Disaggregating proportional multistate lifetables by population heterogeneity to estimate intervention impacts on inequalities

Background Simulation models can be used to quantify the projected health impact of interventions. Quantifying heterogeneity in these impacts, for example by socioeconomic status, is important to understand impacts on health inequalities. We aim to disaggregate one type of Markov macro-simulation model, the proportional multistate lifetable, ensuring that under business-as-usual (BAU) the sum of deaths across disaggregated strata in each time step returns the same as the initial non-disaggregated model. We then demonstrate the application by deprivation quintiles for New Zealand (NZ), for: hypothetical interventions (50% lower all-cause mortality, 50% lower coronary heart disease mortality) and a dietary intervention to substitute 59% of sodium with potassium chloride in the food supply. Methods We developed a disaggregation algorithm that iteratively rescales mortality, incidence and case-fatality rates by time-step of the model to ensure correct total population counts were retained at each step. To demonstrate the algorithm on deprivation quintiles in NZ, we used the following inputs: overall (non-disaggregated) all-cause mortality & morbidity rates, coronary heart disease incidence & case fatality rates; stroke incidence & case fatality rates. We also obtained rate ratios by deprivation for these same measures. Given all-cause and cause-specific mortality rates by deprivation quintile, we derived values for the incidence, case fatality and mortality rates for each quintile, ensuring rate ratios across quintiles and the total population mortality and morbidity rates were returned when averaged across groups. The three interventions were then run on top of these scaled BAU scenarios. Results The algorithm exactly disaggregated populations by strata in BAU. The intervention scenario life years and health adjusted life years (HALYs) gained differed slightly when summed over the deprivation quintile compared to the aggregated model, due to the stratified model (appropriately) allowing for differential background mortality rates by strata. Modest differences in health gains (HALYs) resulted from rescaling of sub-population mortality and incidence rates to ensure consistency with the aggregate population. Conclusion Policy makers ideally need to know the effect of population interventions estimated both overall, and by socioeconomic and other strata. We demonstrate a method and provide code to do this routinely within proportional multistate lifetable simulation models and similar Markov models.

Pharmacokinetic Parameter Driven Outcomes Model Predicts a Reduction in Bleeding Events Associated with BAY 81-8973 Versus Antihemophilic Factor (Recombinant) Plasma/Albumin- Free Method in a Chinese Healthcare Setting

Background: Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population. Methods: Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year. Results: Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3%, 9.3% and 19.3%, respectively for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM. Conclusion: Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.

Cost-Effectiveness of Upper Extremity Dry Needling in Chronic Stroke

Introduction: Dry needling is a non-pharmacological approach that has proven to be effective in different neurological conditions. Objective: The aim of this study was to evaluate the cost-effectiveness of a single dry needling session in patients with chronic stroke. Methods: A cost-effectiveness analysis was performed based on a randomized controlled clinical trial. The results obtained from the values of the EuroQol-5D questionnaire and the Modified Modified Ashworth Scale were processed in order to obtain the percentage of treatment responders and the quality-adjusted life years (QALYs) for each alternative. The cost analysis was that of the hospital, clinic, or health center, including the equipment and physiotherapist. The cost per respondent and the incremental cost-effectiveness ratio of each alternative were assessed. Results: Twenty-three patients with stroke were selected. The cost of DN treatment was EUR 14.96, and the data analysis showed a favorable cost-effectiveness ratio of both EUR/QALY and EUR/responder for IG, although the sensitivity analysis using limit values did not confirm the dominance (higher effectiveness with less cost) of the dry needling over the sham dry needling. Conclusions: Dry needling is an affordable alternative with good results in the cost-effectiveness analysis—both immediately, and after two weeks of treatment—compared to sham dry needling in persons with chronic stroke.

