Budget impact analyses for treatment of heart failure. A systematic review.

Heart failure (HF) is increasing globally and turning out to be a serious worldwide public health problem with significant morbidity and mortality. This study aims to systemically review the budget impact analysis of heart failure treatments on health care expenditure worldwide. Scientific databases such as PubMed, Web of Science, Scopus, and Google Scholar were searched for budget impact analysis and heart failure treatments, over January 2001 to August 2023. The quality assessment of the selected studies was evaluated through ISPOR practice guideline. Nineteen studies were included in this systematic review. Based on ISPOR recommendations, most studies were performed on a 1-year time horizon and used a government (public health) or health system perspective. Data for selected studies was mainly collected from randomized clinical trials, published literature, pharmaceutical companies, and registry data. Only direct costs were reported in the studies. Sensitivity analyses were stated in almost all studies. However, studies conducted in high-income countries reported sensitivity analyses more elaborately than those performed in low- and middle-income countries. In many published articles related to the budget impact analyses of heart failure treatment, addition of new treatments to the health system's formularies can lead to a reduction in cardiovascular hospitalization rates, re-hospitalization rates, cardiac-associated mortality rates, and an improvement in heart failure class, which can decrease the costs of hospitalizations, specified care visits, primary care visits, and other related treatments.

Economic effects of next-generation sequencing diagnostics in unspecific sepsis patients - a budget impact analysis from the healthcare providers' perspective in Germany.

PURPOSE: Next-generation sequencing (NGS) tools have clinical advantages over blood culture but are more expensive. This study assesses the budget impact and break-even point of NGS testing costs from a healthcare provider's perspective in Germany. METHODS: The budget impact was calculated based on aggregated data of German post-operative surgery cases. Simulated cost savings were calculated based on a simulated reduction in hospital length of stay (LOS) of four or eight days with a positivity rate of 71% and compared to the costs of one (scenario A) or two tests (scenario B) per case. Furthermore, the break-even point of the cost of two tests compared to saved costs through shortened LOS was conducted. RESULTS: For 9,450 cases, an average budget impact for scenario A and scenario B of €1,290.41 [95% CI €1,119.64 - €1,461.19] and - €208.59 [95% CI - €379.36 - - €37.81] was identified for gastrointestinal and kidney surgery cases, and €1,355.58 [95% CI €1,049.62 - €1,661.55] and €18.72 [95% CI - €324.69 - €287.24] for vascular artery surgery cases, respectively. The break-even analysis showed that using two tests per case could achieve a minimum positive contribution margin with an average of 1.9 tests per case across the study population. CONCLUSION: The results revealed a positive budget impact for one NGS test and a slightly negative budget impact for two NGS tests per case. Findings suggest that largest cost savings are generated for more severe cases and are highly dependent on the patient population.

Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 to 2019.

OBJECTIVES: Between 2013 to 2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value allocated to 4 manufacturers vs society. METHODS: For 2015 to 2019, we estimated the incremental impact of DAAs on HCV health outcomes and costs. We used the Center for Disease Analysis Foundation Polaris Observatory database to estimate utilization. Per-patient projections of lifetime quality-adjusted life-years (QALYs) gained and medical costs avoided were based on a standard 9-state HCV disease-progression model for DAA treatment vs alternatives. Annual QALY gains were valued at $114 000 per QALY. Outcomes and costs were discounted at 3%. Estimated revenues were based on reported sales. RESULTS: An estimated 1 080 000 patients received DAAs: 81.5% would not have received the pre-DAA standard of care. On average, these patients were projected to gain 4.4 QALYs and save $104 400 in lifetime healthcare costs, generating $531.8 billion in value. Those who would have received treatment gained 1.7 QALYs and saved $41 500 in lifetime costs, generating $47.4 billion in economic value. As treatment costs fell nearly 75%, the 4 manufacturers reported $37.4 billion from DAA sales-an allocation of 6.5% of the total value. CONCLUSIONS: The significant majority (∼90%) of the economic value of curing HCV with DAAs were health benefits to patients and net cost-savings to society. DAA manufacturers received a minority share (6.5%) of the aggregate economic value generated.

