© 2020

Design and Analysis of Subgroups with Biopharmaceutical Applications

  • Naitee Ting
  • Joseph C. Cappelleri
  • Shuyen Ho
  • (Din) Ding-Geng Chen

Part of the Emerging Topics in Statistics and Biostatistics book series (ETSB)

Table of contents

  1. Front Matter
    Pages i-xviii
  2. Subgroups in Clinical Trial Design and Analysis

    1. Front Matter
      Pages 1-1
    2. Hui Wang, Ilana Belitskaya-Lévy, Ying Lu
      Pages 19-31
    3. Alex Dmitrienko, Ilya Lipkovich, Aaron Dane, Christoph Muysers
      Pages 33-91
  3. Subgroup Identification and Personalized Medicine

    1. Front Matter
      Pages 115-115
    2. Pierre Bunouf, Alex Dmitrienko, Jean-Marie Grouin
      Pages 117-145
    3. Wei-Yin Loh, Peigen Zhou
      Pages 147-165
    4. Lei Shen, Hollins Showalter, Chakib Battioui, Brian Denton
      Pages 181-197
    5. Ying Ding, Yue Wei, Xinjun Wang
      Pages 209-228
    6. Yizhao Zhou, Ao Yuan, Ming T. Tan
      Pages 229-243
    7. Xin Huang, Yihua Gu, Yan Sun, Ivan S. F. Chan
      Pages 245-270
    8. Yuan Chen, Ying Liu, Donglin Zeng, Yuanjia Wang
      Pages 271-297
  4. General Issues About Subgroup Analysis, Including Regulatory Considerations

    1. Front Matter
      Pages 299-299
    2. Li Ming Dong, Heng Li, Ram Tiwari, Lilly Q. Yue
      Pages 301-308
    3. Oliver N. Keene, Daniel J. Bratton
      Pages 309-330
    4. Yang Liu, Lijiang Geng, Xiaojing Wang, Donghui Zhang, Ming-Hui Chen
      Pages 331-345

About this book


This book provides an overview of the theories and applications on subgroups in the biopharmaceutical industry. Drawing from a range of expert perspectives in academia and industry, this collection offers an overarching dialogue about recent advances in biopharmaceutical applications, novel statistical and methodological developments, and potential future directions.

The volume covers topics in subgroups in clinical trial design; subgroup identification and personalized medicine; and general issues in subgroup analyses, including regulatory ones. Included chapters present current methods, theories, and case applications in the diverse field of subgroup application and analysis. Offering timely perspectives from a range of authoritative sources, the volume is designed to have wide appeal to professionals in the pharmaceutical industry and to graduate students and researchers in academe and government.


subgroup analysis subgroup identification Bayesian statistics precision medicine personalized medicine benefit-risk confirmatory subgroup analysis meta-analysis network meta-analysis pharmaceutical

Editors and affiliations

  • Naitee Ting
    • 1
  • Joseph C. Cappelleri
    • 2
  • Shuyen Ho
    • 3
  • (Din) Ding-Geng Chen
    • 4
  1. 1.Biostatistics & Data SciencesBoehringer Ingelheim CorporationRidgefieldUSA
  2. 2.Pfizer IncGrotonUSA
  3. 3.UCB Biosciences Inc.RaleighUSA
  4. 4.School of Social WorkUniversity of North CarolinaChapel HillUSA

About the editors

Naitee Ting is a fellow of the American Statistical Association (ASA) and a director in the Department of Biostatistics and Data Sciences at Boehringer-Ingelheim Pharmaceuticals, Inc. (BI). Before joining BI in September 2009, he was at Pfizer, Inc. for 22 years (1987-2009).

Joseph C. Cappelleri is an executive director of biostatistics in the Statistical Research and Data Science Center at Pfizer, Inc. As an adjunct professor, he has served on the faculties at Brown University (biostatistics), Tufts Medical Center (medicine), and the University of Connecticut (statistics). He has co-authored approximately 1000 external presentations and 500 publications on clinical and methodological topics, including on regression-discontinuity designs, meta-analysis, and health measurement scales. Dr. Cappelleri is the lead author of the book Patient-Reported Outcomes: Measurement, Implementation and Interpretation (2013, with Zou, Bushmakin, Alvir, Alemayehu, and Symonds), and has co-authored or co-edited two other books (one on Phase II clinical trials, the other on health economics and outcomes research). He is a Fellow of the American Statistical Association.

Shuyen Ho is a lead research biostatistician at UCB BioSciences in Raleigh, North Carolina. Prior to UCB, he was a biostatistics director at PAREXEL and GlaxoSmithKline (GSK), and a group leader and research statistician at Merck. He has been involved in developing allergy, asthma, COPD (chronic obstructive pulmonary disease) and some other rare disease medications. Dr. Ho has also actively served the International Chinese Statistical Association (ICSA) as a board director, symposium and program committee chair, and executive director.

Ding-Geng (Din) Chen is the Wallace H. Kuralt Distinguished Professor and Director of the Consortium for Statistical Development and Consultation (CSDC) in the School of Social Work, and is jointly appointed as a clinical professor in the Department of Biostatistics at the UNC Gillings School of Global Health. He is an elected fellow of American Statistical Association. As a professor in biostatistics, he is interested in developing biostatistical methodologies in clinical trials, meta-analysis, Bayesian statistics and their applications to public health. As a professor in social work, he is interested in developing Bayesian social and health intervention research, cusp catastrophe modelling, statistical causal inferences, propensity score and structural-equation models (SEM). He is PI/Co-PI for several NIH R01 research projects in biostatistical methodology development and public health applications.

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