Pharmacoepidemiology Measurement of Outcomes

MEASUREMENT OF OUTCOMES IN
PHARMACOEPIDEMIOLOGY
The outcome measures include the studies on,
1. Functional status (level of functioning, Supervision
required, ability to work)
2. Symptom status (days free of pain / an event)
3. Patient satisfaction with various aspects of care (delivery
of care, effects on daily activities or life satisfaction) and
4. QOL studies

 The therapeutic outcomes may be classified as cure,

improvement, no change or deterioration.

 On the other hand they can also be classified as success or

failure.

 In any event, clinical judgement is required in establishing

outcomes.
Morbidity & mortality are the most commonly used measures
of out come
 Morbidity is measured as the number of cases of disease or
events that occur per unit of population (per 100), unit of
time (per year) or both (events/100/year)
 Other measures of morbidity are,The number of
hospitalisations resulting from drug use or prevented by drug
use or days of hospitalization (or days avoided) and deaths
due to or prevented by the use of drugs
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Pharmacoepidemiology Measurement of Outcomes

The measurement of outcomes in PEY can be done by two

approaches:
1. Outcome measures
2. Drug use measures
OUT COME MEASURES
The occurrence of pharmacoepidemiological outcomes is
commonly expressed by measurements such as,
a. Prevalence
b. Cumulative incidence and
c. Incidence rate
 Prevalence
 It is concerned with the disease status
 It is the proportion of people affected with a disease or
exposed to a particular drug in a population at a given
time
 It is usually determined by surveying the population of
interest
 Prevalence varies between 0-1, it can also be
expressed as a percentage
Ex. The prevalence of schizophrenia is 1% in Europe.
 Mathematically,
Prevance = a/b

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a- number of population with disease at a given time

b- total number of population at a given time

Ex. In the year 2003, among 7.8 lac population of Mysore city, 1.2
lac suffered from chikungunya.
What is the prevalence of Chikungunya in Mysore in the year
2003?
P= 0.15 or 15%

Incidence:
• It is a measure of the risk of developing some new condition
within a specified period of time
• it is better expressed as a proportion or as a rate.

Cumulative incidence (incidence proportion):

• It is the number of new cases within a specified time period
divided by the size of the population initially at risk
Ex. if a population initially contains 1,000 non-diseased persons
and 28 develop a condition over two years of observation, the
incidence proportion is 28 cases per 1,000 persons, i.e. 2.8%.
Incidence rate:
• It is the number of new cases per unit of person-time at risk.

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Pharmacoepidemiology Measurement of Outcomes

• It describes the probability of a new case occurring during a

given time interval
What is person-time?
• It is an estimate of the actual time-at-risk in years, months or
days that all persons contributed to study.
• In certain studies people are followed for different lengths of
time as some will remain disease-free longer than others.
• A subject is eligible to contribute person-time to the study
only so long as that person remains disease-free and
therefore, still at risk of developing the disease of interest.
• By knowing the number of new cases of disease and the
person-time-at-risk contributed to the study, an investigator
can calculate incidence rate.
• IR= the no. of new cases of disease during a period of
time/the person-time-at-risk.
• The denominator for IR (person-time) is a more exact
expression of the population at risk during the period of time
when the change from non-disease to disease is being
measured .
• The denominator for IR changes as persons originally at risk
develop disease during the observation period and are
removed from the denominator
Drug use measures
1. Monetary units

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2. Numbers of prescription
3. Units of drug dispensed
4. Defined daily doses
5. Prescribed daily doses
6. Medication adherence measurement

1. Monetary units
• Drug use has been measured in monetary units to quantify
the amounts being consumed by population
• It can indicate the burden on a society from drug use
• Monetary units are convenient & can be converted to a
common unit, which then allows for comparison
• The disadvantage is quantities of drugs actually consumed
are not known & prices may vary widely

2. Number of prescriptions.
• It has been used in research due to the availability & ease
• The disadv is, quantities dispensed vary greatly as duration
of treatment
Ex: Treatment with antibiotics, provide a fairly good estimate of
the no. Of people exposed & of the no. Of treatment episodes .