Burden of Oral Cancer on the 10 Most Populous Countries from 1990 to 2019: Estimates from the Global Burden of Disease Study 2019

Background: Oral cancer (OC) is a common tumour that poses a threat to human health and imposes a heavy burden on countries. This study assessed the burden imposed by OC on the 10 most populous countries from 1990 to 2019 on the basis of gender, age and socio-demographic index. Methods: Data on incidence, mortality, disability-adjusted life years (DALY) and corresponding age-standardised rates (ASR) for OC in the 10 most populous countries from 1990 to 2019 were derived from the Global Burden of Disease Study 2019. Estimated annual percentage changes were calculated to assess the trends of morbidity, mortality and DALY. The indicator that served as a proxy for survival rate was the supplement of mortality-to-incidence ratio (SMIR) (1 − (M/I)). Results: The number of new cases, deaths and DALY have increased in all 10 countries in the past 30 years. Trends in age-standardised incidence rates (ASIR), age-standardised mortality rate (ASMR) and age-standardised DALY for OC in the 10 most populous countries varied. The SMIR increased in all countries, with most countries having an SMIR between 30% and 50%. In 2019, the United States had the highest SMIR at 76%, whereas Russia had the lowest at 21.7%. Incidence and mortality were close between male and female subjects in Japan, Indonesia, Mexico, India, Bangladesh and Pakistan. The incidence and mortality in male subjects in the United States, Russia, China and Brazil were two or more times those of female subjects. Gender difference was highest among patients aged 40–69 years. Conclusion: Trends and gender differences in ASIR, ASMR and age-standardised DALY for OC vary in the 10 most populous countries. Government cancer programs are often expensive to run, especially in countries with large populations. Policy makers need to take these differences into account when formulating policies.

The non-fatal burden of cancer in Belgium, 2004–2019: a nationwide registry-based study

Background The importance of assessing and monitoring the health status of a population has grown in the last decades. Consistent and high quality data on the morbidity and mortality impact of a disease represent the key element for this assessment. Being increasingly used in global and national burden of diseases (BoD) studies, the Disability-Adjusted Life Year (DALY) is an indicator that combines healthy life years lost due to living with disease (Years Lived with Disability; YLD) and due to dying prematurely (Years of Life Lost; YLL). As a step towards a comprehensive national burden of disease study, this study aims to estimate the non-fatal burden of cancer in Belgium using national data. Methods We estimated the Belgian cancer burden from 2004 to 2019 in terms of YLD, using national population-based cancer registry data and international disease models. We developed a microsimulation model to translate incidence- into prevalence-based estimates, and used expert elicitation to integrate the long-term impact of increased disability due to surgical treatment. Results The age-standardized non-fatal burden of cancer increased from 2004 to 2019 by 6 and 3% respectively for incidence- and prevalence-based YLDs. In 2019, in Belgium, breast cancer had the highest morbidity impact among women, followed by colorectal and non-melanoma skin cancer. Among men, prostate cancer had the highest morbidity impact, followed by colorectal and non-melanoma skin cancer. Between 2004 and 2019, non-melanoma skin cancer significantly increased for both sexes in terms of age-standardized incidence-based YLD per 100,000, from 49 to 111 for men and from 15 to 44 for women. Important decreases were seen for colorectal cancer for both sexes in terms of age-standardized incidence-based YLD per 100,000, from 105 to 84 for men and from 66 to 58 for women. Conclusions Breast and prostate cancers represent the greatest proportion of cancer morbidity, while for both sexes the morbidity burden of skin cancer has shown an important increase from 2004 onwards. Integrating the current study in the Belgian national burden of disease study will allow monitoring of the burden of cancer over time, highlighting new trends and assessing the impact of public health policies.

