Individually randomized intervention trials for disease prevention and control

Stat Methods Med Res. 1999 Dec;8(4):287-309. doi: 10.1177/096228029900800403.

Abstract

It is argued that randomized, controlled trials should fulfil a critical role in the identification of practical approaches to the prevention and control of chronic diseases. Because of the great public health potential of chemopreventive and behavioural approaches to chronic disease prevention there is need for a major interdisciplinary scientific effort aimed at intervention development. Because of the cost and duration of controlled trials to evaluate specific interventions there is a need for well-conducted feasibility, pilot and intermediate outcome trials, to inform and to justify corresponding full-scale trials having clinical disease outcomes. Compared to therapeutic trials, prevention trials need to have a greater emphasis on overall benefit versus risk assessment. Such trials need to be large enough, and of sufficient duration, to yield powerful tests of key hypotheses, and informative benefit versus risk summary statements. These requirements have a range of implications for intervention trial design, conduct, monitoring and reporting, which are reviewed and discussed. The clinical trial component of the ongoing Women's Health Initiative provides illustration throughout this discussion.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Aged
  • Chronic Disease* / epidemiology
  • Chronic Disease* / therapy
  • Female
  • Health Promotion / statistics & numerical data*
  • Humans
  • Middle Aged
  • Randomized Controlled Trials as Topic / methods*
  • Research Design*
  • Risk Factors
  • Women's Health