Biopharmaceutical Applied Statistics Symposium

1. Front Matter

   Pages i-xv

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2. Targeted Learning of Optimal Individualized Treatment Rules Under Cost Constraints

   • Boriska Toth, Mark van der Laan

   Pages 1-22

3. Overview of Omics Biomarker Discovery and Design Considerations for Biomarker-Informed Clinical Trials

   • Weidong Zhang, Bo Huang, Jing Wang, Sandeep Menon

   Pages 23-52

4. Phase 3 Oncology Trials of Personalized Medicines with Adaptive Subpopulation Selection

   • Cong Chen, Wen Li, Xiaoyun (Nicole) Li, Robert A. Beckman

   Pages 53-64

5. High-Dimensional Data in Genomics

   • Dhammika Amaratunga, Javier Cabrera

   Pages 65-73

6. Synergy or Additivity—The Importance of Defining the Primary Endpoint and the Approach to Its Statistical Analysis—A Case Study

   • Bruce E. Rodda

   Pages 75-89

7. Recycling of Significance Levels in Testing Multiple Hypotheses of Confirmatory Clinical Trials

   • Mohammad Huque, Sirisha Mushti, Mohamed Alosh

   Pages 91-118

8. Statistical Testing of Single and Multiple Endpoint Hypotheses in Group Sequential Clinical Trials

   • Mohammad Huque, Sirisha Mushti, Mohamed Alosh

   Pages 119-149

9. Expanded Statistical Decision Rules for Interpretations of Results of Rodent Carcinogenicity Studies of Pharmaceuticals

   • Karl K. Lin, Mohammad A. Rahman

   Pages 151-183

10. A Prematurely Halted, Randomized, Controlled Clinical Trial of Alendronate Treatment in Patients with Gaucher Disease

    • Shumei S. Sun

    Pages 185-191

11. Mediation Modeling in Randomized Trials with Non-normal Outcome Variables

    • Jing Cheng, Stuart A. Gansky

    Pages 193-217

12. Statistical Considerations for Quantitative Imaging Measures in Clinical Trials

    • Ying Lu

    Pages 219-240

13. Interesting Applications from Three Decades of Biostatistical Consulting

    • Karl E. Peace, Uche Eseoghene Okoro, Kao-Tai Tsai

    Pages 241-260

14. Uncovering Fraud, Misconduct, and Other Data Quality Issues in Clinical Trials

    • Richard C. Zink

    Pages 261-276

15. Design and Analysis of Biosimilar Studies

    • Shein-Chung Chow, Fuyu Song

    Pages 277-305

16. Causal Estimands: A Common Language for Missing Data

    • Steven A. Gilbert, Ye Tan

    Pages 307-337

17. Development of Prognostic Biomarker Signatures for Survival Using High-Dimensional Data

    • Richard Simon

    Pages 339-351

18. Validation, Multivariate Modeling, and the Construction of Heat-Map Prediction Matrices for Survival in the Context of Missing Data

    • Shankar S. Srinivasan, Albert Elion-Mboussa, Li Hua Yue

    Pages 353-374

19. Tepee Plots, Graphics for Structured Tabular Data, with Biopharmaceutical Examples

    • Shankar S. Srinivasan, Vatsala Karwe, Li Hua Yue

    Pages 375-395

20. Some Methods for Longitudinal and Cross-Sectional Visualization with Further Applications in the Context of Heat Maps

    • Shankar S. Srinivasan, Li Hua Yue, Rick Soong, Mia He, Sibabrata Banerjee, Stanley Kotey

    Pages 397-420

This BASS book Series publishes selected high-quality papers reflecting recent advances in the design and biostatistical analysis of biopharmaceutical experiments – particularly biopharmaceutical clinical trials.  The papers were selected from invited presentations at the Biopharmaceutical Applied Statistics Symposium (BASS), which was founded by the first Editor in 1994 and has since become the premier international conference in biopharmaceutical statistics. The primary aims of the BASS are: 1) to raise funding to support graduate students in biostatistics programs, and 2) to provide an opportunity for professionals engaged in pharmaceutical drug research and development to share insights into solving the problems they encounter.


The BASS book series is initially divided into three volumes addressing: 1) Design of Clinical Trials; 2) Biostatistical Analysis of Clinical Trials; and 3) Pharmaceutical Applications.  



This book is the third of the 3-volume book series. The topics covered include: Targeted Learning of Optimal Individualized Treatment Rules under Cost Constraints, Uses of Mixture Normal Distribution in Genomics and Otherwise, Personalized Medicine – Design Considerations, Adaptive Biomarker Subpopulation and Tumor Type Selection in Phase III Oncology Trials, High Dimensional Data in Genomics; Synergy or Additivity - The Importance of Defining the Primary Endpoint, Full Bayesian Adaptive Dose Finding Using Toxicity Probability Interval (TPI), Alpha-recycling for the Analyses of Primary and Secondary Endpoints of Clinical Trials, Expanded Interpretations of Results of Carcinogenicity Studies of Pharmaceuticals, Randomized Clinical Trials for Orphan Drug Development,  Mediation Modeling in Randomized Trials with Non-normal Outcome Variables, Statistical Considerations in Using Images in Clinical Trials, Interesting Applications over 30 Years of Consulting, Uncovering Fraud, Misconduct and OtherData Quality Issues in Clinical Trials, Development and Evaluation of High Dimensional Prognostic Models, and Design and Analysis of Biosimilar Studies.

• Optimal Individualized Treatment Rules
• Mixture Normal Distribution
• Personalized Medicine
• Phase III Oncology Genomics Trials
• Defining the Primary Endpoint
• Genomics
• Toxicity Probability Interval
• Alpha-recycling
• Carcinogenicity Studies
• Clinical Trials

• Jiann-Ping Hsu College of Public Health, Georgia Southern University, Statesboro, USA

  Karl E. Peace

• School of Social Work and Gillings School of Global Public Health, University of North Carolina, Chapel Hill, USA

  Ding-Geng Chen

• Boston University, Cambridge, USA

  Sandeep Menon

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