Budget impact analysis of a Lifestyle-integrated Functional Exercise (LiFE) program for older people in Germany: a Markov model based on data from the LiFE-is-LiFE trial.

BACKGROUND: Fall prevention is important for healthy ageing, but the economic impact of fall prevention are scarcely investigated. A recent cost-effectiveness analysis compared a group-delivered Lifestyle-integrated Functional Exercise Program (gLiFE) with an individually-delivered program (LiFE) in community-dwelling people (aged ≥ 70 years) at risk of falling. In addition, the current study aimed to analyze the budget impact of LiFE and gLiFE, compared with standard care in Germany. METHODS: A Markov model was developed to reflect falls and associated care needs for community-dwelling persons over 5 years. The intervention effects of LiFE and gLiFE were shown to be equivalent in a non-inferiority trial, although the costs differed. Outpatient, inpatient, and intervention costs were assessed from a payer's perspective. The effect of parameter uncertainty was assessed in sensitivity analyses. RESULTS: The budget impact due to intervention costs was €510 million for LiFE and €186 million for gLiFE. Over five years, health care expenditures were €35,008 million for those receiving standard care, €35,416 million for those receiving LiFE, and €35,091 million for persons receiving gLiFE. Thereby, LiFE and gLiFE could prevent 2700 deaths and 648,000 falls over 5 years. Parameter uncertainties in the risk of falling, uptake of an intervention offer, and in the intervention effects had a major influence; thus cost savings for LiFE and gLiFE compared with standard care could be achieved for individuals with a high risk of falling. CONCLUSIONS: The results revealed that cost savings for LiFE and gLiFE compared with standard care could only be achieved for individuals at high risk of falling, with gLiFE being superior to LiFE. Future research should consider benefits and aspects of fall prevention beyond falls (e.g., physical activity, social aspects, and personal preferences of participants). TRIAL REGISTRATION: The study was preregistered under underclinicaltrials.gov (identifier: NCT03462654) on 12th March 2018; https://clinicaltrials.gov/ct2/show/NCT03462654 .

The budget impact of implementing atrial fibrillation-screening in European countries.

A budget impact analysis estimates the short-term difference between the cost of the current treatment strategy and a new treatment strategy, in this case to implement population screening for atrial fibrillation (AF). The aim of this study is to estimate the financial impact of implementing population-based AF-screening of 75-year-olds compared with the current setting of no screening from a healthcare payer perspective in eight European countries. The net budget impact of AF-screening was estimated in country-specific settings for Denmark, Germany, Ireland, Italy, Netherlands, Serbia, Spain, and Sweden. Country-specific parameters were used to allow for variations in healthcare systems and to reflect the healthcare sector in the country of interest. Similar results can be seen in all countries AF-screening incurs savings of stroke-related costs since AF treatment reduces the number of strokes. However, the increased number of detected AF and higher drug acquisition will increase the drug costs as well as the costs of physician- and control visits. The net budget impact per invited varied from €10 in Ireland to €122 in the Netherlands. The results showed the increased costs of implementing AF-screening were mainly driven by increased drug costs and screening costs. In conclusion, across Europe, though the initial cost of screening and more frequent use of oral anti-coagulants will increase the healthcare payers' costs, introducing population screening for AF will result in savings of stroke-related costs.

Budget impact analysis of high-priced orphan medicinal products intended for the treatment of rare diseases in China: evidence from a densely populated metropolis of Chengdu.