3. Units of drug dispensed

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• Units of drug dispensed like tablets, vials is easy to obtain &

can be used to compare usage trends within population
• The disadv is that no information is available on the
quantities actually taken by the patient
• Hence difficult to determine the actual no. Of patients
exposed to the drug

4. Defined daily doses

• It is the estimated avg. Maintenance dose per day of a drug
when used in its major indication
• It is normally expressed as DDD/1000 ptns/day or DDD/100
bed/day
• It is helpful in describing & comparing patterns of DU &
provides denominator data for estimation of ADR rates
• Adv
 its usefulness for working with readily available drug
statistics
 It allows comparisons b/n drugs in the same therapeutic
class
• Disadv is problems arises when doses vary widely like with
antibiotics or if the drug has more than 1 major indication
Ex: ASA low dose- avoid cardiac events
moderate dose-pain management

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high dose- for inflammatory condition

Prescribed daily doses

• It is the avg daily dose of a drug that has actually been
prescribed
• Calculated from representative sample of prescriptions.
• Disadv is that it does not indicate no. Of population exposed
to drug.
• However, it provides estimate of no of person-days of
exposure.
•
Medication adherence measurements
I. Biological Assays
• Biological assays measure the concentration of a drug, its
metabolites, or tracer compounds in the blood or urine of a
patient.
• These measures are intrusive and often costly to
administer.
• Patients who know that they will be tested may conciously
take medication that they had been skipping so the tests will
not detect individuals who have been nonadherent.

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• Drug or food interactions, physiological differences, dosing

schedules, and the half-life of the drugs may influence the
results
• Biological tracers that have known half lives and do not
interfere with the medication may be used, but there are
ethical concerns
• All of these methods have high costs for the assays that
limit the feasibility of these techniques

II. Pill Counts.

• Counting the number of pills remaining in a patient's supply
and calculating the number of pills that the patient has taken
since filling the prescription is the easiest method for
calculating patient medication adherence
• Some data indicate that this technique may underestimate
adherence in older populations
• Patterns of non-adherence are often difficult to discern with a
simple count of pills on a certain date weeks to months after
the prescription was filled

III. Weight of Topical Medications

• The weight of a topical medication remaining in a tube is
used as a measure of adherence.

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• When compared with patient log books of daily medication

use, weight estimates of adherence were considerably lower
than patient log estimates.
• In the clinical trials involving topical applications incorporate
medication weights as the primary measure of adherence.
• In a comparison of methods to measure adherence, found
that estimates calculated from medication logs and
medication weights were consistently higher than those of
electronic monitors

IV. Electronic Monitoring

• The Medication Event Monitoring System (MEMS)
manufactured by Aardex corporation allows the assessment
of the number of pills missed during a period as well as
adherence to a dosing schedule
• The system electronically monitors when the pill bottle is
opened, and the researcher can periodically download the
information to a computer
• The availability and cost of this system could limit the
feasibility of its use

V. Pharmacy Records and Prescription Claims

• This method can be used primarily for medications that are
taken for chronic illnesses (such as hypertension)

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• These records provide only an indirect measure of drugs

consumed.
• Patterns of over and under consumption for periods less
than that between refills cannot be assessed

VI. Patient Interviews

• Studies have consistently shown that third-party
assessments of medication adherence by healthcare
providers tend to overestimate patients' adherence
• Interviewing patients to assess their knowledge of the
medications they have been prescribed and the dosing
schedule provide little information as to whether the patient
is adherent with the actual dosing schedule.
• Subjective assessments by interviewers can bias adherence
estimates.
• This method is rarely used in medical research to assess
adherence

VII. Patient Estimates of Adherence

• Direct questioning of patients to assess adherence can be
an effective method.
• However, patients who claim adherence may be
underreporting their nonadherence to avoid caregiver
disapproval

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• Other methods may need to be employed to detect these

patients

 Scaled Questionnaire

 Morisky et al. (1986) developed a 4-item scaled

questionnaire to assess adherence with antihypertensive
treatment.
 Li et al. (2005) developed four instruments to measure
antihypertensive medication adherence in a population of
Chinese immigrants in the US
 The Hill-Bone Compliance to High Blood Pressure Therapy
Scale includes 14 items, 8 of which are directed at assessing
medication taking behavior in hypertensive patients
 Not only is this method relatively simple and economically
feasible to use, but it has the added advantage of soliciting
information regarding situational factors that interfere with
medication adherence (e.g. forgetfulness, remembering to
bring medications along when out of town)
 The Compliance-Questionnaire-Rheumatology (CQR) is a
19-item questionnaire that has been favorably compared
with electronic medication event monitoring (de Klerk et al.
2003). This instrument has good validity and reliability.

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