1005P-STATEMENT OF THE RESEARCH PROBLEM & FORMULATION OF RESEARCH OBJECTIVES

By

Professor Omar Hasan Kasule Sr.

Faculty of Medicine, King Fahad Medical City

Riyadh, Saudi Arabia

18-22 May 2010

TABLE OF CONTENTS

1.0 Pre-Test For Workshop On Research Problems And

Research Questions                                                                 3

2.0 Background Reading I: Discovery By John G Hunter                5                

3.0 Background Reading 2: How To Define A Research

Question By Jan P Vandenbroucke                                        7                          

4.0 Background Reading 3: Research Questions And

Hypotheses By Professor Omar Hasan Kasule Sr.                           9

5.0 Presentation: Over View Of Problem Analysis And

Problem Statement                                                                            12

6.0 Presentation: Analysis And Statement Of The Problem    13

7.0 Presentation: Formulation Of Research Objectives And

Hypotheses                                                                             14

8.0 Discussion Problems At Workshop On Stating

Research Problems And Formulating Research Questions               16

9.0 Your Notes                                                                        18

1005 PRE-TEST FOR WORKSHOP ON RESEARCH PROBLEMS AND RESEARCH QUESTIONS

Prepared for the Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

1. The following statements are true about statistical questions and conclusions

1. Substantive questions are framed as statistical questions for analysis
2. Statistical conclusions may differ from substantive conclusions because of bias
3. Statistical significance is exactly the same as clinical or practical significance
4. Some substantive questions cannot be answered statistically
5. A valid substantive conclusion can be from an invalid statistical conclusion

2. The following statements are true about statistical questions and conclusions

1. Bio-statistics always conclusively answers substantive questions
2. Substantive conclusions require interpretation
3. Statistical inference is mostly deductive
4. A statistical question is framed to be answered quantitatively
5. Data is not necessary in formulating a statistical conclusion

3. The following statements are true about hypotheses and the scientific methods

1. Hypothesis formulation is done only after carrying out the experiment
2. Informal use of hypotheses in science is not acceptable
3. Scientists should be open minded when generating hypotheses
4. A hypothesis is a belief that can be supported but not contradicted by experiments
5. Rejection of a hypothesis affects the credibility of the scientist who suggested it

4. The following statements are true about hypotheses and the scientific method

A.     Use of a priori beliefs in the interpretation of experimental studies is unscientific

B.      Use of intuition has no role in the interpretation of experimental studies

C.      Anecdotal observations are reliable scientific evidence

D.     Some phenomena in the universe can not be tested scientifically

E.      Empirical investigation is not the only source of knowledge

F.      No valid scientific experiment can be carried out without a hypothesis

5. The following statements are true about the null and alternative hypotheses

1. H₀ states that there is no difference between the two comparison groups
2. H₀ assumes that apparent differences between groups are due to sampling error.
3. H_A disagrees with H₀
4. H₀ and H_A are complimentary
5. H₀ and H_A are exhaustive and between them cover all the possibilities.
6. A hypothesis can be proved beyond all reasonable doubt

6. The following statements are true about the null and alternative hypotheses

1. H₀ assumes that there are biological difference between compared groups
2. H_A assumes that apparent biological differences are due to sampling error.
3. H₀ can be accepted if the evidence is overwhelmingly in its favor
4. Scientific hypotheses can never be proved in a conclusive and final way
5. Hypothesis testing is not considered part of the scientific method

