Indirect Medical Costs Research Articles

Study on the disease burden of patients with mucopolysaccharidosis type II in China

BackgroundIn this study, we investigated the patient population of Mucopolysaccharidosis type II (MPS II) in China, understood the basic situation, prevalence and diagnosis and treatment status of the patients, as well as the economic burden of the patients, and analyzed the influencing factors.MethodsA cross-sectional study focusing on patients with MPS II was conducted in China in 2023. Participants in the study were drawn from the Beijing Zhengyu Mucopolysaccharide Rare Disease Care Center, which is the only non-profit organization in mainland China registered with the civil affairs department that focuses on mucopolysaccharidosis. Data were collected through an online questionnaire, which included basic patient information, disease status, self-assessment of quality of life, diagnosis and treatment, as well as direct and indirect medical costs. The demographic and diagnosis and treatment profile of patients were analyzed by descriptive statistics. Furthermore, univariate and multiple linear regression were used to explore the economic burden and influencing factors of patients with MPS II.ResultsThe survival data of 145 patients were collected, the majority (98.62%) were male, and 78 were less than or equal to 10 years old. All patients were covered by medical insurance, mainly urban residents (135 cases). In terms of expenses, the 124 patients in the year before the survey incurred a total cost of about 14.7895 million yuan, and the direct economic burden accounted for 87.19%. Univariate analysis showed that age, number of hospitalizations, length of hospital stay, number of outpatient/emergency departments, hematopoietic stem cell transplantation (HSCT), and enzyme replacement therapy (ERT) were significantly associated with the economic burden of disease. Multiple regression analysis showed that the number of hospitalizations, days of hospitalization, number of outpatient/emergency departments and HSCT treatment were the main influencing factors.ConclusionsThis study found that patients with MPS II were difficult to diagnose and easily misdiagnosed, their physical functions were impaired in many aspects. The existing treatment options are not sufficient in terms of economy and effectiveness, and there is also a lack of corresponding policy guarantees and support, which makes patients and their families have to face huge financial pressure.

Economic Burden of Late-Stage Age-Related Macular Degeneration in Bulgaria, Germany, and the US

Age-related macular degeneration (AMD) is a highly prevalent and debilitating retinal condition that affects more than 200 million people globally, with the severe late-stage forms-neovascular AMD (nAMD) and geographic atrophy (GA)-affecting more than 11 million people globally. However, much is unknown about the economic burden of the disease. To estimate the economic burden associated with late-stage AMD in Bulgaria, Germany, and the US at the societal level. This study used the prevalence approach cost-of-illness economic modeling to estimate costs attributable to late-stage AMD. Data on health care resource utilization, well-being, and productivity were obtained via primary data collection. Additional data required for the model were sourced from available published literature. Data were collected from January 2021 to March 2022, and analyzed from April to July 2022. Participants older than 50 years residing in Bulgaria, Germany, and the US who were diagnosed with late-stage AMD (nAMD or any form of GA) in 1 or both eyes and caregivers who care for people diagnosed with late-stage AMD were recruited through ophthalmological clinics in Bulgaria and Germany and via online newsletters and social media in the US. The main outcomes were direct medical costs (disease-related health care expenditures), indirect medical costs (care support and assistive technology), well-being cost (loss of well-being), and productivity cost (loss in productivity due to the diseases for both patient and caregivers). Of the 128 individuals with late-stage AMD in this study, 80 (62%) were female, and 120 (94%) were aged 60 years or older. Of the 61 caregivers, 43 (70%) were female and 55 (91%) were aged 45 years or older. Estimated per-annum total costs attributable to late-stage AMD were €449.5 million ($512.5 million) in Bulgaria, €7.6 billion ($8.6 billion) in Germany, and €43.2 billion ($49.4 billion) in the US. Across all countries, 10% to 13% of the total cost incurred was attributed to direct medical costs. In Germany and Bulgaria, the biggest contributor to the total economic burden was reduced well-being (67% and 76%, respectively), whereas in the US, loss of productivity (42%) was the biggest contributor. The findings of this study indicate a substantial burden of late-stage AMD on patients and caregivers in the US, Germany, and Bulgaria. Across the 3 countries, reduced well-being and loss of productivity were relatively large contributors to the total economic burden. Implementing measures to reduce AMD incidence, delay disease progression, and alleviate humanistic burden may help reduce the economic burden of late-stage AMD.

Matched Comparison Examining the Effect of Obesity on Clinical, Economic, and Humanistic Outcomes in Patients with BipolarI Disorder.

