Comparing the cost-per-QALYs gained and cost-per-DALYs averted literatures.
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ABSTRACT:
SUBMITTER: Neumann PJ
PROVIDER: S-EPMC5801595 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:IntroductionCalculating the cost per disability-adjusted life years (DALYs) averted associated with interventions is an increasing popular means of assessing the cost-effectiveness of strategies to improve population health. However, there has been no systematic attempt to characterize the literature and its evolution.MethodsWe conducted a systematic review of cost-effectiveness studies reporting cost-per-DALY averted from 2000 through 2015. We developed the Global Health Cost-Effectiveness Analysis (GHCEA) Registry, a repository of English-language cost-per-DALY averted studies indexed in PubMed. To identify candidate studies, we searched PubMed for articles with titles or abstracts containing the phrases "disability-adjusted" or "DALY". Two reviewers with training in health economics independently reviewed each article selected in our abstract review, gathering information using a standardized data collection form. We summarized descriptive characteristics on study methodology: e.g., intervention type, country of study, study funder, study perspective, along with methodological and reporting practices over two time periods: 2000-2009 and 2010-2015. We analyzed the types of costs included in analyses, the study quality on a scale from 1 (low) to 7 (high), and examined the correlation between diseases researched and the burden of disease in different world regions.ResultsWe identified 479 cost-per-DALY averted studies published from 2000 through 2015. Studies from Sub-Saharan Africa comprised the largest portion of published studies. The disease areas most commonly studied were communicable, maternal, neonatal, and nutritional disorders (67%), followed by non-communicable diseases (28%). A high proportion of studies evaluated primary prevention strategies (59%). Pharmaceutical interventions were commonly assessed (32%) followed by immunizations (28%). Adherence to good practices for conducting and reporting cost-effectiveness analysis varied considerably. Studies mainly included formal healthcare sector costs. A large number of the studies in Sub-Saharan Africa addressed high-burden conditions such as HIV/AIDS, tuberculosis, neglected tropical diseases and malaria, and diarrhea, lower respiratory infections, meningitis, and other common infectious diseases.ConclusionThe Global Health Cost-Effectiveness Analysis Registry reveals a growing and diverse field of cost-per-DALY averted studies. However, study methods and reporting practices have varied substantially.
| S-EPMC5179084 | biostudies-literature
Project description:BackgroundThe iDSI reference case, originally published in 2014, aims to improve the quality and comparability of cost-effectiveness analyses (CEA). This study assesses whether the development of the guideline is associated with an improvement in methodological and reporting practices for CEAs using disability-adjusted life-years (DALYs).MethodsWe analyzed the Tufts Medical Center Global Health CEA Registry to identify cost-per-DALY averted studies published from 2011 to 2017. Among each of 11 principles in the iDSI reference case, we translated all methodological specifications and reporting standards into a series of binary questions (satisfied or not satisfied) and awarded articles one point for each item satisfied. We then calculated methodological and reporting adherence scores separately as a percentage of total possible points, measured as normalized adherence score (0% = no adherence; 100% = full adherence). Using the year 2014 as the dissemination period, we conducted a pre-post analysis. We also conducted sensitivity analyses using: 1) optional criteria in scoring, 2) alternate dissemination period (2014-2015), and 3) alternative comparator classification.ResultsArticles averaged 60% adherence to methodological specifications and 74% adherence to reporting standards. While methodological adherence scores did not significantly improve (59% pre-2014 vs. 60% post-2014, p = 0.53), reporting adherence scores increased slightly over time (72% pre-2014 vs. 75% post-2014, p<0.01). Overall, reporting adherence scores exceeded methodological adherence scores (74% vs. 60%, p<0.001). Articles seldom addressed budget impact (9% reporting, 10% methodological) or equity (7% reporting, 7% methodological).ConclusionsThe iDSI reference case has substantial potential to serve as a useful resource for researchers and policy-makers in global health settings, but greater effort to promote adherence and awareness is needed to achieve its potential.
