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n a health care economy with limited resources, the provi-
Study Design. Narrative overview.
sion of service that yields the greatest health benefit at the
Objective. To provide clinicians with a basic understanding of
lowest cost is a priority. Informed decision making in value-
economic studies, including cost-benefit, cost-effectiveness, and
based care requires information on benefits and costs. Orga-
cost-utility analyses.
nizing, prioritizing, and analyzing the many variables that
Summary of Background Data. As decisions regarding public
must be considered in making common health care decisions
health policy, insurance reimbursement, and patient care incorporate
is difficult for even the most experienced clinician. Rather than
factors other than traditional outcomes such as satisfaction or
performing a rigorous formal analysis of the options and their
symptom resolution, health economic studies are increasingly
possible beneficial and detrimental outcomes, most caregivers
prominent in the literature. This trend will likely continue, and
rely on informal heuristics to make the many decisions they
it is therefore important for clinicians to have a fundamental
are required to make each day. Understanding health care
understanding of the common types of economic studies and be
economics analyses and methods may empower the clinician
able to read them critically. In this brief article, the basic concepts
to participate in value-based health care decision making and
of economic studies and the differences between cost-benefit, cost-
guide choices toward the provision of optimal health care.
effectiveness, and cost-utility studies are discussed.
A formal, logical process that incorporates complex data
Methods. An overview of the field of health economic analysis is
and models the clinical decision-making process can help the
presented.
clinician to understand the potential impact of different fac-
Results. Cost-benefit, cost-effectiveness, and cost-utility studies
tors in the likely outcomes of an intervention. Economic stud-
all integrate cost and outcome data into a decision analysis model.
ies, including cost-benefit analysis (CBA), cost-effectiveness
These different types of studies are distinguished mainly by the way
analysis (CEA), and cost-utility analysis (CUA) organize the
in which outcomes are valued. Obtaining accurate cost data is often
available clinical and economic data and can help under-
difficult and can limit the generalizability of a study.
stand possible answers to the increasingly relevant question:
Conclusion. With a basic understanding of health economic
“what intervention gives the greatest benefit proportional to
analysis, clinicians can be informed consumers of these important
its cost?”.1
studies.
The purpose of this article is to introduce the topic of
Key words: health economic analysis, cost-effectiveness analysis,
economic studies in health care. It is intended to introduce a
cost-benefit analysis, cost-utility analysis, discount rate, decision
reader to some of the key elements of economic studies and
analysis, quality-adjusted life year.
to spur further in-depth reading about the subject. It is hoped
Spine 2014;39:S9–S15
that after reading this article, one can understand the meth-
odology of health care economic analysis and approach eco-
From the *Department of Neurological Surgery, Columbia University College
nomic studies with a critical and discerning eye that will guide
of Physicians and Surgeons, New York, NY; and †Department of Orthopaedic optimal care.
Surgery, University of California, San Francisco, CA.
Acknowledgment date: April 18, 2014. Revision date: June 25, 2014. MEDICAL DECISION ANALYSIS
Acceptance date: July 9, 2014.
Medical decision analysis is a formal method of organizing
The manuscript submitted does not contain information about medical
device(s)/drug(s).
and connecting probabilities of events and their outcomes in
Supported by AO Spine North America, Inc. Analytic support for this work
a way that allows an assessment of the possible impacts of a
was provided by Spectrum Research, Inc., with funding from the AO Spine decision.2 The decision model is based upon an index health
North America. state and the probability of change with a specific interven-
Relevant financial activities outside the submitted work: board membership, tion. The resultant analysis enables the clinician, administra-
expert testimony, grants, payment for lectures, royalties, stocks.
tor, or policymaker to evaluate a probability distribution of
Address correspondence and reprint requests to Peter D. Angevine, MD,
MPH, Department of Neurological Surgery, Columbia University College of
expected outcomes and to understand the cost of the index
Physicians and Surgeons, 710 W 168th St, Room 510, New York City, NY decision on the total potential cost of care. The model is
10032; E-mail: pda9@columbia.edu based upon clinical, epidemiological, patient-centered, and
DOI: 10.1097/BRS.0000000000000576 economic data.
Spine www.spinejournal.com S9
Copyright © 2014 Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited.
