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A PILOT STUDY EXAMINING CRITERIA USED TO
SELECT DRUGS FOR HOSPITAL, PROVINCIAL AND
NATIONAL FORMULARIES
Robertson J, Newby DA, Pillay T, Walkom EJ
The University of Newcastle, Australia
WHO Collaborating Centre for Training in Pharmacoeconomics and Rational Pharmacotherapy
Abstract
A Pilot Study Examining Criteria Used to Select Drugs for Hospital, Provincial and National Formularies
Robertson J, Newby DA, Pillay T, Walkom EJ
the University of Newcastle, Australia
Problem Statement: Little information is available on the selection criteria used to choose drugs for formularies at
different levels of health care systems.
Objectives: To compare and contrast the importance of 22 criteria in selecting drugs for inclusion in hospital, provincial
and national drug lists in a number of emerging and developed countries.
Design: Questionnaire survey of members of the Australian Pharmaceutical Benefits Advisory Committee (PBAC) and
Economics Sub-Committee (ESC) (national level), 6 provincial drug selection Committees in South Africa and
participants in an international short course on pharmacoeconomics who reported >1 year involvement in drug
selection at hospital, area or national level (emerging and developed economies).
Outcome Measures: Criteria were categorised as “always or almost always important”, “sometimes important” or
“never or almost never important” for drug selection decisions and grouped as clinical factors, cost factors, drug
characteristics and supply issues, and other factors influencing decision-making.
Results: Responses were obtained from 11 PBAC/ESC members, 72 members of South African provincial drug
committees and 22 short course participants. Clinical factors were always or almost always important at all levels
of decision-making and dominant issues in drug selection. Quality of life considerations were less important than
drug efficacy and safety. Cost factors were important at all levels, particularly the likely total cost (budgetary
impact). Cost-effectiveness is mandated for Australian PBAC decision-making, and the potential for non costeffective use was also important. Cost-effectiveness was reported as always or almost always important for
around 90% of South African provincial decision makers, although the extent to which this is formally measured
and considered is unclear. There were differences in the reported importance of cost-offsets for decision-making –
potential savings to health care budgets through shorter hospital stays and reduced nursing time. The availability
of alternative treatment options was an important criterion for decision-making; the age of the patients and whether
or not the patient may have contributed to the development of the disease were less important factors in drug
selection decisions. The importance of drug characteristics and supply-related issues varied with the decisionmaking setting. External pressure to list a drug was sometimes important in all decision-making settings.
Conclusions: While a questionnaire cannot capture the complexity of many drug selection decisions, there were a
number of clinical and cost criteria that seemed to be important in all settings. Identifying the common key
elements may assist in developing formal processes to support objective and rational drug selection decisions.
Study Funding: Management Sciences for Health
Introduction
Bodies responsible for selecting drugs for formularies or
medicines lists face increasingly difficult decisions about which
drugs can be funded. New drugs may offer only small benefits
over existing therapies, but at higher cost. Social, ethical and
political pressures will determine health priorities and may
influence drug selection.
A variety of information about a new therapy must be evaluated
in making drug selection decisions. While clinical efficacy, safety
and acquisition costs are important, these are not the only
factors considered by decision-making committees. Some
factors may be context specific, i.e. relevant to hospital, area or
national level decision-making.
Objectives
to determine the importance of 22 criteria in selecting drugs
for inclusion in hospital, provincial or national drug lists
to compare and contrast the importance of the criteria for drug
selection by different groups of decision makers
Methods
A questionnaire survey was developed and administered to
members of the Australian Pharmaceutical Benefits Advisory
Committee (PBAC) and its Economics Sub-Committee (ESC),
national level decision makers
members of six provincial drug selection committees (PTCs)
in South Africa, area level decision makers,
participants in a WHO-sponsored short course in
pharmacoeconomics who reported >1 year involvement in
drug selection at hospital, area and/or national level,
developed and emerging economies
Likert-scale responses were used to classify decision-making
criteria as “always or almost always” important, “sometimes”
important, or “never or almost never” important.
