Contents
Contributors
Foreword Clifford A. Hudis, MD and Richard L. Schilsky, MD
Preface
PART I. BACKGROUND AND INTRODUCTION TO ONCOLOGY CLINICAL TRIALS
1. The Changing Landscape of Clinical Research and Trials
Susan Halabi and William Kevin Kelly
2. Historical Perspectives of Oncology Clinical Trials
Ada H. Braun and David M. Reese
3. Ethical Principles Guiding Clinical Research
Jackson Bruce Smith
4. Industry Collaboration When Developing Novel Agents in Oncology
Hong Xie
5. The Trials and Tribulations of Writing and Conducting an Investigator Initiated Trial
Jake Vinson, Josh Buddle, Julie Filipenko, Christine Tran, Kristofer Prepelica, and Sarah Wise
6. Writing a Consent Form
Christine Grady
7. Why Do Clinical Trials Fail?
Laurence Collette, Jan Bogaerts, and Xavier Paoletti
PART II. DESIGNING ONCOLOGY CLINICAL TRIALS
8. Choice of Endpoints in Cancer Clinical Trials
Mei-Yin Polley, Wenting Wu, and Daniel J. Sargent
9. Design, Testing, and Estimation in Clinical Trials
Barry Kurt Moser
10. Innovative Phase I Clinical Trials
Nolan A. Wages
11. Pharmacokinetics in Clinical Oncology
Jill M. Kolesar
12. Dose Finding Using the Continual Reassessment Method
Mark R. Conaway
13. Design of Phase II Trials
Hongkun Wang and Gina R. Petroni
14. Biomarkers in Con¿rmatory Clinical Trials
Thomas Gwise
15. Bayesian Designs in Clinical Trials
Gary L. Rosner, B. Nebiyou Bekele, and Yuan Ji
16. Selection Designs
Suzanne E. Dahlberg
17. Phase III Oncology Clinical Trials
Antje Hoering and John Crowley
18. Design of Noninferiority Trials in Oncology
Lei Nie and Zhiwei Zhang
19. Design of Quality of Life Studies
Amylou C. Dueck and Katie L. Kunze
20. Adaptive Designs
Tze L. Lai, Ying Lu, and Ka Wai Tsang
PART III. CONDUCTING ONCOLOGY CLINICAL TRIALS
21. Randomization
Susan Groshen
22. Case Report Form Development
Susan Barry
23. Monitoring, Assessing, and Reporting Adverse Events
Amy Callahan, Elizabeth Ness, and Helen Chen
24. Dose Modi¿cation and Use of Ancillary Treatments in Investigational Studies in Clinical Trials
Yoshihito David Saito, Pamela Harris, Ming Poi, and Robert Wesolowski
25. Assessment of Patient-Reported Outcomes in Industry-Sponsored Clinical Trials
Ari Gnanasakthy and Ethan Basch
26. Recruitment of Research Participants
Christopher Gantz
27. Barriers to Oncology Clinical Trials
Chethan Ramamurthy and Yu-Ning Wong
28. The Role of Novel Imaging Techniques in Clinical Trials
Binsheng Zhao and Lawrence H. Schwartz
29. Practical Issues With Correlative Studies
David McConkey and Woonyoung Choi
30. The Development of Companion Diagnostics in Oncology Clinical Trials
Zixuan Wang and Stephen C. Peiper
PART IV. ANALYZING RESULTS OF ONCOLOGY CLINICAL TRIALS
31. Interim Analysis and Data Monitoring
Scott R. Evans and William T. Barry
32. Reporting of Results: Data Analysis and Interpretation
Donna Niedzwiecki
33. Statistical Considerations for Developing and Validating Prognostic Models of Clinical Outcomes
Susan Halabi and Lira Pi
34. Statistical Evaluation of Surrogate Endpoints in Cancer Clinical Trials
Marc Buyse, Geert Molenberghs, Xavier Paoletti, Koji Oba, Ariel Alonso, Wim Van der Elst, and Tomasz Burzykowski
35. Development and Validation of Genomic Signatures
Stefan Michiels, Nils Ternès, and Federico Rotolo
36. Competing Risks Analysis in Clinical Trials
Solange Bassale, Jeong Youn Lim, and Motomi Mori
37. Systematic Reviews and Meta-Analysis
Claire Vale, Sarah Burdett, David Fisher, Larysa Rydzewska, and Jayne Tierney
38. Statistical Methods for Genomics-Driven Clinical Studies
Richard Simon
39. Handling Missing Data in Oncology Clinical Trials
Xiaoyun (Nicole) Li, Cong Chen, and Xiaoyin (Frank) Fan
PART V. SPECIAL CONSIDERATIONS IN ONCOLOGY CLINICAL TRIALS
40. Health-Related Quality of Life Studies in International Randomized Controlled Oncology Clinical Trials
Andrew Bottomley, Corneel Coens, Murielle Mauer, Madeline Pe, and Francesca Martinelli
41. The Economics of Oncology Clinical Trials
Michaela A. Dinan and Shelby D. Reed
42. Special Considerations in Immunotherapy Trials
Claire F. Friedman, Katherine S. Panageas, and Jedd D. Wolchok
43. Special Considerations in Radiation Therapy Trials
Amanda J. Walker, Hyun Kim, Paul G. Kluetz, Julia A. Beaver, Gideon Blumenthal, and Richard Pazdur
44. Clinical Trials in Hematologic Malignancies
Neil Palmisiano, Bradley M. Haverkos, Sameh Gaballa, Joanne Filicko-O'Hara, Pierluigi Porcu, and Margaret Kasner
45. Issues in Recruiting Elderly, Underserved, Minority, and Rural Populations (and Solutions)
Cecilia R. DeGraf¿nreid, Jill Oliveri, Chasity Washington, Cathy Tatum, and Electra D. Paskett
46. Telemedicine and Clinical Trials
Ana Maria Lopez
PART VI. COOPERATIVE GROUPS, REGULATORY AND GOVERNING BODIES
47. Cooperative Groups and Global Clinical Trials in the Future
Cooperative Groups: An American and Canadian Perspective
Joseph A. Sparano, Judith Manola, and Robert L. Comis
Cooperative Groups: A Japanese Perspective
Kenichi Nakamura, Haruhiko Fukuda, and Yasuo Ohashi
Cooperative Groups: The Australian Perspective
Prudence A. Francis, Katrin Sjoquist, and Linda Mileshkin
Cooperative Groups: A Latin American Perspective
Gustavo Werutsky
48. The Evolution of the Drug Evaluation Process in Oncology: Regulatory Perspective
The Evolution of Oncology Drug Evaluation at the FDA
Steven J. Lemery, Gideon Blumenthal, Paul G. Kluetz, Patricia Keegan, Amy McKee, and Richard Pazdur
The Evolution of the Drug Evaluation Process in the EU
Francesco Pignatti, Emmanuelle Kempf, and Pierre Demolis
The Evolution of the Drug Evaluation Process in Japan
Hiroyuki Sato, Tomohiro Yamaguchi, Yuki Ando, and Takahiro Nonaka
49. Clinical Trials in the Year 2025
Apostolia M. Tsimberidou, Peter Müller, and Richard L. Schilsky
Index