BipolarI disorder (BD-I) is associated with an increased risk of obesity, but few studies have evaluated the real-world clinical, humanistic, and economic effects associated with obesity in people with BD-I. This was a retrospective, cross-sectional analysis of responses to the 2016 and 2020 National Health and Wellness surveys. Respondents (18-64years) with a self-reported physician diagnosis of BD-I were matched to controls without BD-I based on demographic and health characteristics. Respondents were categorized by body mass index as underweight/normal weight (< 25kg/m2), overweight (25 to < 30kg/m2), or obese (≥ 30kg/m2). Multivariable regression models were used to compare obesity-related comorbidities, healthcare resource utilization (HCRU), health-related quality of life (HRQoL), work productivity, and indirect and direct costs. Before matching, the BD-I cohort was younger than the non-BD-I cohort and included more female and white respondents and a greater proportion covered by Medicaid or Medicare. After matching, the BD-I and non-BD-I cohorts had similar characteristics. A total of 5418 respondents (BD-I, n = 1806; matched controls, n = 3612) were analyzed. Obese respondents with BD-I reported the highest adjusted prevalences of high blood pressure (50%), high cholesterol (35%), sleep apnea (27%), osteoarthritis (17%), type2 diabetes (12%), and liver disease (4%). Obesity in respondents with BD-I was associated with the lowest HRQoL scores. Measures of work impairment were highest in respondents with BD-I and obesity, as was HCRU. Respondents with BD-I and obesity had the highest associated total indirect and direct medical costs ($25,849 and $44,482, respectively). Obese respondents with BD-I had greater frequencies of obesity-related comorbidities, higher HCRU, lower HRQoL, greater work impairments, and higher indirect and direct medical costs. These findings highlight the real-world burden of obesity in people with BD-I and the importance of considering treatments that may reduce this burden.

Determining direct, indirect healthcare and social costs for diabetic retinopathy management: a systematic review

IntroductionDiabetic retinopathy (DR) is a rapidly growing global public health threat; it affects 1 in 3 people with diabetes and is still the leading cause of blindness among the working-age population. The management of diabetic retinopathy is becoming more advanced and effective but is highly expensive compared to other ocular diseases.AimTo report direct medical, indirect medical, and nonmedical costs of diabetic retinopathy in developed and developing countries through a systematic review.MethodsRelated articles published in the PubMed, Google Scholar, and EMBASE electronic databases from 1985 to 2022 were identified using the keywords direct medical and indirect medical and social costs of diabetic retinopathy. However, previous systematic reviews, abstracts, and case reports were excluded.ResultsThirteen articles were eligible for assessing the economic burden of diabetes management and its complications. Our analysis revealed that increasing prevalence and severity of diabetic retinopathy (DR) are associated with higher direct and indirect healthcare expenditures. The impact of DR on working-age adults, leading to irreversible blindness in advanced stages, underscores the urgent need for cost-effective prevention and management strategies.DiscussionThis study systematically reviewed the direct medical, indirect medical, and nonmedical costs of DR in developed and developing countries. Our findings highlight the significant economic burden of DR, emphasizing the importance of implementing effective prevention and management measures to alleviate costs and enhance patient outcomes.ConclusionThe substantial financial burden of DR necessitates a re-evaluation of current screening and management programs. Revision of these programs is crucial to improve quality of care, reduce costs, and ultimately achieve Sustainable Development Goal 3, which aims to ensure good health and well-being for all.

The cost of care for children hospitalized with respiratory syncytial virus (RSV) associated lower respiratory infection in Kenya

