The Role of Nonrandomized Trials in the Evaluation of Oncology Drugs

被引：53

作者：

Simon, R. ^{[1
]}

Blumenthal, G. M. ^{[2
]}

Rothenberg, M. L. ^{[3
]}

Sommer, J. ^{[4
]}

Roberts, S. A. ^{[5
]}

Armstrong, D. K. ^{[6
]}

LaVange, L. M. ^{[2
]}

Pazdur, R. ^{[3
]}

机构：

[1] NCI, Bethesda, MD 20892 USA

[2] US FDA, Silver Spring, MD USA

[3] Pfizer Inc, New York, NY USA

[4] Chordoma Fdn, Durham, NC USA

[5] Friends Canc Res, Washington, DC 20036 USA

[6] Johns Hopkins Kimmel Canc Ctr, Baltimore, MD USA

来源：

CLINICAL PHARMACOLOGY & THERAPEUTICS | 2015年 / 97卷 / 05期

关键词：

ADMINISTRATION APPROVAL; CLINICAL-TRIALS; END-POINTS; PHASE-II; SURVIVAL; CANCER; THERAPIES;

D O I：

10.1002/cpt.86

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Although randomized trials provide the most reliable evidence of a drug's safety and efficacy, there are situations where randomized trials are not possible or ethical. In this article we discuss when and how single-arm trials can be used to support full approval of oncology drugs. These include situations in which an unprecedented effect on tumor response is observed in a setting of high unmet medical need, clinical trial patients have been well characterized, enabling a target population to be clearly defined, experience exists in a sufficient number of patients to allow adequate assessment of the risk:benefit relationship, and a proper historical context can be provided for analysis. We also discuss how response rates might be considered predictive of long-term outcomes or clinically meaningful in and of themselves in certain contexts.

引用

页码：502 / 507

页数：6

共 20 条

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