Reduction in burden of illness: a new efficacy measure for prevention trials

Stat Med. 1994 Sep 30;13(18):1807-14. doi: 10.1002/sim.4780131803.

Abstract

A new efficacy measure is developed for use in prevention trials of interventions which may affect both disease incidence and disease severity. We assign a severity score to each incident case and sum severity scores over all incident cases within each treatment group to create a burden-of-illness score for each treatment group. Efficacy is evaluated by the difference between the burden-of-illness per randomized subject in the two randomized treatment groups. Since the numbers of summands in each burden-of-illness score is a random variable, standard methods of analysis are not directly applicable. The asymptotic distribution and sampling properties of the net reduction in the burden-of-illness score are derived for trials designed to stop either after a fixed length of follow-up or after the occurrence of a fixed number of cases. We illustrate the method with data from a clinical trial of a human rotavirus vaccine.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Data Interpretation, Statistical
  • Diarrhea / epidemiology
  • Diarrhea / etiology
  • Diarrhea / prevention & control
  • Humans
  • Incidence
  • Models, Statistical*
  • Primary Prevention / statistics & numerical data*
  • Randomized Controlled Trials as Topic / statistics & numerical data*
  • Rotavirus Infections / epidemiology
  • Rotavirus Infections / prevention & control
  • Sample Size
  • Severity of Illness Index
  • Treatment Outcome
  • Viral Vaccines

Substances

  • Viral Vaccines