This document discusses health systems research and the research process. It defines research as seeking answers to unanswered questions through planned and systematic collection, analysis, and interpretation of data. The objectives of research can be theoretical, factual, or aimed at application. Characteristics of research include using valid methods to gather new knowledge or data and arriving at careful conclusions. Health systems research seeks to generate knowledge to promote population health. The research process involves selecting a problem, formulating hypotheses, developing a study plan, collecting and analyzing data, and formulating results.

1. M U H A M M A D S A L M A N
L E C T U R E R
P U N J A B U N I V E R S I T Y C O L L E G E O F
P H A R M A C Y
Health System Research (HSR)

2. Research?
 Research simply seeks the answer of certain
questions which have not been answered so far and
the answers depend upon human efforts

3.  Example of the moon
 Some years ago man did not know what exactly the
moon is?
 Was this problem which had no solution?
 Man could only make some assumptions about it but
the man know this time by his efforts, he went to the
moon brought the soil of the moon and studied it
 The man is now able to give concrete answer of the
problem what is the moon?

4.  Now the question arises
 “Is the answer of the question in examination also
research”?
 The answer is ‘no’, because the answers of these
questions are available (text-books, class-notes etc)
 Research answers only those questions of which the
answers are not available in literature i.e., in human
knowledge

5.  Therefore, we can say research seeks the answer only of those
questions of which the answers can be given on the basis of
available facilities
 Research is simply the process of arriving as dependable
solution to a problem through the planned and systematic
collection, analysis and interpretation of data
 It is the most important process for advancing knowledge for
promoting progress
 Also enables man to relate more effectively to his environment
to accomplish his purpose and to resolve his conflicts

6. Term “Research”
 Therefore, research means to observe the
phenomena again and again from different
dimensions

7.  “Research is a systematized effort to gain new
knowledge.” (V. Redman and A.V.H. Mory)
 “Research is an honest exhaustive, intelligent
searching for facts and their meanings or
implications with reference to a given problem. The
product or findings of a given piece of research
should be an authentic, verifiable and contribution
to knowledge in the field studied.” (P.M. Cook)

8.  He emphasized the following characteristics of
research in his definition:
 It is an honest and exhaustive process.
 The facts are studied with understanding.
 The facts are discovered in the light of problem. Research is
problem-centred.
 The findings are valid and verifiable.
 Research work should contribute new knowledge in that field

9. Objectives of research
 Theoretical objective
 Formulate the new theories, principles or laws.
 Such type of research is explanatory because it explains the
relationship of certain variables.
 These researches contribute some basic knowledge to the
human knowledge.
 The researches in different disciplines such ads physics,
chemistry, mathematics etc. have theoretical objective.

10.  Factual objective
 Researches whose objective is factual find out new facts
 This objective is by nature descriptive
 Application objective
 Research having application objective does not contribute a
new knowledge in the fund of human knowledge but suggests
new applications. By application we mean improvement and
modification in practice.
 For example if anyone gives a new application of electricity
then such type of research has application objective

11. General characteristics of research
 Gathers new knowledge or data from primary or first –hand sources
 Places emphasis upon the discovery of general principles
 An exact systematic and accurate investigation
 Uses certain valid data gathering devices
 Logical and objective
 Researcher resists the temptation to seek only the data that support
his/her hypotheses
 Researcher eliminates personal feelings and preferences
 Endeavors to organize data in quantitative terms
 Patient and unhurried activity
 Researcher is willing to follow his/her procedures to the conclusions
that may be unpopular and bring social disapproval
 Research is carefully recorded and reported conclusions and
generalizations are arrived at carefully and cautiously

12.  Health research → A systematic collection, analysis and
interpretation of health and health-related data to answer a
certain question or to solve a problem
 Health systems research also known as health services
research or health policy and systems research
 Health systems research → "The people, institutions, and
activities whose primary purpose is to generate high quality
knowledge that can be used to promote, restore, and or
maintain the health status of populations. It can include the
mechanisms adopted to encourage the utilization of research.“
(HRSA, WHO)

13. Classification of Research
 Basic level
 It is designed to add an organized body of scientific knowledge
and does not necessarily produce results of immediate
practical value
 Applied level
 Applied research is undertaken to solve an immediate practical
problem and the goal of adding to scientific knowledge is
secondary

14. Kinds of research
 There are various bases to classify the research
 On the basis of objective of the research
 Fundamental research
 Action research
 On the basis of approach of the research
 Longitudinal research
 Cross sectional research
 On the basis of precision in research findings
 Experimental research
 Non experimental research

15.  On the basis of nature of findings
 Explanatory research
 Descriptive research
 According to national science foundation
 Basic research: Those researches which embrace origin or
unique investigation for the advancement of knowledge
 Applied research: which may be characterized as utilization in
practice
 Developmental research: use of scientific knowledge for the
production of useful materials, devices, systems, methods for
processes excluding design and production engineering.

