Marc Buyse

Marc Buyse is the founder of One2Treat, the International Drug Development Institute (IDDI), and CluePoints, and is Associate Professor of Biostatistics at Hasselt University in Belgium. He is a biostatistician specializing in clinical trial methodology, statistical methods for drug development, and the evaluation of treatment benefit, with a particular focus on approaches that better capture clinically meaningful outcomes.


Read about Marc’s mission with One2Treat – from frustration to innovation.


Marc holds degrees from Université libre de Bruxelles (ULB), Cranfield School of Management, and a doctorate in biostatistics from Harvard University. Before founding IDDI in 1991, he worked at the European Organisation for Research and Treatment of Cancer (EORTC) in Brussels and the Dana-Farber Cancer Institute in Boston.

Research in Net Treatment Benefit and Generalized Pairwise Comparisons

His research has contributed to clinical trial design, generalized pairwise comparisons (GPC), Net Treatment Benefit (NTB), meta-analysis, validation of biomarkers and surrogate endpoints, randomization methods, and statistical methods in oncology. His work on generalized pairwise comparisons established a framework for comparing treatments across multiple prioritized outcomes, providing the statistical basis for measures such as Net Treatment Benefit, success odds, and the win ratio.

Through One2Treat, Marc continues to develop and apply these statistical approaches to patient-centred clinical trial design. A particular focus is on moving beyond single endpoints or conventional composite endpoints to evaluate treatment benefit across multiple outcomes that can reflect what matters most to patients, clinicians, and other stakeholders.

Marc has published extensively on Net Treatment Benefit and generalized pairwise comparisons, including research on their application to oncology, benefit-risk assessment, censored data, and clinical trial design. His recent work includes research on Restricted Net Treatment Benefit in oncology, which extends NTB to settings involving time-restricted survival outcomes, as well as applications of NTB to the formal assessment of benefit-risk balance in clinical trials.

Scientific Leadership

He has served as President of the International Society for Clinical Biostatistics and President of the Quetelet Society, and is a Fellow of the Society for Clinical Trials. His scientific and methodological work spans more than three decades of clinical research, from the development and analysis of clinical trials to newer approaches for quantifying overall treatment benefit.

Selected publications on Net Treatment Benefit and Generalized Pairwise Comparisons

This page brings together selected publications and scientific contributions from Marc Buyse relating specifically to Net Treatment Benefit and Generalized Pairwise Comparisons, providing a central resource for researchers, statisticians, clinical development professionals, and others interested in the methodology and application of NTB in clinical trials.

For a complete list of publications, see https://publicationslist.org/marc.buyse

2026

The Oncologist – Benefit-risk balance of S-1 versus UFT as adjuvant chemotherapy for stage II/III rectal cancer assessed using Net Treatment Benefit

2025

The Lancet Oncology – Dose optimisation to improve access to effective cancer medicines

Handbook of Generalized Pairwise Comparisons – Methods for patient centric analysis

JAMA Network – Net Treatment Benefit to support shared decision-making in the CODA trial

2024

Journal of Clinical Epidemiology – Restricted Net Treatment Benefit in oncology

2023

Statistics in Medicine – Individualized Net Benefit estimation and meta-analysis using generalized pairwise comparisons in N-of-1 trials