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Key Words: Cost minimization analysis, Cost benefit analysis, Cost utility analysis, Cost effectiveness analysis, Health care
economics, Pharmacoeconomics
Introduction
Pharmacoeconomics can be defined as the branch of time to market any new chemical entity as a drug to fulfill
economics that uses cost-benefit, cost-effectiveness, cost- various research requirements (Newdy 2003). Since 1961,
minimization, cost-of-illness and cost-utility analyses to pharmaceuticals are fallen under price regulation in India. A
compare pharmaceutical products and treatment strategies total of 343 drugs accounting for, 85% of the drug market was
(Brinsmead 2003). Pharmacoeconomics and health outcomes under price control in 1979. With successive polices, the
research are playing an increasingly important role in number diminished and now a mere 15 - 20% of the drug
informing clinical development and market access decisions of market is under price control. Therefore drug prices are
new innovative medicines. It mainly works on the health quickly spinning out of reach of the common man.
economics which particularly focuses upon the costs and Pharmacoeconomics adopts and applies the principles and
benefits of drug therapy. methodology of health economics to the field of
It is the part of health economics that focuses on the economic pharmaceutical policy (Basskin 1998). Pharmacoeconomic
evaluation of pharmaceuticals. Health outcomes research, and evaluation therefore makes use of the broad range of
patient-reported outcomes (PRO) in particular, aim at techniques used in health economics evaluation to the specific
understanding patient value in terms of impact of disease and context of medicines management.
its treatment on physical functioning and psychosocial
wellbeing, known also as “health-related quality of life”
(HRQL). Components of pharmacoeconomics are given as
Fig.1. It is the description and analysis of the costs of drug
therapy to healthcare systems and society. The importance of
pharmacoeconomic information to healthcare decision makers
will depend upon the viewpoint from which the analysis is
conducted. Pharmacoeconomic research in the managed care
system is growing. It is currently being used to make
formulary decisions (complementing clinical data), design
disease management programs and measuring the cost- Figure 1: Components of pharmacoeconomics
effectiveness of interventions and programs in managed care Pharmacoeconomics: Needs and Challenges
(Lopert 2003). The demand for and the cost of health care Multi-factorial Variability of Drug Pricing
are increasing in all countries as the improvement in and Enumerable factors affect the prices of drug; some of them are
sophistication of health technologies. The increase in health as follows:
care spending is mainly because of increased life expectance, i. The sector in which medicines are purchased: The price is
increased technology, increased standard of living and often higher in the private sector due to distilentor’s costs
increased demand in health care quality and services. and profiteering.
Medicines form a small but significant proportion of total ii. The types of procurement agent: e.g. different prices may
health care cost. The writing of a prescription is the most be paid for the same product by a public sector purchaser,
common therapeutic intervention in medicine. Cost of such as Ministry of Health (Garber 2004).
medicines are growing constantly as new medicines are iii. The distribution route. A patient who purchases a medicine
marketed and are under patent law, preference of drug therapy at a hospital pharmacy may have to pay more if the hospital
over invasive therapy, discovering various off label uses of pharmacy purchased the product from a local wholesaler
existing drugs and the irrational drug prescription. than if it has been purchased by tender and supplied
Pharmaceutical companies have to spend a lot of money and through public health sector distribution system10. Many
times hospital pharmacy may have limited stock of the
Address for Correspondence generic drugs which one is cheaper than the branded drugs
Email ID: baldiashish@gmail.com prescribed to the patient on routine basis and patient has to
Tel No.: +91-896842384 purchase the branded drugs in the emergency condition.
Received: 10/08/2013
Accepted: 18/10/2013
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iv. The patient status: The price of patented medicine is often Components
higher than that of their generic equivalent at least while Pharmacoeconomic analysis will play a central role in future
the patient is in force (Moayyedi et al 2004). assessments of new technologies and the guidance of pediatric
Many other factors influence the total cost of drugs is as care (Weinstein 1996). The components of pharmacoeconomic
follows: analysis are given in Figure 2.
