Handbook of Generalized Pairwise Comparisons Methods for Patient-Centric Analysis

Abbreviations

Foreword

Preface

Editors

Contributors

I. Introduction

1. Generalized Pairwise Comparisons for patient-centric treatment decisions, Marc Buyse and Geert Molenberghs

II. Statistical Theory

2. Measures of Treatment Effect, Johan Verbeeck and Mickael De Backer

3. Inference, Brice Ozenne and Johan Verbeeck

4. Right-censored Data, Vaiva Deltuvaite-Thomas, Brice Ozenne, Julien Peron, and Mickael De Backer

5. Restricted GPC Statistics, Mickael De Backer and Michal Kicinski

6. Missing Data, Vaiva Deltuvaite-Thomas and Tomasz Burzykowski

7. Challenges and Limitations of NTB, Tomasz Burzykowski

III. Advanced Topics

8. GPC and Rank Procedures, Edgar Brunner

9. Stratification and non-parametric adjustment, Mickael De Backer and Michael Fay

10. Covariate adjustment for GPC, Olivier Thas

11. Cure Rate Models, Mickael De Backer and Catherine Legrand

12. Group Sequential Designs, Stephen Schuurhuis and Frank Konietschke

13. The Desirability Of Outcome Ranking (DOOR), Toshimitsu Hamasaki and Scott R. Evans

14. Theoretical Properties of GPC Statistics, Johan Verbeeck

IV. Software and Datasets

15. Illustrating GPC through a graphical user interface, Jean-Christophe Chiem and Samuel Salvaggio

16. GPC through a command line interface, Brice Ozenne and Mickael De Backer

V. Applications

17. Cardiovascular Disease, Johan Verbeeck, Jan Verwerft, and Pascal Vranckx

18. Oncology, Everardo D. Saad and Julien Peron

19. Rare Diseases, Vaiva Deltuvaite-Thomas and Johan Verbeeck

20. Regulatory considerations for GPC analyses, Marc Buyse and Samuel Salvaggio

21. Quantitative benefit-risk assessment, Johan Verbeeck and Julien Peron

VI. Patient-centricity

22. GPC for N − of − 1 Trials, Joris Giai and Matthieu Roustit

23. Elicitation of Patient Preferences for GPC, Thibaut Reverdy, Laura Del Carpio, Alexandra Dima, and Julien Peron

24. Shared Decision-Making, Oriana Ciani, Vittoria Ardito, Natalia Oprea

25. Patient centricity and participation, Stef Baas, Richard J. Boucherie, and Peter Kapitein

26. Open Science and Patient-level Data Sharing, Frank Rockhold and Rebecca Wilgus

Postface

Bibliography

Index

Biography

Marc Buyse holds a ScD in biostatistics from the Harvard School of Public Health, Boston, MA. He is Associate Professor of Biostatistics at U Hasselt, Belgium, and the founder of companies providing statistical services and software to the biopharmaceutical industry: International Drug Development Institute (IDDI), CluePoints and One2Treat.

Johan Verbeeck trained as a biotechnologist and biostatistician and transitioned from the pharmaceutical industry to academia. Currently, he serves as a post-doctoral biostatistical researcher at the Data Science Institute at U Hasselt, Belgium, specializing in statistical methods for analyzing multivariate outcomes, especially in small sample trials and cardiology.

Mickael De Backer received a PhD in biostatistics from UCLouvain, Belgium, in 2018, with a dissertation focused on quantile regressions in survival analysis. Since then, his work and research have centered on generalized pairwise comparisons in both academic and industry settings. He also serves as an invited lecturer in biostatistics at UCLouvain.

Vaiva Deltuvaite-Thomas obtained a PhD in biostatistics from U Hasselt, Belgium, after working as a community pharmacist in Lithuania, Belgium, Ireland, and France, which made her acutely aware of the need to understand and explain treatment-related information to patients. She is currently providing consultancy services as statistical scientist at International Drug Development Institute (IDDI).

Everardo D. Saad trained and practiced as a medical oncologist before shifting his career to clinical research and education. He serves as Medical Director at International Drug Development Institute (IDDI), Belgium, and is the founder of Dendrix, a medical education and research company in Sao Paulo, Brazil.

Geert Molenberghs received a PhD in biostatistics from U Antwerpen, Belgium. He was President of the International Biometric Society. He is Fellow of the American Statistical Association. He is Professor and founding director of the Center for Statistics at U Hasselt and of the Interuniversity Institute for Biostatistics and statistical Bioinformatics (U Hasselt and KU Leuven).

“This book stands as a guiding beacon for developers, researchers, and regulators, sparking the evolution of fit-for-dossier trials into agile studies tailored for informed decisions.” Francesco Pignatti, Head of the Office of Oncology and Haematology, European Medicines Agency, Amsterdam, the Netherlands.

“The editors of this book and the chapter authors are to be commended for consolidating the considerable advances in GPC statistical methods into a single comprehensive resource that should serve as a standard for many years to come.” Gene Pennello, Mathematical Statistician, US Food and Drug Administration, Bethesda, MD.