This slide contains B.Pharm 8th Sem Biostatistics and research methodology, Unit-3. Topic covered: Designing the methodology, Sample size determination and Power of a study, Report writing and presentation of data, Protocol, Cohorts studies, Observational studies, Experimental studies, Designing clinical trial, various phases.

1. Designing the Methodology
UNIT – 3 PART - 4
MR. HIMANSHU SHARMA
SHRI RAM COLLEGE OF PHARMACY
Biostatistics and Research Methodology
B.Pharm 4th Year 8th Sem

2. Designing the Methodology
 Sample size determination
 Power of a study
 Report writing and presentation of data
 Protocol
 Cohorts Studies and Its types
 Observational Studies
 Experimental Studies
 Designing clinical trials

3. Designing the Methodology
It Refers to the development of a system or a method for a unique situation
Nowadays the term is more often applied to technological field.
 Components of Design Methodology:
• The key to design methodology is to find the best solution for each design
situation whether it is to be in industrial or architecture or technology.
• Design methodology stresses the use of brainstorming to encourage
innovative ideas and collaboration of thinking to work through each
proposed idea and arrive at the best solution.
• Meeting the needs and wants of the end user is the most critical concern.

4. Designing the Methodology
• Design methodology employes basic research methods such as analysis and
testing.
• While design methodology is employed in many industries, it is commonly
applied in technological field including those using the internet, software
and information system development.
• Several design methodology approaches have been developed in the
technology industry.

5. Sample Size Determination & Power of Study
• Sample size determination is a mathematical estimation of a number of
subjects or unit to be included in a study.
• When a representative sample is taken from a population, the finding are
generalized to the population.
• Optimum scale size determination is required for the following reasons:
 To allow appropriate analysis
 To provide desired level of accuracy
 To allow the validity of significance test.

6. Sample sizes may be chosen and different several ways
• Experience a choice of small sample sizes, through sometimes necessary,
can result in wide confidence interval or risk of error in statistical
hypothesis testing.
• Using our target variance for an estimate to be derived from the sample
eventually obtained i.e., if a high precision is required this translates to a
low target variance of the estimator.
• Using a target for the power of a statistical test to be applied once the
sample is collected.
• Using a confidence level i.e., the larger the required confidence level, the
larger the sample size (given the constant position requirement).

7. Report Writing & Presentation of Data
 Report writing.
• Research is a condensed form of description or a brief details of the research
work done by a researcher/scientist. It involves several steps to present the report
in the form of thesis or dissertation.
• A mean to convey some information to others
• An organize, factual and objective presentation of information.
• Report is a comprehensive document and covers all aspects of the subjects matter
of study.

8. Report Writing & Presentation of Data
 Purpose of Report writing.
• Have implications or recommendation in clinical practice, education or administration.
• Reports are written to present and discuss research findings
• provide the reader with the rationale for the research, a description of the method used to
conduct the research, the findings, results, a logical discussion, and
conclusions/recommendations.
 Presentation of data
Method by which the people organize, summarize and communicate information using a
variety of tools such as stable graphs and diagrams.

9. Protocol
It is defined as the standard step to be followed for doing any research.
 It include following steps:
 Identifying the problem
 Exhaustic literature review
 Preparation of hypothesis
 Methodology adopted
 Recording of observation
 Presentation of data
 Conclusion and compilation of data

10. Cohorts Studies
• A cohort study is a type of epidemiological study in which a group of people with a
common characteristic is followed over time to find how many reach a certain health
outcome of interest (disease, condition, event, death, or a change in health status or
behavior)
• Cohort study is a type of analytical study which is undertaken to obtain additional evidence
to refuse or support existence of association between suspected cause and disease.
• Other name of cohort study are:
 Longitudinal Study
 Incidence Study
 Forward Looking Study

11. Features of Cohort Studies
• Cohorts are identifies prior to appearance of disease under investigation.
• To study groups are observed over a period of time to determine the frequency of disease
among them.
• The study proceeds from cause to effects.
 Indicators of Cohort study:
• When the exposure is rare, but incidence of disease among exposed is high.
• When people attrition can be minimized.
• When resources are ample.

12. Observational Studies
• Observation studies are a fundamental part of epidemiologic research.
• They are called observational studies because the investigator observes
individual without manipulation or intervention.
• In fields, such as epidemiology, social science, psychology and statistics, an
observational study inference from a sample to a population where the
independent variable is not under the control of the research because of
ethical concern or logistics constraint.

13. Observational Studies
 Types of Observation Studies:
• Case-control study
Study originally developed in epidemiology in which two existing groups
differing in outcomes are identified and compared on the basis of some
supposed casual attribute.
• Cross-sectional study:
It involves data collection from a population or a representative subset, at one
specific point in time.

14. Experimental Studies
• In contrast to observational studies, experimental studies involves the experimental
allocation subjects to different exposure and then observing difference in a disease
(or other health), outcomes between exposure groups.
• They are time consuming and expensive, therefore, in experimental studies- we
compare intervention groups and control groups.
 Principles:
The individuals (for communities) will be assigned to the drug or intervention
randomly, ensuring that the group are very similar except for the intervention,
removing the much of uncertainty of observational studies.

15. Experimental Studies
• To follow up.
• Incidence
• Temporarily is clear, no confounding/bias
• They have a gold standard to compare against.
Example:
Some patients who already have a disease will be, allocated a new treatment (for
example new drug or new type of surgery) and others will be allocated the standard
treatment or placebo (clinical trials).

16. Designing Clinical Trials
• Clinical study design is the formulation of trials and experiments as well as
observational studies in medical, clinical and other types of research (e.g.
epidemiological) involving human being.
• The goal of a clinical study is to assess the safety, efficacy and/or the mechanism
of action of an investigational medicinal product or procedure, or new drug or
device by a health authority (e.g. FDA).
 Treatment studies:
• Randomized controlled trial
• Blind trial
• Non-blind trial
• Adaptive clinical trial
• Non-randomized trial

17. Various Phases of Clinical Trials
Phase
I
• Phase I Clinical trials is the first time the new treatment is tested in people.
• Phase I trials test for safety and tolerance of the new treatment by recruiting and closely monitoring a small group
of people.
Phase
II
• Phase II clinical trials continue testing the safety and effectiveness of the new treatment.
• Phase II trials recruit and closely monitor a larger group of peoples.
Phase
III
• Phase III Clinical trials focus on determining whether the new product is more effective than the current
treatment option.
• Phase III clinical trials recruits 100-10000s of people.
Phase
IV
• Phase IV clinical trials are new treatment that have been reviewed and approved by regulatory bodies and are now
available to the public.
• Phase IV trials continue monitoring the long term risk and benefits of the newly approved treatment.

18. THANK YOU