Incremental net benefit in randomized clinical trials

Stat Med. 2001 Jun 15;20(11):1563-74. doi: 10.1002/sim.789.


There are three approaches to health economic evaluation for comparing two therapies. These are (i) cost minimization, in which one assumes or observes no difference in effectiveness, (ii) incremental cost-effectiveness, and (iii) incremental net benefit. The latter can be expressed either in units of effectiveness or costs. When analysing data from a clinical trial, expressing incremental net benefit in units of cost allows the investigator to examine all three approaches in a single graph, complete with the corresponding statistical inferences. Furthermore, if costs and effectiveness are not censored, this can be achieved using common two-sample statistical procedures. The above will be illustrated using two examples, one with censoring and one without.

MeSH terms

  • Amiodarone / therapeutic use
  • Anti-Arrhythmia Agents / therapeutic use
  • Antineoplastic Agents / therapeutic use
  • Cost-Benefit Analysis / economics
  • Cost-Benefit Analysis / methods
  • Defibrillators, Implantable
  • Heart Arrest / prevention & control
  • Heart Arrest / therapy
  • Humans
  • Male
  • Mitoxantrone / therapeutic use
  • Models, Statistical*
  • Prednisone / therapeutic use
  • Prostatic Neoplasms / drug therapy
  • Randomized Controlled Trials as Topic / economics*
  • Randomized Controlled Trials as Topic / methods


  • Anti-Arrhythmia Agents
  • Antineoplastic Agents
  • Mitoxantrone
  • Amiodarone
  • Prednisone