The Importance of Beta, the Type II Error and Sample Size in the Design and Interpretation of the Randomized Control Trial. Survey of 71 "Negative" Trials

N Engl J Med. 1978 Sep 28;299(13):690-4. doi: 10.1056/NEJM197809282991304.

Abstract

Seventy-one "negative" randomized control trials were re-examined to determine if the investigators had studied large enough samples to give a high probability (greater than 0.90) of detecting a 25 per cent and 50 per cent therapeutic improvement in the response. Sixty-seven of the trials had a greater than 10 per cent risk of missing a true 25 per cent therapeutic improvement, and with the same risk, 50 of the trials could have missed a 50 per cent improvement. Estimates of 90 per cent confidence intervals for the true improvement in each trial showed that in 57 of these "negative" trials, a potential 25 per cent improvement was possible, and 34 of the trials showed a potential 50 per cent improvement. Many of the therapies labeled as "no different from control" in trials using inadequate samples have not received a fair test. Concern for the probability of missing an important therapeutic improvement because of small sample sizes deserves more attention in the planning of clinical trials.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial

MeSH terms

  • Clinical Trials as Topic
  • Humans
  • Probability
  • Research Design*
  • Research*
  • Therapeutics