Decision analyses, regardless of how apparently simple possible events that result from the decision. Events are
they are, involve many assumptions and choices, some of assumed to flow from left to right through the tree.
which may not be obvious. The most important of these is the Each branch exiting a chance node is assigned a probabil-
perspective of the analysis. With some decisions, only 1 per- ity. The branches are assumed to represent all possible out-
spective may be important or makes sense. On the contrary, comes at the point represented by the node. Consistent with
many problems, particularly those in public health, may have probability theory, the sum of all probabilities at each node
many possible perspectives, such as the individual, commu- must be 1, representing all possible outcomes. Although these
nity, insurance company, hospital, or state or national govern- are modeled as chance events, in reality one can model subse-
ment. For example, in the surgical management of adolescent quent decisions, also. For example, if recurrent radiculopathy
idiopathic scoliosis, the hospital perspective may be limited is modeled as a possible outcome after lumbar microdiscec-
to the episode of care, or the inpatient stay. The insurance tomy a decision tree could include a branch for reoperation.
perspective may be limited to the duration of time that adoles- The decision would be modeled as a simple probability based
cents are covered by the insurance of their parents. The patient on data regarding the proportion of patients who undergo
and the physician share a much longer perspective, and a time reoperation under similar circumstances.
frame that extends for a lifetime. The decision analysis model The probabilities used in a decision analysis are ideally
must be for a specific time frame. This is important both for derived from high-quality data sources. Peer-reviewed litera-
estimating costs and outcomes and for determining the type ture is the usual source for most probabilities used in decision
of model that is appropriate. The latter is an advanced topic analysis of health care problems. In some cases, data are not
that will not be discussed further here but is covered in several available in a form that is directly translatable to probabili-
of the references. ties in a decision tree. In other cases, such as the incidence
Medical decision making is complex and may encompass of complications after a procedure or adverse reactions to a
countless considerations for the patient, the surgeon, the hos- medication, published incidence data may be directly used in
pital, the insurance company, and for public health and social the decision tree.
welfare. Medical modeling involves choosing a finite number Finally, the decision tree is populated with outcomes and
of options and data elements and estimating the distribution cost data. The selection of the types of outcomes used in the
of outcomes based upon the best available data. Branching tree and the methods for determining costs are discussed
decision trees are the most common model used to organize briefly in the following text. For an economic analysis, each
and visualize the possible outcomes of a decision and their path through the tree is associated with a cost and with an
probabilities. A simple decision tree for the treatment of a outcome. A process of “rolling back” probabilities and out-
benign painful condition is shown in Figure 1. The decision comes or costs is used to analyze the tree. This is shown in
on whether or not to have the procedure is represented by Figures 2 and 3. Starting at the final branches, the outcome
the square “node.” The circular nodes represent subsequent value (or cost) of each branch is multiplied by the probability
S10 www.spinejournal.com October 2014
Copyright © 2014 Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited.
MEASURING COSTS
Costs are an important component of any cost-benefit, cost-
effectiveness, or cost-utility model. Estimating costs for the
model is complex, and may vary significantly depending on
the perspective of the stakeholder. The investigator must first
determine what costs should be included in the analysis. A
cost, in economic terms, is any use of a resource. For anal-
ysis, all costs must be in the same units, usually currency.
Time and materials are both types of costs and need to be
appropriately valued and incorporated into the decision
model. Costs include direct costs, which are quantifiable and
measurable, and indirect costs including the costs of keeping
lights on and full staffing in a hospital. Some of the most
significant costs of illness and care include lost productivity
and wages, burden on families, care provider costs, and non-
medical costs. Therefore, direct costs alone underestimate the
Figure 3. The model and analysis for example cost data. These results total cost of an illness. Costs in economic analyses are dis-
and those from Figure 2 are combined in the final base-case analysis tinct from charges. Retail prices are not the same as economic
shown in Table 1.
costs. In health care analyses, this is frequently relevant when
determining hospital costs associated with care delivery.
