Pediatric Clinics of North America - CIPERJ
Pediatric Clinics of North America - CIPERJ
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.