JAMINAN KESEHATAN DAN PENDAPATAN KELUARGA BALITA STUNTING

Balita stunting berkontribusi terhadap 1,5 juta (15%) kematian anak balita di dunia dan menyebabkan 55 juta Disability-Adjusted Life Years (DALYs) yaitu hilangnya masa hidup sehat setiap tahun. Jaminan kesehatan dan pendapatan keluarga merupakan salah satu faktor yang berhubungan tidak langsung dengan kejadian stunting. Akan tetapi dapat mempengaruhi ketersediaan sumber gizi serta perlindungan diri saat menanganai permasalahan infeksi pada balita yang bermuara pada resiko kejadian stunting. Tujuan penelitian : Untuk mengetahui gambaran kepemilikan jaminan kesehatan serta pendapatan keluarga balita stunting. Metodologi penelitian : Jenis penelitian yang digunakan adalah penelitian kuantitatif dengan metode deskriptif. Populasi dalam penelitian ini adalah ibu yang memiliki balita stunting Di Desa Sukamulya Kecamatan Singaparna sebanyak 18 orang. Tehnik pengambilan sampling menggunakan total sampling. Analisis data dilakukan dengan menggunakan analisis univariat dimana hasil disajikan dalam bentuk presentase dan dibuat secara naratif. Hasil penelitian : Terdapat 38,9% keluarga balita stunting yang tidak memiliki jaminan kesehatan, mayoritas jenis jaminan kesehatan yang dimiliki oleh keluarga stunting adalah Kartu Indonesia Sehat serta mayoritas pendapatan keluarga balita stunting di Desa Sukamulya Kecamatan Singaparna adalah dibawah UMR yaitu 72,2%. Kesimpulan dan saran : Keluarga dapat meningkatkan pencarian informasi tentang pentingnya memiliki jaminan kesehatan seghingga dapat memanfaatkan pelayanan yang disediakan sebagai program penyelesaian permasalahan stunting. Selain itu program perbaikan gizi keluarga melalui pemanfaatan lahan masyarakat dapat membantu dalam pemenuhan kebutuhan gizi masyarakat. Kata kunci: Stunting, Kepemilikan jaminan kesehatan, pendapatan keluarga, balita

Assessment of Financial Toxicity Among Patients With Advanced Lung Cancer in Western China

Background: Lung cancer is the leading source of cancer-caused disability-adjusted life years. Medical cost burden impacts the well-being of patients through reducing income, cutting daily expenses, curtailing leisure activities, and depleting exhausting savings. The COmprehensive Score for Financial Toxicity (COST) was created and validated by De Souza and colleagues. Our study intends to measure the financial burdens of cancer therapy and investigate the link between financial toxicity and health-related quality of life (HRQoL) in an advanced lung cancer population.Methods: Patients aged ≥ 18 years with confirmed stage III to IV lung cancer were eligible. The COST questionnaire verified by de Souza et al. was used to identify financial toxicity. Multivariable linear regression analysis with log transformation univariate analysis and Pearson correlations were used to perform the analysis.Results: The majority of the patients (90.8%, n = 138/152) had an annual income of $50,000 ($7,775). The cohort's insurance situation was as follows: 64.5% of the cohort had social insurance, 20.4% had commercial insurance, and 22.0% had both. Patients who were younger age (50–59, P < 0.001), employed but on sick leave, and had lower income reported increased levels of financial toxicity (P < 0.05). The risk factors for high financial toxicity: (i) younger age (50–59), (ii) <1 month of savings, and (iii) being employed but on sick leave. Increased financial toxicity is moderately correlated with a decrease in QoL.Conclusion: Poorer psychological status and specific demographics are linked to increased financial toxicity (lower COST). Financial toxicity has a modest relationship with HRQoL and may have a clear link with HRQoL measurements.

An economic evaluation of triage tools for patients with suspected severe injuries in England

