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Pharmacoepidemiology and Drug Safety Oct 2020There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to... (Review)
Review
PURPOSE
There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to produce real-world evidence (RWE) that provides adequate scientific evidence for regulatory decision-making.
METHODS
This review explores how hybrid study designs that include features of RCTs and studies with real-world data (RWD) can combine the advantages of both to generate RWE that is fit for regulatory purposes.
RESULTS
Some hybrid designs include randomization and use pragmatic outcomes; other designs use single-arm trial data supplemented with external comparators derived from RWD or leverage novel data collection approaches to capture long-term outcomes in a real-world setting. Some of these approaches have already been successfully used in regulatory decisions, raising the possibility that studies using RWD could increasingly be used to augment or replace traditional RCTs for the demonstration of drug effectiveness in certain contexts. These changes come against a background of long reliance on RCTs for regulatory decision-making, which are labor-intensive, costly, and produce data that can have limited applicability in real-world clinical practice.
CONCLUSIONS
While RWE from observational studies is well accepted for satisfying postapproval safety monitoring requirements, it has not commonly been used to demonstrate drug effectiveness for regulatory purposes. However, this position is changing as regulatory opinions, guidance frameworks, and RWD methodologies are evolving, with growing recognition of the value of using RWE that is acceptable for regulatory decision-making.
Topics: Decision Making; Drug Approval; Humans; Observational Studies as Topic; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 31823482
DOI: 10.1002/pds.4932 -
Pediatrics Jun 2020The World Health Organization recommends tummy time for infants because of the benefits of improved motor development and reduced likelihood of plagiocephaly. Because of...
CONTEXT
The World Health Organization recommends tummy time for infants because of the benefits of improved motor development and reduced likelihood of plagiocephaly. Because of poor uptake of these recommendations, the association of tummy time with other health outcomes requires further investigation.
OBJECTIVE
To review existing evidence regarding the association of tummy time with a broad and specific range of infant health outcomes.
DATA SOURCES
Electronic databases were searched between June 2018 and April 2019.
STUDY SELECTION
Peer-reviewed English-language articles were included if they investigated a population of healthy infants (0 to 12 months), using an observational or experimental study design containing an objective or subjective measure of tummy time which examined the association with a health outcome (adiposity, motor development, psychosocial health, cognitive development, fitness, cardiometabolic health, or risks/harms).
DATA EXTRACTION
Two reviewers independently extracted data and assessed their quality.
RESULTS
Sixteen articles representing 4237 participants from 8 countries were included. Tummy time was positively associated with gross motor and total development, a reduction in the BMI- score, prevention of brachycephaly, and the ability to move while prone, supine, crawling, and rolling. An indeterminate association was found for social and cognitive domains, plagiocephaly, walking, standing, and sitting. No association was found for fine motor development and communication.
LIMITATIONS
Most studies were observational in design and lacked the robustness of a randomized controlled trial. High selection and performance bias were also present.
CONCLUSIONS
These findings guide the prioritization of interventions aimed at assisting parents meet the global and national physical activity guidelines.
Topics: Child Development; Humans; Infant; Infant Health; Infant, Newborn; Observational Studies as Topic; Plagiocephaly; Prone Position
PubMed: 32371428
DOI: 10.1542/peds.2019-2168 -
Journal of the American Society of... Aug 2023Target trial emulation has drastically improved the quality of observational studies investigating the effects of interventions. Its ability to prevent avoidable biases... (Review)
Review
Target trial emulation has drastically improved the quality of observational studies investigating the effects of interventions. Its ability to prevent avoidable biases that have plagued many observational analyses has contributed to its recent popularity. This review explains what target trial emulation is, why it should be the standard approach for causal observational studies that investigate interventions, and how to do a target trial emulation analysis. We discuss the merits of target trial emulation compared with often used, but biased analyses, as well as potential caveats, and provide clinicians and researchers with the tools to better interpret results from observational studies investigating the effects of interventions.