BACKGROUND: An increasing number of orphan medicinal products (OMPs) are being included in social health insurance schemes, significantly improving access to medicines for patients with rare diseases. However, high-priced OMPs are still not covered, primarily due to health equity controversies and inadequate data systems required for economic evaluation. The aim of this study was to estimate the burden of drug expenditures and the size of the reimbursement budget required for high-priced OMPs from the perspectives of society and healthcare payers. METHODS: The study performed a budget impact analysis using data from multiple sources to estimate the reimbursement budget for high-priced OMPs in Chengdu, a densely populated metropolis in China. The budget analysis consisted of three main elements: the number of patients, the price of drugs, and the simulated policy scenario. By adjusting the combinations of these elements, the budget fluctuations for payers were estimated. Furthermore, the study predicted the budget trend for the next three years to validate its sustainability. RESULTS: The analysis indicated that 98 rare disease patients in Chengdu required high-priced OMPs in 2019. This suggested a projected budget of CNY 179 million for these patients without reimbursement policies, from a societal perspective. Under six assumed policy scenarios, this budget ranged from CNY 32 million to CNY 156 million. Over the next three years, the annual budget was estimated to range from CNY 200 million to CNY 1.303 billion. CONCLUSION: Integration of multi-source data helps to obtain more scientifically reliable results on budget impacts. The study found that the budgetary impacts of high-priced OMPs on society and payers are relatively limited. Health policymakers can choose appropriate reimbursement strategies based on financial affordability among a diverse mix of elements. The results of related studies provided insights for optimizing the allocation of health resources and improving patient access to medications.

Evaluation of an acute trust Domestic Abuse Coordinator role: Impact findings and a budget impact model.

Domestic Abuse Coordinators (DACs) work strategically across National Health Service (NHS) hospital and other off-site clinical settings to support clinical staff in domestic abuse enquiry and response, and to co-lead the development and implementation of effective clinical policies and procedures for the management of domestic abuse and the support of survivors. Drawing on data from a large NHS acute trust in central London, we analyse the impact of the DAC role in increasing the rate of referrals of high-risk domestic abuse cases, and generate plausible estimates of the budget impact of the DAC role in respect of costs accrued to NHS trusts. Using eight quarters of clinical data and an interrupted time series design, we find that evidence that implementation of a DAC role is linked with an increase in the rate of high-risk referrals of between 18% and 21% per quarter, indicating improved responses to victim-survivors at highest risk of imminent harm. Under a range of reasonable assumptions, initiation of the DAC role is shown to be cost-saving to an employing acute trust. Future work should seek to quantify the direct impacts to survivor health and wellbeing of the implementation of the DAC role.

Budget impact analysis of introducing digital breast tomosynthesis in breast cancer screening in Italy.

PURPOSE: This study quantifies the impact on budget and cost per health benefit of implementing digital breast tomosynthesis (DBT) in place of digital mammography (DM) for breast cancer screening among asymptomatic women in Italy. METHODS: A budget impact analysis and a cost consequence analysis were conducted using parameters from the MAITA project and literature. The study considered four scenarios for DBT implementation, i.e., DBT for all women, DBT for women aged 45-49 years, DBT based on breast density (BI-RADS C + D or D only), and compared these to the current DM screening. Healthcare provider's perspective was adopted, including screening, diagnosis, and cancer treatment costs. RESULTS: Introducing DBT for all women would increase overall screening costs by 20%. Targeting DBT to women aged 45-49 years or with dense breasts would result in smaller cost increases (3.2% for age-based and 1.4-10.7% for density-based scenarios). The cost per avoided interval cancer was significantly higher when DBT was applied to all women compared to targeted approaches. The cost per gained early-detected cancer slightly increases in targeted approaches, while the assumptions on the clinical significance and overdiagnosis of cancers detected by DBT and not by DM have a strong impact. CONCLUSIONS: Implementing DBT as a primary breast cancer test in screening programs in Italy would lead to a substantial increase in costs. Tailoring DBT use to women aged 45-49 or with dense breasts could enhance the feasibility and sustainability of the intervention. Further research is needed to clarify the impact of DBT on overdiagnosis and the long-term outcomes.

[Pharmacoeconomic analysis of anti-angiogenic drugs for diabetic macular edema]. / Farmakoekonomicheskii analiz primeneniya antiangiogennykh preparatov pri diabeticheskom makulyarnom oteke.

