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CC = Colorado Convention Center   H = Hyatt Regency Denver at Colorado Convention Center
* = applied session       ! = JSM meeting theme

Activity Details


Register 1
Sun, 7/28/2019, 12:30 PM - 1:50 PM H-Centennial Ballroom F
SPAIG Committee Speaker with Lunch (Added Fee) — Roundtables Speaker with Lunch
Stats. Partnerships Among Academe Indust. & Govt. Committee, Biopharmaceutical Section, Section on Bayesian Statistical Science
SL01: Considerations in Bayesian Framework Implementation and Impact on Small-Population Clinical Development
Freda Cooner, Amgen
 
 

9 * !
Sun, 7/28/2019, 2:00 PM - 3:50 PM CC-111
Impact of Using Surrogate Endpoints on Drug Development — Invited Papers
WNAR, International Chinese Statistical Association, Biopharmaceutical Section
Organizer(s): Ying Lu, Stanford University
Chair(s): Ying Zhang, University of Nebraska Medical Center
2:05 PM Havrda and Charvat Entropy-Based Measures to Assess Longitudinal Surrogate Endpoints in Clinical Trials
María del Carmen Pardo , Complutense University; Ying Lu, Stanford University; Hua Jin, South China Normal University; Qian Zhao, Guangzhou Medical University
2:25 PM Statistical Considerations for Biomarker-Based Surrogate Endpoints
Marc Buyse, IDDI Inc.
2:45 PM An Information-Theoretic Approach for the Evaluation of Surrogate Endpoints Based on Causal Inference
Presentation
Ariel Alonso Abad, KUleuven
3:05 PM Model Free Approach to Quantifying the Proportion of Treatment Effect Explained by a Surrogate Marker
Presentation
Lu Tian, Stanford University School of Medicine; Tianxi Cai, Harvard University ; Xuan Wang, Harvard University; Layla Parast, RAND
3:25 PM Discussant: Ying Lu, Stanford University
3:45 PM Floor Discussion
 
 

17 * !
Sun, 7/28/2019, 2:00 PM - 3:50 PM CC-104
New Frontiers in Adaptive Clinical Trial Designs — Topic Contributed Papers
Biopharmaceutical Section, Biometrics Section, International Chinese Statistical Association
Organizer(s): Lei Gao, Vertex Pharmaceuticals
Chair(s): Lei Gao, Vertex Pharmaceuticals
2:05 PM A Case Study of Phase II/III Seamless Adaptive Design
Hui Quan, Sanofi US; Yi Xu, Sanofi; Yixin Chen, Sanofi; Lei Gao, Vertex Pharmaceuticals; Xun Chen, Sanofi
2:25 PM Bayesian Adaptive Approach for Neoadjuvant/Adjuvant Oncology Trial
Jing Zhao, Merck Research Labs
2:45 PM Sample Size Re-Estimation in Action: Design Consideration, Charter Development, and Implementation of Analyzes in a Trial with Survival Endpoints
Presentation
Adam Hamm, Cytel, Inc.
3:05 PM Similarity-Based Artificial Intelligence for Adaptive Clinical Trial and Beyond
Mark Chang, Veristat; Susan Hwang, Boston University
3:25 PM An Efficient Sample Size Adaptation Strategy with Adjustment of Randomization Ratio
Yijie Zhou, Vertex
3:45 PM Floor Discussion
 
 

24 * !
Sun, 7/28/2019, 2:00 PM - 3:50 PM CC-102
Emerging Opportunities for Utilizing Real-World Evidence to Impact Drug Development and Regulatory Decision-Making — Topic Contributed Panel
Biopharmaceutical Section, Health Policy Statistics Section, Section on Statistics in Epidemiology
Organizer(s): Joo-Yeon Lee, U.S Food and Drug Administration
Chair(s): Hana Lee, U.S Food and Drug Administration
2:05 PM Emerging Opportunities for Utilizing Real-World Evidence to Impact Drug Development and Regulatory Decision-Making
Presentation 1 Presentation 2 Presentation 3 Presentation 4
Panelists: David Martin , FDA
David Benkeser, Emory
Jessica M Franklin, Brigham and Women’s Hospital and Harvard Medical School
Weili He, AbbVie
3:40 PM Floor Discussion
 
 

32
Sun, 7/28/2019, 2:00 PM - 3:50 PM CC-106
Statistical Methods in Dose-Finding Studies — Contributed Papers
Biopharmaceutical Section, International Society for Bayesian Analysis (ISBA), Section on Bayesian Statistical Science
Chair(s): Kevin Gan, GlaxoSmithKline
2:05 PM Flexible Bayesian Semiparametric Designs for Dose-Finding with Multiple Populations
Presentation
Jianchang Lin, Takeda Pharmaceuticals; Mo Li, Yale University; Rachael Liu, Takeda Pharmaceuticals ; Veronica Bunn, Takeda Pharmaceuticals; Hongyu Zhao, Yale
2:20 PM Design Consideration in Phase 2 Dose Response Trial in the Presence of Possible Non-Monotonicity -- Comparison of Bayesian Emax and NDLM Model
Feng Liu, AstraZeneca; Stephen Walters, University of Sheffield; Steven Julious, University of Sheffield
2:35 PM Bayesian Hierarchical EMAX Model for Dose-Response in Early Phase Efficacy Clinical Trials
Presentation
Byron Gajewski, University of Kansas Medical Center, The University of Kansas Cancer; Caitlyn Meinzer, Medical University of South Carolina; Scott Berry, Berry Consultants; Gayland L Rockswold, Hennepin County Medical Center; William G Barsan, University of Michigan; Frederick K Korley, University of Michigan; Renee' H Martin, Medical University of South Carolina
2:50 PM Bayesian Method Based Dose Escalation in Clinical Trials with Combination Therapy
Presentation
Shanmei Liao, BeiGene; Theis Lange, University of Copenhagen, section of biostatistics
3:05 PM A Comparison of the Up-And-Down or Biased Coin Design to the Continual Reassessment Method for Phase I Dose Finding Studies
Robert A. Perera, VCU Department of Biostatistics; Roy T Sabo, Virginia Commonwealth University; Adam Sima, Virginia Commonwealth University
3:20 PM Bayesian Dose-Finding Model with Adaptive Time-To-Event Weight Incorporating Cycle Information for Immuno-Oncology Studies
Zhaowei Hua, Alnylam Pharmaceuticals, Inc.; Yutong Li, University of Illinois at Urbana-Champaign; Ying Yuan, Takeda Pharmaceutical Company Ltd ; Dan Zhao, Takeda Pharmaceutical Company Ltd
3:35 PM Floor Discussion
 
