Study Types

Study type strengths Limitations

A. Observational and descriptive Studies
Case series, Case report 1. Help describe rare 1. Purely descriptive and considered to
Case reports and series are manifestations and new be the weakest form of evidence.
helpful in recognizing and diseases. 2. Lack of control group (no
describing new disease 2. Identify emerging health comparison)
processes or rare conditions. 3. Can not be used to test for the
manifestations and identify 3. Better suited to describing the presence of a valid statistical
emerging health conditions. characteristics of the disease association.
“documents unusual adverse than identify causality. 4. Based on the experience of only one
events observed in single person.
patients”

Cross-sectional study 1. Fast and inexpensive. Cannot estimate incidence

1.
Prevalence study 2. Avoids the problem of loss to Difficult to establish causal
2.
“It is a survey of the total follow-up. relationship
population for the exposure 3. A cross-sectional study 3. Impractical for the study of rare
and disease (or drug use conducted at the beginning of a diseases and requires a large sample
problems) at a single point cohort or clinical trial provides size
in time”. demographic and clinical 4. Only a snapshot at a time leading to
characteristics at baseline. a misinformation
5. The result not representative of the
whole population (only individuals
who participate in the study).
B. Observational and analytical Studies
Case-control study 1. Relatively inexpensive 1. Low internal validity (data
Compare cases with a because of smaller sample collection is based on event recall).
disease to controls without sizes. 2. High chance of bias because of
the disease, looking for 2. It is useful for studying a separate sampling of cases and
differences in exposures relatively rare disease controls and retrospective
(multiple possible causes of 3. Multiple etiologic factors can measurement of various predictor
a single disease). be studied. variables.
4. Useful for generating 3. Cannot estimate incidence or
hypotheses about the causes prevalence.
of an outcome variable. 4. Only one outcome can be studied,
5. One does not need an ethical whereas cohort and cross-sectional
consent. studies can evaluate multiple
6. There is no risk to the subject outcome variables
.

Cohort study 1. Can measure incidence. 1. Expensive because of large sample

Can be prospective or 2. Establish temporal association size and long follow-up.
retrospective 3. Good for common diseases. 2. Not good for rare diseases.
In a prospective cohort 4. Good for rare exposures. 3. Confounding factors
study, patients are identified 5. Possible to study associations 4. A number of potential biases must be
at the beginning of the study of an exposure with several taken into account in conducting
based on their exposure outcome variables. cohort studies.
status (exposed or not a. Bias in assessment of the outcome
exposed) and followed up b. Information bias or recall bias.
for a period from exposure c. Biases from lack of response due to loss to
to outcome follow-up.
d. Analytic bias.
 Both designs are identical, comparing exposed and non-exposed populations. The only difference is calendar
time.
 The duration of follow-up and cost, associated with prospective cohort studies may be reduced by conducting
retrospective cohort studies.
 C. Intervention Study (Experimental)
Randomized controlled 1. Exposure is under the control 1. Expensive
study of investigator. 2. Subject exclusion may limit ability to
Subjects in a population are 2. Randomization generalize findings to other patients.
randomly allocated into 3. Blinding eliminates bias 3. A long period of time is often
groups, usually called study 4. Control on time span required to reach a conclusion.
and control groups to receive 5. Confounding factors can be 4. A large number of participants may
and not receive an controlled be required.
experimental preventive or 6. Best method to study causal 5. Need an ethical consent.
therapeutic procedure. relationship 6. Subjects may not comply with
7. We can confirm or refute treatment assignments.
"The investigator applies an etiological hypothesis on 7. May expose participants to potential
intervention and observes evidence. harm.
the effect on one of more 8. More than one outcome can
outcomes". be measured.
Clinical trials
Preclinical The first step in development of a new drug is bench research using tissue cultures or
animal models. Information on mechanism of action, efficacy, toxicity,
pharmacokinetics, and pharmacodynamics is obtained from these studies
Phase I trial This phase emphasizes safety. It involves 20 to 80 healthy volunteers. Information on
the drug’s most frequent adverse effects, drug metabolism, and excretion are obtained
from phase I studies
Phase II trial The goal of phase II trials is to obtain preliminary data on whether the drug works in
patients who have a certain disease or condition. It typically involves hundreds of
patients. Safety continues to be evaluated, and short-term adverse effects are studied.
Phase III trial Phase III trials typically involve hundreds or thousands of patients and gather more
information on safety and efficacy (RCT)
New Drug Application If the phase III trial is successful, the sponsor applies for an New Drug Application to the
review FDA. This process includes a review of the proposed professional labeling and
inspection of the manufacturing facility. If the review is favorable, the FDA may
approve the drug for marketing.
Phase IV or postmarketing This trial happen after the FDA has approved medication. This phase involves thousands
surveillance of participants and can last for many years.
Investigators use this phase to get more information about the medication’s long-term
safety, effectiveness, and any other benefits.
14/2/2019
‫انواع الدراسات *‪*STUDY DESING‬‬
‫فيه ثالث انواع منها‪-:‬‬
‫وصفية ‪Descripitive‬‬
‫باملالحظة ‪Observatinal‬‬
‫بالتجربة ‪Experimental‬‬

