Oncology Clinical Trials

William Kevin Kelly, DO, is Professor, Department of Medical Oncology and Urology, Sidney Kimmel Medical College at Thomas Jefferson University, Sidney Kimmel Cancer Center, Philadelphia, PA. Susan Halabi, PhD, Associate Professor, Department of Biostatistics and Bioinformatics, Duke University Medical Center.

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 Confirmatory 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 Modification 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. DeGraffinreid, 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