BackgroundRespiratory syncytial virus (RSV) is one of the main causes of hospitalization for lower respiratory tract infection in children under five years of age globally. Maternal vaccines and monoclonal antibodies for RSV prevention among infants are approved for use in high income countries. However, data are limited on the economic burden of RSV disease from low- and middle-income countries (LMIC) to inform decision making on prioritization and introduction of such interventions. This study aimed to estimate household and health system costs associated with childhood RSV in Kenya.MethodsA structured questionnaire was administered to caregivers of children aged < 5 years admitted to referral hospitals in Kilifi (coastal Kenya) and Siaya (western Kenya) with symptoms of acute lower respiratory tract infection (LRTI) during the 2019–2021 RSV seasons. These children had been enrolled in ongoing in-patient surveillance for respiratory viruses. Household expenditures on direct and indirect medical costs were collected 10 days prior to, during, and two weeks post hospitalization. Aggregated health system costs were acquired from the hospital administration and were included to calculate the cost per episode of hospitalized RSV illness.ResultsWe enrolled a total of 241 and 184 participants from Kilifi and Siaya hospitals, respectively. Out of these, 79 (32.9%) in Kilifi and 21(11.4%) in Siaya, tested positive for RSV infection. The total (health system and household) mean costs per episode of severe RSV illness was USD 329 (95% confidence interval (95% CI): 251–408 ) in Kilifi and USD 527 (95% CI: 405– 649) in Siaya. Household costs were USD 67 (95% CI: 54–80) and USD 172 (95% CI: 131– 214) in Kilifi and Siaya, respectively. Mean direct medical costs to the household during hospitalization were USD 11 (95% CI: 10–12) and USD 67 (95% CI: 51–83) among Kilifi and Siaya participants, respectively. Observed costs were lower in Kilifi due to differences in healthcare administration.ConclusionsRSV-associated disease among young children leads to a substantial economic burden to both families and the health system in Kenya. This burden may differ between Counties in Kenya and similar multi-site studies are advised to support cost-effectiveness analyses.

COST OF ILLNESS ANALYSIS OF EPILEPSY OUTPATIENTS USING VALPROIC ACID, PHENYTOIN AND CARBAMAZEPINE THERAPY IN HOSPITAL IN INDONESIA

Epilepsy is a condition of the onset of attacks in the form of abnormal, irregular brain nerve cell movements, occurring repeatedly and causing temporary motor, sensory or mental function disorders. The treatment of epilepsy has costs that must be known, including direct medical costs, indirect medical costs, and indirect costs; this study aims to determine the cost of illness of epilepsy in one hospital in banyuasin regency with retrospective costing in outpatients. The research method used in this study is a cross-sectional study using patient medical record data and cost data taken from BPJS claims data and hospital rate patterns. This study uses the BPJS perspective and societal perspective, the cost data taken from the BPJS perspective is the result of BPJS claims according to the INACBGs. Package tariff and for the socital perspective the data taken is doctor consultation data, administrative costs, laboratory costs, drug costs and transportation costs and lost productivity costs carried out by interview method for 12 months. The results of this study indicate that the cost of epilepsy outpatients from a societal perspective is Rp.3,141,414 and for BPJS perspective is Rp.2,338,567.

Global perspective on nonalcoholic fatty liver disease and nonalcoholic steatohepatitis - prevalence, clinical impact, economic implications and management strategies.

The metabolically-based liver disease, nonalcoholic fatty liver disease (NAFLD), is the most common cause of chronic liver disease currently affecting 38% of the world's adult population. NAFLD can be progressive leading to nonalcoholic steatohepatitis (NASH), liver transplantation, liver cancer, liver-related mortality and is associated with decreased quality of life from impaired physical functioning and increased healthcare resource utilisation. However, screening for NAFLD is cost-prohibitive but screening for high risk NAFLD (NAFLD with F2 fibrosis or greater) is imperative. To review the global perspective on NAFLD and NASH METHODS: We retrieved articles from PubMed using search terms NAFLD, prevalence, clinical burden, economic burden and management strategies. NAFLD/NASH shows geographical variation across the globe. Highest prevalence rates are in South America and the Middle East and North Africa; lowest prevalence is in Africa. NAFLD's economic impact is from direct and indirect medical costs and loss in worker productivity. It is projected that, over the next two decades, the total cost of NAFLD and diabetes will exceed $1.5 trillion (USD). Risk stratification algorithms identifying "high risk NAFLD" were made following non-invasive tests for NAFLD identification and fibrosis development. These algorithms should be used in primary care and endocrinology settings so timely and appropriate interventions (lifestyle and cardiometabolic risk factor management) can be initiated. To reduce the burgeoning burden of NAFLD/NASH, management should include risk stratification algorithms for accurate identification of patients, linkage to appropriate settings, and initiation of effective treatment regimens.

