Early Detection of Pancreatic Cancer: Study Design and Analytical Considerations in Biomarker Discovery and Early Phase Validation Studies

Authors

Lynette M Smith
Douglas W Mahoney
William R Bamlet
Fang Yu
Suyu Liu
Michael G Goggins
Sourat Darabi
Shounak Majumder
Qiao-Li Wang
Gregory A Coté
Michael J Demeure
Zhen Zhang
Sudhir Srivastava
Akhil Chawla
Grant Izmirlian
Janet E Olson
Brian M Wolpin
Jeanine M Genkinger
Kenneth S Zaret
Randall Brand
Eugene J Koay
Ann L Oberg

Publication Date

12-1-2024

Journal

Pancreatology

DOI

10.1016/j.pan.2024.10.012

PMID

39516175

PMCID

PMC11780679

PubMedCentral® Posted Date

12-1-2025

PubMedCentral® Full Text Version

Author MSS

Abstract

Objectives: Pancreatic ductal adenocarcinoma (PDAC) is a highly lethal disease that is challenging to detect at an early stage. Biomarkers are needed that can detect PDAC early in the course of disease when interventions lead to the best outcomes. We highlight study design and statistical considerations that inform pancreatic cancer early detection biomarker evaluation.

Methods: We describe experimental design strategies in this setting useful for streamlining biomarker evaluation at each Early Detection Research Network (EDRN) phase of biomarker development. We break the early EDRN phases into sub-phases, proposing objectives, study design strategies, and biomarker performance benchmarks.

Results: The goal of early detection in populations at high-risk of PDAC is described. Evaluating biomarker behavior in patients under surveillance without disease can winnow candidate biomarkers. Potential resources for biomarker validation studies are described.

Conclusions: Multisite and multidisciplinary collaboration can facilitate study design strategies in this lethal but low incidence disease and streamline the path from biomarker discovery to clinical use. Improvements in analytical and experimental design methods could help accelerate biomarker evaluation through the phases of biomarker development.

Keywords

Humans, Pancreatic Neoplasms, Biomarkers, Tumor, Early Detection of Cancer, Carcinoma, Pancreatic Ductal, Research Design, Biomarker development, Early detection, Experimental design, Pancreatic cancer, Validation

Published Open-Access

yes

Recommended Citation

Smith, Lynette M; Mahoney, Douglas W; Bamlet, William R; et al., "Early Detection of Pancreatic Cancer: Study Design and Analytical Considerations in Biomarker Discovery and Early Phase Validation Studies" (2024). Faculty, Staff and Student Publications. 4604.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/4604