• Prices of patented, non-patented and generic drugs;
• Retail and wholesale mark-ups,
• Pharmacists' professional fees,
• Changes in the composition of total population, e.g.
proportion of older persons,
• Changes in prescribing habits of physicians,
• Changes in the utilization of drugs, i.e. number of drugs
used per patient,
• Trends towards using newer drug therapy instead of other
treatments,
Need of Pharmacoeconomics
Pharmacoeconomics identifies measures and compares the
cost and consequences of pharmaceutical products and
services and describe the economic relationship involving drug
research, drug production distribution, storage, pricing and
used by the people (Rawlins et al 2004). It runs through the
thread of our socioeconomic system, which regulates and
Figure 2: Components of pharmacoeconomics analysis
influences all the sectors involved in pharmaceuticals.
It is implicitly understood that reducing costs and rationing
The demand for and the cost of health care are increasing in all
resources is not compatible with the achievement of optimal
countries as the improvement of health technologies. All over
health outcomes. Decisions on resource allocation must be
the world, patients are affected by high price of medicines. In a
based on comparisons of cost and benefits as much as possible
developing country like India, 85% of total health expenditure
(Sackett et al 2000). In an era when resources for health and
is financed by house-hold out of pocket expenditure. Many
health care are scarce, the costs of clinical decisions must be
poor people frequently face a choice between buying
recognized and accommodated. Pharmacoeconomic analysis is
medicines or buying food or other necessities due to limited
a powerful tool for such adaptation. In this, the movement
resources and high pricing of drug (Russell 1996). So medicine
toward outcomes research has been fueled by a number
prices do matter. Pharmacoeconomics has become more
of factors, including increasing prevalence of chronic
important over the past 20 years, due to an increases emphasis
disease among children, the desire of patients (and their
on efficient drug therapies for disease, which increase health
parents) to participate in decision making, the limited
cost etc.
generalizability or external validity of clinical trial data
Pharmacoeconomics is a subdivision of health economics and
(particularly for a pediatric population), evidence of wide
results from that discipline coming of age through
variation in clinical practice norms that cannot be
consolidation to diversification (Siegel 1996). Health
accounted for on the basis of evidence, growing interest
economics, as a branch of economics is itself relatively young.
in cost containment and acceptance of the need for
Basically the pharmacoeconomics is needful in following
accountability on the part of clinical decision makers
manner:
(Abarca 2005) and the perceived threat of cost containment
• In Industry- Deciding among specific research and to the overall quality of patient care. The target sector and
development alternatives. assessment methodology adopted for pharmacoeconomic
• In Government- Determining program benefits and prices evaluation is given as Figure 3 and Figure 4.
paid.
• In Private Sector- Designing insurance benefit coverage.
Pharmacoeconomic Challenges
This is a challenging time for the pharmaceutical industry,
with blockbuster drugs going off-patent, generics competition
and a pressing need for innovative medicines, associated with
rising drug development costs (Vermeulen et al 2000). The
role for pharmacoeconomics could not be timelier.
Pharmacoeconomic evaluations will be able to assist in the
development of performance based pricing and reimbursement
agreements. Figure 3: Target health care sector for pharmacoeconomic
The main challenges for pharmacoeconomics continue to be: evaluation
1. Establishing guidelines or standards of practice. Aims and Objectives of Pharmacoeconomic Evaluation
2. Creating a cadre of trained producers and consumers of Implementation of pharmacoeconomic evaluation will help to
pharmacoeconomic work. reduce monetary burden on the consumers (by insuring
3. Continuing education on the relevant features of this global pricing strategy) for the effective management of health
discipline for practitioners, government officials, private sector care system as the principle of pharmacoeconomic evaluation
executives. is to make more efficient use of limited resources for
4. Stable funding to support applied pharmacoeconomic maximization of health care benefit at lower cost (Sculpher et
research. al 2005). In this way, it can be applied in practice to take
Pharmacoeconomic Evaluations decisions about drug therapies.