Economic burden of systemic lupus erythematosus in Malaysia

Introduction Our cost-of-illness (COI) model adopted the perspective of both payer and society over a time horizon of 5 years to measure the economic burden of systemic lupus erythematosus (SLE) in Malaysia. Methodology Our COI model utilized a prevalence-based model to estimate the costs and economic consequences of SLE in Malaysia. The clinical parameters were obtained from published literature and validated using the Delphi panel. Direct and indirect medical costs were measured, including disease management, transient events, and indirect costs. One-way sensitivity analysis was also performed. Results The number of target Malaysian patients with SLE in the COI model was 18,121. At diagnosis, the numbers of SLE patients with mild, moderate, and severe phenotypes were 2,582, 13,897, and 1,642, respectively. The total SLE cost in Malaysia over 5 years from both payer and society perspectives was estimated at MYR 678 million and 2 billion, respectively. The results showed a considerable cost burden due to productivity losses resulting from SLE-related morbidity and mortality. Over a 5-year time horizon, the costs per patient per year from the payer and society perspectives were MYR 7,484 ($4766) and 24,281($15,465), respectively. Conclusion Our study demonstrated the substantial economic burden of SLE in Malaysia over a time horizon of 5 years. It affects adults of working age, in addition to the costs of SLE management and its consequences, such as flares, infection, and organ damage. Our COI model indicated that disease management costs among patients with higher disease severity were higher than those among patients with a mild phenotype. Hence, more attetion should be paid to limiting the progression of SLE and the occurrence of flares, with the need for further economic evaluation of novel treatments that could lead to better outcomes.

Cost of Illness Analysis of Invasive Meningococcal Disease Caused by Neisseria Meningitidis Serogroup B in the Netherlands-a Holistic Approach.

Invasive meningococcal disease (IMD) caused by Neisseria meningitidis is a rapidly progressing, rare disease that often presents as meningitis or sepsis. It mostly affects infants and adolescents, with high fatality rates or long-term sequelae. In the Netherlands, serogroup B (MenB) is most prevalent. We aimed to estimate the economic burden of MenB-related IMD between 2015 and 2019, including direct and indirect medical costs from short- and long-term sequelae, from a societal perspective. IMD incidence was based on laboratory-based case numbers from the Netherlands Reference Laboratory for Bacterial Meningitis (Amsterdam UMC, Amsterdam, the Netherlands); there were 74 MenB cases on average per year in the study period 2015-2019. Case-fatality rate (3.8%) and percentage of patients discharged with sequelae (46%) were derived from literature. Direct costs included treatment costs of the acute phase, long-term sequelae, and public health response. Indirect costs were calculated using the human capital (HCA) and friction costs (FCA) approaches, in which productivity losses were estimated for patients and parents during the acute and sequelae phases. Costs were discounted by 4% yearly. Estimated costs due to MenB IMD in an annual cohort were €3,094,199 with FCA and €9,480,764 with HCA. Direct costs amounted to €2,974,996, of which 75.2% were related to sequelae. Indirect costs related to sequelae were €52,532 with FCA and €5,220,398 with HCA. Our analysis reflects the high economic burden of MenB-related IMD in the Netherlands. Sequelae costs represent a high proportion of the total costs. Societal costs were dependent on the applied approach (FCA or HCA).

The Economic Costs of Tobacco Related Illnesses in Kenya.

To estimate the economic costs of selected tobacco-related illnesses (TRI) in Kenya in 2022. This study was conducted in 2 phases. Phase 1, conducted between 2021 and 2022, entailed conducting a cross-sectional study conducted in 4 national public referral hospitals in Kenya. Patients with cardiovascular disease, cancer, chronic obstructive pulmonary disease, or tuberculosis were interviewed to compute the indirect and direct medical costs related to the illness. Activity-Based Costing approach was used to capture costs for services along the continuum of care pathway. In the second phase, the Tobacco Attributable Factor was used to estimate the direct, indirect, and ultimately economic cost due to tobacco smoking. The estimated health care cost attributed to tobacco use in Kenya is US$396,107,364. Among TRIs included in the study, myocardial infarction had the highest health care cost at US$158,687,627, followed by peripheral arterial disease and stroke with health care cost of US$64,723,181 and US$44,746,700 respectively. The main cost driver across all the illnesses is the cost for medication accounting for over 90% of the total health care cost. The productivity losses from the diseases ranged between US$148 to US$360 and accounted for 27% to 48% of the economic costs. The total cost attributable to tobacco use to Kenya's economy for the selected TRIs was between US$544.74 million and US$756.22 million. Tobacco related illnesses impose a significant economic burden as reported for direct and indirect costs. These findings underscore the need for strengthened implementation of the provision of the Framework Convention on Tobacco Control and the Tobacco Control Act (2007) to facilitate a reduction in tobacco consumption in the population.

Risk Prediction and Management of Chronic Kidney Disease in People Living with Type 2 Diabetes Mellitus.