Diabetic macular edema (DME) is a degenerative disease of the macular area in diabetes mellitus and can lead to vision loss, disability, and significantly reduced quality of life. Faricimab is the only bispecific antibody for DME therapy that targets two pathogenic pathways (Ang-2 and VEGF-A). PURPOSE: This study comparatively evaluates the clinical and economic feasibility of faricimab and other angiogenesis inhibitors in patients with DME. MATERIAL AND METHODS: This article analyzed literature on the efficacy and safety of intravitreal injections (IVI) of ranibizumab 0.5 mg, aflibercept 2 mg, and faricimab 6 mg. A model of medical care was developed for patients with DME receiving anti-angiogenic therapy. Pharmacoeconomic analysis was performed using cost minimization and budget impact analysis (BIA) methods. Modeling time horizon was 2 years. The research was performed from the perspective of the healthcare system of the Russian Federation. RESULTS: The efficacy and safety of faricimab in a personalized regimen (up to one IVI in 16 weeks) are comparable to those of aflibercept and ranibizumab, administered in various regimens. The use of faricimab is associated with the lowest number of IVIs. Over 2 years, the maximum costs of drug therapy were associated with the use of ranibizumab (about 914 thousand rubles), while the minimum costs were associated with the use of faricimab (614 thousand rubles). The reduction in inpatient care costs with faricimab therapy was 36% compared to aflibercept (216 and 201 thousand rubles in inpatient and day hospitals, respectively) and 82% compared to ranibizumab (486 and 451 thousand rubles in inpatient and day hospitals, respectively). BIA demonstrated that the use of faricimab will reduce the economic burden on the healthcare system by 11.3 billion rubles (9.8%) over 2 years. CONCLUSION: The use of faricimab is a cost-effective approach to treatment of adult patients with DME in Russia.

New tuberculosis vaccines in India: modelling the potential health and economic impacts of adolescent/adult vaccination with M72/AS01E and BCG-revaccination.

BACKGROUND: India had an estimated 2.9 million tuberculosis cases and 506 thousand deaths in 2021. Novel vaccines effective in adolescents and adults could reduce this burden. M72/AS01E and BCG-revaccination have recently completed phase IIb trials and estimates of their population-level impact are needed. We estimated the potential health and economic impact of M72/AS01E and BCG-revaccination in India and investigated the impact of variation in vaccine characteristics and delivery strategies. METHODS: We developed an age-stratified compartmental tuberculosis transmission model for India calibrated to country-specific epidemiology. We projected baseline epidemiology to 2050 assuming no-new-vaccine introduction, and M72/AS01E and BCG-revaccination scenarios over 2025-2050 exploring uncertainty in product characteristics (vaccine efficacy, mechanism of effect, infection status required for vaccine efficacy, duration of protection) and implementation (achieved vaccine coverage and ages targeted). We estimated reductions in tuberculosis cases and deaths by each scenario compared to the no-new-vaccine baseline, as well as costs and cost-effectiveness from health-system and societal perspectives. RESULTS: M72/AS01E scenarios were predicted to avert 40% more tuberculosis cases and deaths by 2050 compared to BCG-revaccination scenarios. Cost-effectiveness ratios for M72/AS01E vaccines were around seven times higher than BCG-revaccination, but nearly all scenarios were cost-effective. The estimated average incremental cost was US$190 million for M72/AS01E and US$23 million for BCG-revaccination per year. Sources of uncertainty included whether M72/AS01E was efficacious in uninfected individuals at vaccination, and if BCG-revaccination could prevent disease. CONCLUSIONS: M72/AS01E and BCG-revaccination could be impactful and cost-effective in India. However, there is great uncertainty in impact, especially given the unknowns surrounding the mechanism of effect and infection status required for vaccine efficacy. Greater investment in vaccine development and delivery is needed to resolve these unknowns in vaccine product characteristics.

Management of perioperative iron deficiency anemia as part of patient blood management in France: A budget impact model-based analysis based on real world data.

