The use of prognostic factors in clinical trials.

Abstract:

:Numerous examples exist of prognostic factors that have been conclusively established and that can segregate the target patient population into subgroups with vastly different outcomes. At the same time, we can expect, in the majority of trials being conducted, that if a new treatment turns out to be successful, it generally results in only a modest improvement in patient outcome. This has contributed to a major emphasis on the identification and use of prognostic factors in clinical trials. Besides the inherent descriptive information for predicting patient outcome, prognostic factors are increasingly used in trial design and analysis with the hope of reducing or correcting bias that could otherwise occur from simple patient allocation schemes and unadjusted comparisons of treatment groups, and tailoring therapy so that the best treatments are selected in patient subgroups. Although the above objectives are reasonable, numerous misunderstandings and misuses of prognostic factors regularly occur. These involve such issues as the inappropriate application of clinical/statistical criteria for identification and confirmation of such factors, the sometimes undue emphasis on selection of best subdivisions of prognostic factors, the overreliance on prognostic factors and statistical models by proponents of historical control studies, the exaggerated importance of prognostic factors in special randomization schemes, etc. However, if a thorough understanding of such issues is combined with a careful approach to the use of prognostic factors, significant benefit to the design, conduct, and ultimate scientific information from clinical trials will result.

journal_name

Cancer

journal_title

Cancer

authors

Sather HN

doi

10.1002/1097-0142(19860715)58:2+<461::aid-cncr2820

subject

Has Abstract

pub_date

1986-07-15 00:00:00

pages

461-7

issue

2 Suppl

eissn

0008-543X

issn

1097-0142

journal_volume

58

pub_type

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