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Decision analysis
Authors: Mark S Roberts, MD, MPP, Joel Tsevat, MD, MPH
Section Editor: Mark D Aronson, MD
Deputy Editor: Carrie Armsby, MD, MPH

All topics are updated as new evidence becomes available and our peer review process is complete.

Literature review current through: Apr 2022. | This topic last updated: Oct 30, 2019.

INTRODUCTION

Decision analysis is a quantitative evaluation of the outcomes that result from a set of choices in a specific clinical situation.
With the exception of the word quantitative, this definition is no different from the clinical decision-making process conducted
by clinicians every day. When faced with a particular problem, clinicians develop an array of possible actions ranging from doing
nothing, to obtaining more information by performing diagnostic tests, to recommending various therapeutic strategies. This
process is often implicit and occurs in the context of internal algorithms and heuristics (mental shortcuts) that the clinician has
developed and acquired over time. Decision analysis, by requiring a specific model structure and assessment of the various
likelihoods and values of the outcomes, makes the decision process explicit and much more amenable to examination,
discussion, and intellectual challenge.

Decision models are often used as an analytic tool to conduct cost-effectiveness analyses since decision analysis methodology
can be used to find the expected value of most any outcome. Cost-effectiveness analysis is discussed separately. (See "A short
primer on cost-effectiveness analysis".)

TYPES OF PROBLEMS APPROPRIATE FOR DECISION ANALYSIS


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The range of clinical problems appropriate for decision analysis is vast. The two major requirements for its use include:

● The problem focuses upon a specific decision that must be made


● There is a tradeoff involved in the decision

"Tradeoff" means that one of the choices considered should not be unambiguously superior. As an example, a diagnostic test
might carry some risk, but the tradeoff is more appropriate therapy when treatment is directed by the results of that test.

The scope of the problem should be manageable. Asking "Should patients with elevated cholesterol levels be treated with
statins?" poses a question so broad and complex that it is unanswerable. In contrast, a question such as, "What is the benefit of
treating high cholesterol levels in patients with varying 10-year risk of developing symptomatic cardiovascular disease?" is
sufficiently specific to be amenable to decision analysis [1]. This specific question illustrates another characteristic of problems
appropriate for decision analysis: there should be debate or uncertainty regarding the best choice. It is unlikely that decision
analysis would be useful if it addresses the exact question already answered definitively by randomized controlled trials.

Questions relating to clinical decisions that do not have direct answers in the literature are remarkably common. One may be
faced with a patient who is significantly older or has more comorbidities than the study population in a published trial or who
has a set of unique attributes that make the direct application of results from a published study problematic. In general,
decision analyses have been developed to:

● Assist in clinical decision-making for a specific individual patient


● Estimate optimal strategies for classes of patients with specific clinical characteristics in given situations
● Link estimates of both clinical and economic outcomes (cost-effectiveness analysis) to help inform health policy questions
● Provide estimates of expected outcomes in situations where classic methods such as randomized trials are either
impossible or impractical

There are several advantages of using decision analysis to investigate options involving a single patient [2]. Placing the problem
in an explicit, analytic context forces clinicians to make their assumptions clear, and the presence of a structured model rapidly
focuses clinical disagreements. Decision analyses can directly incorporate issues regarding quality of life and how the particular
patient values various outcomes. As an example, if possible death in the near-term is the trade-off for a potential cure, then

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maximizing life expectancy may not be the most important outcome for a terminally ill patient who wants to live to witness a
grandchild's graduation from college several months hence. Such individual patient preferences can be incorporated directly
into decision analyses by altering the value of the various outcomes.

Decision analyses are performed more commonly to evaluate the appropriate strategy for a class of patients identified by a set
of clinical characteristics. Often this will involve combining data from a variety of different sources to provide a comprehensive
analysis of the problem. An example of such a decision analysis is an analysis weighing the risks and benefits of prophylactic
mastectomy and oophorectomy for patients with BRCA1 or BRCA2 mutations [3]. The model combined several kinds of data,
including baseline cancer incidence, the risks of the various procedures, and the estimates of the increased risks of ovarian and
breast cancer for women with the mutations. The analysis demonstrated, using the best available data at the time, that life
expectancy for women who carry those mutations was increased by as much as 5.3 years with prophylactic mastectomy and by
as much as 1.7 years with prophylactic oophorectomy.

Decision analysis can also be used to provide estimates of risks and benefits for groups of patients with characteristics
somewhat different from patients studied in previous randomized controlled trials. This application was exemplified by
Krumholz and coworkers, who constructed a decision model of thrombolytic therapy for older adult patients with myocardial
infarction (see 'Example' below) [4]. Their paper also illustrates how a decision analysis of a clinical problem can be blended with
economic outcomes to produce a cost-effectiveness analysis. (See "A short primer on cost-effectiveness analysis".)

CONDUCTING A DECISION ANALYSIS OF A SPECIFIC PROBLEM

Developing and constructing a decision analysis follows a logical series of steps. Problems or errors in any step can alter the
eventual results; thus, proper adherence to each of these steps is important both from the view of a researcher conducting an
analysis and a clinician interpreting the results of a published study. Throughout the remainder of this topic review, the basic
techniques of decision analysis are described in a primer-based style, with no assumption of prior knowledge or experience. In
addition, we have annotated and explained the decision analysis by Krumholz and colleagues in an example below, which is
linked at each step to illustrate how these methods were applied in a published decision analysis [4]. For a more detailed
introduction to decision analysis, the reader is referred to a series of introductory articles in Medical Decision Making [5-9].

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Step 1: Frame the question — The process of defining the scope and boundaries of the particular clinical situation to be
analyzed is similar to developing selection criteria for a randomized controlled trial. The purpose is to define the particular
population of patients, conditions, and intervention strategies that are appropriate to the analysis.

One of the most important decisions to be made in a decision analysis is the perspective from which the analysis is to be
conducted. As an example, a decision analysis conducted from the point of view of an individual patient may look different
when analyzed from society's point of view, where secondary effects on others in the population (eg, from transmission of an
infectious disease) would need to be included. Examples of multiple perspectives can be found in decision analyses of the
decision to use preventive therapy in patients with latent tuberculosis infection [10,11].

The time horizon of the analysis needs to be specified and be consistent with the clinical reality of the condition. A time horizon
of one month, for example, would be inappropriate for a decision analysis of a cholesterol lowering medication since gains in
survival may not be realized for years. On the other hand, a decision analysis of different treatments for urinary tract infection
might not need to consider outcomes beyond one or two months after treatment. In general, the time frame should match the
natural history of the disease process.

Step 2: Structure the clinical problem — Structuring the problem simply means constructing a decision model that represents
the relevant components of the problem. The mathematical representation of a decision model is called a decision tree and is
composed of several discrete elements. Elements combined into trees contain an arbitrary amount of detail. Virtually any
decision can be modeled, but it is important that the choices considered are realistic. As an example, a decision analysis
examining bypass surgery versus medical therapy but ignoring percutaneous coronary intervention might be inappropriate.

There is constant tension between increasing the level of detail to be as clinically realistic as possible versus the feasibility of
model construction, validation, and presentation [12]. The more detail desired, the more difficult the model is to construct,
validate, and present. However, the less detailed the analysis and the greater the number of simplifying assumptions, the more
vulnerable is the analysis to attack for lacking clinically meaningful elements.

All decision trees begin with a decision node, which represents the decision to be made ( figure 1 and figure 2).

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● Figure 1 illustrates a decision with two options, Choice 1 and Choice 2, representing a generic clinical decision. Such a
decision could represent the choice between medical and surgical therapy for a particular condition; empiric treatment for
a disease versus diagnostic testing; or different follow-up intervals after resection of a cancer.

● Following a decision node is one or more chance nodes. In this example, Outcome 1 and Outcome 2 are possible
consequences of Choice 1, and Outcome 3 and Outcome 4 are possible consequences of Choice 2. Each branch of a
chance node is characterized by a probability between 0.0 and 1.0 (p1 through p4), which represents the likelihood that
the particular event will occur. The sum of all probabilities at a chance node must equal 1.0 (100 percent).

● At the end of each branch is a terminal node, which represents a state of health or outcome that results from traversing a
particular path through the tree. As an example, if Choice 1 represents a surgical intervention, Outcome 1 might be
operative mortality, and therefore Value 1 (or V1, which might represent life expectancy) would be the value assigned to
immediate death, which is by convention 0.0.

