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Overestimated treatment effects in randomised phase II trials: What's up doctor?

Accepted version
Peer-reviewed

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Type

Article

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Authors

Michiels, Stefan 

Abstract

Phase II clinical trials of experimental treatments play an essential role in drug development. Historically, trials were conducted sequentially, starting from a phase I trial in which dose and safety are evaluated advancing to phase II, looking for some sign of efficacy on a short-term end-point such as tumour shrinkage to screen out inefficacious experimental agents, and to large-scale randomised phase III trials evaluating properly the efficacy of a new treatment on objective clinical end-points. To expedite this process, phase II trials historically used single-arm designs to treat patients with experimental therapies only [1]. However, the overall success rate of drug development programs in oncology from the start of phase I to registration has been estimated recently to be only 3.4%, much lower compared with that of other diseases [2]. Part of this low success rate has been argued to be due to suboptimal use of randomised trial designs in the phase II space.

Description

Keywords

32 Biomedical and Clinical Sciences, 3211 Oncology and Carcinogenesis

Journal Title

European Journal of Cancer

Conference Name

Journal ISSN

0959-8049
1879-0852

Volume Title

123

Publisher

Elsevier
Sponsorship
MRC (unknown)
Cancer Research UK (18113)