OBJECTIVES: Patient Blood Management (PBM) is defined as a patient-centered, systematic, evidence-based approach to improve patient outcomes by managing and preserving a patient's own blood, while promoting patient safety and empowerment. As a corollary, it also reduces the utilization of allogeneic blood components. However, demonstrating cost-effectiveness depends on the health insurance system considered. This analysis aims to estimate the one-year budget impact of PBM in four elective surgical areas, from French National Health Insurance and hospital perspectives. METHODS: A budget impact model was developed to estimate the difference in the cost of care between scenarios with and without PBM. The impact of hematopoiesis optimization (first pillar of PBM) was studied throughout the management of preoperative anemia and iron deficiency in four types of surgeries: orthopedic, cardiac & cardiovascular, vascular & thoracic, and urologic & visceral surgery. Estimation of model's parameters was based on data collected in 10 French hospitals, literature, and on data from the French national medico-administrative database. RESULTS: A total of 980,125 patients were modeled for all four therapeutic areas. Results shows that implementation of a PBM program could generate annual savings up to €1079 M from the French National Health Insurance perspective (€1018 M from the hospital perspective), and the sparing of 181,451 red blood cells units per year. The deterministic sensitivity analysis showed that PBM generates savings for both perspectives in most parameters tested. CONCLUSION: Implementing PBM programs could result in important savings for the health care system in France.

Budget impact analysis of molecular subtype profiling in endometrial cancer.

OBJECTIVE: This study evaluated the costs associated with four approaches to classifying endometrial cancer (EC), including histomorphological, histomorphological with ancillary immunohistochemical assays, histomolecular and selective molecular classification. METHODS: Direct costs were determined per EC sample from the hospital's perspective. A budget impact analysis and sensitivity analysis were conducted to estimate the mean, minimum and maximum costs per sample and annual institutional costs in adjusted 2022 Canadian dollars. A provincial cost forecast was projected based on expected 2022 EC biopsies. RESULTS: In 2018, our institution performed 190 EC biopsies. The mean cost per biopsy was $158 ($156-$212) for histomorphological classification, $384 ($360-$514) for histomorphological classification with immunohistochemistry and $1297 ($1265-1833) for histomolecular classification. Total annual institutional cost for histomorphological classification was $29,980 and $72,950 with immunohistochemistry. For histomolecular classification, the first year cost was $246,521, accounting for initial educational learning curve, and $233,461 thereafter, assuming a consistent number of biopsies per year. Targeted implementation of histomolecular classification among high-grade, p53 abnormal and/or MMR-deficient ECs (56% of cases) cost $169,688 in the first year and $162,418 annually thereafter. With a projected 3400 EC biopsies in Ontario in 2022, histomorphological classification would annually cost $537,078 and $1,305,677 with immunohistochemistry. Histomolecular classification would cost $4,410,203 in the first year and $4,176,737 annually once established. Selective molecular classification would lead to a cost of $3,044,178 in the first year and $2,913,443 thereafter. CONCLUSIONS: The study highlights the need for informed decision-making when implementing molecular classification in clinical practice, given the substantial incremental healthcare costs associated with these approaches.

Cost-Effectiveness and Budget Impact of Future Developments With Whole-Genome Sequencing for Patients With Lung Cancer.

OBJECTIVES: This study aimed to investigate the cost-effectiveness, budget impact (BI), and impact of uncertainty of future developments concerning whole-genome sequencing (WGS) as a clinical diagnostic test compared with standard of care (SoC) in patients with locally advanced and metastatic non-small cell lung cancer. METHODS: A total of 3 likely scenarios to take place within 5 years (according to experts) were simulated using a previously developed, peer reviewed, and published decision model. The scenarios concerned "WGS results used for treatment selection" (scenario 1), "WGS-based biomarker for immunotherapy" (scenario 2), and "off-label drug approval for WGS results" (scenario 3). Two diagnostic strategies of the original model, "SoC" and "WGS as a diagnostic test" (base model), were used to compare our scenarios with. Outcomes were reported for the base model, all scenarios separately, combined (combined unweighted), and weighted by likelihood (combined weighted). Cost-effectiveness, BI, and value of information analyses were performed for WGS compared with SoC. RESULTS: Total costs and quality-adjusted life-years for SoC in metastatic non-small cell lung cancer were €149 698 and 1.235. Incremental outcomes of WGS were €1529/0.002(base model), -€222/0.020(scenario 1), -€2576/0.023(scenario 2), €388/0.024(scenario 3), -€5041/0.060(combined unweighted), and -€1715/0.029(combined weighted). The annual BI for adopting WGS for this population in The Netherlands ranged between €682 million (combined unweighted) and €714 million (base model). The consequences of uncertainty amounted to €3.4 million for all scenarios (combined weighted) and to €699 000 for the diagnostic yield of WGS alone (combined weighted). CONCLUSIONS: Our findings suggest that it is likely for WGS to become cost-effective within the near future if it identifies more patients with actionable targets and show the impact of uncertainty regarding its diagnostic yield. Modeling future scenarios can be useful to consider early adoption of WGS while timely anticipating on unforeseen developments before final conclusions are reached.