People with type 2 diabetes mellitus have increased risk of chronic kidney disease and atherosclerotic cardiovascular disease. Improved care delivery and implementation of guideline-directed medical therapy have contributed to the declining incidence of atherosclerotic cardiovascular disease in high-income countries. By contrast, the global incidence of chronic kidney disease and associated mortality is either plateaued or increased, leading to escalating direct and indirect medical costs. Given limited resources, better risk stratification approaches to identify people at risk of rapid progression to end-stage kidney disease can reduce therapeutic inertia, facilitate timely interventions and identify the need for early nephrologist referral. Among people with chronic kidney disease G3a and beyond, the kidney failure risk equations (KFRE) have been externally validated and outperformed other risk prediction models. The KFRE can also guide the timing of preparation for kidney replacement therapy with improved healthcare resources planning and may prevent multiple complications and premature mortality among people with chronic kidney disease with and without type 2 diabetes mellitus. The present review summarizes the evidence of KFRE to date and call for future research to validate and evaluate its impact on cardiovascular and mortality outcomes, as well as healthcare resource utilization in multiethnic populations and different healthcare settings.

Socioeconomic per-case costs of stroke, myocardial infarction, and preterm birth attributable to air pollution in Sweden.

Incident cases of stroke, myocardial infarction, and preterm birth have established exposure-response functions associated with air pollution. However, there are no studies reporting detailed costs per case for these health outcomes that are adapted to the cost-benefit tools that guide the regulation of air pollution. The primary objective was to establish non-fatal per-case monetary estimates for stroke, myocardial infarction, and preterm birth attributable to air pollution in Sweden, and the secondary objective was to ease the economic evaluation process of air pollution morbidity effects and their inclusion in cost-benefit assessments. Based on recommendations from the literature, the case-cost analysis considered direct and indirect medical costs, as well as production losses and informal costs relevant for the calculation of the net present value. A literature search was conducted to estimate the costs of each category for each incident case in Sweden. Informal costs were estimated using the quality-adjusted life-years approach and the corresponding willingness-to-pay in the Swedish population. The total average per-case cost was estimated based on specific health outcome durations and severity and was discounted by 3.5% per year. Sensitivity analysis included varying discount rates, severity of health outcome, and the range of societal willingness to pay for quality-adjusted life years. The average net present value cost estimate was €2016 460k (185k-1M) for non-fatal stroke, €2016 24k (16k-38k) for myocardial infarction, and €2016 34k (19k-57k) for late preterm birth. The main drivers of the per-case total cost estimates were health outcome severity and societal willingness to pay for risk reduction. Varying the discount rate had the largest effect on preterm birth, with costs changing by ±30% for the discount rates analysed. Because stroke, myocardial infarction, and preterm birth have established exposure-response functions linking these to air pollution, cost-benefit analyses should include the costs for these health outcomes in order to adequately guide future air pollution and climate change policies.

Subjective and objective financial toxicity among colorectal cancer patients: a systematic review

BackgroundColorectal cancer (CRC) is the third most common cancer type worldwide. Colorectal cancer treatment costs vary between countries as it depends on policy factors such as treatment algorithms, availability of treatments and whether the treatment is government-funded. Hence, the objective of this systematic review is to determine the prevalence and measurements of financial toxicity (FT), including the cost of treatment, among colorectal cancer patients.MethodsMedline via PubMed platform, Science Direct, Scopus, and CINAHL databases were searched to find studies that examined CRC FT. There was no limit on the design or setting of the study.ResultsOut of 819 papers identified through an online search, only 15 papers were included in this review. The majority (n = 12, 80%) were from high-income countries, and none from low-income countries. Few studies (n = 2) reported objective FT denoted by the prevalence of catastrophic health expenditure (CHE), 60% (9 out of 15) reported prevalence of subjective FT, which ranges from 7 to 80%, 40% (6 out of 15) included studies reported cost of CRC management– annual direct medical cost ranges from USD 2045 to 10,772 and indirect medical cost ranges from USD 551 to 795.ConclusionsThere is a lack of consensus in defining and quantifying financial toxicity hindered the comparability of the results to yield the mean cost of managing CRC. Over and beyond that, information from some low-income countries is missing, limiting global representativeness.

