Research Design and Biostatistics

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Chapter 13

Research Design and Biostatistics

Contents

section 1 Introduction

Critical review of medical research is essential for orthopaedic surgeons. Experiments are conducted in both the clinical and basic sciences, and decisions are based on the results of these experiments and associated statistical analyses. The concepts that support or refute these decisions and generalizations must be understood by the astute consumer of medical literature.

Research starts with developing a question that is important to a particular area of investigation or clinical population; then the study population is defined, and the most appropriate outcome measures and variables are selected. It is important that the research team collaborate so that their combined expertise can contribute to the study aims.

This chapter describes some important concepts to consider in designing a research study and in analyzing and interpreting results.

section 2 Common Research Designs and Research Terminology

PROSPECTIVE STUDIES

II RETROSPECTIVE STUDIES

III LONGITUDINAL STUDIES

IV OBSERVATIONAL RESEARCH DESIGNS

    These designs can be prospective, retrospective or longitudinal (Figures 13-1 and 13-2). Common observational designs are as follows:

Case reports

Case series

Case-control studies

Cohort study

Cross-sectional study designs

RESEARCH TERMINOLOGY

Instrument validity:

Instrument reliability:

Clinical studies can be designed to determine superiority of one treatment over another, whether one treatment is no worse than another (noninferiority), or whether both treatments are equally effective (equivalency).

Clinical research can be designed to assess outcomes data that are reported by the patient (subjective) or collected by an examiner (objective).

VI EXPERIMENTAL RESEARCH DESIGNS

VII POTENTIAL PROBLEMS WITH RESEARCH DESIGNS

Internal validity concerns the quality of a research design and how well the study is controlled and can be reproduced. External validity concerns the ability of the results to generalize to a whole population of interest.

Confounding variables are factors extraneous to a research design that potentially influence the outcome. Conclusions regarding cause-effect relationships may be explained by confounding variables, instead of by the treatment or intervention being studied and must therefore be controlled or accounted for.

Bias is unintentional, systematic error that threatens the internal validity of a study. Sources of bias include selection of subjects (sampling bias), loss of subjects to follow-up (nonresponder bias), observer/interviewer bias, and recall bias.

Protection against these threats can be achieved through randomization (i.e., random allocation of one or more treatments) to ensure that bias and confounding factors are distributed equally among the study groups. Single blinding (examiner or patient is unaware of to which study condition the patient is assigned) or double blinding (both examiner and patient are unaware of assignment of study condition) is important for minimizing bias.

Control groups can help account for the potential placebo effect of interventions.

Control subjects are often matched on the basis of specific characteristics (e.g., gender, age), which helps account for potential confounding variables that may influence the interpretation of research findings.

The strongest research design involves the use of random allocation, blinding, and use concurrent control subjects who are matched to the experimental group(s).

VIII DESCRIPTIVE AND CONTROLLED LABORATORY STUDIES

These studies are common in basic science research, but they may involve many of the common concepts of clinical research, and similar statistical methods and design methods are used to protect against sources of bias and confounding.

section 4 Concepts of Epidemiology

DEFINITIONS

    Epidemiology is the study of the distribution and determinants of disease. The following measures are commonly used in this type of research.

Prevalence is the proportion of existing cases or conditions of injuries or disease within a particular population.

Incidence (absolute risk) is the proportion of new injuries or disease cases within a specified time interval (a follow-up period is required).

Relative risk (RR) is a ratio between the incidences of an outcome in two cohorts. Typically, a treated or exposed cohort (in the numerator of the ratio) is compared with an untreated or unexposed (control) group (in the denominator of the ratio). Values can range from 0 to infinity and are interpreted as follows:

Odds ratios are calculated from the probabilities of an outcome in two cohorts.

Interpreting relative risk and odds ratio:

II CLINICAL USEFULNESS OF DIAGNOSTIC TESTS

2×2 contingency table (Figure 13-3) can be used to plot the occurrences of a disease or outcome of interest among patients whose diagnostic test results were positive or negative.

Analysis of diagnostic ability

1. Sensitivity:

2. Specificity:

3. Positive predictive value:

4. Negative predictive value: