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Pediatric Clinics of North America - CIPERJ

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294 O’BRIEN<br />

contains the values an analyst believes are closest to the actual state <strong>of</strong> affairs.<br />

Analyzing a decision tree involves comparing the overall benefits expected<br />

from choosing each strategy, defined as the expected utility [22].<br />

For example, in Fig. 1, assume that Medication A has an 80% chance <strong>of</strong><br />

success and, therefore, a 20% chance <strong>of</strong> failure. If the medication succeeds,<br />

the patient has perfect health or a utility <strong>of</strong> 1. If the medication fails, the patient<br />

remains ill and has a utility <strong>of</strong> 0.7. The expected utility for this chance<br />

node is the sum <strong>of</strong> the product <strong>of</strong> each <strong>of</strong> the probabilities multiplied by its<br />

utility, or (0.8 1) þ (0.2 0.7) ¼ 0.94. This process is repeated for every<br />

chance node, moving from right to left. The process is called folding back or<br />

rolling back and typically performed using decision analysis s<strong>of</strong>tware. The<br />

expected utility can be measured using any scale an analyst chooses: number<br />

<strong>of</strong> blood transfusions, gain in life expectancy, or quality <strong>of</strong> life (utilities), as<br />

chosen in this example. Details on the rolling back process can be found in<br />

several reviews [10,23,24].<br />

The end result <strong>of</strong> rolling back is that each clinical strategy in the model is<br />

assigned a final value. In a decision analysis for which morbidity or mortality<br />

is the primary outcome measure, the strategy with the lowest value is the<br />

preferred option. If QALYs are the primary outcome measure, the strategy<br />

with the highest value is preferred. In a cost-effectiveness analysis (the most<br />

common type <strong>of</strong> decision analysis), the s<strong>of</strong>tware program presents each clinical<br />

strategy in ascending order by total cost and compares the strategies using<br />

an incremental cost-effectiveness ratio. This ratio is defined as the extra<br />

cost <strong>of</strong> a strategy divided by its extra clinical benefit as compared with the<br />

next least expensive strategy. Any strategy that costs more but is less effective<br />

than an alternative strategy is considered dominated and removed from<br />

further consideration. Although there is no absolute threshold for costeffectiveness,<br />

incremental cost-effectiveness ratios <strong>of</strong> less than $50,000 to<br />

$100,000 per healthy life year (QALY) gained typically are considered<br />

cost effective [25]. These proposed ratios, more than 20 years old, however,<br />

have not been adjusted for inflation and have not been considered independently<br />

for the pediatric population [26].<br />

In most decision analysis studies, there is some uncertainty about the inputs<br />

used in model construction. Sensitivity analysis, always performed after<br />

the base-case analysis, is an important tool for handling the uncertainty inherent<br />

in any decision analysis model and evaluates the effect <strong>of</strong> alternative<br />

assumptions on the final result. In this process, the probabilities, costs, and<br />

utilities <strong>of</strong> a model can be changed systematically, and the results <strong>of</strong> the<br />

analysis are recalculated multiple times. For example, in Fig. 1, the probability<br />

<strong>of</strong> treatment success with Medication A can be changed from 80% to<br />

50%, 90%, or any other number chosen by the analyst. If changing a variable<br />

over a reasonable range <strong>of</strong> values changes the preferred strategy, the<br />

model is considered sensitive to that variable. Typically, a model is sensitive<br />

to variation <strong>of</strong> some parameters and insensitive to variation <strong>of</strong> others. A<br />

model that is insensitive to variation <strong>of</strong> most parameters is a robust model.

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