Pharmacoepidemiology

Introduction, Definition & Scope

1. Epidemiology is the study of the distribution and determinants of

diseases in populations
2. Epidemics is the study of chronic/ infectious diseases in large
populations
3. Pharmacoepidemiology is the application of the principles of
epidemiology to drug effects and drug use. Hence,
Pharmacoepidemiology is the study of the use of and the effects of
drugs in larger population
4. It involves the examination of a single individual or large groups of
people followed for many years
5. It involves gathering & analysis of information in order to identify
possible causation & related factors that can be applied in clinical
practice to group of people & also to individuals undergoing treatment.

In general, Pharmacoepidemiology examines the relationship between the drug

exposure and health outcomes in a defined population.

Origin & evolution.

1. ADRs to drugs were as old as modern pharmacotherapy which was
developed in 20th century.
2. Drug resistance, drug abuse & variations in rates of clinical effectiveness
were the other therapeutic problems which emerged.
3. In 1961, the case reports of maternal use of thalidomide with
malformations in offspring results in awareness of the potential for drugs to
cause ADRs.
4. Since then, a greater attention was focused on the detection, prevention &
management of ADRs & the era of Pharmacoepidemiology has began.
5. The important ADRs detected through these systems include,
 Grey baby syndrome due to Chloramphenicol
 Vaginal cancer in off springs of women who took diethylstilbestrol
during pregnancy
 Isotretinoin induced birth defects
 Triazolam induced CNS disturbances
 Suicidal ideation with Fluoxetine
 Deaths with Fenoterol
 Venous thromboembolism with OCs.
6. ISPE was formed,to obtain more data on risk & benefits of drugs in
population and to discuss, develop & disseminate information about
Pharmacoepidemiological methods
7. In early 1960, the related field of drug utilisation was developed along with
the study of ADRs
8. Previously, DU studies were conducted mostly for marketing purposes and
data were not available for use by health authorities
9. As a result of wide variations in the pattern & extent of drug prescribing,
growing concern about ADRs & cost of the drugs, the
Pharmacoepidemiological methods were developed
10.According to WHO, DU is the marketing, distribution, prescription & use of
drugs in a society with special emphasis on the resulting medical, social &
economic consequences
11.In Europe, DU research developed at the national & international level with
a common methodology for comparative DU studies using a relatively
economic & readily available drug statistical sources
In NA, DU research was developed on a smaller scale & was most
emphasized on the qualitative aspects of prescribing (antibiotics)
12. DUR is an authorized, structured & continuing program that reviews,
analyses & interprets patterns of drug use against predetermined standards
13. In Europe, the medical audit concept was implemented & was defined as a
searching examination of the way in which drugs are used in clinical
 practice carried out at interval frequent enough to maintain generally
accepted standard of prescribing
14. It was mainly focused on medical practitioners with aim of improving the
RDU

Why Pharmacoepidemiology?
1. Lack of alternative models to investigate some drug events
E.g.: to evaluate teratogenic effects of a new medicine
2. CTs are inadequate to answer questions about drug safety, as they lack
adequate statistical power
3. If at all adequate for establishing effectiveness, the sample size are
inadequate to detect less common ADRs
4. CTs are conducted on highly selected patients without any co-morbidities &
who taking no other medications.
5. CTs does not involve elderly, paediatric or pregnant patients
6. CTs investigate the single indication
7. Hence, CTs fail to provide adequate information related to safety & efficacy
of a drug under non-trial conditions & in other indications
In contrast, Pharmacoepidemiological models provide alternative
approaches to evaluate drug effects.

Aims of PEY
1. Signal generation:
Identification of new, sometimes serious ADRs
Also helps to detect new applications of a drug
Ex: Minoxidil is used as Antihypertensive and Hirsutism
2. Risk quantification:
Risk quantification is the process of evaluating the risks that have been identified and developing the
data that will be needed for making decisions
 3. Hypothesis testing:
It requires the use of comparison groups to determine whether there
are differences in variables of interest

Applications of PEY
1. Estimation of the risks of drug use:
 The risk involved in drug use can be quantified
 The benefits & risks of use of a drug may be weighed
 Risk estimation also helps to identify risk situation
Ex: Case reports of triazolam induced psychiatric disturbances
appeared soon after its introduction to market
The drug was withdrawn in some countries. The reaction was likely
due to dose related; hence the problem was abated by
recommending a lower dose

2. Use in patient counseling:

Collection & analysis of observational data from other studies may

help to address certain issues through counseling the patients

Ex:A pregnant patient may wish to terminate pregnancy if there is a

substantial risk for producing a seriously malformed child, but would
also wish to proceed with the pregnancy if the risk is low.

3. Formulation of public health policy decisions:

Qualitative as well as quantitative information from PEY studies

helps to address many issues

Ex: If an inappropriate prescribing is observed among prescribers,

regulatory agencies may require educational intervention or may
impose restrictions on specific drugs or on practitioners
 PEY studies also help the policy makers to assess whether a drug should be
withdrawn from the market or allowed to remain

4. Facilitation of pharmacoeconomic evaluations:

Data from PEY studies can be used to measure the effects of drugs on
overall health care costs & resource consumption

Ex: Hospitalization due to serious adverse effects of a drug leads to more

expenses as well as resource consumption, which could be avoidable.