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Observational Studies: Why Are They So Important

Mariani, A.W. and Pêgo-Fernandes, P.M. Observational studies: why are they so important. Sao Paulo Med J. 132(1): 1-2. 2014.

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56 views2 pages

Observational Studies: Why Are They So Important

Mariani, A.W. and Pêgo-Fernandes, P.M. Observational studies: why are they so important. Sao Paulo Med J. 132(1): 1-2. 2014.

Uploaded by

Andre Lanzer
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© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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DOI: 10.1590/1516-3180.2014.

1321784 editorial

Observational studies: why are they so important?


Estudos observacionais: por que são tão importantes?
Alessandro Wasum MarianiI, Paulo Manuel Pêgo-FernandesII
Instituto do Coração (InCor), Hospital das Clínicas (HC), Faculdade de Medicina da Universidade de São Paulo (FMUSP), São Paulo, Brazil

MD. Thoracic Surgeon, Instituto do Coração


I
Randomized clinical trials (RCTs) are known to be the gold standard for medical research,
(InCor), Hospital das Clínicas (HC), Faculdade
de Medicina da Universidade de São Paulo
and this study design is preferred for investigating the efficacy of new interventions. Many
(FMUSP), São Paulo, Brazil. authors and editors believe that the RCT design always surpasses other research designs. How-
II
MD, PhD. Associate Professor, Discipline ever, this is not an unquestionable truth.
of Thoracic Surgery, Instituto do Coração
The main advantage of RCTs is that they provide better control over possible bias through
(InCor), Hospital das Clínicas (HC), Faculdade
de Medicina da Universidade de São Paulo randomization and blinding. In other words, the great strength of an RCT is its high inter-
(FMUSP), São Paulo, Brazil. nal validity. On the other hand, rigid design control could reduce the ability to generalize the
results.1 Another issue relating to RCTs is the fact that recruitment, randomization and blind-
ing are not always possible because of technical issues (e.g. surgical procedures) or ethical issues
(e.g. in a hypothetical trial to prove the association between smoking and lung cancer, it would
be unethical to randomly assign a group to smoke).
But what should be done when RCTs are unfeasible or unethical? The answer could be a
well-designed observational study. This term describes a range of study designs that includes
cross-sectional, case-control, prospective and retrospective cohort studies. The main character-
istic of observational studies is that any intervention is not determined by the protocol, but by
clinical practice.2
Cross-sectional studies are used to estimate prevalence. They are relatively quick and cheap,
and can be used to study multiple outcomes. However, they do not differentiate between cause
and effect or within the sequence of events. They are useful for identifying associations that can
then be more rigorously studied using a cohort study or randomized controlled study. The most
important problem with this type of study is in differentiating between cause and effect from a
simple association.3
Case-control studies are considered to be simple to organize and useful for hypothesis gen-
eration. They retrospectively compare two groups in order to identify predictors of an outcome.
They allow calculation of odds ratios. The disadvantages are that they can only evaluate one out-
come and that the presence of bias is usually high and difficult to assess. The major difficulty
could lie in determining an adequate control group.3
Cohort studies are excellent for estimating the incidence and natural history of a condition.
They may be prospective or retrospective and sometimes two cohorts are compared. They ana-
lyze predictors (risk factors) that enable relative risk calculation. Since they measure events in
temporal sequence, they can distinguish causes from effects. Retrospective cohorts are consid-
ered to be cheaper and quicker, but may have fragile results, particularly if the database is inad-
equate. Prospective cohorts are more accurate and, with a good protocol (with adequate sample
size and follow-up) can have results that are as reliable as those of RCTs.3
The main problem in observational studies is the presence of confounders and selection
bias (which are prevented in RCTs through randomization and blinding). A confounder can be
defined as any factor that is related not only to the intervention (e.g. treatment) but also to the
outcome and could affect both.4 One good example is age: in a study on the relationship between
smoking (exposure) and lung cancer (outcome), age could be implicated as a factor that would
increase the incidence of the outcome. Thus, if one of the groups (smokers or non-smokers) has
an older population, the increase in lung cancer could be influenced by age (as a confounder),
and not by the exposure studied.

Sao Paulo Med J. 2014; 132(1):1-2 1


EDITORIAL | Mariani AW, Pêgo-Fernandes PM

However, advanced statistical tools may enable good and reliable control over many confounders. Some tools like propensity
scores and sensitivity analysis, when correctly performed, could drastically reduce the bias caused by the lack of randomization.5
Some authors have studied the results from RCTs, compared with similar observational studies. Concato et al. published an evalu-
ation of meta-analyses that compared outcomes between RCTs and observational studies and reached the following conclusion: “The
results from well-designed observational studies (with either a cohort or a case-control design) do not systematically overestimate the
magnitude of the effects of treatment, as compared with those in randomized, controlled trials on the same topic”.6 In other words, if
the observational study has good methodological quality, the results are quite similar.
Many investigators have pointed out that the main strength of observational studies is their greater proximity to “real life situa-
tions”, since RCTs have stricter inclusion criteria and rigid protocols that may not reflect clinical practice. By definition, observational
studies have greater heterogeneity of medical interventions and patient populations that are closer to clinical practice.2 Other advan-
tages of observational studies are that they are usually cheaper than RCTs and can be used to investigate rare outcomes and to detect
unusual side effects, and that some designs are easily and quickly performed.
Observational studies also are important for creating new hypotheses, proving the external validity of RCTs already performed,
establishing the sample size for an RCT and evaluating which patient subsets really benefit from each alternative intervention of effec-
tive alternative therapies.1 In this way, it can be said that observational studies can be complementary to RCTs.
Although the evidence level of observational studies appears to be lower than that of RCTs, it is clear that this kind of investiga-
tion is crucial for elucidating many scientific questions. Not only authors but also editors around the world are giving more attention
to these studies. The take-home message is that the study question and the quality of the methodology applied to answer it are much
more important than the study design. In this context, observational studies may be the best way to answer the many medical ques-
tions in situations in which the classical RCT approach does not apply.

REFERENCES Sources of funding: none


1. Hannan EL. Randomized clinical trials and observational studies: Conflict of interests: none
guidelines for assessing respective strengths and limitations. JACC
Cardiovasc Interv. 2008;1(3):211-7. Date of first submission: October 23, 2013
2. Yang W, Zilov A, Soewondo P, et al. Observational studies: going Last received: October 23, 2013
beyond the boundaries of randomized controlled trials. Diabetes Res Accepted: November 13, 2013
Clin Pract. 2010;88 Suppl 1:S3-9.
3. Mann CJ. Observational research methods. Research design II: cohort, Address for correspondence:
cross sectional, and case-control studies. Emerg Med J. 2003;20(1):54-60. Alessandro Wasum Mariani
4. McNamee R. Confounding and confounders. Occup Environ Med. Rua Treze de Maio, 1.217 — apto 31
2003;60(3):227-34; quiz 164, 234. Bela Vista — São Paulo (SP) — Brasil
5. Joffe MM, Rosenbaum PR. Invited commentary: propensity scores. CEP 01327-001
Am J Epidemiol. 1999;150(4):327-33. E-mail: [email protected]
6. Concato J, Shah N, Horwitz RI. Randomized, controlled trials,
observational studies, and the hierarchy of research designs. N Engl J
Med. 2000;342(25):1887-92.

2 Sao Paulo Med J. 2014; 132(1):1-2

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