Background Many health care systems triage injured patients to major trauma centres (MTCs) or local hospitals by using triage tools and paramedic judgement. Triage tools are typically assessed by whether patients with an Injury Severity Score (ISS) ≥ 16 go to an MTC and whether patients with an ISS < 16 are sent to their local hospital. There is a trade-off between sensitivity and specificity of triage tools, with the optimal balance being unknown. We conducted an economic evaluation of major trauma triage tools to identify which tool would be considered cost-effective by UK decision makers. Methods A patient-level, probabilistic, mathematical model of a UK major trauma system was developed. Patients with an ISS ≥ 16 who were only treated at local hospitals had worse outcomes compared to being treated in an MTC. Nine empirically derived triage tools, from a previous study, were examined so we assessed triage tools with realistic trade-offs between triage tool sensitivity and specificity. Lifetime costs, lifetime quality adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were calculated for each tool and compared to maximum acceptable ICERs (MAICERs) in England. Results Four tools had ICERs within the normal range of MAICERs used by English decision makers (£20,000 to £30,000 per QALY gained). A low sensitivity (28.4%) and high specificity (88.6%) would be cost-effective at the lower end of this range while higher sensitivity (87.5%) and lower specificity (62.8%) was cost-effective towards the upper end of this range. These results were sensitive to the cost of MTC admissions and whether MTCs had a benefit for patients with an ISS between 9 and 15. Conclusions The cost-effective triage tool depends on the English decision maker’s MAICER for this health problem. In the usual range of MAICERs, cost-effective prehospital trauma triage involves clinically suboptimal sensitivity, with a proportion of seriously injured patients (at least 10%) being initially transported to local hospitals. High sensitivity trauma triage requires development of more accurate decision rules; research to establish if patients with an ISS between 9 and 15 benefit from MTCs; or, inefficient use of health care resources to manage patients with less serious injuries at MTCs.

Cost-Effectiveness Analysis of Imaging Modalities for Breast Cancer Surveillance Among BRCA1/2 Mutation Carriers: A Systematic Review

BackgroundBRCA1/2 mutation carriers are suggested with regular breast cancer surveillance screening strategies using mammography with supplementary MRI as an adjunct tool in Western countries. From a cost-effectiveness perspective, however, the benefits of screening modalities remain controversial among different mutated genes and screening schedules.MethodsWe searched the MEDLINE/PubMed, Embase, Cochrane Library, Scopus, and Web of Science databases to collect and compare the results of different cost-effectiveness analyses. A simulated model was used to predict the impact of screening strategies in the target group on cost, life-year gained, quality-adjusted life years, and incremental cost-effectiveness ratio (ICER).ResultsNine cost-effectiveness studies were included. Combined mammography and MRI strategy is cost-effective in BRCA1 mutation carriers for the middle-aged group (age 35 to 54). BRCA2 mutation carriers are less likely to benefit from adjunct MRI screening, which implies that mammography alone would be sufficient from a cost-effectiveness perspective, regardless of dense breast cancer.ConclusionsPrecision screening strategies among BRCA1/2 mutation carriers should be conducted according to the acceptable ICER, i.e., a combination of mammography and MRI for BRCA1 mutation carriers and mammography alone for BRCA2 mutation carriers.Systematic Review RegistrationPROSPERO, identifier CRD42020205471.

Cost-Effectiveness Analysis of Tyrosine Kinase Inhibitors in Gastrointestinal Stromal Tumor: A Systematic Review

Background: The introduction of tyrosine kinase inhibitor (TKI) therapy has dramatically improved the clinical effectiveness of patients with locally advanced and/or metastatic gastrointestinal stromal tumors (GIST), and this systematic review was conducted aiming at the cost-effectiveness analysis of TKIs in GIST.Methods: A thorough literature search of online databases was performed, using appropriate terms such as “gastrointestinal stromal tumor or GIST,” “cost-effectiveness,” and “economic evaluation.” Data extraction was conducted independently by two authors, and completeness of reporting and quality of the evaluation were assessed. The systematic review was conducted following the PRISMA statement.Results: Published between 2005 and 2020, 15 articles were incorporated into the systematic review. For advanced GIST, imatinib followed by sunitinib was considered cost-effective, and regorafenib was cost-effective compared with imatinib re-challenge therapy in the third-line treatment. For resectable GIST, 3-year adjuvant imatinib therapy represented a cost-effective treatment option. The precision medicine-assisted imatinib treatment was cost-effective compared with empirical treatment.Conclusion: Although identified studies varied in predicted costs and quality-adjusted life years, there was general agreement in study conclusions. More cost-effectiveness analysis should be conducted regarding more TKIs that have been approved for the treatment of GIST.Systematic Review Registration:https://www.crd.york.ac.uk/, PROSPERO: CRD42021225253.