| S-EPMC6493721 | biostudies-literature
Project description:ImportanceAdjuvant therapies are often approved based on improvements in disease-, progression-, or relapse-free survival (ie, an event). An important estimate in adjuvant therapies is the cost per event averted.ObjectiveTo characterize the costs per event averted of anticancer drugs approved by the US Food and Drug Administration (FDA) between January 2018 and March 2022.Design, setting, and participantsIn this cross-sectional study, all approvals were reviewed from the FDA website. Approvals selected for the analysis needed to be drawn from randomized trials involving anticancer drugs studied in the adjuvant treatment of solid tumors. Treatments of nonsolid tumors; nonrandomized, noncontrolled trials; and unpublished trials were excluded. Approvals between January 2018 and March 2022 were included, and 11 trials met inclusion criteria. The monthly costs of each agent were abstracted from the Micromedex RED BOOK database. All variables included in calculations were derived from the original trial publication. Information abstracted for each approval included the name of the drug approved, name of the trial, primary end point, dosing regimen in the trial, median duration of treatment, tumor type, monthly costs per drug, and the number needed to treat for the primary end point. Data were analyzed in March 2022.Main outcomes and measuresThe cost per event averted for each agent studied in each clinical trial; the monthly cost of each drug, the cost per patient for each drug, the primary clinical end point studied in each trial; the study's design and setting.ResultsA total of 11 approvals were included in the study. From January 2018 to March 2022, all approvals were based on a surrogate end point as the primary end point, with no trials demonstrating, to date, an overall survival benefit. The median cost per event averted of drugs in the adjuvant setting was $1 610 000 (range, $820 000 to $2 640 000). The median cost of a complete adjuvant treatment was $158 000 per patient.Conclusions and relevanceIn this cross-sectional study, anticancer agents in the adjuvant setting were approved based on disease-, progression-, event-, or relapse-free survival. Costs per event averted were high. These findings suggest that these drugs may not achieve wide or global use unless their efficacy improves or their prices decrease.
| S-EPMC9187950 | biostudies-literature
Project description:Background Large amounts of data are collected in neonatal intensive care units, which could be used for research. It is unclear whether these data, usually sampled at a lower frequency, are sufficient for retrospective studies. We investigated what to expect when using one-per-minute data for descriptive statistics. Methods One-per-second inspiratory oxygen and saturation were processed to one-per-minute data and compared, on average, standard deviation, target range time, hypoxia, days of supplemental oxygen, and missing signal. Results Outcomes calculated from data recordings (one-per-minute = 92, one-per-second = 92) showed very little to no difference. Sub analyses of recordings under 100 and 200 h showed no difference. Conclusion In our study, descriptive statistics of one-per-minute data were comparable to one-per-second and could be used for retrospective analyses. Comparable routinely collected one-per-minute data could be used to develop algorithms or find associations, retrospectively.
| S-EPMC9133439 | biostudies-literature
Project description:To review existing evidence on the potential of incentives to undermine or "crowd out" intrinsic motivation, in order to establish whether and when it predicts financial incentives to crowd out motivation for health-related behaviors.We conducted a conceptual analysis to compare definitions and operationalizations of the effect, and reviewed existing evidence to identify potential moderators of the effect.In the psychological literature, we find strong evidence for an undermining effect of tangible rewards on intrinsic motivation for simple tasks when motivation manifest in behavior is initially high. In the economic literature, evidence for undermining effects exists for a broader variety of behaviors, in settings that involve a conflict of interest between parties. By contrast, for health related behaviors, baseline levels of incentivized behaviors are usually low, and only a subset involve an interpersonal conflict of interest. Correspondingly, we find no evidence for crowding out of incentivized health behaviors.The existing evidence does not warrant a priori predictions that an undermining effect would be found for health-related behaviors. Health-related behaviors and incentives schemes differ greatly in moderating characteristics, which should be the focus of future research.