Topics: Causality; Observational Studies as Topic
PubMed: 37131279
DOI: 10.1681/ASN.0000000000000152 -
European Journal of Endocrinology Dec 2020The name of the study should properly reflect the actual conduct and analysis of the study. This short paper provides guidance on how to properly name the study design....
The name of the study should properly reflect the actual conduct and analysis of the study. This short paper provides guidance on how to properly name the study design. The first distinction is between a trial (intervention given to patients to study its effect) and an observational study. For observational studies, it should further be decided whether it is cross-sectional or whether follow-up time is taken into account (cohort or case-control study). The distinction prospective-retrospective has two disadvantages: prospective is often seen as marker of higher quality, which is not necessarily true; there is no unifying definition that makes a proper distinction between retrospective and prospective possible.
Topics: Case-Control Studies; Clinical Trials as Topic; Cohort Studies; Cross-Sectional Studies; Humans; Observational Studies as Topic; Prospective Studies; Research Design; Retrospective Studies; Terminology as Topic
PubMed: 33055302
DOI: 10.1530/EJE-20-0873 -
JAMA Surgery Aug 2021
Topics: Biomedical Research; Checklist; Guidelines as Topic; Humans; Meta-Analysis as Topic; Observational Studies as Topic; Research Design
PubMed: 33825847
DOI: 10.1001/jamasurg.2021.0522 -
JBI Evidence Synthesis Jul 2020The objective of the review was to identify, critically appraise and synthesize evidence on the impact of professional interpreters on outcomes for hospitalized children...
OBJECTIVE
The objective of the review was to identify, critically appraise and synthesize evidence on the impact of professional interpreters on outcomes for hospitalized children from migrant and refugee families with limited English proficiency.
INTRODUCTION
A key strategy to facilitate effective communication between limited-English-proficient migrant and refugee families and healthcare providers is the use of professional interpreters. Despite awareness of the importance of professional interpreters, interpreters are underutilized. This results in suboptimal care, including poor access to health care, increased risk of serious medical events, increased admission to hospital and inadequate health literacy.
INCLUSION CRITERIA
Limited-English-proficient migrant, refugee or asylum-seeker families with a hospitalized child aged 0 to 18 years, who had used a professional interpreter, were considered for this review. Comparators included standard care or no interpreter, and reported results of comparisons of the duration, frequency or mode of interpreter delivery. Outcomes were length of stay in hospital, unplanned readmission rates to hospital, non-attendance at hospital clinic appointments and ambulatory care, child and/or parent satisfaction with care in hospital, adherence to treatment, medication errors, and other adverse events relating to patient safety. The review considered all studies conducted using a quantitative approach.
METHODS
A three-step search strategy was used. Databases were searched in December 2018 for published and unpublished articles. Studies published in English were considered for inclusion. The recommended JBI approach to critical appraisal, study selection, data extraction and data synthesis was used.
RESULTS
Six articles reporting results from three randomized controlled trials and one observational study were included. Studies were undertaken in the United States involving Spanish-speaking participants. Three studies were in emergency departments. There was a total of 1813 families, of whom 1753 had limited English proficiency. Migrant and refugee families with limited English proficiency reported greater satisfaction with aspects of care when a professional interpreter service was used compared with using ad hoc interpreters. Using professional in-person interpreters resulted in a shorter total emergency department throughput time compared to using professional interpreters via telephone. There was no difference in concordance of child's discharge diagnoses between parents assigned professional interpreters and those assigned bilingual physicians. Video interpretation provided better understanding of diagnoses than phone interpretation.
CONCLUSION
There is evidence that use of ad hoc interpreters or no interpreter is inferior to use of professional interpreters of any mode. Although video and in-person interpreters are more favorable for some outcomes, mode of delivery may not be as important as the fact that a professional interpreter is being used. The mode of professional interpreter delivery should be based on accessibility, availability, language requirements and patient preference.