7. The following statements are true about the null and alternative hypotheses

A.    In obvious cases, there is no need to state H₀ and H_A before the experiment

B.     H₀ and H_A should always be stated together

C.     H₀ may be accepted if the sample size is large enough

D.    If H₀ is rejected, H_A is automatically proved

E.     Only 1 valid experiment is necessary to prove/disprove a hypothesis

8. The following statements are true about a statistically significant test

A.     H₀ is false

_B.        H₀ is not rejected

C.     Observations are not compatible with H₀

D.     Observed differences are due to sampling variation

E.      Observations may be or may not be real/true biological phenomena.

9. The following statements are true about a statistically non significant test

A.    H₀ is not false

B.     H₀ is true

C.     H₀ is not rejected

D.    Observations are compatible with H₀

E.     Observations are due to sampling variation or random errors of measurement

F.      Observations are artificial, apparent and not real biological phenomena.

10. The following statements are true about the interpretation of hypothesis tests

A.    A statistically significant may have no clinical significance

B.     A statistically significant may have no clinical importance

C.     A statistically significant may have no practical significance

D.    A statistically significant may have no practical importance

E.     Confounding and wrong measurements can lead to misleading statistical significance

F.      Clinically important differences may not be statistically significant for small samples

11. The following statements are true about the interpretation of hypothesis tests

A.     Statistical significance implies practical or clinical significance

B.     Statistical significance could be clinically insignificant

C.     Clinically significant findings may not reach statistical significance

D.     Prior experience is needed in interpreting clinical significance of hypothesis testing

E.      The level of significance used depends on the type of study

1005 RESEARCH QUESTIONS AND HYPOTHESES

Prepared for the Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

1.0 PURPOSES OF RESEARCH

The main purposes of epidemiological research can be listed as exploration, description, explanation, and prediction. Exploratory studies are preliminary and have the objective of obtaining basic information about a disease and its potential causes in order to enable formulation of causal hypotheses that can be tested in more sophisticated studies. Descriptive studies characterize a disease in terms of place, time, and person. Explanatory studies seek to establish causal relations between a disease and its risk factors. Prediction uses existing epidemiological profiles of a disease and its exposures to predict future disease patterns.

2.0 STEPS OF AN EPIDEMIOLOGIC INVESTIGATION

An epidemiological investigation proceeds in several stages. It starts by identifying the problem i.e. a decision has to be made that a public health or medical problem exists. This is followed by description of the extent and distribution of the problem. Hypotheses are then formulated about the causes of the problem using the procedures of the scientific method. Appropriate studies are designed to test the hypotheses. Epidemiological information is sourced from existing data or studies (observational or experimental).  An epidemiologic study involves data collection, data analysis, and data interpretation. Biostatistics is the technology of the scientific method that enables sophisticated data analysis and interpretation.

Epidemiological methodology, following the scientific method, is empirical, inductive, and refutative. Epidemiology relies on and respects only empirical findings. Empiricism refers to reliance on physical proof. Induction is building a theory on several individual observations. Refutation is basically refusal of a supposition until it is proved otherwise. Epidemiological investigation is not as deterministic as laboratory investigation but is cheap and easy.

3.0 RESEARCH QUESTIONS & CONCLUSIONS: STATISTICAL vs. SUBSTANTIVE:

An investigator starts with a substantive question. This is formulated as a statistical question. Data is then collected and is analyzed to answer the statistical question. The answer to the statistical question is the statistical conclusion. The investigator uses the statistical conclusion and other knowledge available to him to reach a substantive conclusion. Statistics therefore gives statistical and not substantive answers.

A substantive question is the subject matter stated in ordinary language. Technical terminology may or may not be used. The less technical the formulation is, the better to enable statisticians who are not specialists in the subject matter can understand. Care must be taken to make sure that accuracy and exactness are not sacrificed for the sake of simplification.

A statistical question is when the substantive question is stated using statistical language. Since the language of statistics is mathematical, the statistical question is stated as numbers, parameters, relations of equality, and relations of inequality.

A statistical conclusion is the result of mathematical manipulation of parameters or data. Statistical conclusions are made about groups and not individuals. Any inference to the individual is to a hypothetical individual. In other words the statistical conclusion is depersonalized. 

A substantive conclusion is the translation of the statistical conclusion back to normal language to answer the substantive question that was posed at the start.

4.0 FORMULATION OF HYPOTHESES:

Epidemiology, as a scientific discipline, uses the procedures of the scientific method.  The procedures require stating a hypothesis, collecting and analyzing data to test the hypothesis, and reaching conclusions about the hypothesis.

Continuous improvement of knowledge is by generating and testing hypotheses. The hypothesis is based on previous knowledge or data. It may also be based on purely theoretical or intuitive considerations.

An epidemiological hypothesis is formulated to relate two phenomena: the disease and the putative cause of the disease (the exposure or risk factor). Four methods are generally used in such a formulation: difference, agreement, concomitant variation, and analogy.

A causal hypothesis can be generated by looking for the difference between two situations, one that leads to disease and the other does not. The difference(s) between the two situations may be the putative cause of the disease.

Agreement involves similarities between different situations that lead to the same disease. The common factor on which the similarity or agreement is based could be the putative cause of disease. If variation in a putative causal factor is always associated with concomitant variation in disease occurrence, then that factor is likely to be etiologically important.

The method of analogy is used to generate a causal hypothesis by looking at a causal relation between two phenomena and understanding its mechanism. If that mechanism is relevant to another disease situation, then the cause in the first instance could also be the cause in the second instance.

Hypotheses must be specific and testable. Empirical data is from experimentation and observation. The conclusions from testing a hypothesis can be rejection/non-rejection and never acceptance. A new hypothesis is generated from the conclusion and the process is repeated. Use of the scientific method implies among other things that epidemiological knowledge is never stable. It keeps on changing and getting nearer the truth as new information is discovered.