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B. “Utility” units - Utility is an economist’s word for
satisfaction, or sense of well being, and is an attempt to
evaluate the quality of a state of health, and not just its
quantity (Sanchez 1999). Utility estimates can be obtained
through direct measurement (using techniques such as time
trade off or standard gambles, or by imputing them from the
literature or expert opinion. They are often informed by
measures of quality of life in different disease states.
The Approach
A pharmacoeconomic evaluation may be conducted as an
economic assessment incorporated into clinical trials (Sanchez
1999). It is difficult to justify a pharmacoeconomic trial as a
separate, independent trial primarily due to the increase in
costs (Weinstein et al 2003). The evaluation may be done at
any time during phase I or II of the clinical trials.
Figure 4: Nature of pharmacoeconomic assessments The reasons for conducting a pharmacoeconomic trial include:
The Fundamentals 1. Pricing of a new drug;
Types of Cost 2. Repricing of an old drug;
Costs involved in pharmacoeconomic evaluation can be 3. Convincing a drug formulatory;
mainly divided into financial cost (mandatory cost) and 4. Generation of data for promotional material; and
economic cost (resource for which no mandatory payment is 5. Mandatory legislative requirement for drug licensing and
made) opportunity cost is the benefit foregone when selecting medical reimbursement (in some countries).
one therapy alternative over the next best alternative (Doubilet An ideal approach of economic evaluation in health café sector
et al 1986). Several costs can be measured when weighing up is given in Figure 5.
the cost of any invention. The first step in any cost analysis is
identification of the various costs. These can be direct, indirect
and intangible.
Direct i.e. costs from the perspective of the healthcare funder:
including staff costs, capital costs, drug acquisition costs. It
includes physicians’ fees, cost of administering the
medication, costs of treating an adverse drug reaction, etc.
Indirect i.e. costs from the perspective of society as a whole:
for example, these might include loss of earnings, loss of
productivity, loss of leisure time, due to the illness, and cost of
travel to hospital etc (Drummond et al 1997). This would
include not just the patient themselves but also their family
and society as a whole.
Intangible i.e. the pain, worry or other distress; which a
patient or their family might suffer. These may be impossible
to measure in monetary terms, but are sometimes captured in
measures of quality of life.
The cost can be measured in following ways:
Figure 5: An ideal approach of pharmacoeconomic evaluation
• Cost / unit
• Cost / treatment
Methods of Pharmacoeconomic Evaluation
• Cost / person
Pharmacoeconomic evaluations include any study designed to
• Cost / person / year
assess the costs (resources consumed) and consequences
• Cost / case prevented
(clinical, humanistic) of alternative therapies The evaluation
• Cost / life saved
mechanisms delineated were often helpful in demonstrating
• Cost / DALY (disability-adjusted life year)
the cost impact of innovative treatments, therefore granting
Outcomes
them greater acceptance by healthcare providers,
The second fundamental component of a pharmacoeconomic
administrators, and the public.
study is outcomes or benefits. A cost-benefit analysis
There are basically four types of pharmacoeconomic studies.