Health Technology Assessment-Informed Decision Making by the Federal Joint Committee/Institute for Quality and Efficiency in Health Care in Germany and the National Institute for Health and Care Excellence in England: The Role of Budget Impact.

OBJECTIVES: This study aimed to test (official) evaluation criteria including the potential role of budget impact (BI) on health technology assessment (HTA) outcomes published by the Federal Joint Committee (Gemeinsamer Bundesausschuss [GBA]) and the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen [IQWiG]) in Germany as well as the National Institute for Health and Care Excellence (NICE) in England. METHODS: Data were extracted from all publicly available GBA decisions and IQWiG assessments as well as NICE single technology appraisals between January 2011 and June 2018, and information with regard to evaluation criteria used by these agencies was collected. Data were analyzed using logistic regression to estimate the effect of the BI on the HTA outcomes while controlling for criteria used by GBA/IQWiG and NICE. RESULTS: NICE recommendations are largely driven by the incremental cost-effectiveness ratio and, if applicable, by end-of-life criteria (P < .01). While IQWiG assessments are significantly affected by the availability of randomized controlled trials and patient-relevant endpoints (P < .01), GBA appraisals primarily focus on endpoints (P < .01). The BI correlated with NICE single technology appraisals (inverted-U relationship, P < .1) and IQWiG recommendations (increasing linear relationship, P < .05), but not with GBA decisions (P > .1). Nevertheless, given that IQWiG assessments seem to be more rigorous than GBA appraisals regarding the consideration of evidence-based evaluation criteria, decisions by GBA might be negatively associated with the BI. CONCLUSIONS: Results reveal that GBA/IQWiG and NICE follow their official evaluation criteria consistently. After controlling for all significant variables, the BI seems to have an (independent) effect on HTA outcomes as well.

Budget impact analysis of a digital monitoring platform for COPD.

BACKGROUND: Chronic obstructive pulmonary disease (COPD) is a progressive debilitating condition with frequent exacerbations that have a high burden for patients and society. Digital tools may help to reduce the economic burden for patients and payers by improving outcomes. The Propeller platform is a digital self-management tool that facilitates passive monitoring of inhaler medication utilization, potentially assisting the healthcare team to identify patients at risk of a COPD exacerbation who may require further intervention. This study estimated the budget impact of Propeller from commercial payer and Medicare fee-for-service payer perspectives. METHODS: An Excel-based model was used to estimate the budget impact of Propeller for COPD patients in commercial and Medicare population sizes of 5 million members. Data on prevalence, baseline healthcare resource utilization (HCRU), and baseline use of rescue and controller inhaler medications with unit costs (adjusted to 2020 US dollars) were obtained from peer-reviewed literature. Data on reductions in HCRU during Propeller usage were based on direct evidence. Estimates for costs of remote monitoring were obtained from publicly available information. All patients were assumed to have insurance claims related to ongoing remote monitoring. RESULTS: The estimated number of annual eligible COPD patients for commercial and Medicare was 212,200 and 606,600, respectively. Propeller decreased costs by an estimated $2,475 (commercial) and $915 (Medicare) per enrolled patient. The greatest increase in expenditure was for remote monitoring related expenses. After accounting for estimated reductions in hospitalizations, emergency department visits and short-acting beta-agonist use, total net savings were approximately $1.60 and $1.70 per-member per-month for commercial and Medicare payers, respectively. CONCLUSION: Propeller is projected to be cost saving from both the commercial and Medicare payer perspectives.

