Early detection of pancreatic cancer: Study design and analytical considerations in biomarker discovery and early phase validation studies.
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Smith LM, Mahoney DW, Bamlet WR, Yu F, Liu S, Goggins MG, Darabi S, Majumder S, Wang QL, Coté GA, Demeure MJ, Zhang Z, Srivastava S, Chawla A, Izmirlian G, Olson JE, Wolpin BM, Genkinger JM, Zaret KS, Brand R, Koay EJ, Oberg AL
Pancreatology 24 (8) 1265-1279 [2024-12-00; online 2024-10-29]
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. 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. 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. 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.
PubMed 39516175
DOI 10.1016/j.pan.2024.10.012
Crossref 10.1016/j.pan.2024.10.012
mid: NIHMS2034711
pmc: PMC11780679
pii: S1424-3903(24)00784-1