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Improving cost-effectiveness of epidemiological studies via designed missingness strategies.

ABSTRACT: Modern epidemiological studies face opportunities and challenges posed by an ever-expanding capacity to measure a wide range of environmental exposures, along with sophisticated biomarkers of exposure and response at the individual level. The challenge of deciding what to measure is further complicated for longitudinal studies, where logistical and cost constraints preclude the collection of all possible measurements on all participants at every follow-up time. This is true for the National Children's Study (NCS), a large-scale longitudinal study that will enroll women both prior to conception and during pregnancy and collect information on their environment, their pregnancies, and their children's development through early adulthood-with a goal of assessing key exposure/outcome relationships among a cohort of approximately 100 000 children. The success of the NCS will significantly depend on the accurate, yet cost-effective, characterization of environmental exposures thought to be related to the health outcomes of interest. The purpose of this paper is to explore the use of cost saving, yet valid and adequately powered statistical approaches for gathering exposure information within epidemiological cohort studies. The proposed approach involves the collection of detailed exposure assessment information on a specially selected subset of the study population, and collection of less-costly, and presumably less-detailed and less-burdensome, surrogate measures across the entire cohort. We show that large-scale efficiency in costs and burden may be achieved without making substantive sacrifices on the ability to draw reliable inferences concerning the relationship between exposure and health outcome. Several detailed scenarios are provided that document how the targeted sub-sampling design strategy can benefit large cohort studies like the NCS, as well as other more focused environmental epidemiologic studies.

SUBMITTER: Strauss WJ

PROVIDER: S-EPMC2882677 | biostudies-literature | 2010 Jun

REPOSITORIES: biostudies-literature

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Improving cost-effectiveness of epidemiological studies via designed missingness strategies.

Strauss Warren J WJ Ryan Louise L Morara Michele M Iroz-Elardo Nicole N Davis Mark M Cupp Matthew M Nishioka Marcia G MG Quackenboss James J Galke Warren W Ozkaynak Haluk H Scheidt Peter P

Statistics in medicine 20100601 13

Modern epidemiological studies face opportunities and challenges posed by an ever-expanding capacity to measure a wide range of environmental exposures, along with sophisticated biomarkers of exposure and response at the individual level. The challenge of deciding what to measure is further complicated for longitudinal studies, where logistical and cost constraints preclude the collection of all possible measurements on all participants at every follow-up time. This is true for the National Chil ...[more]

PMID: 20527011

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Project description:Osteoporosis affects many men, with significant morbidity and mortality. However, the best osteoporosis screening strategies for men are unknown. We developed an individual-level state-transition cost-effectiveness model with a lifetime time horizon to identify the cost-effectiveness of different osteoporosis screening strategies for US men involving various screening tests (dual-energy X-ray absorptiometry [DXA]; the Osteoporosis Self-Assessment Tool [OST]; or a fracture risk assessment strategy using age, femoral neck bone mineral density [BMD], and Vertebral Fracture Assessment [VFA]); screening initiation ages (50, 60, 70, or 80 years); and repeat screening intervals (5 years or 10 years). In base-case analysis, no screening was a less effective option than all other strategies evaluated; furthermore, no screening was more expensive than all strategies that involved screening with DXA or the OST risk assessment instrument, and thus no screening was "dominated" by screening with DXA or OST at all evaluated screening initiation ages and repeat screening intervals. Screening strategies that most frequently appeared as most cost-effective in base-case analyses and one-way sensitivity analyses when assuming willingness-to-pay of $50,000/quality-adjusted life-year (QALY) or $100,000/QALY included screening initiation at age 50 years with the fracture risk assessment strategy and repeat screening every 10 years; screening initiation at age 50 years with fracture risk assessment and repeat screening every 5 years; and screening initiation at age 50 years with DXA and repeat screening every 5 years. In conclusion, expansion of osteoporosis screening for US men to initiate routine screening at age 50 or 60 years would be expected to be effective and of good value for improving health outcomes. A fracture risk assessment strategy using variables of age, femoral neck BMD, and VFA is likely to be the most effective of the evaluated strategies within accepted cost-effectiveness parameters. DXA and OST are also reasonable screening options, albeit likely slightly less effective than the evaluated fracture risk assessment strategy. © 2016 American Society for Bone and Mineral Research.

Project description:BACKGROUND:Exposure to certain pesticides has been associated with several chronic diseases. However, to determine the role of pesticides in the causation of such diseases, an assessment of historical exposures is required. Exposure measurement data are rarely available; therefore, assessment of historical exposures is frequently based on surrogate self-reported information, which has inherent limitations. Understanding the performance of the applied surrogate measures in the exposure assessment of pesticides is therefore important to allow proper evaluation of the risks. OBJECTIVE:The Improving Exposure Assessment Methodologies for Epidemiological Studies on Pesticides (IMPRESS) project aims to assess the reliability and external validity of the surrogate measures used to assign exposure within individuals or groups of individuals, which are frequently based on self-reported data on exposure determinants. IMPRESS will also evaluate the size of recall bias on the misclassification of exposure to pesticides; this in turn will affect epidemiological estimates of the effect of pesticides on human health. METHODS:The IMPRESS project will recruit existing cohort participants from previous and ongoing research studies primarily of epidemiological origin from Malaysia, Uganda, and the United Kingdom. Consenting participants of each cohort will be reinterviewed using an amended version of the original questionnaire addressing pesticide use characteristics administered to that cohort. The format and relevant questions will be retained but some extraneous questions from the original (eg, relating to health) will be excluded for ethical and practical reasons. The reliability of pesticide exposure recall over different time periods (<2 years, 6-12 years, and >15 years) will then be evaluated. Where the original cohort study is still ongoing, participants will also be asked if they wish to take part in a new exposure biomonitoring survey, which involves them providing urine samples for pesticide metabolite analysis and completing questionnaire information regarding their work activities at the time of sampling. The participant's level of exposure to pesticides will be determined by analyzing the collected urine samples for selected pesticide metabolites. The biomonitoring measurement results will be used to assess the performance of algorithm-based exposure assessment methods used in epidemiological studies to estimate individual exposures during application and re-entry work. RESULTS:The project was funded in September 2017. Enrollment and sample collection was completed for Malaysia in 2019 and is on-going for Uganda and the United Kingdom. Sample and data analysis will proceed in 2020 and the first results are expected to be submitted for publication in 2021. CONCLUSIONS:The study will evaluate the consistency of questionnaire data and accuracy of current algorithms in assessing pesticide exposures. It will indicate where amendments can be made to better capture exposure data for future epidemiology studies and thus improve the reliability of exposure-disease associations. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID):PRR1-10.2196/16448.

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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.

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Improving cost-effectiveness of epidemiological studies via designed missingness strategies.

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Improving cost-effectiveness of epidemiological studies via designed missingness strategies.

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