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This chapter focuses on the foundations of study design and statistical analysis in psychological research. It explores strategies for ensuring internal validity, such as randomization, control groups, and large sample sizes. Additionally, it addresses the complexity of human behavior by exploring multivariate experiments and the use of artificial intelligence and machine learning in neuroscience. The chapter also discusses the replication crisis and the emergence of open science practices, encouraging students to think critically about isolated scientific findings and offering tools for identifying credible research. Lastly, it critiques null hypothesis significance testing and p-values while providing an overview of key statistical topics like correlation coefficients, standardized mean differences, and regression.
The traditional case register involved assembling records of people with a given condition in order to support cohort studies to describe and investigate the course of their condition and other outcomes. This old design has been resurrected and revolutionised following the widespread implementation of fully electronic healthcare records over the past few decades, providing ‘big data’ resources that are both large and very detailed. These, in turn, are being further enhanced through linkages with complementary administrative data (both health and non-health) and through natural language processing generating structured meta-data from source text fields. This chapter provides an overview of this rapidly developing research infrastructure, considering and advising on some of the challenges faced by researchers planning studies using clinical data and by those considering future resource development.
This chapter explores the various steps involved in conducting research, including defining the research problem, formulating research questions, selecting an appropriate research design, choosing participants, employing data collection methods, processing and analyzing data, interpreting results, and writing research reports. Understanding these steps is important as it provides a structured framework for approaching research, making the entire process less daunting. Special emphasis is placed on determining the appropriate research design and selecting participants. Additionally, the chapter introduces various data collection methods, techniques, and tools used by applied linguistics researchers. The importance of data processing and analysis will also be highlighted. Moreover, you will explore how to develop purpose statements for both qualitative and quantitative research and learn to identify the strengths of a well-conducted study.
Although decentralized research is being used more frequently, few data are available regarding barriers for potential subjects related to engaging in decentralized research with remote biospecimen collection, especially within pregnancy and birth cohorts that include individuals of diverse racial and ethnic backgrounds.
Methods:
Focus groups and individual interviews with pregnant and postpartum women were conducted in English and Spanish. Thematic analysis was used to identify motivators and barriers to participation in decentralized research involving biospecimens.
Results:
Sixty women (35% Hispanic/Latino, 23% Black, 18% Asian, 15% non-Hispanic White) participated in 10 focus groups (English = 8, Spanish = 2) and 11 individual interviews (English = 7, Spanish = 4). Three themes emerged about factors that could promote participation in decentralized biospecimen collection: 1) convenience, 2) autonomy, and 3) benefit (to self, community or society). Four themes emerged about potential barriers: 1) lack of interaction with trained professionals, 2) inability to coordinate with existing clinical care, 3) discomfort and invasiveness, and 4) concerns about data transparency and security. Overall, participants felt more comfortable providing biospecimens for themselves compared to their child and with biospecimens perceived as less painful or invasive to obtain.
Discussion:
Our findings suggest that transparency about the purposes and use of collecting biospecimen and clear instructions (such as written and instructional videos) could improve biospecimen collection in decentralized pregnancy and birth cohorts. Additionally, opportunities for virtual interaction with study staff and options related to collection of certain biospecimens such as blood (mobile collection unit with trained staff versus a self-collection device) may also improve participant engagement.
Graphical displays are often utilised for high-quality reporting of meta-analyses. Previous work has presented augmentations to funnel plots that assess the impact that an additional trial would have on an existing meta-analysis. However, decision-makers, such as the National Institute for Health and Care Excellence in the United Kingdom, assess health technologies based on their cost-effectiveness, as opposed to efficacy alone. Motivated by this fact, this article outlines a novel approach, developed for augmenting funnel plots, based on the ability of an additional trial to change a decision regarding the optimal intervention. The approach is presented for a generalised class of economic decision models, where the clinical effectiveness of the health technology of interest is informed by a meta-analysis, and is illustrated with an example application. The ‘decision contours’ produced from the proposed methods have various potential uses not only for decision-makers and research funders but also for other researchers, such as meta-analysts and primary researchers designing new studies, as well as those developing health technologies, such as pharmaceutical companies. The relationship between the new approach and existing methods for determining sample size calculations for future trials is also considered.