16. Health research triangle
 three operational interlinked categories of biomedical, health services and behavioral
research, the so-called health research triangle
Bio-medical Behavioral
Health services

17. Research process
 Selection of Research problem
 Formulation of hypothesis
 Research plan
 Data collection
 Analysis of data
 Formulation of result and conclusions

18. Selection and formulation of the problem
?

19. Hypothesis
 Carefully constructed statements about a
phenomenon in the population
 Generated by deductive reasoning, or based on
inductive reasoning from prior observations
 One of the most useful tools of health research is the
generation of hypotheses which, when tested, will
lead to the identification of the most likely causes of
disease or changes in the condition being observed

20.  Mill’s canons of inductive reasoning are frequently
utilized in the forming of hypotheses which relate
association and causation.
 Method of difference - when the frequency of a
disease is markedly dissimilar under two
circumstances, and a factor can be identified in one
circumstance and not the other, this factor, or its
absence, may be the cause of the disease
 for example → the difference in frequency of lung
cancer in smokers and nonsmokers

21.  Method of agreement - if a factor, or its absence is
common to a number of different circumstances that
are found to be associated with the presence of a
disease, that factor, or its absence may be causally
associated with the disease
 The occurrence of hepatitis A is associated with
patient contact, poor sanitation and hygiene, each
conducive to the transmission of the hepatitis virus

22.  Method of concomitant variation, or the dose response
effect
 The increasing expression of endemic goiter with
decreasing levels of iodine in the diet
 The increasing frequency of leukemia with increasing
radiation exposure
 The increase in prevalence of elephantiasis in areas of
increasing filarial endemicity

23.  Method of analogy – the distribution and frequency
of a disease or effect may be similar enough to that of
some other disease to suggest commonality in cause
 e.g. hepatitis B virus infection and cancer of the liver

24. Research plan/design
 Defining the population, characteristics of interest
and probability distributions
 Type of study
 observational or experimental

25.  method of data collection, management and analysis:
 sample selection
 measuring instruments (reliability and validity of instruments)
 training of interviewers
 quality control of measurements
 computerization, checking and validating measurements
 the issue of missing observations
 statistical summarization of information
 testing of hypothesis
 ethical considerations
 Conclusions

26.  Deductive Reasoning
 Thinking precedes from general assumption to specific
application
GENERAL → SPECIFIC
 Inductive reasoning
 Conclusions about events are based on information generated
through many individual and direct observations
SPECIFIC → GENERAL

27. Inductive reasoning Deductive reasoning
SPECIFIC → GENERAL GENERAL → SPECIFIC
Every rabbit has been observed has
lungs. Therefore, every rabbit has lungs
Every mammal has lungs. All rabbits
are mammals. Therefore, every rabbit
has lungs.

28. Epidemiological studies

30. Classification
 Non-international
 AKA observational studies
 Researchers do not manipulate situations/objects
 Only describes or analyses situations/objects
 Interventional
 Participants are assigned to receive one or more interventions
(or no intervention) so that researchers can evaluate the effects
of the interventions on biomedical or health-related outcomes.

31. Non-interventional studies
 Descriptive studies
 Case series
 Community diagnosis or need assessment
 Epidemiological description of disease occurrence
 Descriptive cross-sectional studies
 Ecological descriptive studies
 Analytical studies
 Case-control studies
 Cohort studies
 Prospective cohort studies
 Retrospective cohort studies
 Analytical cross-sectional studies

32. Descriptive studies
 When an epidemiological study is not structured
formally as an analytical or experimental study i.e.
when it is not aimed specifically to test a hypothesis,
it is called a descriptive study
 The wealth of material obtained in most descriptive
studies allows the generation of hypotheses, which
can then be tested by analytical or experimental
designs

33.  Case series
 based on reports of a series of cases of a specific condition, or a
series of treated cases, with no specifically allocated control
group
 These represent the numerator of disease occurrence, and
should not be used to estimate risks

34.  Community diagnosis or needs assessment
 This kind of study entails collection of data on
 existing health problems
 Programs
 Achievements
 Constraints
 social stratification
 leadership patterns
 focal points of resistance or high prevalence, or groups at highest
risk.
 Its purpose is to identify existing needs and to provide baseline
data for the design of further studies or action

35.  Epidemiological description of the disease occurrence
 This common use of the descriptive approach involves the collection of
data on the occurrence and distribution of disease in populations
according to
 specific characteristics of individuals
 Age
 Sex
 Education
 Smoking habits
 Religion
 Occupation
 social class
 Marital status
 Health status
 Personality
 Place (rural/urban, local, national, international)
 Time (epidemic, seasonal, cyclic, secular)

