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Non-randomised studies. Methodologies for a new era summer school School of Applied Social Studies, University College Cork 22 June 2011 Dr Paul Montgomery Jennifer Burton. Why bother?. - PowerPoint PPT Presentation
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Non-randomised studies
Methodologies for a new era summer school
School of Applied Social Studies, University College Cork
22 June 2011
Dr Paul MontgomeryJennifer Burton
Why bother?
The data from a good study can be analysed in The data from a good study can be analysed in many ways, but no amount of clever analysis many ways, but no amount of clever analysis
can compensate for the problems with the can compensate for the problems with the design of a study.design of a study.
(Altman, 1991)(Altman, 1991)
Introduction
It is sometimes impossible or undesirable to influence events in a human sample You may not be able to control group allocation It may be unethical to expose or withhold an
intervention
An appreciation of the varieties of study designs available can reduce the need to reinvent the wheel and to rediscover the mistakes of others. It can also help to end the “scandal of research”’
(Altman, 1993)
Aims
Identify types of questions that can be answered using non-randomised methods
Describe several non-randomised designsHighlight the strengths and weaknesses of
common non-randomised designs
Question to design
Prevalence/ incidenceRisk and protective factorsPrognosisHarmEffectiveness
Internally Valid
Evidence of Effectiveness
Meta-Analysis of Randomised Trials Randomised Trials
Non-randomised studies Cohort studies Case-Control studies
Non-comparative studies Case Series (open trial)
Case reports
Expert opinion
The Hierarchy of EvidenceFor Intervention Studies
Illustrative examples, hypothesis generating studies
Question to Design
In practice, methods tend to be complementary in answering questions
Each method may be used to answer several types of questions
Several studies may help tease apart a question
Prevalence
How many people have mental health disorders?
Surveys
Census (one-time full population) Ideal but expensive, difficult, likely to miss people
from marginal groups
Cross section (one-time sample) Must consider many potential biases due to
geography, time, etc.
Cross-Sectional Survey
Identify a sample of adults representative of the population
Measure symptoms of mental health problems
Calculate the number of people above a given threshold
Surveys
Usefully estimate prevalence or incidence and associations
Comparisons may be made between different subgroups to identify associations
Risk and Protection
Does smoking cause cancer?
Risk and Protection
What factors can predict falls in the elderly?
Retrospective Cohort
Take all elderly people in OxfordLook at
Characteristics of their homes Individual factors (e.g. medication
use) Other predictors?
Look for association between these factors and falls
Retrospective Cohort
Backward looking surveyRelatively inexpensive and practicalGood for detecting latent outcomesProne to several sources of bias (selection,
participant recall, etc.)
Risk and Protection
What are the factors that contribute to chronic fatigue syndrome?
Prospective Cohort
Take all babies born in a given period in 1970
Survey them regularlyLook for correlations between
variables (e.g. maternal depression) and outcomes (e.g. chronic fatigue)
Prospective Cohort
Identifies temporal relationshipsCan examine multiple effects of exposure Loss-to-follow-up can be a problem Inefficient for the evaluation of rare problems
unless the attributable risk is high e.g. 1970 British Cohort Study (ongoing)
http://www.cls.ioe.ac.uk/
Risk and Protection
Is fish oil good for my mental health?
Ecological Studies
Compare countries’ consumption of fish oil to their rates of depression
Increased consumption of fish oil lowers a nation’s rate of depression (Hibbeln 2001), but eating fish is not the only difference among countries
Ecological Studies
Large unit of analysis (e.g. countries)May identify population-level risk and protective
factorsBecause the unit of observation is not the
individual subject, they are subject to the ecological fallacy when they overlook important sources of variance
Risk and Protection
Is running bad for my knees?
Case Control
Identify a group of runnersThen find a group of people who
don’t run matched for age, sex, weight and other variables
Test for associations between knee problems and being a runner
Case Control
Inexpensive and practicalGood for generating hypothesesLacks a temporal dimensionUnless data come from a population-based
survey, cannot give incidence and prevalence data
Prognosis
My husband has just taken 4 times the recommended dose of purple pills.
What’s going to happen?
Case Report
An otherwise healthy 60 year old man was prescribed 10 mg of vardenafil (Levitra, Bayer) for sexual dysfunction. Because this was ineffective, he increased the dose to 40 mg. Three hours later, he had a tonic-clonic seizure, seen by his relatives.
On admission to hospital, neurological examination, brain magnetic resonance imaging, and electroencephalography after sleep deprivation were normal. Stress electrocardiography, echocardiography, and cardiac scan with dipyridamole test as well as carotid doppler ultrasonography did not show concomitant cardiac diseases. The man was told to stop using vardenafil.
Two months later he had a new tonic-clonic seizure, four hours after taking 30 mg of vardenafil. At eight months' follow-up he is seizure-free without treatment.
Pasquale Striano, Federico Zara, Carlo Minetti (professor of paediatrics), Salvatore Striano (2006). Epileptic seizures can follow high doses of oral vardenafil. BMJ;333:785.
Case Report
Inexpensive and quickMay draw attention to important
clinical and research issuesIn rich detail, describes conditions
and outcomes May not be representative, does
not usually provide evidence of causation
Case Series
Several case studiesDraws attention to patterns in client populationsCommon in aetiological research
Break
Effectiveness
Does abstinence education reduce the likelihood of premarital sex?
Pre-post (single group)
Take a class of kidsAsk them if they will have sex before marriageThey attend an abstinence-based education
programmeAsk them if they will have sex before marriage
Pre-post (single group)
Inexpensive, generally easier than controlled studies
Provides some evidence of temporal relationships
Usually lacks a plausible counterfactual (i.e. what would have happened in the absence of intervention)
Effectiveness
Does Head Start improve IQ?
Between Group
Look at all the kids in New York born in 1980 who were eligible for Head Start
Compare those who attended to those who did not attend
If possible, collect measures before and after attendance
Between Group
Provides a counterfactual scenario, can give evidence of temporal relationships.
Groups may differ on both measured and unmeasured variables, observed differences may be attributable to factors other than the intervention.
Effectiveness
Does having regular contact with a social worker improve outcomes for fostered children?
Historical Control
Compare children in foster care since the 1944 education act to children in foster care before then.
If possible, include measures before and after enrolment for children in each group.
Historical Control
Provides a counterfactual scenarioGroups may differ on both
measured and unmeasured variables, observed differences may be attributable to factors other than the intervention
Effectiveness
Do intensivepolice
crackdowns reduce gun
violence?
Time-Series
Identify areas with high levels of gun crime and identify peak times
Repeatedly use crackdowns during periods of high crime
Compare times with the intervention to periods without the intervention
Time-Series
Provides a counterfactual scenarioTimes may be different Often requires complicated
statistical analyses to control for differences in baseline variables, time trends, etc.
Conclusion
What is your question?What types of study design might
contribute to an answer?Think ‘Horses for Courses’