See below for a more detailed example.

Step 3: Estimate the relevant probabilities — Once a decision tree has been structured, the numeric values of the various
probabilities need to be determined. There are many sources of data that can be used to make these determinations. Although
there is a generally accepted hierarchy of study quality [13], such ratings are not always useful for decision analysis since the
particular study type may not be conducive to estimating a given parameter. As an example, a randomized controlled trial is an
excellent source for comparing one therapy versus another, but it is a poor source of data regarding the incidence of a
particular disease. Thus, it is important to tailor the data source to the type of data required ( table 1).

It is rarely the case that all of the data needed to parameterize a decision model can be found in a single study. If that were
true, it would be likely that the question being asked had already been answered.

The sources used to estimate probabilities need to take into consideration potential differences in characteristics of populations
between published trials and the population of interest to the decision analyst. As an example, carotid endarterectomy was
found to be beneficial in patients with asymptomatic carotid stenosis in a study in which the operative mortality rate was 3.5
percent [14]. The local rate should be used in the analysis if it is different from the published rate.

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Step 4: Estimate the values of the outcomes — The structure of the problem defines the specific outcome measure to be
used. As noted above, life expectancy is not likely to be a relevant outcome for an analysis of different treatment strategies for
urinary tract infection. In contrast, if death is a possible outcome of one or more strategies, life expectancy would be an
appropriate outcome measure. The most important aspect in assigning outcomes is that they be measured in the same units
across all branches.

A useful feature of decision analysis is that a given model can be evaluated using different outcome measures. As an example,
in addition to survival, the investigator may want to track the effect of different therapies upon the rate of stroke, myocardial
infarction, pulmonary embolization, etc, across various treatment options. The decision model can be analyzed using any of
those outcomes.

It is intuitively obvious that a year of life in full health is not the same as a year with severe angina or a year of life following a
stroke or an amputation. Patients place different values on those health states and are willing to accept risks (undergo
operations, take medications with side effects) to avoid them. In decision analyses, one can consider quality of life and length of
life simultaneously.

The important attribute of quality of life measures for decision models is that they allow quantitative comparisons among
different health states. In other words, quality of life adjustments for decision models must be able to make comparisons of the
form: "One year of life with a stroke is worth X percent of a year of life in full health." When measured in this manner, decision
analyses produce estimates of quality-adjusted life years (QALYs). These methods are summarized in a classic review [15].
Methods to incorporate quality of life are available [16].

Step 5: Analyze the tree: The mechanics of the analysis — The "answer" to a decision analysis problem is the strategy that
maximizes the expected value of the outcome. As an example, if the outcome of interest were life expectancy, the result of a
decision analysis would be of the form: "the average life expectancy with strategy A is 10.2 years versus 9.8 years with strategy
B; therefore strategy A is the optimal strategy."

These "expected values" or "expected utilities" are determined by recursive evaluation of the tree (known as averaging out and
folding back) from right to left, replacing each chance node with the expected value of the combination of branches that arise

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from that chance node. The value of each branch at a decision node is the arithmetic average of the branches that follow. For
the simple tree in the figure ( figure 1):

● Expected value of Choice 1 = (p1*V1) + (p2*V2)


● Expected value of Choice 2 = (p3*V3) + (p4*V4)

More concretely, the figure illustrates the calculation for hypothetical probabilities and life expectancies ( figure 3). Such a
tree might represent the choice between two therapies, one with little difference between its possible outcomes (Choice 1) and
the other offering a chance of longer life at the risk of a shorter life (Choice 2). In this example, Choice 2 is the preferred
strategy in terms of life expectancy.

Step 6: Test the model's assumptions: Sensitivity analysis — The results obtained from a decision analysis depend upon the
accuracy of the data used to estimate the probabilities and outcomes. It is rarely the case that estimates are known with
complete certainty; even in data from very large population-based studies, estimates of mortality and effectiveness are couched
in confidence limits.

One of the major advantages of decision analysis models is their ability to rapidly test their assumptions and input data. As an
example, in referring to the hypothetical example in Figure 2, suppose the life expectancy estimate of 10 years for patients who
experienced Outcome 1 came from a relatively small study, and the confidence limits on life expectancy ranged from 8 to 12
years. That range could be used to recalculate the model several times in order to examine whether such differences would
change which strategy was optimal ( table 2).

The table represents a one-way sensitivity analysis since it varies the value of only a single variable ( table 2). More than one
parameter can be varied, producing two-, three-, and multiway sensitivity analyses that relate the expected value of the choices
to simultaneous variations in the values of several variables.

Sensitivity analysis is also a helpful tool in constructing and validating decision models. The model's answer can be compared
with the "true" answer to validate the model structure by evaluating a tree using parameters for which the result is known a
priori. As an example, in a choice between a more effective (but riskier) surgical therapy and a less effective (but safer) medical
therapy, a sensitivity analysis that postulated a zero mortality rate for the surgical intervention should advocate the surgical

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arm since there would be no downside risk. If the model did not indicate that the surgical arm was preferred, an error or "bug"
in the model would likely be present.

Step 7: Interpret the results — Interpreting the results of a decision analysis is often the most complicated task. Unlike other
experimental designs or study types, it is often not the explicit result from a decision analysis that becomes its most important
contribution to decision-making. Rather, the ability of a decision analysis to explore how the optimal strategy in a particular
clinical situation changes with variation in assumptions (and therefore to identify areas for further data needs) is often one of
the most powerful attributes of this type of analysis.

Several conditions must be met before the results can be incorporated into day-to-day practice [17,18]:

● Relevant competing strategies must be included


● All clinically relevant outcomes given those strategies must be described
● The data used for calibration (both probabilities and outcomes) must be acquired and summarized in a clear and
reasonable manner
● Appropriate sensitivity analyses must be performed.

Furthermore, the consumer of a decision analysis should examine several details of an analysis prior to using the results to
change practice or policy.

● The patient population should closely match the patients seen by the clinician. A decision analysis exploring coronary
artery bypass graft versus percutaneous coronary intervention in young male patients with chronic stable angina probably
will not inform the same decision as in older adult women with crescendo angina.

● The reader needs to assess the strength of the result. Decision analyses do not contain statistical indicators such as p-
values. Thus, one relies on the sensitivity analysis to indicate whether outcomes change over appropriate ranges of
relevant variables. If sensitivity analyses indicate that the optimal choice is strongly dependent upon a given parameter,
one should try to develop accurate measures of the parameter estimate.

EXAMPLE
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The issue of thrombolytic therapy in older adult patients with myocardial infarction (MI) provides a good example of how
decision analysis can be used in clinical practice.

Step 1: Frame the question — Clinical trials of thrombolytic agents in acute MI have demonstrated benefit in the patient
populations studied, but the evidence of their benefit in older adults has been mixed. Noting that no trial had directly
addressed this issue, one group performed a decision analysis to address the problem [4]. Their approach represents a
common use of decision analysis: extending tried and true therapies into populations somewhat dissimilar from those initially
studied.

The thrombolysis issue has several characteristics that make it appropriate for decision analysis:

● There is a specific choice (to give thrombolytic therapy or not)


● There are accurate data on the characteristics and outcomes of MIs in older adults
● There is strong evidence of efficacy in certain study populations

The following paragraphs describe the decision analysis of Krumholz and colleagues [4]. The published version of this analysis
did not include a figure of the decision model, but we present the model so that the reader may follow and replicate the
analysis.

Step 2: Structure the clinical problem — The decision tree models two mutually exclusive strategies: whether to give
thrombolytic therapy to an older adult patient with acute MI or provide only supportive care ( figure 4).

● If thrombolysis is chosen, the patient may experience a complication of therapy (eg, cerebral hemorrhage). In this analysis,
complications of thrombolysis are assumed to be equivalent to death. However, because a cerebral hemorrhage may not
always be tantamount to death, their assumption biases the analysis against thrombolysis by making the complications
worse than they might actually be.

● The diagnostic criteria for MI are not perfect. Thus, some patients given thrombolytics have actually not had an MI (false
positives). For this reason, a chance node representing actual MI versus no MI follows the complication/no complication
node.

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● The patient may die or survive whether or not she has an MI, although the likelihood of death will be much higher with MI.

The no thrombolysis strategy looks the same structurally except for the absence of the complication/no complication branch,
since no drug is administered.

Step 3: Estimate the relevant probabilities — The structure of the tree and the number of branches at chance nodes define
the probabilities required to "parameterize" the tree. In this model, estimates of the probability of MI, the mortality rates with
and without infarction, and the efficacy and risks of thrombolytic therapy are required ( table 3). Most of the data for this
analysis came from published trials including the Groupo Italiano per lo Studio della Streptochinasi nell'Infarcto Miocardio
(GISSI [19]) and the Second International Study of Infarct Survival (ISIS-2 [20]) trials. Other data were collected from the
literature. The estimated probabilities used in their analysis are growing older, but this represents one of the benefits of
developing a model: The parameters can be updated with more current information as it becomes available.

Step 4: Estimate the values of the outcomes — The values at the end of each terminal node for the basic analysis are simply
the value of surviving (set to 1) or dying (0) in the hospital. The expected value of each branch will be the average probability of
in-hospital survival for a cohort of patients treated under that strategy (thrombolysis or no thrombolysis).

The authors also needed to estimate the number of years of life saved and the costs of the interventions for the purposes of
also conducting a cost-effectiveness analysis (beyond the scope of this topic review) (see "A short primer on cost-effectiveness
analysis"). They obtained these values from a cardiovascular risk prediction model, the Coronary Heart Disease Policy Model
[21]. As an example, they estimated that a 70-year-old patient would have a life expectancy of 5.5 years after MI. For calculating
average life expectancy (rather than probability of survival) under each strategy, the specific life expectancies for each outcome
would be inserted in their appropriate terminal nodes, with the value of death remaining at zero.

Step 5: Analyze the tree (average out/fold back) — The tree is analyzed using the standard averaging-out and folding back
approach described above. The tree is folded back from right to left, and the average value at each chance node is calculated (
figure 5). Given the baseline assumptions provided in Figure 4, the expected value of complication-free survival is 0.7864 for
the thrombolytic arm versus 0.7559 for the standard therapy strategy.

The results can also be presented in two other forms:

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● The number needed to treat to save one additional life


● Life expectancy

In order to calculate life expectancies, one would need to know the life expectancy after MI for a patient of a given age,
estimated in this study to be 5.5 years for a 70-year-old male [4]. Using standard life expectancy tables for survival without MI,
the relevant life expectancies could be entered as the values of the terminal node, and the analysis repeated to find the
expected life expectancy for each strategy.

Step 6: Test assumptions (sensitivity analysis) — We have provided a simple one-way sensitivity analysis as an introduction,
changing only the probability of a fatal complication of thrombolytic therapy over a reasonable range of values (0 to 6 percent).
This can be seen in a figure ( figure 6). As expected, the probability of surviving under the thrombolysis strategy decreases as
the risk of fatal complication rises. At a probability of fatal complication greater than 0.042 (the so-called "threshold"), the
thrombolytic strategy becomes inferior to the no-thrombolytic strategy.

The published paper presents several examples of two-way sensitivity analyses, where two variables (eg, probability of MI and
effectiveness of thrombolytic therapy) are varied simultaneously to determine when thrombolytic therapy is beneficial [4]. All of
the analyses demonstrated that for reasonable ranges of values surrounding their baseline estimates, thrombolysis remained
the preferred strategy.

Step 7: Interpret the results — This analysis has most of the features of a sound decision analysis. For patients presenting
with suspected MI, the model presents two options: thrombolysis versus no thrombolysis. One could question why primary
percutaneous coronary intervention was not considered, as this option has been included in many of the MI interventional
studies. The data used to calibrate the model come from several large trials and the outcomes modeled were reasonable,
although one could have modeled minor complications as well. Several sensitivity analyses were performed, all indicating that
the results were quite robust.

EXTENSIONS

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Simple node and branch decision trees can be used to develop models of arbitrary complexity, but the methodology is not
particularly well suited to modeling events that occur repetitively over time, or to analyzing interventions that alter the risk of
future events. Such problems are more appropriately structured using methods that explicitly include the element of time. One
such technique is the Markov process. Markov models are an extremely powerful complement to decision analysis for
expanding the types and complexity of clinical situations that can be effectively analyzed [22]. Complex models of underlying
physiologic processes have been incorporated into decision models, allowing a clinically realistic representation of the disease
process [23].

Decision models are often used as the analytic "engine" to conduct cost-effectiveness analyses since decision analysis
methodology can be used to find the expected value of most any outcome. Cost-effectiveness analysis is a methodology that
examines the simultaneous effect of different strategies upon clinical and economic outcomes (see "A short primer on cost-
effectiveness analysis"). By including both costs and clinical effects in one model, analyses can provide estimates of the cost per
year of life saved, the cost per quality-adjusted life year saved, etc.

Two professional societies, the Society for Medical Decision Making and the Professional Society for Health Economics and
Outcomes Research, developed a series of position papers outlining good research practices for using decision models to
address problems in health care. The papers provide an overview of decision modeling [24]; recommendations for
conceptualizing a decision problem and a model [25]; methods for constructing state transition models [26], discrete event
simulation models [27], and dynamic transmission models [28]; methods to estimate the values of model parameters and
include uncertainty [29]; and a description of methods to validate models [30]. Although not a primer, they are an excellent
resource for the appropriate use of modeling methodologies.

SUMMARY

● Decision analysis is a quantitative evaluation of the outcomes that result from a set of choices in a specific clinical
situation. With the exception of the word quantitative, this definition is no different from the clinical decision-making
process conducted by clinicians every day. (See 'Introduction' above.)

● The range of clinical problems appropriate for decision analysis is vast. The two major requirements for its use include:
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• The problem focuses upon a specific decision that must be made


• There is a tradeoff involved in the decision

(See 'Types of problems appropriate for decision analysis' above.)

● Decision analysis is performed using estimates of probabilities of events and values placed on outcomes. These outcomes
may include patient values such as quality-adjusted life years or rates of events such as death. (See 'Conducting a decision
analysis of a specific problem' above.)

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19. Effectiveness of intravenous thrombolytic treatment in acute myocardial infarction. Gruppo Italiano per lo Studio della
Streptochinasi nell'Infarto Miocardico (GISSI). Lancet 1986; 1:397.

20. Randomised trial of intravenous streptokinase, oral aspirin, both, or neither among 17,187 cases of suspected acute
myocardial infarction: ISIS-2. ISIS-2 (Second International Study of Infarct Survival) Collaborative Group. Lancet 1988; 2:349.

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21. Weinstein MC, Coxson PG, Williams LW, et al. Forecasting coronary heart disease incidence, mortality, and cost: the
Coronary Heart Disease Policy Model. Am J Public Health 1987; 77:1417.

22. Sonnenberg FA, Beck JR. Markov models in medical decision making: a practical guide. Med Decis Making 1993; 13:322.
23. Eddy DM, Schlessinger L, Kahn R. Clinical outcomes and cost-effectiveness of strategies for managing people at high risk
for diabetes. Ann Intern Med 2005; 143:251.
24. Caro JJ, Briggs AH, Siebert U, et al. Modeling good research practices--overview: a report of the ISPOR-SMDM Modeling
Good Research Practices Task Force-1. Med Decis Making 2012; 32:667.

25. Roberts M, Russell LB, Paltiel AD, et al. Conceptualizing a model: a report of the ISPOR-SMDM Modeling Good Research
Practices Task Force-2. Med Decis Making 2012; 32:678.

26. Siebert U, Alagoz O, Bayoumi AM, et al. State-transition modeling: a report of the ISPOR-SMDM Modeling Good Research
Practices Task Force-3. Med Decis Making 2012; 32:690.
27. Karnon J, Stahl J, Brennan A, et al. Modeling using discrete event simulation: a report of the ISPOR-SMDM Modeling Good
Research Practices Task Force-4. Med Decis Making 2012; 32:701.
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Practices Task Force Working Group-5. Med Decis Making 2012; 32:712.
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SMDM Modeling Good Research Practices Task Force Working Group-6. Med Decis Making 2012; 32:722.

30. Eddy DM, Hollingworth W, Caro JJ, et al. Model transparency and validation: a report of the ISPOR-SMDM Modeling Good
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GRAPHICS

Decision tree

A decision tree starts with a decision node (square), which


represents the decision to be made, followed by chance nodes
(circles), which represent the possible events given the choices. Each
outcome is associated with a probability (p1 through p4) that
represents its likelihood of occurring. Each branch eventually ends in
a terminal node or outcome (boxes) representing the value of that
outcome.

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Decision analysis elements

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Sources of data for a decision analysis problem

Sources of data Comments

Randomized Excellent source of data on effects of therapies. Often the study requirements (entry and exclusion criteria) will
controlled trials make the results of the trial applicable to only a narrow range of patients.
(RCTs)

Cohort studies Useful for risk factor determination, natural history.

Administrative Excellent sources for broad population-based estimates of disease; often more accurate as estimate of
databases effectiveness for various procedures than RCTs, which, in general, estimate efficacy. However, it is difficult to be
sure that confounding variables are controlled for.

Electronic health Provides additional detail above what administrative databases are able to provide. Very useful for determining the
records (EHRs) distribution of a particular variable in different diseases.

Meta- Provides summary measures over several different studies for a particular parameter. Generally restricted to RCTs,
analyses/systematic so often global population data are not well-represented.
reviews

Expert opinions Often plagued by biases and dependent upon the method used to assess opinion. Most closely resembles the
methods used to make clinical decisions in current clinical practice (use of consultants, etc); therefore, the use of
expert opinion has face validity.

Investigator A last resort, if there are no other good sources of data. When combined with appropriate sensitivity analysis, can
estimates be a useful first start.

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Evaluation of a simple decision tree

The expected value of any node is simply the probability-weighted


average of the outcomes across all of the branches of the node. This
is calculated by taking the sum of each branch's value times its
probability. For Choice 1 above, the average life expectancy of a
cohort of patients undergoing that therapy would consist of the
combination of 45 percent who lived 10 years and 55 percent who
lived 12 years, for an average of 11.1 years.

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Sensitivity analysis on the life expectancy of Outcome 1

Value of V1 (life expectancy of Expected value of Expected value of Preferred


Outcome 1) Choice 1 Choice 2 strategy

8.0 10.2 11.5 Choice 2

9.0 10.7 11.5 Choice 2

10.0 11.1 11.5 Choice 2

11.0 11.6 11.5 Choice 1

12.0 12.0 11.5 Choice 1

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Decision tree for thrombolysis in the elderly


(example data for decision analysis)

MI: myocardial infarction.

Created with data from: Krumholz HM, Pasternak RC, Weinstein MC, et al, N Engl J
Med 1992; 327:7.

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Baseline probabilities for thrombolytic decision tree (example for decision analysis)

For illustration purposes only

Parameter Percent

Probability that the patient is having a myocardial infarction (MI) 83

If the patient is having an MI, probability of death 29

If the patient is not having an MI, probability of death 2

Probability of a patient having a severe or fatal complication of thrombolytic therapy 0.40

In a patient having an MI, relative reduction in mortality from treatment with thrombolytic therapy 13

Data from: Krumholz HM, Pasternak RC, Weinstein MC, et al, N Engl J Med 1992; 327:7.

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Decision tree for thrombolysis in the elderly


(example data for decision analysis)

Each chance node is replaced by its expected value: the arithmetic


average of the value of its branches. As an example, the expected
value of the MI branch of No complication is 0.7506 [(0.7506 x 1) +
(0.2494 x 0)].

MI: myocardial infarction.

Created with data from: Krumholz HM, Pasternak RC, Weinstein MC, et al, N Engl J
Med 1992; 327:7.

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One-way sensitivity analysis (example for decision


analysis)

One-way sensitivity analysis of the probability of a complication from


thrombolytic therapy. As the likelihood of a complication (x-axis)
rises, the expected value of the thrombolysis strategy declines. The
point at which the probability of survival is equivalent between the
two strategies is called the threshold.

Data from: Krumholz HM, Pasternak RC, Weinstein MC, et al. N Engl J Med 1992;
327:7.

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Contributor Disclosures
Mark S Roberts, MD, MPP No relevant financial relationship(s) with ineligible companies to disclose. Joel Tsevat, MD, MPH No relevant
financial relationship(s) with ineligible companies to disclose. Mark D Aronson, MD No relevant financial relationship(s) with ineligible
companies to disclose. Carrie Armsby, MD, MPH No relevant financial relationship(s) with ineligible companies to disclose.

Contributor disclosures are reviewed for conflicts of interest by the editorial group. When found, these are addressed by vetting through a
multi-level review process, and through requirements for references to be provided to support the content. Appropriately referenced content
is required of all authors and must conform to UpToDate standards of evidence.

Conflict of interest policy

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