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In an adequately powered, randomized controlled trial conducted over 3 years, a specific serious side effect (i.e., reduction in leukocytes) with conventional therapy is seen in 0.5% of the study sample. In patients who receive a newly discovered drug, only 0.45% experience the same side effect. Based on these results, the minimum number of patients that would need to receive the new drug for 3 years to statistically demonstrate the prevention of one episode of this side effect in at least one patient (i.e., NNT) is:

  1. 20.

  2. 150.

  3. 200.

  4. 2000.

The correct answer is: 2000.

The minimum number of patients that would need to receive the new drug for 3 years to statistically demonstrate the prevention of one episode of this side effect in at least one patient (NNT) is 2000. This means that for every 2000 patients who receive the new drug, one episode of this specific serious side effect can be prevented. Option A is incorrect because 20 patients is too small of a sample size to have a significant result. Option B is also incorrect because 150 is still relatively small considering the study is over 3 years. Option C is incorrect because although it is a larger number, it is still not large enough to have a statistically significant result. Option D is the correct answer because 2000 is a large enough number to allow for a statistically significant result.