Decision-making criteria assessed:
clinical factors – drug efficacy and safety, severity of disease,
other treatment options available, quality of life, whether for
treatment of disease or symptom relief
cost factors – cost to treat one patient, numbers of patients
requiring treatment, budgetary impact (total cost), costeffectiveness, potential for non cost-effective use, allows
earlier hospital discharge, reduces nursing time
‘pharmacological’ factors – likely compliance with treatment,
addiction/abuse potential of the drug, generic or branded
product, reputation of the pharmaceutical company
‘other’ factors – age of patients, patient’s contribution to the
condition being treated, local clinical experience with the drug,
already listed on other formularies, external pressure to make
the drug available
Results 1
Clinical factors: Efficacy and safety were the most important
factors; the availability of other treatment options was a key
consideration for all groups. Quality of life was less important
than efficacy and safety. Whether the drug provided treatment of
disease or symptom relief was a less important criterion for all
groups (Figure 1).
Figure 1
100
90
Percentage of respondents
80
70
60
50
40
30
20
PBAC
South Africa
10
0
Effectiveness
of medicine
Short Course
Side effect
profile
Already other
treatment options
available
Severity of
disease
Whether medicine
improves quality of
life
Treats underlying
disease or symptom
relief
Clinical factors rated as “always or almost always” important (block colour) or
“sometimes” important (shaded colour)
Results 2
Cost factors: Consideration of total costs (budget impact) and
cost-effectiveness are mandated for Australian decision makers;
the potential for non cost-effective use was important for these
respondents. The importance of cost-effectiveness for SA
respondents was surprising as there are no formal methods for
using this information in decision-making. Short course
participation was often prompted by the limited use of costeffectiveness information in their own country. Cost-offsets with
reduced hospital length of stay and nursing time were of slightly
greater importance to SA respondents (Figure 2).
Figure 2
100
90
Percentage of respondents
80
70
60
50
40
30
PBAC
20
South Africa
10
Short Course
0
Likely total
annual cost
Cost-effectiveness
of treatment
Potential for non
cost-effective use
No. of patients
w ho might need
medicine
Cost of treatment
for one patient
Allow s patient to
be discharged
sooner
Given in a w ay that
w ill reduce nursing
time
Cost/cost-offset factors rated as “always or almost always” important (block colour) or
“sometimes” important (shaded colour)
Results 3
The importance of ‘pharmacological factors’ varied by setting.
The reputation of the pharmaceutical company and whether the
product was branded or generic were not important criteria for
Australian respondents but ‘always or almost always’ important
for 24-43% of SA and short course respondents. Compliance
with therapy was ‘always or almost always’ an important criterion
for 76% of SA respondents but only 38% and 27% of short
course and Australian respondents. Potential for addiction or
abuse of the drug was also a greater concern for SA
respondents.
Figure 3
100
PBAC
South Africa
90
Short Course
Percentage of respondents
80
70
60
50
40
30
20
10
0
Age of population
group to use
medicine
Whether person's ow n
actions could bring on
illness
Doctors have already
been using medicine
on a limited basis
Listed on other
medicines lists
Other factors rated as “always or almost always” important (block colour) or
“sometimes” important (shaded colour).
External pressure
to make medicine
available
Results 4
‘Other factors’: The age of the population being treated; whether
a person’s own actions contributed to the illness and whether
the drug was listed on other formularies were more important
criteria for SA respondents than for Australian and short course
participants. External pressure to list a drug was recognized in
all settings. Clinical experience with the drug was always or
sometimes important in all settings (Figure 3).
Conclusions / Implications
Survey methods cannot capture the complexity of decisionmaking and general questions may conceal the importance of
some criteria in particular circumstances. However, the
identification of criteria that are important in all settings may
assist in identifying standard information requirements to support
objective and rational drug selection decisions.