As we progress through this part and the next, you will be introduced to the different ways in which epidemiologists go about analysing the factors that are associated with people becoming ill or getting better. Each of these has a role to play in building up our knowledge about what influences human health. Our objective here is to provide an overview of the range of techniques that are available and to develop your understanding of which of these might be more appropriate in any given situation. One way to think about these techniques is as a set of tools for tackling a range of problems, much as a carpenter has a box full of tools for tackling different aspects of building a house. No one tool is useful in every situation, and some are more useful at certain stages of the construction process than others. Some even have features that make them useful in a variety of situations. Of course, context is everything, so even when a tool might not look like it’s the ‘right’ one in a particular situation, if the results are robust and reliable then that might be all that matters.
Insufficient sample sizes threatened the fidelity of the primary research trials. Even if the research group recruits a sufficient sample size, the sample may lack diversity, reducing the generalizability of the results of the study. Evaluating the effectiveness of online advertising platforms (e.g., Facebook & Google Ads) versus traditional recruitment methods (e.g., flyers, clinical participation) is essential.
Methods:
Patients were recruited through email, electronic direct message, paper advertisements, and word-of-mouth advertisement (traditional) or through Google Ads and Facebook Ads (advertising) for a longitudinal study on monitoring COVID-19 using wearable devices. Participants were asked to wear a smart watch-like wearable device for ∼ 24 hours per day and complete daily surveys.
Results:
The initiation conversion rate (ICR, impressions to pre-screen ratio) was better for traditional recruitment (24.14) than for Google Ads, 28.47 ([0.80, 0.88]; p << 0.001). The consent conversion rate (CCR, impressions to consent ratio) was also higher for traditional recruitment (66.54) than for Google Ads, 2961.20 ([0.015, 0.030]; p << 0.001). Participants recruited through recommendations or by paper flier were more likely to participate initially (Χ2 = 23.65; p < 0.005). Clinical recruitment led to more self-reporting white participants, while other methods yielded great diversity (Χ2 = 231.47; p << 0.001).
Conclusions:
While Google Ads target users based on keywords, they do not necessarily improve participation. However, our findings are based on a single study with specific recruitment strategies and participant demographics. Further research is needed to assess the generalizability of these findings across different study designs and populations.
In order to examine our three questions, we need objective research methods. Estimating whether a treatment can work, does work, and has value requires a wide range of research strategies. Evidence establishing that a treatment works under controlled conditions does not necessarily assure benefits when the intervention is applied in clinical practice. This chapter considers the development of a research protocol, and biases that might be attributable to participant recruitment, enrollment, retention, and dissemination of findings. In practice, establishing the value of a treatment should consider an examination of the existing literature, development of thorough research plans, recognition of the strengths and weaknesses of the chosen research methods, and integration of study results within a wider body of knowledge. We challenge beliefs in a hierarchy of methods that assumes some methods, such as the RCT, are free from bias.
It was identified in the largest graduate unit of the Faculty of Medicine of a major Canadian University that there was a critical unmet curricular need for an introductory statistics and study design course. Based on the collective findings of an external institute review, both quantitative and qualitative data were used to design, develop, implement, evaluate, and refine such a course.
Methods
In response to the identified need and inherent challenges to streamlining curriculum development and instructional design in research-based graduate programs representing many biomedical disciplines, the institute used the analyze, design, develop, implement and evaluate instructional design model to guide the data-driven development and ongoing monitoring of a new study design and statistics course.
Results
The results demonstrated that implementing recommendations from the first iteration of the course (Fall 2021) into the second iteration (Winter 2023) led to improved student learning experience (3.18/5 weighted average (Fall 2021) to 3.87/5 (Winter 2023)). In the second iteration of the course, a self-perceived statistics anxiety test was administered, showing a reduction in statistics anxiety levels after completing the course (2.41/4 weighted average before the course to 1.65/4 after the course).
Conclusion
Our experiences serve as a valuable resource for educators seeking to implement similar improvement approaches in their educational settings. Furthermore, our findings offer insights into tailoring course development and teaching strategies to optimize student learning.