‫_)‪_Descripitive(case report and case series‬‬

‫الكيس ريبورت ملا االحظ على املريض عرض جديد يكون ريبورت على مريض واحد‬
‫اكثر من مريض هذا سيرس‬
‫لو كان مريض واحد وفيه اكثر من دكتور كتب عن هذا العرض هذي برضوا سيرس‬
‫من ايجابياتها تساعدني على انشاء فرضية جديدة‬
‫سلبياتها اني ماقدر اسوي مزامنة بني املرض والعرض وبرضوا ماتعلمني السبب ال‪Causality‬‬

‫_‪_Observatinal‬‬
‫عن طريق املالحظة الي هي‬
‫‪Control‬‬
‫كلماتها املفتاحية ‪ rare‬و‪orphan‬‬
‫ايضا ريترو يعني ارجع باملاضي‬ ‫تعتبر ً‬
‫‪Cohort‬‬
‫فيها نوعني ريترو رجوع للماضي‬
‫وبرو تنبؤ مستقلبي اربطوها مثال بايفون برو شيء فاخر يتيح لك بالتنبأ بنتائج مستقلبية‬
‫مثال لو جاك كيس وقالك راقبنا املرضى ونبغى نشوف تطور املرضى دام فيها تطور هذيي ع طول برو‬
‫ايضا ‪Incidence study‬‬ ‫تسمى ً‬

‫‪Cross sectional‬‬
‫كلماتها املفتاحية لو قالك سوينا‬
‫دراسة او نشرنا استبيان او جلسنا مع املرضى او حدد لك مدينة معينة‬
‫ايضا ‪prevalence study‬‬ ‫تسمى ً‬

‫‪Experimental‬‬
‫وتعبتر االقوى واالفضل واالغلى برضو‬
‫‪Meta‬‬
‫االقوى ويجيك السؤال فيها عن القوة او مثال يقولك اجمع دراسات عشان اسوي خاتمة وحده هذي ميتا اوقات يجيبون تعريفها بطريقة‬
‫توهمك انها سيستماتيك يقول انها توزعت بطريقة نظامية او ‪ systematic arrangement‬بالوهله االولى بتقول سيستماتيك ذي لكن‬
‫الجواب الصح هي ميتا‬
‫‪Systematic review‬‬
‫نفس فكرة ميتا بالضبط وتعتبر ثاني اقوى دراسة‬
‫‪Randomized‬‬
‫الي تعلمني السبب او ال‪causality‬‬
 Single exposure
Single outcome

Hypothesis - short duration

Rare - Inexpensive No causality

Prevalence

Just Retrospective

‫إلي عندهم‬
‫أمراض وإلي ما‬
‫عندهم‬
Rare disease Odds ratio - Multiple exposure Only one outcome - Recall bias

Several outcome
Relative risk
Incidence

Case control: Study based on presence or absent of the disease

Cohort study: Study based on presence or absent of the exposure or risk

as
case
control
 SAIF ALGHAMDI
!‫توكلت على ا‬

study design ‫ا‬

1- If a study is to follow a group of smokers and a group of nonsmokers time, and see which ones eventually
develop lymphoma and which.
What do you call this study design
A. Retrospective cohort study
B. Prospective cohort study
C. Cross-sectional study
D. Case-series