36.  Descriptive cross-sectional studies
 AKA population/community surveys
 Entail the collection of data on, as the term implies, a cross-section
of the population, which may comprise the whole population or a
proportion (sample) of it
 Many cross-sectional studies do not aim at testing a hypothesis about
an association
 Provide a prevalence rate at a particular point in time → point
prevalence
 Or over a period of time → period prevalence

37.  Ecological descriptive studies
 When the unit of observation is an aggregate (e.g. family, clan
or school)
 Or an ecological unit (a village, town or country) the study
becomes an ecological descriptive study

38. Analytical strategies of epidemiological studies
 Observational studies, where establishing a relationship
between a ‘risk factor’ (etiological agent) and an outcome
(disease) is the primary goal, are termed analytical
 hypothesis testing is the primary tool of inference

39.  Case-control studies
 Simplest and most commonly used analytical strategy in
epidemiology
 Designed primarily to establish the causes of diseases by
investigating associations between exposure to a risk factor
and the occurrence of disease
 Design is relatively simple, except that it is backward-looking
(retrospective) based on the exposure histories of cases and
controls

40.  one investigates an association by contrasting the exposure of
a series of cases of the specified disease with the exposure
pattern of carefully selected control groups free from that
particular disease

41. Advantages Disadvantages
Relatively quick and inexpensive great risk of bias in the selection of
cases and
Controls
feasible for rare disease Prone to recall bias (it may be very
difficult or impossible to obtain
information on exposure if the recall
period is long)
Requires a smaller sample size than
cohort study
Cannot measure incidence, only relative
risk is measured
Little problem with attrition, as when
follow-up requires periodic
investigations and some subjects refuse
to continue to cooperate

42. Cohort studies
 Prospective cohort study
 The common strategy of cohort studies is to start with a reference
population (or a representative sample thereof)
 Some of whom have certain characteristics or attributes relevant to
the study (exposed group), with others who do not have those
characteristics (unexposed group)
 Both groups should, at the outset of the study, be free from the
condition or conditions under consideration
 Both groups are then observed over a specified period to find out the
risk each group has of developing the condition(s) of interest

43. Design of a cohort (prospective) study

44. Example of Prospective cohort study design

45.  Design features
 Selection of cohort
 a community cohort of specific age and sex
 an exposure cohort, e.g. radiologists, smokers, users of oral
contraceptives
 a birth cohort, e.g. school entrants
 an occupational cohort, e.g. miners, military personnel
 a diagnosed or treated cohort, e.g. cases treated with radiotherapy,
surgery, hormonal treatment
The usual procedure is to locate or identify the cohort, which
may be a total population in an area or sample thereof

46.  Data to be collected
 data on the exposure of interest to the study hypotheses
 data on the outcome of interest to the study hypotheses
 characteristics of the cohort that might confound the association
under study
 Methods of data collection
 Several methods are used to obtain the above data, which should
be on a longitudinal basis.
 These methods include
 Interview surveys with follow-up procedures
 Medical records monitored over time
 Medical examinations and laboratory testing

47.  Measures of frequency
 Cumulative incidence
 Incidence density (Person-year approach)

48.  In September 2009, 3000 Birmingham university freshman enrolled in a study
of health behaviors that aimed to track them over 4 years of college. Data from
September 2012 are shown below
 What is the cumulative incidence of obesity over 4 years?
No. of
new
cases
No of
students
“at risk”
at the
begining
Admitted to the emergency room for an alcohol-related
injury
400 3500
Unintended pregnancy? 252 2000
Diagnosed with depression? 310 3000
Obese? 543 2100

49.  Person-time approach/incidence density
 This approach is an improvement over the conventional
measure of incidence, because it takes into consideration both
the number observed and the duration of observation for each
individual
 If 30 individuals were observed as follows: 10 for two years, 5
for three years, and 15 for four years, they would contribute
(10x2)+(5x3)+(15x4) = 95 person-years of observation, which
would become the denominator. The numerator is the number
of new cases observed in these groups over the specified period
of time………

50. Advantages Disadvantages
Allow calculation of incidence rates,
relative risk , attributable risk etc.
Expensive
If a probability sample is taken from the
reference population, it is possible to
generalize from the sample to the
reference population with a known
degree of Precision
long-term and sometimes are not
always feasible
capable of identifying other diseases
that may be related to the same risk
factor
Sample sizes required for cohort studies
are extremely large
The most serious problem is that of
attrition

51.  Historical/retrospective cohort study

52.  Analytical cross-sectional study
 Investigator measures exposure and disease simultaneously in
a representative sample of the population
 By taking a representative sample, it is possible to generalize
the results obtained in the sample for the population as a
whole
 Cross-sectional studies measure the association between the
exposure variable and existing disease (prevalence)
 Unlike cohort studies, which measure the rate of developing
disease (incidence)