Exploration of an alternative reconstructed individual patient data-based approach for budget impact analysis of anticancer drugs.

BACKGROUND: The duration of treatment (DOT) of the initial intervention and subsequent treatment is the key to determining the accuracy of anticancer-drug budget impact analysis (BIA) calculations. However, existing studies only use simple assumptions as a proxy for DOT, resulting in a high degree of bias. OBJECTIVES: To enhance the accuracy and reliability of anticancer-drug BIA and solve the problem regarding DOT, we propose an alternative individual patient data (IPD)-based approach that reconstructs IPD from the published Kaplan Meier survival curves to estimate DOT. METHODS: We developed a four-step methodological framework for this new approach, taking the use of pembrolizumab in treating microsatellite-instability-high (MSI-H) advanced colorectal cancer as an example: (1) reconstructing the IPD; (2) calculating the total DOT of the initial intervention and subsequent treatment for each patient; (3) assigning a randomized time and DOT; and (4) multiple replacement sampling and calculation of the mean value. RESULTS: Using this approach, the average DOT for the initial intervention and subsequent treatment in each year of the BIA time horizon can be calculated and used to calculate the resources consumed and costs in each year. In our example, the average DOT for the initial intervention with pembrolizumab from the first to the fourth year was 4.90, 6.60, 5.24, and 5.06 months, respectively, while the average DOT for subsequent treatment was 0.75, 2.84, 2.99, and 2.50 months, respectively. CONCLUSIONS: The reconstructed IPD-based approach can improve the accuracy and reliability of anticancer-drug BIA compared with conventional methods, and can be widely used, especially for anticancer drugs with excellent efficacy.

Costs associated with the use of multigene panel tests in three solid tumor types and the impact on insurance premiums.

Aim: This study assessed the costs associated with multigene panel tests (MGPTs) in the USA and the impact of coverage on insurance premiums. Materials & methods: We conducted a retrospective claims analysis to estimate total patient costs associated with MGPT use in three solid tumors: advanced non-small-cell lung cancer, advanced melanoma and metastatic colorectal cancer. A decision analytic model was constructed to estimate the premium impact of a 1 million member commercial health plan. Results: In all three tumor types, mean total costs for patients receiving or not receiving MGPTs were not significantly different (p > 0.05). The estimated change in premiums per enrollee per month was estimated to be US$0.040. Conclusion: MGPTs were not associated with higher costs and coverage is expected to have minimal impact on insurance premiums.

Costs & premiums for tests of biomarkers We examined whether using tests for many biomarkers at once in three cancer types would lead to higher costs and increase insurance premiums for patients with private insurance. Using real-world data, we found that use of these tests was not linked to higher costs. Furthermore, we estimated that insurance coverage of these tests would result in small changes to insurance premiums.

Effectiveness and cost-effectiveness of MicroShunt implantation versus standard trabeculectomy for open-angle glaucoma (a SIGHT study): study protocol of a multicentre randomised controlled trial.

