Faculty Collaboration Database

Of Endpoints and Equipoise: Reforming Clinical Trials in Colorectal Polyp Resection. Curr Gastroenterol Rep 2025 Sep 18;27(1):62

Date

09/18/2025

Pubmed ID

40965773

DOI

10.1007/s11894-025-01013-2

Scopus ID

2-s2.0-105016592118 (requires institutional sign-in at Scopus site)   1 Citation

Abstract

PURPOSE OF REVIEW: To review and assess current design approaches in endoscopic mucosal resection (EMR) trials, identify areas where traditional methodologies may limit relevance or generalizability, and propose a forward-looking framework that incorporates methodological innovations aligned with clinical and stakeholder priorities.

RECENT FINDINGS: Despite major procedural advances in EMR, trial design has evolved more slowly - employing binary endpoints, limited patient and clinician input, and enrollment models often led by high-volume proceduralists. Critical design innovations, such as ordinal recurrence classifications, composite outcomes ranked by clinical severity, and proceduralist-aware statistical models can help to overcome these limitations. Introducing methodology such as generalized pairwise comparisons yielding a win ratio, while useful for analyzing hierarchical composite endpoints (HCEs), represent just one facet of a broader strategy. Drawing from innovations in cardiovascular and other procedural disciplines, this review highlights how diverse design elements can be adapted to the EMR space. Improving EMR trials demands a shift in trial architecture. By combining stakeholder-informed outcome hierarchies, advanced analytic methods, and strategies to mitigate operator bias, a modern framework capable of producing more meaningful, reproducible, and generalizable evidence is possible. This evolution in design reflects a necessary progression for procedural trials and sets the stage for a new standard in colorectal polyp resection research.

Author List

Smith ZL, Forbes N, Madhavan S, Pohl H, Schauer JM, Steinbrück I, von Renteln D

Authors

Srivats Madhavan MBBS Associate Professor in the Medicine department at Medical College of Wisconsin
Zachary Smith DO Associate Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Clinical Trials as Topic
Colonic Polyps
Colorectal Neoplasms
Endoscopic Mucosal Resection
Endpoint Determination
Humans
Research Design
Therapeutic Equipoise