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Collaborative clinical trials: quality or quantity?

Identifieur interne : 001294 ( Istex/Corpus ); précédent : 001293; suivant : 001295

Collaborative clinical trials: quality or quantity?

Auteurs : Ian F. Tannock

Source :

RBID : ISTEX:A9EDF66D16AE9C7255E48A6038058063F1F97E23

English descriptors

Abstract

Abstract: Purpose: To review the merits of using the limited available resources — patients, money, clinical scientists, and ideas — in various types of clinical trial. Conclusions: Two types of trial represent a poor use of resources: (a) nonrandomized trials that provide no insight into biologic mechanisms and are not precursors to testing new strategies in comparison with standard treatment in randomized trials; and (b) small randomized trials that are difficult to interpret because of a high rate of false-positive and false-negative trials. Very large trials that can detect small differences in survival for patients with common tumors are appropriate, but a similar design to detect transient improvements due to palliative therapy represent a poor use of resources. Larger gains in therapeutic index will require the recognition of tumor heterogeneity and the conduct of small trials that are based on biologic hypotheses, and which provide mechanistic information in patients; two examples are provided. Ultimately, strategies that may be individualized among a group of patients with histologically similar tumors will need to be evaluated against the current standard (and homogeneous) treatment.

Url:
DOI: 10.1016/S0360-3016(00)01500-5

Links to Exploration step

ISTEX:A9EDF66D16AE9C7255E48A6038058063F1F97E23

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<affiliation>Department of Medical Oncology and Hematology, Princess Margaret Hospital and University of Toronto, Toronto, Canada</affiliation>
<affiliation>E-mail: ian.tannock@uhn.on.ca</affiliation>
<description>Reprint requests to: Dr. Ian F. Tannock, Department of Medical Oncology and Hematology, Princess Margaret Hospital and University of Toronto, Toronto, ON M5G 2M9, Canada</description>
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<abstract lang="en">Abstract: Purpose: To review the merits of using the limited available resources — patients, money, clinical scientists, and ideas — in various types of clinical trial. Conclusions: Two types of trial represent a poor use of resources: (a) nonrandomized trials that provide no insight into biologic mechanisms and are not precursors to testing new strategies in comparison with standard treatment in randomized trials; and (b) small randomized trials that are difficult to interpret because of a high rate of false-positive and false-negative trials. Very large trials that can detect small differences in survival for patients with common tumors are appropriate, but a similar design to detect transient improvements due to palliative therapy represent a poor use of resources. Larger gains in therapeutic index will require the recognition of tumor heterogeneity and the conduct of small trials that are based on biologic hypotheses, and which provide mechanistic information in patients; two examples are provided. Ultimately, strategies that may be individualized among a group of patients with histologically similar tumors will need to be evaluated against the current standard (and homogeneous) treatment.</abstract>
<note type="content">Section title: ICTR 2000</note>
<note type="content">Table 1: Problems that prevent single arm studies from giving useful information about therapeutic benefit</note>
<note type="content">Table 2: Factors that can lead to false-positive and false-negative results among randomized trials</note>
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<identifier type="ISSN">0360-3016</identifier>
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<date>2001</date>
<detail type="volume">
<number>49</number>
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<number>2</number>
<caption>no.</caption>
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<start>299</start>
<end>616</end>
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<end>343</end>
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<identifier type="DOI">10.1016/S0360-3016(00)01500-5</identifier>
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<accessCondition type="use and reproduction" contentType="copyright">©2001 Elsevier Science Inc.</accessCondition>
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