compares the costs and outcomes of alternative therapies and
the outcome is then expressed in monetary terms (Brien et al • Cost-minimization analysis (CMA)
1997). Cost-benefit analysis allows researchers to make • Cost-effectiveness analysis (CEA)
comparisons across a wide variety of alternatives. It compares • Cost-utility analysis (CUA)
the costs involved in implementing a programme with the • Cost-benefit analysis (CBA)
value of the outcome. Since the endpoints are measured in Cost-Minimization Analysis (CMA)
monetary terms, different endpoints can be studied, such as a Cost-minimization is a tool used in pharmacoeconomics and is
surgical procedure compared with a pharmaceutical applied when comparing multiple drugs of equal efficacy and
intervention equal tolerability. This is done when the outcomes are the
The expected benefits from an intervention might be same for the two interventions. In this, only the input, i.e. the
measured in: cost, is considered. The option that has the least cost is
A. “Natural” units e.g. years of life saved, strokes prevented, selected, e.g. if a hospital decides to introduce compulsory
peptic ulcers healed etc. prescribing of generic names of drugs instead of their brand
names, then the pharmacoeconomic evaluation of this would
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be done by CMA (Rawlins 1999). The objective of this method utility measurement used is a quality-adjusted life year
is to select the least costly among multiple equivalent (QALY) gained. QALY is a common measure of health status
interventions. It cannot be used to evaluate programmes or used in CUA, combining morbidity and mortality data. Results
therapies that lead to different outcomes. of CUA are also expressed in a ratio, a cost-utility ratio (C:U
CMA is a relatively straightforward and simple method for ratio). Most often this ratio is translated as the cost per QALY
comparing competing programs or treatment alternatives as gained or some other health-state utility measurement. The
long as the therapeutic equivalence of the alternatives being preferred treatment alternative is that with the lowest cost per
compared has been established. If no evidence exists to QALY (or other health-status utility). QALYs represent the
support this, then a more comprehensive method such as cost- number of full years at full health that are valued equivalently
effectiveness analysis should be employed. However, CMA to the number of years as experienced. For example, a full year
shows only a “cost savings” of one program or treatment over of health in a disease free patient would equal 1.0 QALY,
another. Employing CMA is appropriate when comparing two whereas a year spent with a specific disease might be valued
or more therapeutically equivalent agents or alternate dosing significantly lower, perhaps as 0.5 QALY, depending on the
regimens of the same agent (Tan et al 2006). disease (Eisenberg 1989). An example of QALY measurement
Cost Effective Analysis (CEA) for two different treatment approaches is given in Figure 6
CEA is a technique designed to assist a decision-maker in .
identifying a preferred choice among possible alternatives.
Generally, cost-effectiveness is defined as a series of analytical
and mathematical procedure that aid in the selection of a
course of action from various alternative approaches.
CEA evaluates multiple drug treatments for the same
condition. The cost of the drug treatments are weighed against
the effectiveness of the drug (Thwaits et al 1998). The costs of
drug treatments include acquisition costs, physician
involvement, and nursing costs for administration of the drug.
The effectiveness of drug treatment is measured in tangible
measures such as length of hospital stay, duration of treatment
required, and mortality rate. The results of a CEA are
expressed as cost/outcome for both therapies. Figure 6: Calculating QALYs - A simple example
Pharmacoeconomic analysis should be incorporated in the
clinical trial itself. However, for the majority of drugs, CEA is Cost Benefit Analysis (CBA)
done on the basis of pre-existing data available in the medical CBA is the most comprehensive and the most difficult of all
literature. economic evaluation techniques. In this technique, the benefits
CEA is the most commonly applied form of economic analysis are also assigned a monetary value so that costs and benefits
in the literature, and especially in drug therapy (Lee et al can be easily compared. Thus, totally different interventions
1991). It does not allow comparisons to be made between two can be compared, making it a useful tool (like CUA) for
totally different areas of medicine with different outcomes. resource allocation by policy-makers. It is a basic tool that
The key measure of these evaluations is the incremental cost allows for the identification, measurement, and comparison of
effectiveness ratio (ICER), which can be determined as the benefits and costs of a program or treatment alternative.
follows: CBA should be employed when comparing treatment
Cost of drug A - alternatives in which the costs and benefits do not occur
Cost of drug B simultaneously. CBA also can be used when comparing
Incremental cost effectiveness ratio= programs with different objectives because all benefits are
Benefits of drug A -
Benefits of drug B converted into dollars and to evaluate a single program or
Cost Utility Analysis (CUA) compare multiple programs.
