-cel is a cost-effective treatment alternative to SoC for 2L treatment of adults with r/r LBCL.

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