5.0 TYPES OF HYPOTHESES

A hypothesis is a statement of belief in something. Unlike other types of beliefs, scientific beliefs are subject to experimental verification. Two hypotheses are always stated for proper scientific investigation: the null and the alternative hypotheses.

The null hypothesis or research hypothesis, H₀, states that there is no difference between the two comparison groups and that the apparent difference seen is due to sampling error.

The alternative hypothesis, H_A, disagrees with the null hypothesis and states that there is a real difference not explained by sampling error. H₀ and H_A are complimentary and exhaustive in that between them they cover all the possibilities. H_A could be vague. When H₀ is rejected, we cannot accept H_A we only fail to reject it.

The aim of hypothesis testing is to make a conclusion about H0. The conclusion is in the form of rejecting or not rejecting the hypothesis. If H0 is rejected, HA becomes the new working hypothesis. A hypothesis cannot be proved; you only give an objective measure of probability of its truth

6.0 IMPLICATIONS OF STATISTICAL SIGNIFICANCE

Implications of statistically significant

·                H₀ is false

·                H₀ is rejected

·                Observations are not compatible with H0

·                Observations are not due to sampling variation

·                Observations are real/true biological phenomenon

Implications of not statistically significant

·                H₀ is not false (we do not say true)

·                H₀ is not rejected

·                Observations are compatible with H0

·                Observations are due to sampling variation or random errors of measurement.        

·                Observations are artificial, apparent and not real biological phenomena

Statistical and practical significance

Statistically significant may have no clinical/practical significance/importance. This may be due to (a) other factors being involved and not studied here (b) measurements that are not valid. Clinically important difference may not reach statistical significance due to 2 main reasons: (a) small sample size (b) measurement that are not discriminating enough

1005 PRESENTATION: OVER VIEW OF PROBLEM ANALYSIS AND STATEMENT

Presented at a Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

1.0  THE PROBLEM

1.1 Question: What is the problem and why should it be studied?

1.2 Action: selection, analysis, and statement of the research problem

1.3 Outcomes: problem identification, prioritizing the problem, analysis of the problem, and justification

2.0  INFORMATION

2.1  Question: What information is available?

2.2   Action: literature review

2.3   Literature and other available information

3.0  WHY?

3.1  Question: Why do we carry out the research? What do we hope to achieve?

3.2  Action: Formulation of research objectives

3.3  Outcomes: general and specific objectives & hypotheses

1005 PRESENTATION: ANALYSIS AND STATEMENT OF THE PROBLEM

Presented at a Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

1.0 STEPS IN ANALYSING THE PROBLEM

1.2 Clarify views of stakeholders on the problem

1.3 Further specify and describe the core problem

1.4 Analyze the problem: underlying factors organized in larger categories (socio cultural, service, disease-related, KAP