CUA is a type of evaluation in which drugs/interventions with The benefits realized from a program or treatment alternative
different outcomes can be compared. CUA is the most are compared with the costs of providing it. Both the costs and
appropriate method to use when comparing programs and the benefits are measured and converted into equivalent dollars
treatment alternatives that are life extending with serious side in the year in which they will occur. Future costs and benefits
effects (e.g., cancer chemotherapy), those which produce are discounted or reduced to their current value. These costs
reductions in morbidity rather than mortality (e.g., medical and benefits are expressed as a ratio (a benefit-to-cost ratio), a
treatment of arthritis), and when HRQOL is the most net benefit, or a net cost (Bootman et al 2005). The most
important health outcome being examined (Bootman 1995). difficult and challenging part of CBA lies in calculating the
CUA is employed less frequently than other economic benefits in economic terms. Some benefits are easy to convert,
evaluation methods because of a lack of agreement on others need subjective judgment. CBA may ignore intangible
measuring utilities, difficulty comparing QALYs (quality benefits (pain, anxiety, stress) that are difficult to express in
adjusted life years) across patients and populations, and monetary terms (Freund et al 1992).
difficulty quantifying patient preferences. Analysis of Results in Pharmacoeconomic Evaluation
Pharmacoeconomists sometimes want to include a measure of Health economics, and particularly its branch precision health
patient preference or quality of life when comparing economics, help us choose between two alternatives, which we
competing treatment alternatives. CUA is a method for compare with regard to their clinical benefit on one hand and
comparing treatment alternatives that integrates patient their cost on the other. The users of pharmacoeconomic
preferences and HRQOL. CUA can compare cost, quality, and evaluations are not interested in the results themselves but in
the quantity of patient-years. Cost is measured in dollars, and where and how these results can help them for a better care of
therapeutic outcome is measured in patient-weighted utilities their patients (Barner et al 2003).
rather than in physical units (Hepler et al 1990).Often the
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Health benefits and costs can be accessed from a different pharmaceuticals and medical devices as viewed from the
viewpoint called "perspective". There are three common types standpoint of cost-effectiveness, the techniques are
of perspectives in economic studies: a producer's perspective, a applicable in a diversity of domains (Druglis et al 1989).
payer's perspective and a social perspective. The perspective of IV. Pharmacoeconomics provides precious input in studies of
an economic assessment is important. In general, the social product portfolios by pharmaceutical firms and
perspective is the broadest and most appropriate for making manufacturers of medical devices. Because QALYs may be
financial decision because it leads to optimal decisions but equated to pharmaceutical value per se, the aggregate
other perspectives are also valid (Blumenschein et al 1999). QALYs of a firm's products may be equated to its corporate
Consider the four possible results arising in a CEA given in value (Drummond at al 1997).
Fig.7. First, if costs are lower and health benefits higher for V. Healthcare practitioners, regardless of practice setting, can
one drug relative to another, the former is said to dominate and benefit from applying the principles and methods of
would be the preferred treatment. Second, the opposite applies, pharmacoeconomics to their daily practice settings.
i.e. the new drug is more expensive and less effective, and thus Applied pharmacoeconomics is defined as putting
is considered inferior and not recommended. The third and pharmacoeconomic principles, methods, and theories into
most common case is where the new drug is both more practice to quantify the value of pharmacy products and
effective and more expensive than the standard; on the basis of pharmaceutical care services used in real-world
ICERs, a judgment must be made regarding whether the environments (Cox 2003).Today’s practitioners
additional benefits are worth the extra costs of the new drug increasingly are required to justify the value of the products
and, therefore, whether it is ‘cost-effective’. This might be and services they provide. Applied pharmacoeconomics
defined by a previously agreed ICER threshold value (Thar can provide the means or tools for this valuation.
1992). The fourth case is similar to the third, with the roles of VI. Pharmacoeconomic analysis is important since payers such
the new therapy and the standard reversed; the question now is as third-party payers or government/private health plans
whether the extra benefits provided by the standard justify the utilise them when determining whether to reimburse a
additional costs of retaining it as the preferred treatment when claim. These results in decreased adherence, lack of
the option of a new, cheaper but less effective drug exists. prescription filling, decreased physician visits and
increased emergency room care (Sanchez 1994).
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