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Innovative Thinking on Endpoint Selection in Clinical Trials.

Publication ,  Journal Article
Chow, S-C; Huang, Z
Published in: J Biopharm Stat
2019

In clinical trials, selection of appropriate study endpoints is critical for an accurate and reliable evaluation of safety and effectiveness of a test treatment under investigation. In practice, however, there are usually multiple endpoints available for measurement of disease status and/or therapeutic effect of the test treatment under study. For example, in cancer clinical trials, overall survival, response rate, and/or time to disease progression are usually considered as primary clinical endpoints for evaluation of safety and effectiveness of the test treatment under investigation. Once the study endpoints have been selected, sample size required for achieving a desired power is then determined. It, however, should be noted that different study endpoints may result in different sample sizes. In practice, it is usually not clear which study endpoint can best inform the disease status and measure the treatment effect. Moreover, different study endpoints may not translate one another although they may be highly correlated one another. In this article, we intend to develop an innovative endpoint namely therapeutic index based on a utility function to combine and utilize information collected from all study endpoints. Statistical properties and performances of the proposed therapeutic index are evaluated theoretically. A numerical example concerning a cancer clinical trial is given to illustrate the use of the proposed therapeutic index.

Duke Scholars

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Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2019

Volume

29

Issue

5

Start / End Page

941 / 951

Location

England

Related Subject Headings

  • Thinking
  • Statistics & Probability
  • Product Surveillance, Postmarketing
  • Organizational Innovation
  • Humans
  • Endpoint Determination
  • Clinical Trials as Topic
  • Antineoplastic Agents
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

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Chow, S.-C., & Huang, Z. (2019). Innovative Thinking on Endpoint Selection in Clinical Trials. J Biopharm Stat, 29(5), 941–951. https://doi.org/10.1080/10543406.2019.1657140
Chow, Shein-Chung, and Zhipeng Huang. “Innovative Thinking on Endpoint Selection in Clinical Trials.J Biopharm Stat 29, no. 5 (2019): 941–51. https://doi.org/10.1080/10543406.2019.1657140.
Chow S-C, Huang Z. Innovative Thinking on Endpoint Selection in Clinical Trials. J Biopharm Stat. 2019;29(5):941–51.
Chow, Shein-Chung, and Zhipeng Huang. “Innovative Thinking on Endpoint Selection in Clinical Trials.J Biopharm Stat, vol. 29, no. 5, 2019, pp. 941–51. Pubmed, doi:10.1080/10543406.2019.1657140.
Chow S-C, Huang Z. Innovative Thinking on Endpoint Selection in Clinical Trials. J Biopharm Stat. 2019;29(5):941–951.

Published In

J Biopharm Stat

DOI

EISSN

1520-5711

Publication Date

2019

Volume

29

Issue

5

Start / End Page

941 / 951

Location

England

Related Subject Headings

  • Thinking
  • Statistics & Probability
  • Product Surveillance, Postmarketing
  • Organizational Innovation
  • Humans
  • Endpoint Determination
  • Clinical Trials as Topic
  • Antineoplastic Agents
  • 1115 Pharmacology and Pharmaceutical Sciences