53. Design of a cross-sectional study

54. Experimental study designs
 An experiment is the best epidemiological study
design to prove causation
 Viewed as a final or definitive step in the research
process
 A mechanism for confirming or rejecting the validity
of ideas, assumptions, postulates and hypotheses
about the behavior of objects, or effects upon them
which result from interventions under defined sets of
conditions

55.  Investigator has control of the subjects, the
intervention, outcome measurements, and sets the
conditions under which the experiment is conducted
 In particular, the experimenter determines who will
be exposed to the intervention and who will not
 This selection is done in such a way that the
comparison of outcome measure between the
exposed and unexposed groups is as free of bias as
possible

56. General experimental study design

57.  The most commonly encountered experiment in
health science research, and the research strategy by
which evidence of effectiveness is measured, is the
randomized, controlled, double-blind clinical trial,
commonly known as an RCT

59.  Clinical trials may be done for various purposes
 Some of the common types of clinical trial (according
to purpose) are:
 prophylactic trials, e.g. immunization, contraception
 therapeutic trials, e.g. drug treatment, surgical procedure
 safety trials, e.g. side-effects of oral contraceptives and
injectable
 Risk-factor trials, e.g. withdrawing the agent (e.g. smoking)
through cessation

60.  Therapeutic trials may be conducted to test efficacy
does a therapeutic agent work in an ideal, controlled
situation?
 or to test effectiveness (e.g. after having established
efficacy, if the therapy is introduced to the
population at large, will it be effective when having
to deal with other co-interventions, confounding,
contamination, etc.?

61.  The intervention in a clinical trial may include
 drugs for prevention, treatment or palliation
 clinical devices, such as intrauterine devices
 surgical procedures, rehabilitation procedures
 medical counselling
 diet, exercise, change of other lifestyle habits
 risk factors
 communication approaches, e.g. face-to-face communication versus
pamphlets
 different categories of health personnel, e.g. doctors versus nurses
 treatment regimens, e.g. once-a-day dispensation versus three times
a day

62.  Usually, clinical trials of new therapies or devices
pass through four phases
 Phase I
 Phase II
 Phase III
 Phase IV

63. Phase I clinical trial
 This first phase in humans is preceded by
considerable research
 Including pharmacological and toxicological studies
in experimental animals
 To establish that the new agent is effective and may
be suitable for human use,
 And to estimate roughly the dose to be used in man

64.  Phase I trials include studies of volunteers who
receive, initially, a fraction of what the anticipated
dose is likely to be
 And are monitored for effects on body functions,
such as
 Hepatic
 Cardiovascular
 Renal
 Gastroinstestinal
 Endocrinal functions

65.  The metabolism of the drug may also be investigated
at this stage
 These studies are normally done on volunteers, who
are usually institutionalized, and occupy what are
called ‘research beds’
 Require close supervision
 short duration

66. Phase II clinical trial
 Also carried out on volunteers selected according to
strict criteria
 Purpose of Phase II is
 to assess the effectiveness of the drug or device
 to determine the appropriate dosage
 to investigate its safety
 Further information on the pharmacology, especially
the dose-response relationship of the drug, is
collected

67. Phase III clinical trials
 This is the classical phase
 The one usually referred to as a ‘clinical trial’ and
reported in health research journals
 Performed on patients, who should consent to being
in a clinical trial
 Strict criteria for inclusion in and exclusion
 purpose of this phase is
 to assess the effectiveness
 to assess safety in continue use of the drug or device in a larger
and more heterogeneous population than in Phase II

68.  It includes more detailed studies and monitoring
than those given in a normal service situation
 Usually carried out on hospital inpatients, but may
be performed on outpatients with intense monitoring
follow-up
 It requires superior clinical and epidemiological
skills, in addition to the required laboratory
technology

69.  Results from these trials are used by regulatory
agencies to evaluate whether a new product or device
should be licensed for general public use

70. Phase IV
 To identify and evaluate the long-term effects of new
drugs and treatments over a lengthy period for a
greater number of patients.
 Phase IV research takes place after the FDA approves
the marketing of a new drug
 Through Phase IV clinical studies, new drugs can be
tested continuously to uncover more information
about efficacy, safety and side effects after being
approved for marketing

71. Factors that affect the design of and analysis of
trial (phase III)
 The agent, treatment or experimental factor
 Conditions to be treated
 The target population
 Ethical issues
 Outcomes to be measured
 Side-effects
 Study instruments
 Blinding
 Stopping rule
 Plans for the analysis
 Selective attrition
 Methods for ensuring the integrity of the data
 The choice of design
 Time required

72. Community intervention trials
 CITs are usually carried out in hospitals or clinics,
and are usually directed at a patient group with
specific health conditions
 a test of whether the introduction of iron-fortified
salt in the community would reduce the incidence of
anemia in the community
 The major difference from the RCT is that the
randomization is done on communities rather than
individuals