Closing Evidence Gaps in Clinical Prevention (2022) / Chapter Skim
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Appendix B: Research Methods
Appendix B: Research Methods
Pages 145-164
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From page 145... ...
New designs for RCTs and innovative methods for observational studies have gained increasing traction in health care delivery research in the past two decades. Modern study designs could enable researchers to address evidence gaps in the USPSTF analytic framework where traditional individual-level RCTs are not feasible for a variety of reasons.
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One statement, on abdominal aortic aneurysm screening, suggested that high-quality modeling studies for women could be informative if new trials were not available. Of the nine USPSTF recommendations reviewed containing letter grades and no I statements, six specifically requested additional RCTs.
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Risk prediction study, including test performance Longitudinal follow-up (in RCT or cohort study) Cross-sectional study Research in subgroups Dissemination or Implementation study Notes Part 1.
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From page 148... ...
Risk prediction study, including test performance Longitudinal follow-up (in RCT or cohort study) Cross-sectional study Research in subgroups Dissemination or Implementation study Notes High Blood Pressure in Children and RCTs could be Adolescents: Screening difficult because 1 1 1 1 this screening is routine in usual care Prevention and Cessation of Tobacco Use in Children and Adolescents: Primary Care 1 1 Interventions Unhealthy Drug Use: Screening 1 1 1 Illicit Drug Use in Children, Adolescents, and Young Adults: Primary Care–Based 1 1 1 Interventions Bacterial Vaginosis in Pregnant Persons to Prevent Preterm Delivery: Screening 1 1
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From page 149... ...
Cognitive Impairment in Older Adults: Calls for more Screening consistent 1 1 definitions of outcomes Abdominal Aortic Aneurysm: Screening Suggested high quality modeling 1 1 1 1 1 1 studies could be useful Elevated Blood Lead Levels in Children and RCTs could be Pregnant Women: Screening difficult because 1 1 1 this screening is routine in usual care Atrial Fibrillation: Screening with Mentions ongoing Electrocardiography 1 1 RCTs Peripheral Artery Disease and Cardiovascular Mentions ongoing Disease: Screening and Risk Assessment with 1 1 1 RCTs the Ankle-Brachial Index Cardiovascular Disease: Risk Assessment Requests studies of with Nontraditional Risk Factors incremental benefit 1 1 1 1 in real-world practice Total 4 11 10 5 2 7 3 149 continued
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Risk prediction study, including test performance Longitudinal follow-up (in RCT or cohort study) Cross-sectional study Research in subgroups Dissemination or Implementation study Notes Part 2.
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Other statements calling for research on the benefits of a screening, treatment, or other intervention were classified as "Intervention Effectiveness Study, unspecified." 151
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From page 152... ...
in non-randomized studies of interventions and to assess the magnitude of bias in different domains, including confounding, selection, misclassification of intervention status, deviations from intended interventions, missing data, outcomes measurement, and reporting. SELECTION OF STUDY DESIGNS For many of the USPSTF recommendations reviewed in Table 5-1, it would be possible to address a research need using either an RCT or an observational study design.
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From page 153... ...
Limitations of Individual-Level Randomized Controlled Trials RCTs are not practically feasible or are not the optimal approach in some situations. Examples include preventive services where: • The service is already widely accepted as standard of practice, such as lead screening for children.
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Here, the committee discusses selected designs that may be useful in clinical prevention. Examples of modern trial designs include • Cluster-randomized trials, in which the unit of intervention is a group of patients clustered based on an organizing factor such as a clinic, a medical center, or a geographic area.
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From page 155... ...
For example, the USPSTF recommends colorectal cancer screening and calls for more research to understand uptake and adherence to individual screening tests, including repeated colonoscopy after 10 years and repeated stool tests annually, as well as research on the accu racy and effectiveness of serum- and urine-based tests ("liquid biopsy" screening) and capsule endoscopy tests, which could enhance adherence.
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For example, multiple observational studies prior to the late 1990s indicated that the use of postmenopausal hormone replacement therapy was associated with substantial reduction in coronary heart disease risk (Humphrey et al., 2002)
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. Modern analytic methods include • Propensity score methods, which are analytic approaches used in observational studies to balance the intervention and compari son groups on observed pre-intervention characteristics.
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From page 158... ...
For example, if a health care financing policy changed to provide access to a preventive service that was previously unavailable to a group of patients, and if uptake of the preventive service were brisk, this design could be used to study the result ing changes in outcomes. PREDICTIVE ANALYTICS Most of the recent USPSTF statements the committee reviewed called for predictive analytics -- risk prediction studies using tests, questionnaires, or clinical characteristics to identify patients at high risk for a condition.
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Patient monitoring technology that gathers highly detailed data, such as wearable patches for heart rhythm monitoring in atrial fibrillation, is also increasing. These trends are likely to increase the needs for predictive analytics, and for modeling studies that combine predictive analytic methods with other methods to give a comprehensive overview of the projected benefits and harms of a preventive service.
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From page 160... ...
In addition, many patients have comorbidities that set up competing priorities with the impetus to deliver preventive care. To present realistic projections of the impact of preventive services in actual practice, it would be helpful for models to not assume 100 percent adherence to an intervention and to account for the fact that harms and benefits data from randomized trials may need to be adjusted to reflect real-world outcomes.
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From page 161... ...
The USPSTF uses several types of processes where methods for group decision-making could be further tested and refined, including the selection of topics to review, the decision about a recommendation on a given topic once the evidence is reviewed, and in the future, the prioritization among evidence gaps that should be filled for a given topic. Learning more about how to best apply such methods in developing recommendations on preventive services and associated research would enhance the robustness of decisions and optimize their downstream impact.
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2002. Risks and benefits of estrogen plus progestin in healthy postmenopausal women: Principal results from the women's health initiative randomized controlled trial.
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U.S. Preventive Services Task Force pro cedure manual.
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