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A Whirlwind Tour of Alternative Study Designs

Philip J Clare

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Overview

  1. Introduction
  2. ‘Standard’ designs
  3. Experimental Study Designs
  4. Descriptive Study Designs
  5. Other designs
  6. Conclusions
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1. Introduction

  • Descriptive vs experimental
  • Most important thing is to use the best design for the specific question being asked
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1. Introduction

  • Descriptive studies:
    • Involve observing and assessing
    • Can be used for things where it isn’t ethical to randomise
  • Experimental studies:
    • Involve the researchers changing something
    • Tend to be more difficult and expensive
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2. 'Standard' Designs

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2. 'Standard' Designs

  • Three standard designs we should all know:
    • Randomised controlled trials
    • Cross-sectional surveys
    • Longitudinal surveys
  • These tend to form the standard by which other study designs are considered
    • eg how is a particular design better or worse than an RCT
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2. 'Standard' Designs

Randomised Controlled Trials

  • Randomise to group; give each group a different intervention; follow-up and compare the groups
  • Gold standard for causation
    • Ignorability
  • Efficacy vs Effectiveness
  • Not always appropriate
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2. 'Standard' Designs

Randomised Controlled Trials

Randomised Controlled Trials

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2. 'Standard' Designs

Cross-sectional survey

  • Tends to be exploratory
    • Hard to design an RCT when you don’t know what the intervention should be
  • Can be cheap and easy to design, run, analyse and report
  • Difficult to recruit representative samples
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2. 'Standard' Designs

Prospective Cohort

  • In some ways, just an extension of cross-sectional
    • But there are a range of specific issues that arise in longitudinal research that do not apply in cross-sectional research
    • There are also specific benefits
  • Can be either descriptive or analytic
  • With limitations, can be used for causal inference
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2. 'Standard' Designs

Descriptive Prospective Cohort

Descriptive Prospective Cohort

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2. 'Standard' Designs

Analytic Prospective Cohort

Analytic Prospective Cohort

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3. Experimental Designs

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3. Experimental Designs

  • There are lots of other experimental designs:
    • Pre/post experimental trials
    • Cluster RCTs
    • Stepped-wedge
    • Cross-over Trials
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3. Experimental Designs

Pre/post Designs

  • Measure participants, then administer intervention and measure again
  • Participants form their own control
  • Within subjects designs reduces variance, so requires smaller sample sizes
  • Don’t know what would have happened without the experiment
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3. Experimental Designs

Pre/post Designs

Pre/post Designs

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3. Experimental Designs

Cluster RCTs (1)

  • Instead of randomisation at the individual level, randomises at the cluster eg hospital, school
  • Good for systemic interventions eg new hospital process
  • Also good for interventions where there is risk of cross-contamination eg participants talking to each other while in hospital
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3. Experimental Designs

Cluster RCTs

Cluster RCTs

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3. Experimental Designs

Stepped Wedge (2)

  • Instead of randomising clusters to intervention or control, randomises the order that intervention begins
  • Eventually rolls out to all clusters
  • Pragmatic design – not great for efficacy trials
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3. Experimental Designs

Stepped Wedge

Stepped Wedge

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3. Experimental Designs

Cross-over Trials (3)

  • Randomise to order; give participants intervention; wait for effect to fade; give participants intervention they didn’t receive in the first round
  • Very good for experiments with multiple, short-acting interventions
  • Similar benefit to pre/post – ppnts form their own control
  • Allows for testing of multiple interventions
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3. Experimental Designs

Cross-over Trials

Cross-over Trials

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4. Non-experimental Designs

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4. Non-experimental Designs

  • These include:
    • Qualitative studies
    • Case studies
    • Case-control studies
    • Ecological studies
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4. Non-experimental Designs

Qualitative Studies

  • Very in-depth
  • Great for explaining WHY something happens
  • Don't generalise (and aren't intended to)
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4. Non-experimental Designs

Case Studies (4)

  • In-depth
  • Illustrative, rather than representative
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4. Non-experimental Designs

Case-control Studies (5)

  • Find ‘cases’ (ie people where the outcome has occurred), then see how they differ from others in whom the outcome did not occur
  • Good for rare outcomes that might not be expected to occur in a prospective study
  • Can be unreliable – relies on data that has already been collected
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4. Non-experimental Designs

Case-control Studies

Case-control Studies

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4. Non-experimental Designs

Ecological Studies (6)

  • Defined by the 'level' of measurement
    • Namely, measurement is at a macro rather than individual level
  • Can be used to measure very rare diseases etc
  • Can lead to the 'ecological fallacy'
    • Where the nature of the group is falsely used to describe the nature of individuals in the group
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5. Other Designs

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5. Other Designs

Interrupted Time Series (7)

  • Measures the change in a trend that is ‘interrupted’
  • Technically involves an intervention – but often out of the researchers hands
  • Sometimes the only way to assess whether high-level interventions have any effect.
  • Can be problematic because effects often aren't instant
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5. Other Designs

Interrupted Time Series

Interrupted Time Series

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5. Other Designs

Linkage Studies (8)

  • Can be either descriptive or experimental
  • Can involve linking administrative data to an existing study such as a cohort study, or linking different administrative datasets to each other
  • Can be very complex, and involve extremely large datasets
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6. Conclusions

  • There are lots of study designs that can be used
  • Most important thing is to use the best design for the specific question being asked
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7. References

  1. Murray DM, Varnell SP, Blitstein JL. Design and Analysis of Group-Randomized Trials: A Review of Recent Methodological Developments. Am J Public Health. 2004;94(3):423-432.
  2. Hemming K, Haines TP, Chilton PJ, Girling AJ, Lilford RJ. The stepped wedge cluster randomised trial: rationale, design, analysis, and reporting. BMJ. 2015;350:h391.
  3. Johnson DE. Crossover experiments. WIREs Comp Stat. 2010;2:620-625.
  4. Yin RK. Case study research: design and methods. SAGE Publications. 2013.
  5. Mann CJ. Observational research methods. Research design II:cohort, cross sectional, and case-control studies. Emerg Med J. 2003;20:54-60.
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7. References

  1. Morgenstern H. Ecologic studies in epidemiology: concepts, principles, and methods. Annu Rev Public Health. 1995;16:61-81.
  2. Kontopantelis E. Regression based quasi-experimental approach when randomisation is not an option: interrupted time series analysis. BMJ. 2015;350:h2750.
  3. Bohensky MA, Jolley D, Sundararajan V, Evans S, Pilcher DV, Scott I, Brand CA. Data Linkage: A powerful research tool with potential problems. BMC Health Services Research. 2010;10:346.
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Overview

  1. Introduction
  2. ‘Standard’ designs
  3. Experimental Study Designs
  4. Descriptive Study Designs
  5. Other designs
  6. Conclusions
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