Involving participants in the design of clinical trials should improve the overall success of a study. For this to occur, streamlined mechanisms are needed to connect the populations potentially impacted by a given study or health topic with research teams in order to inform trial design in a meaningful and timely manner. To address this need, we developed an innovative mechanism called the “ResearchMatch Expert Advice Tool” that quickly obtains volunteer perspectives from populations with specific health conditions or lived experiences using the national recruitment registry, ResearchMatch. This tool does not ask volunteers to participate in the trial but allows for wider community feedback to be gathered and translated into actionable recommendations used to inform the study’s design. We describe early use cases that shaped the current Expert Advice Tool workflow, how results from this tool were incorporated and implemented by studies, and feedback from volunteers and study teams regarding the tool’s usefulness. Additionally, we present a set of lessons learned during the development of the Expert Advice Tool that can be used by other recruitment registries seeking to obtain volunteer feedback on study design and operations.
In this chapter, we look at the analytic studies that are our main tools for identifying the causes of disease and evaluating health interventions. Unlike descriptive epidemiology, analytic studies involve planned comparisons between people with and without disease, or between people with and without exposures thought to cause (or prevent) disease. They try to answer the questions, ‘Why do some people develop disease?’ and ‘How strong is the association between exposure and outcome?’. This group of studies includes the intervention, cohort and case–control studies that you met briefly in Chapter 1. Together, descriptive and analytic epidemiology provide information for all stages of health planning, from the identification of problems and their causes to the design, funding and implementation of public health solutions and the evaluation of whether these solutions really work and are cost-effective in practice.
The overarching goal of public health is to maximise the health of the population, and to achieve this we need evidence about what works and what does not work. Good studies are difficult to design and implement, and interpretation of their results and conclusions is not always as straightforward as we might hope. How, then, can we make the best use of this information? In the next three chapters we look at ways to identify, appraise, integrate and interpret the literature to generate the evidence we need to inform policy and practice. In this chapter we focus on interpreting the results from a single study, because if they are not valid they will be of limited value. The central question we have to answer when we read a study report is, ‘Are the results of the study valid?’
Confounding refers to a mixing or muddling of effects that can occur when the relationship we are interested in is confused by the effect of something else. It arises when the groups we are comparing are not completely exchangeable and so differ with respect to factors other than their exposure status. If one (or more) of these other factors is a cause of both the exposure and the outcome, then some or all of an observed association between the exposure and outcome may be due to that factor.
In the previous chapter we alluded to what is sometimes called ‘secondary’ prevention, where instead of trying to prevent disease from occurring, we try to detect it earlier, in the hope that this will enable more effective treatment and thus improved health outcomes. This is an aspect of public health that has great intuitive appeal, especially for serious conditions such as cancer, where the options for primary prevention can be very limited. However, screening programs are usually very costly exercises and they do not always deliver the expected benefits in terms of improved health outcomes. In this chapter we introduce you to the requirements for implementing a successful screening program and to some of the problems that we encounter when trying to determine whether such a program is actually beneficial in practice.
The New Jersey Kids Study (NJKS) is a transdisciplinary statewide initiative to understand influences on child health, development, and disease. We conducted a mixed-methods study of project planning teams to investigate team effectiveness and relationships between team dynamics and quality of deliverables.
Methods:
Ten theme-based working groups (WGs) (e.g., Neurodevelopment, Nutrition) informed protocol development and submitted final reports. WG members (n = 79, 75%) completed questionnaires including de-identified demographic and professional information and a modified TeamSTEPPS Team Assessment Questionnaire (TAQ). Reviewers independently evaluated final reports using a standardized tool. We analyzed questionnaire results and final report assessments using linear regression and performed constant comparative qualitative analysis to identify central themes.
Results:
WG-level factors associated with greater team effectiveness included proportion of full professors (β = 31.24, 95% CI 27.65–34.82), team size (β = 0.81, 95% CI 0.70–0.92), and percent dedicated research effort (β = 0.11, 95% CI 0.09–0.13); age distribution (β = −2.67, 95% CI –3.00 to –2.38) and diversity of school affiliations (β = –33.32, 95% CI –36.84 to –29.80) were inversely associated with team effectiveness. No factors were associated with final report assessments. Perceptions of overall initiative leadership were associated with expressed enthusiasm for future NJKS participation. Qualitative analyses of final reports yielded four themes related to team science practices: organization and process, collaboration, task delegation, and decision-making patterns.