Modelling the Potential Public Health Impact of Different COVID-19 Vaccination Strategies with an Adapted Vaccine in Singapore

ABSTRACT Background Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) causing COVID-19 has been a dynamically changing virus, requiring the development of adapted vaccines. This study estimated the potential public health impact alternative vaccination strategies for COVID-19 in Singapore. Research Design and Methods The outcomes of alternative vaccination strategies with a future adapted vaccine were estimated using a combined Markov decision tree model. The population was stratified by high- and standard-risk. Using age-specific inputs informed by local surveillance data and published sources, the model estimated health (case numbers, hospitalizations, and deaths) and economic (medical costs and productivity losses) outcomes in different age and risk subpopulations. Results Booster vaccination in only the elderly and high-risk subpopulation was estimated to avert 278,614 cases 21,558 hospitalizations, 239 deaths, Singapore dollars (SGD) 277 million in direct medical costs, and SGD 684 million in indirect medical costs. These benefits increased as vaccination was expanded to other subpopulations. Increasing the booster vaccination coverage to 75% of the standard-risk population averted more deaths (3%), hospitalizations (29%), infections (145%), direct costs (90%), and indirect costs (192%) compared to the base case. Conclusions Broader vaccination strategies using an adapted booster vaccine could have substantial public health and economic impact in Singapore.

Costs associated with informal health care pathway for patients with suspected Lyme borreliosis

ObjectivesTo compare the direct and indirect medical costs for patients with suspected Lyme borreliosis according to whether or not they had used an informal care pathway. Patients and methodsWe retraced the care pathways of participating patients by a prospective questionnaire survey and a retrospective analysis of care records. Direct and indirect costs were estimated using a micro-costing method from different perspectives. We compared the costs of patients who had consulted a “Lyme Doctor” (informal care pathway) with those who had only used the formal care pathway. Non-parametric tests were appraised the significance of the differences between the two groups of patients. ResultsOut of 103 eligible patients, 49 (including 12 having used an informal health care pathway) agreed to be investigated. Five expenditure items entirely borne by patients were significantly higher for patients following an informal care pathway: productivity loss (3041 ± 6580 vs 194 ± 1177 euros, p = 0.01), alternative therapies (3484 ± 7308 vs 369 ± 956 euros), biological tests sent abroad (571 ± 1415 vs 17 ± 92 euros, p < 0.01), self-medication (918 ± 1998 vs 133 ± 689, p = 0.02) and transport (3 094 ± 3456 vs 1 123 ± 1903p = 0.01). ConclusionsFrom the patient’s standpoint, the informal care pathway involving consultation with a Lyme Doctor is far more expensive than the formal care pathway. More specifically, the patient has to bear the costs of alternative treatments and repeated, non-recommended examinations.

Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States

Introduction: Oral complement inhibition therapy has the potential to transform the care of patients with paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening hematological disorder characterized by intravascular hemolysis, thrombosis, and bone marrow failure. Current standard-of-care (SOC) for PNH are monoclonal antibodies targeting the distal complement component 5 (C5) and include the intravenous agents eculizumab and ravulizumab. However, this distal mechanism of action allows extravascular hemolysis to persist. Intravenous therapy also requires patient and nursing time spent for infusion care. Iptacopan is one oral proximal complement pathway inhibitor that selectively inhibits complement factor B. Clinical trial data shows that iptacopan controls both intravascular and extravascular hemolysis, resulting in increased transfusion independence. Iptacopan was granted breakthrough therapy designation for expedited review by the FDA. The potential approval is anticipated by the end of 2023, after which point pricing will be set. A cost-effective price for iptacopan is currently not known. We sought to fill this gap by conducting the first cost-effectiveness analysis of oral versus intravenous complement inhibitor therapy in PNH to determine a cost-effective monthly price threshold for iptacopan monotherapy and to estimate patient and nursing time saved with transition to oral therapy. Methods: In this independent analysis free of industry influence, we built a Markov model to first determine the cost-effectiveness of iptacopan monotherapy at a monthly cost equivalent to SOC. Second, we solved for the cost-effective monthly maximum threshold cost of iptacopan across accepted willingness-to-pay thresholds in US dollars per quality-adjusted life-year (QALY). Transition probabilities for transfusion burden and breakthrough hemolysis were informed by the phase III study results (NCT 04558918). Probabilities of transfusion-associated adverse events were sourced from hemovigilance reporting in the 2015 National Blood Collection and Utilization Survey. Costs were assessed in 2023 US dollars and were informed from the Centers for Medicare &amp; Medicaid Services and PNH-specific health resource utilization literature. The latter accounted for country- and PNH-specific direct and indirect medical costs, derived after adjustment for baseline characteristics, and isolated for transfusion-dependent treatment-experienced patients with PNH by comparison to transfusion-independent, treatment-experienced patients with PNH. Effectiveness was calculated in quality-adjusted life-years (QALY), and employed PNH-specific utilities. The two primary outcomes for iptacopan monotherapy were 1) the incremental cost-effectiveness ratio (ICER) or, the incremental net monetary benefit (iNMB) if the therapy was found to be cost saving, and 2) the cost-effective maximum monthly threshold price of iptacopan monotherapy, as compared to the SOC. The secondary outcome was the aggregated patient and nursing time saved with the use of oral versus intravenous treatment. We concluded with deterministic sensitivity analysis to isolate parameters that the model results are most sensitive to, and probabilistic sensitivity analysis to capture uncertainty in all parameters simultaneously over 10,000 Monte Carlo simulations. Results: In the base-case (i.e., where monthly price of iptacopan is equivalent to SOC), therapy with iptacopan versus C5 inhibition accrued 15.6 and 13.5 QALYs at costs of $9.7 million and $14.0 million, respectively. The iNMB with iptacopan was $4.4 million (95% credible interval $2.7-6.5 million) and threshold analysis for the maximum monthly price of iptacopan was $61,000. Iptacopan therapy projects to save patients and nurses approximately 730 and 2920 hours for ravulizumab and eculizumab, respectively, in PNH-specific care averted. Model results were most sensitive to iptacopan price. Conclusion: If the monthly price is no more than 149% of SOC, iptacopan monotherapy can be a cost-effective therapeutic option for transfusion-dependent, treatment-experienced patients with PNH. This is achieved by increasing the proportion of transfusion-independence in this patient population and, in turn, decreasing the burden of both the risks and costs associated with health resource utilization for patients with PNH.

