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Rethinking clinical trials for cytostatic drugs

Nature Reviews Cancer volume 3, pages 540–545 (2003)Cite this article

Abstract

The failure of many cytostatic agents in Phase III clinical trials for treatment of common cancers has led researchers to question current approaches to trial development. Recent studies offer some clues as to what is wrong with two particular aspects of clinical trial design — survival as an end point and simultaneous combination with cytotoxic chemotherapy — and indicate possible alternatives.

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Figure 1: Scheme for clinical trials of cytostatic agents as monotherapy.

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Author information

Authors and Affiliations

  1. The Lawns, Winterbrook Lane, Wallingford, OX10 9EF, Oxford, UK

    Andrew W. Millar

  2. Associate Medical Director of Novartis, Australia

    Kevin P. Lynch

Authors
  1. Andrew W. Millar
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  2. Kevin P. Lynch
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Corresponding author

Correspondence to Andrew W. Millar.

Related links

Related links

DATABASES

Cancer.gov

breast cancer

colorectal cancer

head and neck cancer

lung cancer

ovarian cancer

pancreatic cancer

prostate cancer

stomach cancer

LocusLink

EGF

ERBB2

KIT

PDGF

VEGF

Glossary

BAYESIAN STATISTICAL METHODS

Statistical methods in which expectation and ongoing observation are used to increase efficiency and reduce the number of observations (patient numbers) that are necessary to reach reliable conclusions. These are particularly efficient compared with 'frequentist' statistical approaches when multiple decisions are necessary — for example, in dose comparisons.

OVERALL SURVIVAL

The duration for which a patient with cancer survives after the start of treatment.

PROGRESSION-FREE SURVIVAL

The duration for which a patient with cancer survives after the start of treatment without evidence of disease progression.

SURROGATE END-POINT

A biomarker that is used to monitor disease progression and expected to predict clinical benefit based on epidemiological, therapeutic, pathophysiological or other scientific evidence.

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Millar, A., Lynch, K. Rethinking clinical trials for cytostatic drugs. Nat Rev Cancer 3, 540–545 (2003). https://doi.org/10.1038/nrc1124

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