| S-EPMC3906839 | biostudies-other
Project description:For unresectable or metastatic melanoma, first-line ipilimumab has demonstrated long-term survival benefits over a 7-year period. First-line treatment with BRAF inhibitors has demonstrated efficacy in clinical trials with up to 3 years of follow-up. The long-term comparative efficacy and costs of ipilimumab and BRAF inhibitors are unknown.Patient-level data from 12 clinical studies for ipilimumab were used. Survival data were extracted from included clinical trials for BRAF inhibitors based on a systematic literature review. Different parametric survival models, including exponential, Gompertz, log-normal, and Weibull models, were used to fit reported overall survival (OS) data and to project long-term survival for BRAF inhibitors. Survival benefits were measured in terms of total life-months gained as calculated by the area under the curve of OS Kaplan-Meier curves for the observed ipilimumab data and projected BRAF inhibitor data. Total life-months gained and cumulative costs per life-month gained were compared between ipilimumab and BRAF inhibitors.The systematic literature review identified six randomized-controlled trials of BRAF inhibitors for subsequent analyses. With 7-year follow-up, ipilimumab was associated with a total of 28.5 life-months gained. Based on the Weibull model, the extrapolated total life-months gained for BRAF inhibitors were 26.5 months for dabrafenib, 21.3 months for trametinib, 14.3 months for vemurafenib, and 24.6 months for dabrafenib + trametinib. In sensitivity analyses, extrapolated total life-months gained varied across the three other models, ranging from 13.7 to 36.8 months across therapies. Cumulative costs per life-month gained with ipilimumab decreased steadily over time, while the costs remained constant for BRAF inhibitors due to continuous dosing. By year 3, cumulative costs per life-month gained were the lowest with ipilimumab; by year 7, the costs were $4281 for ipilimumab, compared with $8920 for dabrafenib, $10,211 for trametinib, $11,002 for vemurafenib, and $19,132 for the dabrafenib + trametinib combination therapy.Ipilimumab was associated with a better long-term cost-per-life month compared to BRAF agents. Long-term extrapolation of survival with BRAF agents was uncertain, and showed no evidence of prolonged survival compared to ipilimumab.
| S-EPMC4827222 | biostudies-other
Project description:ObjectivesWhile almost 60% of the world has received at least one dose of COVID-19 vaccine, the global distribution of vaccination has not been equitable. Only 4% of the population of low-income countries (LICs) has received a full primary vaccine series, compared with over 70% of the population of high-income nations.DesignWe used economic and epidemiological models, parameterised with public data on global vaccination and COVID-19 deaths, to estimate the potential benefits of scaling up vaccination programmes in LICs and lower-middle-income countries (LMICs) in 2022 in the context of global spread of the Omicron variant of SARS-CoV2.SettingLow-income and lower-middle-income nations.Main outcome measuresOutcomes were expressed as number of avertable deaths through vaccination, costs of scale-up and cost per death averted. We conducted sensitivity analyses over a wide range of parameter estimates to account for uncertainty around key inputs.FindingsGlobally, universal vaccination in LIC/LMIC with three doses of an mRNA vaccine would result in an estimated 1.5 million COVID-19 deaths averted with a total estimated cost of US$61 billion and an estimated cost-per-COVID-19 death averted of US$40 800 (sensitivity analysis range: US$7400-US$81 500). Lower estimated infection fatality ratios, higher cost-per-dose and lower vaccine effectiveness or uptake lead to higher cost-per-death averted estimates in the analysis.ConclusionsScaling up COVID-19 global vaccination would avert millions of COVID-19 deaths and represents a reasonable investment in the context of the value of a statistical life. Given the magnitude of expected mortality facing LIC/LMIC without vaccination, this effort should be an urgent priority.