Conclusions:
We identified several correlates of team effectiveness in a team science initiative's early planning phase. Extra effort may be needed to bridge differences in team members' backgrounds to enhance the effectiveness of diverse teams. This work also highlights leadership as an important component in future investigator engagement.
This chapter describes the basics of scientific figures. It provides tips for identifying different types of figures, such as experimental protocol figures, data figures, and summary figures. There is a description of ways to compare groups and of different types of variables. A short discussion of statistics is included, describing elements such as central tendency, dispersion, uncertainty, outliers, distributions, and statistical tests to assess differences. Following that is a short overview of a few of the more common graph types, such as bar graphs, boxplots, violin plots, and raincloud plots, describing the advantages that each provides. The end of the chapter is an “Understanding Graphs at a Glance” section which gives the reader a step-by-step outline for interpreting many of the graphs commonly used in neuroscience research, applicable independently of the methodology used to collect those data.
Network studies follow an explicit form, from framing questions and gathering data, to processing those data and drawing conclusions. And data processing leads to new questions, leading to new data and so forth. Network studies follow a repeating lifecycle. Yet along the way, many different choices will confront the researcher, who must be mindful of the choices they are making with their data and the choices of tools and techniques they are using to study their data. In this chapter, we describe how studies of networks begin and proceed, the life-cycle of a network study
Research articles in the clinical and translational science literature commonly use quantitative data to inform evaluation of interventions, learn about the etiology of disease, or develop methods for diagnostic testing or risk prediction of future events. The peer review process must evaluate the methodology used therein, including use of quantitative statistical methods. In this manuscript, we provide guidance for peer reviewers tasked with assessing quantitative methodology, intended to complement guidelines and recommendations that exist for manuscript authors. We describe components of clinical and translational science research manuscripts that require assessment including study design and hypothesis evaluation, sampling and data acquisition, interventions (for studies that include an intervention), measurement of data, statistical analysis methods, presentation of the study results, and interpretation of the study results. For each component, we describe what reviewers should look for and assess; how reviewers should provide helpful comments for fixable errors or omissions; and how reviewers should communicate uncorrectable and irreparable errors. We then discuss the critical concepts of transparency and acceptance/revision guidelines when communicating with responsible journal editors.
At the core of epidemiology is the use of quantitative methods to study health, and how it may be improved, in populations. It is important to note that epidemiology concerns not only the study of diseases but also of all health-related events. Rational health-promoting public policies require a sound understanding of causation. The epidemiological analysis of a disease or activity from a population perspective is vital in order to be able to organize and monitor effective preventive, curative and rehabilitative services. All health professionals and health-service managers need an awareness of the principles of epidemiology. They need to go beyond questions relating to individuals to challenging fundamentals such as ‘Why did this person get this disease at this time?’, ‘Is the occurrence of the disease increasing and, if so, why?’ and ‘What are the causes or risk factors for this disease?’
The United States Congress passed the 21st Century Cures Act mandating the development of Food and Drug Administration guidance on regulatory use of real-world evidence. The Forum on the Integration of Observational and Randomized Data conducted a meeting with various stakeholder groups to build consensus around best practices for the use of real-world data (RWD) to support regulatory science. Our companion paper describes in detail the context and discussion of the meeting, which includes a recommendation to use a causal roadmap for study designs using RWD. This article discusses one step of the roadmap: the specification of a sensitivity analysis for testing robustness to violations of causal model assumptions.
Methods:
We present an example of a sensitivity analysis from a RWD study on the effectiveness of Nifurtimox in treating Chagas disease, and an overview of various methods, emphasizing practical considerations on their use for regulatory purposes.
Results:
Sensitivity analyses must be accompanied by careful design of other aspects of the causal roadmap. Their prespecification is crucial to avoid wrong conclusions due to researcher degrees of freedom. Sensitivity analysis methods require auxiliary information to produce meaningful conclusions; it is important that they have at least two properties: the validity of the conclusions does not rely on unverifiable assumptions, and the auxiliary information required by the method is learnable from the corpus of current scientific knowledge.
Conclusions:
Prespecified and assumption-lean sensitivity analyses are a crucial tool that can strengthen the validity and trustworthiness of effectiveness conclusions for regulatory science.