06. Study Designs

Study design: Overview

  • ANALYTIC: attempts to quantify the effect of an exposure on an outcome.
    • Experimental: the exposure is manipulated, prospective.
    • Observational analytic: an exposure and its effect on the outcome are measured, can be retrospective or prospective.
  • SYSTEMATIC REVIEW: examines existing evidence from multiple sources to answer a specific research question.
    • Systematic review: rigorous identification and examination of published studies with goal of summarizing best available research.
    • Meta-analysis: builds on systematic review, adding a statistical re-analysis of pooled data from all applicable valid studies to obtain a summary estimate of the effect of an intervention on an outcome.
  • DESCRIPTIVE (non-analytic): attempts to describe what is happening. It does not quantify any relationships.

Analytic Studies: Design Characteristics

Analytic studies: quantifies the effect of an exposure on the outcome

Design of study

Description

Pros

Cons

Typical outcomes

Type of question

Examples

Experimental: the exposure is manipulated

RCT

- parallel

- crossover

Prospective, groups randomized to exposure

Minimize bias and confounding

Expensive, time consuming, need for follow-up, ethics

Odds ratio

Relative risk

Therapy

Dabigatran versus Warfarin, early goal directed therapy

Non-randomized controlled trial

Prospective, groups not randomized

Detects association between intervention and outcome

No randomization, lack of control of selective bias, additional cons as above

Odds ratio

Relative risk

Therapy

 

Observational: measures an exposure and its effect on the outcome

Cohort

Prospective or retrospective, examines associations of exposures and outcomes over time

Stronger evidence for causality, yields incidence, relative risk

Confounding, time consuming, need for follow-up, condition must be sufficiently prevalent

Incidence

Relative risk

Hazards ratio

Prognosis

Etiology

Therapy

Framingham Heart Study

Cross-sectional

Surveys sample population at one point in time

Determines prevalence and associations, analysis of diagnostic tests

Single point in time, unable to determine causality, bias, confounding

Prevalence

Diagnosis

Etiology

NHANES (National Health and Nutrition Examination Survey)

Case-control

Compares group with and without disease

Cheap, efficient, rare diseases

Bias, chance, confounding

Odds ratio

Therapy

Prognosis

Harm

Etiology

Food-borne outbreak investigation, diethylstilbestrol and vaginal cancer

 

 

Systematic Review: Design Characteristics

Systematic review: Examines existing evidence from multiple sources

Design of study

Description

Pros

Cons

Type of question

Examples

Systematic review

Rigorous examination of existing data from multiple sources using rigorous criteria and search strategy

Cheap

Limited longevity of relevance depending on quality of studies

Diagnosis

Therapy

Prognosis

Cochrane reviews

Meta-analysis

Statistical analysis of pooled data found in studies obtained through rigorous criteria and search strategy

Produce summary statistics, cheap

Depends on quality of included data (“garbage in, garbage out”)

Diagnosis

Therapy

Prognosis

Safety/efficacy of bare metal vs drug eluting stents

Guidelines

Combination of systemic reviews, meta-analyses, and expert consensus

Expert analysis

May be influenced by sponsoring organization (i.e. government analysis usually more cost-conscious versus societies more aggressive with testing/treatment)

 

Diagnosis

Therapy

Prognosis

Harm

US Preventative Services Taskforce (USPSTF)

Descriptive Studies: Design Characteristics

Descriptive studies: describes what is happening, does not quantify any relationships

Design of study

Description

Pros

Cons

Case series or report

Report on treatment or outcome of individual patient(s)

Describes unique case

Little statistical validity

Survey

Assess opinions, experiences

Cheap, far-reaching

Qualitative

Explore, describe, and explain-health related phenomena

Gives voice, adds human experience