Cost-Effectiveness of Health Literacy Enhancement Intervention on Patients with Type II Diabetes.

Type II diabetes is considered a chronic disease that influences the affected person's quality of life and imposes a high economic burden on the patient and society. The enhancement of health literacy seems essential for self-management and disease control in patients with type II diabetes. Consequently, this study was performed to evaluate the cost-effectiveness of enhanced health literacy in type II diabetes patients. This study was of quantitative and economic evaluation type. A population of 232 patients was selected among those referred to the Tafihan Shiraz clinic based on the quality of entry and exit. The health literacy educational intervention was carried out for three months. In order to collect information, researchers used the SF36 questionnaire and the checklist of costs. The Cost Effectiveness Ratio (CER) and Incremental Cost Effectiveness Ratio (ICER) were calculated. Moreover, the costs of each unit of increasing the quality of life before and after the intervention were calculated. A decision was made to determine the cost-effectiveness of the intervention. According to the results, 40% of the participants were females, and 34.5% were 30-40 years old. Examination of the quality of life in patients before the intervention indicated that the mean and standard deviation of the patients' quality of life before the intervention was 18.43±44.99, and the mean and standard deviation of the quality of life after the intervention was 49.57±16.21. Moreover, the patients' quality of life increased after the educational intervention. The total direct medical costs were $717,484 and $685,620 before and after the intervention, respectively. The total indirect medical costs were $604,122 and $493,011 before and after the intervention, respectively. Moreover, the total indirect costs were $122,535 and $122,119 before and after the intervention, respectively. The study was cost-effective with CER=0.9 and ICER= - 140,000 per increase in the quality of life. Improving health literacy can have a range of benefits and improve the quality of life of patients with type II diabetes and reduce their treatment costs, and thereby, this may be seen as an effective step toward the recovery of patients with type II diabetes.

Modelling the potential public health impact of different vaccination strategies with an omicron-adapted bivalent vaccine in Thailand

ABSTRACTBackgroundSevere acute respiratory syndrome coronavirus 2 (SARS-CoV-2) causing COVID-19 has continuously evolved, requiring the development of adapted vaccines. This study estimated the impact of the introduction and increased coverage of an Omicron-adapted bivalent booster vaccine in Thailand.Research Design and MethodsThe outcomes of booster vaccination with an Omicron-adapted bivalent vaccine versus no booster vaccination were estimated using a combined cohort Markov decision tree model. The population was stratified into high- and standard-risk subpopulations. Using age-specific inputs informed by published sources, the model estimated health (case numbers, hospitalizations, and deaths) and economic (medical costs and productivity losses) outcomes in different age and risk subpopulations.ResultsBooster vaccination in only the elderly and high-risk subpopulation was estimated to avert 97,596 cases 36,578 hospitalizations, 903 deaths, THB 3,119 million in direct medical costs, and THB 10,589 million in indirect medical costs. These benefits increased as vaccination was expanded to other subpopulations. Increasing the booster vaccination coverage to 75% of the standard-risk population averted more deaths (95%), hospitalizations (512%), infections (782%), direct costs (550%), and indirect costs (687%) compared to the base case.ConclusionsBroader vaccination with an Omicron-adapted bivalent booster vaccine could have significant public health and economic benefits in Thailand.