| S-EPMC9471205 | biostudies-literature
Project description:BackgroundThe prices of newly approved cancer drugs have risen over the past decades. A key policy question is whether the clinical gains offered by these drugs in treating specific cancer indications justify the price increases.ObjectivesTo evaluate the price per median and mean life year gained among newly approved cancer therapies from 1995 to 2017.MethodsWe collected data on the price (in 2017 USD) per life-year gained among cancer drug-indication pairs approved by the US Food and Drug Administration (FDA) between 1995 and 2017. We modeled trends using fractional polynomial and linear spline regression models that controlled for route of administration and cancer type fixed effects.ResultsWe found that between 1995 and 2012, price increases outstripped median survival gains, a finding consistent with previous literature. Nevertheless, price per mean life-year gained increased at a considerably slower rate, suggesting that new drugs have been more effective in achieving longer-term survival. Between 2013 and 2017, price increases reflected equally large gains in median and mean survival, resulting in a flat profile for benefit-adjusted launch prices in recent years.ConclusionsAlthough drug costs have been rising more rapidly than median survival gains, they have been rising at about the same rate as mean survival gains. This suggests that when accounting for longer-term survival gains, the benefits of new drugs are roughly keeping pace with their costs, despite rapid cost growth.
| S-EPMC7589784 | biostudies-literature
Project description:BackgroundGiven the variability of intensive care unit (ICU) costs in different countries and the importance of this information for guiding clinicians to effective treatment and to the organisation of ICUs at the national level, it is of value to gather data on this topic for analysis at the national level in Belgium. The objectives of the study were to assess the total cost of ICUs and the factors that influence the cost of ICUs in hospitals in Belgium.MethodsThis was a retrospective cohort study using data collected from the ICUs of 17 Belgian hospitals from January 01 to December 31, 2018. A total of 18,235 adult ICU stays were included in the study. The data set was a compilation of inpatient information from analytical cost accounting of hospitals, medical discharge summaries, and length of stay data. The costs were evaluated as the expenses related to the management of hospital stays from the hospital's point of view. The cost from the hospital perspective was calculated using a cost accounting analytical methodology in full costing. We used multivariate linear regression to evaluate factors associated with total ICU cost per stay. The ICU cost was log-transformed before regression and geometric mean ratios (GMRs) were estimated for each factor.ResultsThe proportion of ICU beds to ward beds was a median [p25-p75] of 4.7% [4.4-5.9]. The proportion of indirect costs to total costs in the ICU was 12.1% [11.4-13.3]. The cost of nurses represented 57.2% [55.4-62.2] of direct costs and this was 15.9% [12.0-18.2] of the cost of nurses in the whole hospital. The median cost per stay was €4,267 [2,050-9,658] and was €2,160 [1,545-3,221] per ICU day. The main factors associated with higher cost per stay in ICU were Charlson score, mechanical ventilation, ECMO, continuous hemofiltration, length of stay, readmission, ICU mortality, hospitalisation in an academic hospital, and diagnosis of coma/convulsions or intoxication.ConclusionsThis study demonstrated that, despite the small proportion of ICU beds in relation to all services, the ICU represented a significant cost to the hospital. In addition, this study confirms that nursing staff represent a significant proportion of the direct costs of the ICU. Finally, the total cost per stay was also important but highly variable depending on the medical factors identified in our results.
| S-EPMC10500739 | biostudies-literature
Project description:Biobanks have facilitated the conduct of large-scale genomics studies, but they are challenged by the difficulty of validating some phenotypes, particularly for complex traits that represent heterogeneous groups ofpatients. The guideline definition of COPD, based on objective spirometry measures, has been preferred in genome-wide association studies (GWAS) conducted with epidemiological cohorts, but spirometry measures are seldom available for biobank participants. Defining COPD based on International Classification of Disease (ICD) codes or self-reported measures is highly feasible in biobanks, but it remains unclear whether the misclassification inherent in these definitions prevent the discovery of genetic variants that contribute to COPD. We found that while there was poor agreement in classification of UK Biobank participants as having COPD based on ICD diagnosis codes, self-reported doctor diagnosis or spirometry measures, contrasting GWAS results for these definitions provided insights into what patient characteristics each trait may capture.
| S-EPMC7233028 | biostudies-literature