research methodology series |
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Indian Pediatr 2021;58:
584-588 |
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Formulation of Research Question and Composing Study
Outcomes and Objectives
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Shashi Kant Dhir, 1 Piyush
Gupta2
From Departments of Pediatrics, 1Guru Gobind Singh Medical College,
Faridkot, Punjab; 2University College of Medical Sciences, New Delhi.
Correspondence to: Piyush Gupta, Professor and Head, Department of
Pediatrics, University College of Medical Sciences, New Delhi, India.
Email:
[email protected]
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Framing an appropriate research question is the most critical and
fundamental part of a study. This helps in developing a hypothesis,
formulating aims and objectives and methodological execution of the
study. Research questions are usually generated by literature backed
thorough analysis of the gaps in previous studies and funnelling it to a
specific focussed issue. The research question should be framed using
the PICO (Population, Intervention/Exposure, Comparator and Outcome)
format and should fulfil the FINER (feasible, interesting, novel,
ethically sound, and relevant) criteria for practical aspects.
Objectives should always be framed in alignment of the research question
using SMART (specific, measurable, achievable, realistic and time
defined) approach. Outcomes are classified as primary and secondary. It
is advisable to have only one primary objective while secondary
objectives can be multiple (usually not exceeding five). This paper
describes a cascade approach starting from framing the research question
and then deciding on the outcomes and study objectives.
Keywords: Aim, Finer, Hypothesis, PICO, Study
design.
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R esearch question (RQ) is the question or the
query which the researcher is trying to answer by conducting an
investigation. The formulation of appropriate research question is the
most fundamental and critical part of a study. All the further steps of
the research i.e., developing a hypothesis, formulating objectives and
methodological execution of the study depend upon the framing of the RQ.
Composing the objectives and outcomes is the natural progression after
framing the RQ in planning a study. Researchers often find it difficult
to frame appropriate RQ and objectives from an inviting idea. This paper
details the step-by-step systematic conversion of an idea to a valid RQ
and translating it further to frame objectives and outcomes.
Getting Ideas for Research
Ideas are everywhere but they have to be converted to
a valid RQ. The choice of RQ may be made from the evaluation of previous
studies, one’s own experiences, from topic of interest or by the need of
the time [1]. Most of the ideas for research come from one’s perceived
gaps in the existing knowledge of a topic. These gaps could be the lack
of clear conclusions or insufficient results from previous studies.
Identification of such gaps could be taken up as a RQ which would then
be used to build on the previous research. Ideas could also be developed
from observations made on the previous work. Systematic analysis of this
observation after brainstorming may also be one of the methods of
finding the correct RQ. Unprecedented circumstances may also yield
multiple RQs on a single topic.
Developing Research Question From an Idea
As a rule of thumb, the broad ideas themselves do not
form a RQ. They should be chiselled to yield one. This is achieved by an
exhaustive and critical analysis of the broad idea by a thorough
literature research. The brainstorming would include searching for
studies conducted in past on same topic, identifying the lacunae in the
existing knowledge or need to replicate the question in different
settings, thinking of a hypothesis, and then generating a RQ [2].
Although multiple RQs may stem from a broad idea, it is best to choose
only one primary RQ for a particular project (choosing the best RQ is
described later). The RQ is the interrogative form of the solution which
we are looking. Simply put, it is the purpose of study written in a
question format [3]. It has to be specific, focused and clearly defined
in terms of population targeted, planned intervention, outcome etc.
For example, while working in pediatric neurology
ward, a student observed that there were a large number of children
coming with breakthrough seizures. He brainstormed with peers to analyze
the reasons for poor control of seizures in children with epilepsy. One
of the RQ which was formed was ‘What are the causes of non-compliance of
treatment among children with epilepsy at a tertiary-care center?’
ESSENTIAL ELEMENTS OF A RESEARCH QUESTION
The RQs are broadly classified into descriptive and
inferential questions based upon the purpose, objective, and clinical
context of the study [4]. Various types of RQs along with examples are
given in Table I.
A RQ has multiple components. The most common
approach to address these is referred to as PICO approach, the acronym
standing for Population, Inter-vention/Exposure, Comparator and Outcome
[5]. Some-times Timeframe, and Effect Size are also added to call it as
PICOTES approach which makes it more compre-hensive. Although most
commonly used in the inferential RQ, some of the elements (P and O) are
also mandatory in the descriptive RQ. The salient features of PICO
elements are explained in Box I.
Box I PICO Elements in a Research Question
Population of Interest, Patient or
Problem to be discussed in the descriptive RQ
Description: The study population
characteristics need to be clearly defined so that there is no
ambiguity.
Example:
• Preterm neonates < 32 wk gestation
• Neonates requiring umbilical arterial
catheter (UAC)
• VLBW Neonates having suspected early onset
sepsis
Intervention or Exposure
The primary variable whose effect is
investigated in the defined population. Could be an intervention
(drug, treatment, procedure) or an exposure.
Example:
• Delayed cord clamping for 60s
• Positioning a high UAC
• Estimating use of CRP in first 24h
Comparator or Control
An alternative condition to compare the
primary variable. Could be the gold standard or placebo.