BACKGROUND: Trabeculectomy is the "gold standard" initial surgical procedure for open-angle glaucoma worldwide. During the last decade, the introduction of less invasive procedures, including new bleb-forming surgery such as the MicroShunt, has altered the approach of glaucoma management. At present, there is insufficient evidence comparing the effectiveness between these procedures nor versus trabeculectomy. Furthermore, there is no data available on patient impact and cost-effectiveness. This study aims to address this gap in evidence and establish whether MicroShunt implantation is non-inferior compared to trabeculectomy with regard to effectiveness and whether it is cost-effective. METHODS: A multicentre, non-inferiority, randomised controlled trial (RCT) studying open-angle glaucoma with an indication for surgery will be conducted. Patients with previous ocular surgery except for phacoemulsification are excluded, as are patients with ocular comorbidity compromising the visual field or requiring a combined procedure. After informed consent is obtained, patients will be randomly allocated to the intervention, a PRESERFLO™ MicroShunt implantation, or the control group, trabeculectomy, using block randomisation (blocks of 2, 4 or 6 patients). In total, 124 patients will be randomised in a 1:1 ratio, stratified by centre. The primary endpoint will be intraocular pressure (IOP) one year after surgery. Secondary outcomes include IOP-lowering medication use, treatment failure, visual acuity, visual field progression, additional interventions, adverse events, patient-reported outcome measures (PROMs), and cost-effectiveness. Study outcomes will be measured up to 12 months postoperatively. DISCUSSION: This study protocol describes the design of a multicentre non-inferiority randomised controlled trial. To this date, cost-effectiveness studies evaluating the MicroShunt have not been undertaken. This multicentre RCT will provide more insight into whether MicroShunt implantation is non-inferior compared to standard trabeculectomy regarding postoperative IOP and whether MicroShunt implantation is cost-effective. TRIAL REGISTRATION: ClinicalTrials.gov, Identifier: NCT03931564 , Registered 30 April 2019.

Economics and Precision Medicine.

Introducing precision medicine strategies into routine practice will require robust economic evidence. Decision-makers need to understand the value of a precision medicine strategy compared with alternative ways to treat patients. This chapter describes health economic analysis techniques that are needed to generate this evidence. The value of any precision medicine strategy can be demonstrated early to inform evidence generation and improve the likelihood of translation into routine practice. Advances in health economic analysis techniques are also explained and their relevance to precision medicine is highlighted. Ensuring that constraints on delivery are resolved to increase uptake and implementation will improve the value of a new precision medicine strategy. Empirical methods to quantify stakeholders' preferences can be effective to inform the design of a precision medicine intervention or service delivery model. A range of techniques to generate relevant economic evidence are now available to support the development and translation of precision medicine into routine practice. This economic evidence is essential to inform resource allocation decisions and will enable patients to benefit from cost-effective precision medicine strategies in the future.

Cost-utility and budget impact analyses of cervical cancer screening using self-collected samples for HPV DNA testing in Thailand.

INTRODUCTION: Cervical cancer ranks as the third most prevalent cancer among women in Thailand. However, the effectiveness of cervical cancer screening programs is limited by several factors that impede the screening rate. The utilization of self-collected samples for screening purposes has the potential to alleviate barriers to screening in Thai women. This study assessed the cost-utility and budget impact of implementing cervical cancer screening using self-collected samples for human papillomavirus (HPV) deoxyribonucleic acid (DNA) testing in Thailand. MATERIALS AND METHODS: We employed a decision tree integrated with a Markov model to estimate the lifetime costs and health benefits associated with the cervical cancer screening program for women aged 25-65. The analysis was conducted from a societal perspective. Four screening policy options were compared: (1) additional self-collected samples for HPV DNA testing, (2) clinician-collected samples for HPV DNA testing only, (3) clinician-collected samples for cytology test (i.e., status quo), and (4) no screening. The model inputs were based on unvaccinated women. The screening strategies and management in those with positive results were assumed followed to the Thai clinical practice guideline. Costs were reported in 2022 Thai baht. Sensitivity analyses were conducted. The ten-year budget impacts of the additional self-collected samples for HPV DNA testing were calculated from a payer perspective. RESULTS: All screening policies were cost-saving compared to no screening. When comparing the additional self-collected samples for HPV DNA testing with the clinician-collected samples policy, it emerged as the dominant strategy. The incremental benefit in cervical cancer prevention achieved by incorporating self-collected samples for screening was observed at any additional screening rate that could be achieved through their use. Sensitivity analyses yielded consistently favorable results for the screening policies. The average annual budget impact of the additional self-collected samples for screening policy amounted to 681 million Thai baht. This budget allocation could facilitate cervical cancer screening for over 10 million women. CONCLUSIONS: An addition of self-collected samples for HPV DNA testing into the cervical cancer screening program is cost-saving. The benefits of this screening policy outweigh the associated incremental costs. Policymakers should consider this evidence during the policy optimization process.