2.0 DECIDING ON THE FOCUS & USEFULNESS OF THE RESEARCH

2.1 Usefulness of the information

2.2 Feasibility

2.3 Are we duplicating previous efforts?

3.0 NEED FOR A GOOD PROBLEM STATEMENT

3.1 Basis for all future work

3.2 Defines what information will be needed

3.3 Makes the research focused and systematic

4.0  CONTENTS OF A GOOD PROBLEM STATEMENT

4.1 Socio-cultural-economic background of the area

4.2 Nature of the problem: size, distribution, severity, who is affected

4.3 Major factors that may influence the problem

4.4 Solutions tried in the past and why the failed

4.5 Type of information expected from the project

4.6 Definitions of crucial concepts

1005 PRESENTATION: FORMULATION OF RESEARCH OBJECTIVES and HYPOTHESES

Presented at a Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

1.0  LEARNING OUTCOMES

1.1 State reasons for writing objectives

1.2 Define and describe differences between general and specific objectives

1.3 Define characteristics of research objectives

1.4 Prepare research objectives in an appropriate format

1.5 Develop further research questions, research hypotheses

2.0  RESEARCH OBJECTIVES: GENERAL AND SPECIFIC

2.1  Objectives summarize what is to be achieved by the study

2.2  Objectives should be related to the problem statement

2.3 General objectives state expected outcomes in general terms

2.4 Specific objectives are logical breakdowns of the general objectives defining: what, where, and what purpose

3.0  ADVANTAGES OF DEVELOPING OBJECTIVES

3.1 Focus the study on the essentials

3.2 Ensure collection of only strictly necessary data

3.3 Organize the study in parts or phases

4.0  CHARACTERISTICS OF GOOD OBJECTIVES

4.1 Comprehensive covering aspects of the problem and its factors

4.2 Coherent and in a logical sequence

4.3 Clearly phrased in operational terms

4.4 Realistic in the local conditions

4.5 Use specific verbs that can be evaluated

5.0  HYPOTHESES

5.1 A hypothesis is a testable explanation of the problem

5.2 A hypothesis is a testable prediction of the relation the problem and its factors

5.3 Health services research unlike clinical research is not amenable to hypothesis

formulation because of limited insight into complex interlinked problems

1005 DISCUSSION PROBLEMS AT WORKSHOP ON STATING RESEARCH PROBLEMS & FORMULATING RESEARCH QUESTIONS

Prepared for the Workshop on Training in Research Methodology held at the Faculty of Medicine King Fahad Medical City Riyadh Saudi Arabia 18^th May 2010 by Dr Omar Hasan Kasule Sr MB ChB (MUK), MPH (Harvard), DrPH (Harvard) Professor of Epidemiology and Bioethics <omarkasule@yahoo.com>

PROBLEM #1.

The Ministry of Health has approached you for advice on what to do about a new disease entity whose exact definition and causes are unknown so far. It started in one of the international schools on the first day of school with 2 children running high fever, coughing, and diarrhea. By the end of the week 30 children in the school had been excused from attending school. By the end of the month more than 1000 similar cases has been identified in many schools in the capital and the problem was spreading outside the capital.

PROBLEM #2:

The Ministry of Health has invited you to attend a presentation by a foreign company of an infra-red thermal device for detecting respiratory infection among passengers arriving at the airport. The device is in the form of a camera that is targeted at each passenger as he/she passes the health control post. You are required to investigate the effectiveness and usefulness of the device.

PROBLEM #3

The Ministry of Health has asked you for advice regarding licensing a new vaccine against cervical HPV infection recently produced by a transnational pharmaceutical company that is interested in marketing it in Malaysia. The vaccine is targeted at young women.

PROBLEM #4

The Ministry of Health in cooperation with the Ministry of Education has approved a 5-year SAR2.0 million nutritional supplementation project for primary school children in fishing villages along the East Coast. However there is no epidemiological information on the matter.

PROBLEM #5

There has been a recent spate of heart attack cases in the country and newspapers have started writing about an epidemic and the need to open cardiac centers in each district. Several individuals and organizations have also called for mass screening of the population to discover risk factors or symptoms of coronary heart disease early in order to start early treatment. The Ministry of Health wants to make policy decisions to address these problems and they have approached you for advice. You check in reports available at the Ministry and medical journals for information on the prevalence of coronary heart disease but found nothing. You then decide to collect the necessary data yourself.

PROBLEM #6

In the past 2 weeks, 10 citizens have been afflicted by a mysterious illness that starts as a common cold but develops into a severe cough, pneumonia, and sudden death. Nothing is known about the nature of the disease or its causes. The cases of the disease have been found all over the country and both genders are affected. Some of the victims are children while others are adults. The victims do not seem to have been in direct contact with one another. There is general fear in the country and the newspapers are calling on the Ministry of Health to take quick action. A policy meeting held at the Ministry of Health could not reach any conclusions because nothing is known about the disease. The meeting assigned you the task of finding out events or socio-demographic factors associated with the disease. You decide to use a case control design.

PROBLEM #7

The Ministry of Health has received complaints that schools in urban areas get better school health services making their children healthier than those of rural schools. The Minister has asked you to investigate this matter and to propose a policy to address the problem. You decide to study the height, weight, and completion of vaccination as indicators of health status.

PROBLEM #8

Statistics at the Center for Disease Control in the Ministry of Health have shown that an increase in cases of dysentery (bloody diarrhea) in the past 6 months in a sea-side village. Newspapers have been speculating that the cause is a new type of fish imported from a neighboring country. The Director of the Center for Disease Control has asked you to investigate the matter and report to him.

PROBLEM #9

The Ministry of Health is concerned about complaints from the public about absenteeism of paramedical workers. The Minister has asked you to investigate the matter. You go to the main hospital and obtain the records of staff in 3 departments: dietetics, physiotherapy, and radiology. You decide to investigate (a) the relation between absence and illness and (b) the relation between days of absence and salary.

 PROBLEM #10

A pharmaceutical manufacturer has applied for a license from the Ministry of Health to start marketing a new anti-influenza vaccine for the elderly in Saudi Arabia. The Minister has asked you to carry out research to establish whether the vaccine for is effective at the community level.

YOUR NOTES: