Question

Difficulty: EasyMeasures of Association and Impact

A randomized controlled trial is conducted to evaluate a novel biologic agent for the treatment of moderate-to-severe plaque psoriasis. The study enrolls 200200 patients, who are randomly assigned in a 1:1 ratio to receive either the biologic agent or a placebo. After 1616 weeks, the patients are evaluated for disease progression. In the placebo group, 3030 of the 100100 patients experience disease progression. In the treatment group, 1010 of the 100100 patients experience disease progression. Based on these study results, what is the number needed to treat (NNT) with the new biologic agent to prevent one additional case of disease progression compared to placebo?

  1. A
    33
  2. 55Answer
  3. C
    1010
  4. D
    2020
  5. E
    3030

Answer

The correct answer is 55.
The Number Needed to Treat (NNT) is determined by first calculating the Absolute Risk Reduction (ARR), which is the difference in the event rates between the control group and the experimental group. In this study, the Control Event Rate is 30/10030/100 (0.300.30) and the Experimental Event Rate is 10/10010/100 (0.100.10). The ARR is 0.30−0.10=0.200.30 - 0.10 = 0.20. The NNT is the inverse of the ARR (1/0.201 / 0.20), which equals 55.

Step-by-Step Solution

1
Calculate the Control Event Rate (CER).
CER=30/100=0.30CER = 30 / 100 = 0.30
To determine the baseline risk of disease progression in patients receiving the placebo.
2
Calculate the Experimental Event Rate (EER).
EER=10/100=0.10EER = 10 / 100 = 0.10
To determine the risk of disease progression in patients receiving the new biologic agent.
3
Calculate the Absolute Risk Reduction (ARR).
ARR=CER−EER=0.30−0.10=0.20ARR = CER - EER = 0.30 - 0.10 = 0.20
To find the absolute difference in risk between the two study groups.
4
Calculate the Number Needed to Treat (NNT).
NNT=1/ARR=1/0.20=5NNT = 1 / ARR = 1 / 0.20 = 5
To calculate the number of patients who need to receive the treatment to prevent one additional bad outcome.

Key Concept

The Number Needed to Treat (NNT) is an epidemiological measure that indicates how many patients need to be treated with a specific intervention to prevent one additional adverse outcome. It is calculated as the inverse of the Absolute Risk Reduction (ARR).
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