Example:
• Immediate cord clamping
• Positioning a low UAC
• Estimating procalcitonin in first 24h
Outcome
The expression that will be assessed at the
end point of the study.
Example:
• Incidence of intraventricular hemorrhage
• Complications related to UAC
• Detection of culture proven neonatal sepsis
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Attributes of a Good Research Question
Some of the framed RQs, although inviting and
lucrative are not possible to do. A good RQ once framed, should be
assessed using the FINER approach given by Hulley, et al. [6]. It should
be feasible, interesting, novel, ethically sound, and relevant. It
should be clearly stated and appropriately complex. This audit should be
done before the start of the study to avoid wastage of manpower and
resources. Doing a pilot study may also help in unearthing real time
issues. These attributes are described in the Table II. The RQ
should be chosen so that it addresses issues common to clinical setup of
a particular area, builds upon previous gaps, ethically sound and is
doable by the researcher in the given time limit.
Framing a Research Question
The RQ should be written in one’s own wording, be
appropriately complex and should not be very broad. It should be in
question format and complete in itself [7]. One example of research
question would be "How efficacious is intravenous phenobarbitone in
comparison to intravenous levetiracetam, as a first line drug, in
controlling neonatal seizures?" RQ should NOT be framed to provide
answer in YES or NO. Binary outcome framing is not a correct approach as
decisions in medicine are based on probabilities and cannot be absolute
(0 or 100). Some examples of errors in framing RQ are shown below.
Example 1
Incorrectly framed: In preterm neonates less than
30 weeks, does formula feeding predispose to necrotizing enterocolitis
(NEC)?
Reason: RQ should not have a binary outcome.
Correct: What is the relationship of formula
feeding with NEC in preterm neonate less than 30 weeks?
Example 2
Incorrectly framed: What measures are being taken
to prevent postoperative wound infection undergoing emergency surgeries?
Reason: Too simple and broad, should be
appropriately complex.
Correct: What are the risk factors associated
with deep surgical site infection following laparotomy for acute
perforation peritonitis?
FORMULATING THE OUTCOMES
The writing of RQ should be followed by the framing
of study outcomes and objectives. Outcomes are the measurable endpoints
of the objectives which are monitored during the study and occurrence
(or absence) of these indicate that result has been achieved. They
should originate and be in alignment with the study objectives. It is of
utmost importance to a priori define an outcome as well as a
standard validated method to measure it as a particular entity may be
definable or measurable by variable methods. The outcomes should be
clearly written such that anyone can easily understand the nature of
what is being measured and replicate the measurements at their research
settings. The ideal outcomes should be reproducible under same
conditions and have minimum inter observer difference, and valid i.e.
should measure what they intend to measure. The primary outcome is the
most important measurable endpoint and should correspond to the primary
objective. The study design and sample size are also based upon the
primary outcome of the study. The additional measurable endpoints
pertaining to the secondary objectives are called as secondary outcomes.
Usually outcomes are analyzed independently but ‘composite outcomes’ can
be used when the individual occurrences are rare, correlate with each
other or a combination is more informative as compared to an isolated
outcome [12]. Mortality or bronchopulmonary dysplasia is a composite
outcome used in RQs pertaining to respiratory support in neonates; death
or presence of a severe neuro-development impair-ment (blindness,
deafness, motor or cognitive disability) by two years of age is another
example of composite outcome used in assessing long term impact of
various interventions in extremely low birth weight neonates.
STUDY OBJECTIVES
The RQ tells us that what we are planning to do. The
objectives of the study give us accurate description of the steps about
how we are going to achieve what we had thought of in the RQ. The
objectives serve as milestones for the ultimate goal [8,9]. The
objectives are classified as primary and secondary. The primary
objective is the most important endpoint and should reflect the RQ. The
additional endpoints which we want to study are termed as secondary
objectives. It is better to have only one primary objective for a
particular RQ. Secondary objec-tives can be multiple; however, should
not exceed five.
Framing the Objectives
Objectives are written in single infinitive sentence
format starting with the word ‘To’ using SMART format. They should be
specific, measurable, achievable, realistic and time defined [10]. The
action verbs from Bloom’s measurable verb list e.g. determine, compare,
verify, establish etc. should be used in the framing of the objective
[11]. The objectives dictate the type of study design and help in
developing the methodology section of the protocol. We should avoid
writing too many objectives and should have a clear flow between the RQ
and objectives as shown in Table III. The relationship between
the individual objectives should have a synergistic impact [8,9].
CONCLUSION
The formulation of RQ is most critical and
fundamental part of a study which should be done carefully and
scientifically. Framing of objectives is a natural corollary after
finalizing the research questions. Defining the outcome (mostly in the
form of a numerical measurable expression) is a necessary intermediary
between the two processes. A summary of the process of developing a
research question and translating it into outcomes and objectives is
exemplified (Table IV).
Contributors: SKD: literature research, drafted
and revised the manuscript, approved final version of manuscript PG:
conceived the idea, supervised the manuscript, reviewed the manuscript,
finalised and approved the manuscript. Both authors have contributed to,
designed and approved the manuscript.
Funding: None; Competing interest: None
stated.
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