SYSTEMATIC REVIEW REGISTRATION NUMBER
PROSPERO CRD42017058161.
Topics: Adolescent; Child; Child, Hospitalized; Child, Preschool; Communication Barriers; Humans; Infant; Infant, Newborn; Limited English Proficiency; Observational Studies as Topic; Refugees; Transients and Migrants; Translating; United States
PubMed: 32813387
DOI: 10.11124/JBISRIR-D-19-00300 -
British Journal of Hospital Medicine... May 2020The numbers of clinical trials have increased exponentially over the last decade, amplifying the pressure to select an appropriate study design to obtain reliable and... (Review)
Review
The numbers of clinical trials have increased exponentially over the last decade, amplifying the pressure to select an appropriate study design to obtain reliable and valid evidence. The ability to find, critically appraise and use evidence to develop new interventions is fundamental to evidence-based medicine. Different study designs have their own advantages and disadvantages, and provide different evidentiary value. This article provides an overview of clinical trials, illustrating that, ultimately, the study design chosen needs to meet experimental and funding limitations, while minimising error.
Topics: Clinical Trials as Topic; Humans; Observational Studies as Topic; Research Design; Systematic Reviews as Topic
PubMed: 32468947
DOI: 10.12968/hmed.2020.0127 -
European Journal of Epidemiology Jan 2023A detailed examination of the 1930 Lanarkshire Milk Experiment (LME) by the famous statistician William Sealy Gossett ("Student"), which appeared in Biometrika in 1931,... (Review)
Review
A detailed examination of the 1930 Lanarkshire Milk Experiment (LME) by the famous statistician William Sealy Gossett ("Student"), which appeared in Biometrika in 1931, is re-examined from a more modern perspective. The LME had a complicated design whereby 67 schools in Lanarkshire were allocated to receive either raw or pasteurised milk but pupils within the schools were allocated to either receive milk or to act as controls. Student's criticisms are considered in detail and examined in terms of subsequent developments on the design and analysis of experiments, in particular as regards appropriate estimation of standard errors of treatment estimates when an incomplete blocks structure has been used. An analogy with a more modern trial in osteoarthritis is made. Suggestions are made as to how analysis might proceed if the original data were available. Some lessons for observational studies in epidemiology are drawn and it is speculated that hidden clustering structures might be an explanation as to why results may vary from observational study to observational study by more than conventionally calculated standard errors might suggest.
Topics: Humans; Animals; Milk; Schools; Observational Studies as Topic
PubMed: 36477576
DOI: 10.1007/s10654-022-00941-x -
Nature Aging May 2023The increasing number of people with dementia globally illustrates the urgent need to reduce dementia's scale and impact. Lifetime social participation may affect... (Review)
Review
The increasing number of people with dementia globally illustrates the urgent need to reduce dementia's scale and impact. Lifetime social participation may affect dementia risk by increasing cognitive reserve, and through brain maintenance by reducing stress and improving cerebrovascular health. It may therefore have important implications for individual behavior and public health policy aimed at reducing dementia burden. Observational study evidence indicates that greater social participation in midlife and late life is associated with 30-50% lower subsequent dementia risk, although some of this may not be causal. Social participation interventions have led to improved cognition but, partly due to short follow-up and small numbers of participants, no reduction in risk of dementia. We summarize the evidence linking social participation with dementia, discuss potential mechanisms by which social participation is likely to reduce and mitigate the impact of neuropathology in the brain, and consider the implications for future clinical and policy dementia prevention interventions.
Topics: Humans; Dementia; Social Participation; Brain; Cognition; Cognitive Reserve; Observational Studies as Topic
PubMed: 37202513
DOI: 10.1038/s43587-023-00387-0 -
The New England Journal of Medicine Oct 2021
Topics: Causality; Confounding Factors, Epidemiologic; Data Interpretation, Statistical; Epidemiologic Research Design; Humans; Observational Studies as Topic; Public Health
PubMed: 34596980
DOI: 10.1056/NEJMp2113319