Clinical Trial Design in Neuroendocrine Tumors

Daniel M Halperin; James C Yao

doi:10.1016/j.hoc.2015.09.011

Clinical Trial Design in Neuroendocrine Tumors

Hematol Oncol Clin North Am. 2016 Feb;30(1):209-17. doi: 10.1016/j.hoc.2015.09.011.

Authors

Daniel M Halperin¹, James C Yao²

Affiliations

¹ Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Unit 426, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
² Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Unit 426, 1515 Holcombe Boulevard, Houston, TX 77030, USA. Electronic address: jyao@mdanderson.org.

PMID: 26614378
DOI: 10.1016/j.hoc.2015.09.011

Abstract

Neuroendocrine tumors (NETs) present tremendous opportunities for productive clinical investigation, but substantial challenges as well. Investigators must be aware of common pitfalls in study design, informed by an understanding of the history of trials in the field, to make the best use of available data and our patient volunteers. We believe the salient issues in clinical trial design and interpretation in the NET field are patient homogeneity, standardized response assessment, and rigorous design and execution. Whether designing or interpreting a study in patients with NET, these principles should drive assessment.

Keywords: Clinical trials; Neuroendocrine tumors; Patient homogeneity; Standardized response assessment; Study design and interpretation.

Publication types

Review

MeSH terms

Clinical Trials as Topic / methods*
Disease-Free Survival
Humans
Neuroendocrine Tumors / therapy*
Outcome Assessment, Health Care / methods
Outcome Assessment, Health Care / statistics & numerical data
Patient Selection*
Quality of Life
Research Design*