Clinical, Economic, and Humanistic Outcomes Associated with Obesity Among People with Bipolar I Disorder in the United States: Analysis of National Health and Wellness Survey Data.

People living with bipolar I disorder (BD-I) have an increased risk for obesity compared with the general population that may be related to genetic, lifestyle, and treatment factors. Few studies have examined possible effects of obesity on those living with BD-I. This study examined relationships between obesity and clinical, humanistic, and economic outcomes among adults with BD-I. This retrospective, cross-sectional study analyzed survey responses from a nationally representative sample of US adults participating in the 2016 or 2020 National Health and Wellness Survey. Respondents (18-64 years) with a self-reported physician diagnosis of BD-I were included and categorized by body mass index: underweight/normal weight (<25 kg/m2), overweight (25 to <30 kg/m2), or obese (≥30 kg/m2). Adjusted analyses assessed comorbidities, health-related quality of life (HRQoL), work productivity, health care resource utilization (HCRU), and economic outcomes. In total, responses from 1,853 participants were analyzed; most were female (65%) and white (62%). Respondents with obesity had the highest prevalence of medical comorbidities, including high blood pressure (52%), sleep apnea (37%), hypercholesterolemia (34%), and type 2 diabetes (12%). Obesity was generally associated with the lowest scores of physical health and HRQoL. Activity impairment scores were highest among respondents with obesity, as were numbers of hospitalizations and emergency department visits in the previous 6 months. Respondents with obesity incurred higher annual indirect and direct medical costs ($28,178 and $37,771, respectively) when compared with the underweight/normal weight ($23,823 and $32,227, respectively) and overweight ($24,312 and $35,231, respectively) groups. In this nationally representative sample, obesity was associated with several outcomes that may negatively affect people living with BD-I, including medical comorbidities, higher HCRU, HRQoL impairments, and greater indirect and direct medical costs. These findings highlight the importance of considering the presence of or risk for obesity and associated medical comorbidities when treating BD-I.

Evaluation of health and economic effects of United States school meal standards consistent with the 2020–2025 dietary guidelines for Americans

BackgroundThe current school meal nutrition standards, established in 2010, are not fully aligned with the 2020–2025 Dietary Guideline for Americans (DGA). This study evaluates the potential short-term and long-term health and economic benefits of strengthening the school meal standards on added sugars, sodium, and whole grains to be aligned with current guidelines. MethodsWe used comparative risk assessment frameworks based on nationally representative data incorporating current demographics, dietary habits, and risk factors of United States children aged 5–18 y from 3 cycles of the National Health and Nutrition Examination Survey (2013–2018). To estimate short-term impact, the model incorporated estimated dietary changes owing to potential new DGA-aligned school meal nutrition standards and the effect of these changes on childhood body mass index (in kg/m2) and blood pressure. To estimate long-term impact, the model further incorporated data on the sustainability of childhood dietary changes into adulthood, and on demographics and risk factors of United States adults, diet-disease associations, and disease-specific national mortality. ResultsIn a best-case scenario assuming full school compliance, implementing new DGA-aligned nutritional standards would lower elementary children’s BMI by an average 0.14 (95% UI: 0.08–0.20) kg/m2 and systolic blood pressure by 0.13 (95% UI: 0.06–0.19) (95% mm Hg. Later in life, the new standards were estimated to prevent 10,600 [95% uncertainty interval (UI): 4820–16,800) annual deaths from cardiovascular disease (CVD), diabetes, and cancer in adulthood; and save 355,000 (95% UI: 175,000–538,000) disability-adjusted life years and $19.3 (95% UI: 9.35–30.3) B in direct and indirect medical costs each year. Accounting for plausible (incomplete) school compliance, implementation would save an estimated 9110 (95% UI: 2740–15,100) deaths, 302,000 (95% UI: 120,000–479,000) disability-adjusted life years, and $15.9 (95% UI: 4.54–27.2) B in healthcare-related costs per year in later adulthood. ConclusionsStronger school meal nutrition standards on added sugars, sodium, and whole grains aligned with the 2020–2025 DGA recommendations may improve diet, childhood health, and future adult burdens of CVD, diabetes, cancer, and associated economic costs.