2- Which one of the following designs could be deemed a study

A. Cohort
‫تعتبر دراسة‬
B. Clinical trials
C. Case-control
D. Cross-sectional

3- Which of the following is an interventional study

A. Cohort
B. Ecological
C. Case-Control
D. Randomized Clinical Trial

4- Researchers compare a group of people who have diabetes with of people who have no diabetes, and looks
back in time to see characteristics of the two groups [Link] of the following is the described study design
A. Prospective cohort
B. Case-Control
C. Experimental
D. Randomized clinical

5- Which of the following studies are more susceptible to recall bias

A. Retrospective cohort
B. Prospective cohort study
C. Randomized controlled trial
D. Non-randomized controlled trial

6- Which of the following is the type of studies considers data from multiple studies of different designs to draw
conclusions
A. Case Series
B. Double-blind study
C. Systematic Review
D. Consensus statement

7- The authors report two isolated gastro-intestinal tuberculosis in renal transplant recipients that illustrates the
difficulty of making this diagnosis and a brief review of the literature on its clinical presentation, diagnosis and
therapeutic approach.
What is the type of study in the above-mentioned abstract
A. Case-series
B. Cohort studies
C. Case-control studies
D. Cross-sectional studies
8- Scenario: A study located a group of subjects with lymphoma and went to identify which subjects are smokers
and which are not.
Which of the following study designs is used to conduct the study in the scenario
A. Case-series
B. Cross-sectional study
C. Prospective cohort study
D. Retrospective cohort study

9- Which of the following terms measures the association between the exposure and the outcome
A. P Value
B. Risk ratio
C. Standard deviate

10- Which of the following study design is the best to use to assess the awareness on the usage of vitamin D
supplements among people of Jeddah
A. Cohort study
B. Case report study
C. Case control study
D. Cross sectional stud

11- A researcher gathered all vitamin E studies from the past ten years. Vitamin E was used for a variety of condition.
The populations studied as well as the vitamin E formulations and doses were all different. The researcher
compared the incidences of cardiovascular-related mortality in those taking vitamin E supplements versus those
that did not.
Which of the following best describe this type of study
A. Meta-analysis Meta-analysis is a research process used to systematically synthesise or merge
B. Cohort study the findings of single, independent studies, using statistical methods to calculate
an overall or 'absolute' effect.2 Meta-analysis does not simply pool data from
C. Observational study smaller studies to achieve a larger sample size.
D. Controlled clinical trial

12- A group of patients with diabetes were interviewed to determine they are currently eating fast food or
[Link] epidemiological study design is this
A. Cohort
B. Case-control
C. Cross-sectional
D. Randomized control trial

13- Which of the following is the dis advantage of conducting randomized Controlled trial
A. Expensive
B. High possibility of bias
C. Difficult to control confounders
D. Cannot be used for hypothesis testing

14- Study that investigate if drinking coffee lead to coronary hear found that there is a correlation between two
variables. Study that investigate if drinking coffee lead to coronary hear found that there is a correlation
between two variables. However drinkers are more often smokers than the average and, correlation between
drinking coffee and nicotine consumption, strong causal correlation between smoking and the incidence of
heart disease. Which of the flowing type of bias affect this study results
A. Measurement errors
B. Hawthorne effect
C. Information bias
D. Confounding
 LEARNING RESOURCES

SAIF ALGHAMDI
!‫توكلت على ا‬

Study Design Quiz

by Laura King, MA, ELS
CORRECT ANSWERS

Directions: Respond to the following questions based on your knowledge of chapter 20

(§20.2 §20.7; pp 838 851 in print) of the AMA Manual of Style.

1. Which type of study assesses the efficacy of the treatment intervention in a

controlled, standardized, and highly monitored setting and usually among highly
selected samples of patients?

randomized controlled trial

case series
sensitivity analysis
cost benefit analysis

2. In which type of study do participants receive more than 1 of the treatments under
investigation, usually in a randomly determined sequence and with a prespecified
amount of time (a “washout period”) between sequential treatments?

parallel design, double blind trial

noninferiority trial
prospective cohort study
crossover trial

Copyright © American Medical Association, 2011. For educational use only.

