One- and Two-Stage Models for Comparing an Experimental Treatment with a Standard.

Abstract

Optimal one- and two-stage plans are developed for selecting one of two medical treatments, an experimental treatment A or a standard treatment B. The response to treatment is normally distributed with unknwon mean mu sub A or mu sub B and known variance sigma squared. It is assumed mu sub A > or = mu sub B but that treatment A is worthwhile adopting only if mu sub A - mu sub B > or = a, where a is fixed. The only cost considered is the ethical one of treating a patient with the 'wrong' treatment. Out of a total of N patients with the disease in question, a fraction 2P of them is to be placed in a clinical trial comparing the two treatments, with the 'winning' treatment to be given to the remaining N(1-2P) patients. Optimal values of P are found for both a one-stage and a two-stage plan, with the latter showing a modest improvement in expected net gain. The case in which mu sub B is known is also considered, and the resultant improvement in expected net gain over the case mu sub B unknown is found to moderate for a one-stage trial. (Author)

Document Details

Document Type
Technical Report
Publication Date
Oct 08, 1971
Accession Number
AD0731397

Entities

People

  • Allan P. Donner

Organizations

  • Harvard University

Tags

Communities of Interest

  • Biomedical

DTIC Thesaurus Topics

  • Clinical Trials
  • Therapy

Fields of Study

  • Mathematics

Readers

  • Mathematics or Statistics
  • Oncology
  • Statistical inference.