Hospital charges include markups over what was paid for
most useful for procedures in which the intended outcome materials or services and, in general should not be used in
of care is an improvement of quality of life. Most discre- economic analyses because they are not directly translatable
tionary spine procedures in adults are amenable to evalua- to direct costs. Instead, the investigator should determine or
tion using the cost per unit of improvement health status, or estimate the true cost of the service, material, or procedure.
utility. CUA is less useful in procedures in which the primary Direct costs are not transparent and hospitals and manufac-
goal of care is to avoid the consequences of disease progres- turers are limited in complete disclosure of cost information.
sion, or to prolong survival. Specifically, CUA in adolescent In some models, the reimbursement from Medicare or other
idiopathic scoliosis may underestimate the value of care in payers may be used as an estimate of cost, but this method-
the patient with limited preoperative health compromise. The ology is limited because the contribution margin (revenue-
value of care in adolescent idiopathic scoliosis is measured direct cost) may be highly variable between procedures and
by the value of avoiding the expected consequences of defor- payers. Published literature may also be a useful resource for
mity progression. Disability-adjusted life year estimation may estimating cost data.
be most useful for preventative interventions. Similarly, an Costs for similar services or materials can vary significantly
en bloc excision of an asymptomatic chordoma of the sacrum between institutions and geographical regions. Bederman
may improve survival, but would not be expected to improve et al10 demonstrated 8-fold variation in the costs of implants
patient preference for health status as a result of care. There- and in total costs of care for common spine procedures. The
fore, CUA studies may be most appropriate for conditions expected audience for the analysis may also be an important
in which the goal of care is improvement of patient reported consideration in deciding whether the variation is important.
health status. Sensitivity analysis can be used to determine the effect of
As with cost estimates, health states that occur in the future changes in cost estimates on the overall results of the study. A
should be discounted. Discounting future costs without dis- final consideration that is relevant to perspective is the neces-
counting future outcomes will result in erroneous results. sity of discounting future costs (and benefits) that occur in
Conventionally, outcomes (and costs) that occur more than the medium term to long term. Generally, it is appropriate to
1 year in the future are discounted. The same discount rate discount costs that occur more than 1 year in the future; in
should be used for costs and outcomes. most situations, the present value of a dollar is greater than
interventions are best understood as estimates based on a spe- hospitals, and health care systems, insurance companies, and
cific model and data that should, similar to other economic governmental policy makers. A single economic study should
analyses, be tested and updated as new information becomes not be interpreted as a definitive demonstration of the cost-
available.17 The robustness of the results of a CUA or other effectiveness of an intervention, but CEA, CBA, and CUA
economic analysis should be thoroughly tested. This sensitiv- that are designed well and conducted can serve as a helpful
ity analysis gives the investigator and the reader an under- decision aid for its intended audience. Physicians and other
standing of the likelihood that the base case results accurately providers, who have a thorough understanding of the clinical
represent the underlying reality. Figures 4 and 2A, B demon- conditions and interventions, are critical to the proper design,
strate examples of probability distributions of outcomes for conduct, and interpretation of economic analyses.
operative and nonoperative care. Using these probability dis- In a health care economy with limited resources, value is
tributions, the investigator can model the likelihood of spe- the priority of health care decisions.18 Informed choice in a
cific outcomes, and the cost of reaching that outcome. If, for value-based health care economy is characteristically made
example, altering cost, outcomes, and probability estimates with incomplete information. Decision analysis is the pro-
for important variables within plausible ranges does not sig- cess of making calculated decisions under circumstances of
nificantly change the results, one can be reasonably assured uncertainty. Medical decision modeling uses the best available
that the model will apply to a fairly wide set of circumstances. data to guide informed choice. The literature on cost analysis
On the contrary, if a relatively small change in one estimate in spine interventions remains limited, and the introduction
dramatically alters the results the investigator or reader will and adoption of new techniques and technologies outpace the
likely want to ensure that estimates of that variable are as studies of value and cost analysis. Reimbursement, coverage
accurate as possible. At the same time, neither the robust- levels, and access to care for patients with spinal disorders are
ness of the model nor its absence indicates the accuracy of dependent upon demonstration of value of specific interven-
the model itself, which should be tested thoroughly against tions. Future research using the techniques reviewed in this
confirmatory data from other sources. article is a priority for guiding an evidence-based approach to
Health economic analyses, similar to randomized clinical spine care in a value-based health care economy.
trials, are most useful at a population level. An individual
patient may have significantly different risks, costs, or values
than the average values used in an overall analysis. Eliciting
these differences may be difficult and is not generally part of
a clinical encounter. A strongly robust model may, however, ➢ Key Points
reassure the patient and clinician that the results are likely to
Health economic analyses integrate cost data
apply in a particular circumstance.
with clinical outcomes data to estimate the
economic impact associated with any gain in
CONCLUSION outcomes comparing one treatment option with
Economic analyses of health interventions are an impor- another.
tant tool for many audiences, including patients, physicians,
S14 www.spinejournal.com October 2014
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