[Link]
 LEARNING RESOURCES

3. In which type of study does the investigator use information already collected to look
for associations?

retrospective study
prospective study
historical study
survey study

4. In which type of study does the investigator identify a group of individuals and then
observe them for a specified period after study initiation?

retrospective study
prospective study
meta analysis
diagnostic test study

5. Which type of study assesses whether 1 or more treatments are superior to the
others?

parallel design, double blind trial

retrospective cohort study
case control study
crossover trial

6. Which type of study follows up a similar group of individuals who are initially free of
the outcome of interest but for whom the outcomes have been defined before the
events occur?

prospective cohort study

crossover study
case series
cost benefit analysis

7. Case control studies are always

prospective
retrospective
overmatched
multivariate

Copyright © American Medical Association, 2011. For educational use only.

[Link]
 LEARNING RESOURCES

8. Which of type of study compares a less expensive treatment or intervention against a

treatment or intervention that is already known to be effective to assess whether the
new intervention is no worse than the existing treatment?

meta analysis
case control study
noninferiority trial
randomized controlled trial

9. Which type of study compares those who have had an outcome or event with those
who have not?

case control study

case series
cohort study
meta analysis

10. Which type of study can be used to describe the experience of an individual or
institution in treating a disease?

case control study

crossover trial
case series
equivalence study

11. Which type of study is a systematic pooling of the results of 2 or more studies to
address a question of interest or hypothesis?

case control study

meta analysis
noninferiority trial
parallel design, double blind trial

12. Which type of study generally yields estimates of likelihood ratios, sensitivity,
specificity, positive predictive values, and negative predictive values?

crossover study
cost effectiveness study
meta analysis
diagnostic test study

Copyright © American Medical Association, 2011. For educational use only.

[Link]
 LEARNING RESOURCES

13. In longitudinal survey studies, the same respondents are surveyed

at several time points

at a single time point
only once
cross sectionally

14. Which type of study converts clinical measures of outcomes into monetary units,
allowing both expenses and advantages to be expressed on a single scale?

cost benefit analysis

cost effectiveness analysis
equivalence study
sensitivity analysis

15. Which type of study benefits from the publication of a flow diagram showing the
flow of participants in the study, including when and why participants dropped out or
were lost to follow up and how many participants were evaluated for the study end
points?

case series
randomized controlled trial
meta analysis
survey study

Copyright © American Medical Association, 2011. For educational use only.

[Link]
 7/19/20

pharmacoeconomics and
pharmacoepidemiology
6th Year – Pharm.D.
SPLE
SAIF ALGHAMDI
Lecture 2 !‫توكلت على ا‬
Prepared by
Dr. Dalia Tantawy
Clinical Pharmacy Department
1440-1441 H
2019-2020 G
1

Learning Outcomes:
q Pharmacoepidemiology:
-By the end of this part, the students will be able to :
● Identify data sources and analytic tools that provide an estimate of
the probability of beneficial or adverse effects of medication use
in large populations.
● Identify application of epidemiological study designs to evaluate
drug use and outcomes in large populations.
● Recognize Methods for continually monitoring unwanted effects
and other safety-related aspects of medication use in large
populations.
● Recognize Pharmacovigilance and medication safety.

1
 7/19/20

Question #1:

of
Rct

Answer #1:

2
 7/19/20

Question #2:

be its observe ionol

5

observational's
Rct y 2g I I

Answer #2:

3
 7/19/20

Question #3:

Answer #3:

4
 7/19/20

Question #4:
start
with Risk

this
is
diseas
Not
risk
case
control
9

Answer #4:

10

5
 7/19/20

Question #5:

11

Answer #5:

12

6
 7/19/20

Question #6: case control

becals he start
q with
disease

o
coo
o I
2 so 02
a oo
o t S
o o2

13

Answer # 6:

14

7
 7/19/20

Question #7:

15

Answer #7:

16