 

46 * !
Sun, 7/28/2019, 4:00 PM - 5:50 PM CC-103
Recent Developments in Novel Clinical Trial Design and Analysis for Precision Medicine — Invited Papers
ENAR, Biometrics Section, Biopharmaceutical Section
Organizer(s): Yingqi Zhao, Fred Hutchinson Cancer Research Center
Chair(s): Yingqi Zhao, Fred Hutchinson Cancer Research Center
4:05 PM Bayesian Clinical Trial Designs to Evaluate Subgroup-Specific Treatment Effects
Presentation
Peter Thall, U.T. M.D. Anderson Cancer Center; Juhee Lee, University of California, Santa Cruz; Thomas Murray, University of Minnesota; Andrew Chapple, Louisiana State University
4:30 PM Adaptive Contrast Weighted Learning and Tree-Based Reinforcement Learning for Multi-Stage Multi-Treatment Decision-Making
Lu Wang, University of Michigan; Yebin Tao, Google; Daniel Almirall, University of Michigan
4:55 PM Designing Precision Medicine Trials in Oncology to Yield Greater Population Impact
Michael LeBlanc, Fred Hutchinson Cancer Research Center; Yingqi Zhao, Fred Hutchinson Cancer Research Center
5:20 PM Online Experimentation and Learning Algorithms in a Clinical Trial
Presentation
Susan Murphy, Harvard University
5:45 PM Floor Discussion
 
 

55 * !
Sun, 7/28/2019, 4:00 PM - 5:50 PM CC-110
Recent Evaluations of Methods for Handling Noncompliance/Dropouts in Clinical Trials for Better Guidance Driven Application — Invited Papers
Biopharmaceutical Section
Organizer(s): Xiang Zhang, Eli Lilly and Company
Chair(s): Xiang Zhang, Eli Lilly and Company
4:05 PM Principal Component Causal Estimands for Equivalence Trials in the Presence of Intercurrent Events
Wanjie Sun, FDA
4:25 PM An Evaluation of the Trimmed Mean Approach in Clinical Trials with Dropout
Presentation
Ming-Dauh Wang, Regeneron ; Craig Mallinckrodt, Biogen; Jiajun Liu, Biogen
4:45 PM Principal Stratification Approach for Bayesian Sequential Monitoring Design to Address Noncompliance in Clinical Trials
Weining Shen, University of California at Irvine
5:05 PM Test of Treatment Effect for Binary Composite Endpoint with Missing Components in Clinical Trials
Yanyao Yi, University of Wisconsin at Madison; Ting Ye, University of Wisconsin at Madison; Xiang Zhang, Eli Lilly and Company; Junxiang Luo, Sanofi-Aventis
5:25 PM Discussant: Ilya Lipkovich, Eli Lilly and Company
5:45 PM Floor Discussion
 
 

73
Sun, 7/28/2019, 4:00 PM - 5:50 PM CC-112
Alternative Designs and Related Topics — Contributed Papers
Biopharmaceutical Section
Chair(s): John Han
4:05 PM Estimating Responder Status in Sequential Multiple Assignment Randomized Trials (SMARTs)
Keighly Bradbrook, Virginia Commonwealth University; Roy T Sabo, Virginia Commonwealth University
4:20 PM Incorporating Auxiliary Data to Improve Conditional Power Estimation
Jian Zhu, Takeda Pharmaceuticals; Xin Li, George Washington University; Godwin Yung, Takeda Pharmaceuticals; Jianchang Lin, Takeda Pharmaceuticals
4:35 PM Group-Sequential Randomized Trial Design Utilizing the MEM Framework for Incorporating Historical Data
Ales Kotalik, University of Minnesota; David Michael Vock, University of Minnesota; Joseph Koopmeiners, University of Minnesota
4:50 PM Improvement on the Design for the Qualification Phase in Human Abuse Potential Studies
Ling Chen, FDA
5:05 PM Two Novel Non-Parametric Methods for the Analysis of Stepped-Wedge Cluster Randomized Trials
Lee Kennedy-Shaffer, Harvard University; Victor De Gruttola, Harvard T.H. Chan School of Public Health; Marc Lipsitch, Harvard T.H. Chan School of Public Health
5:20 PM Big Stick Design Within Arbitrary Boundaries Minimizes the Selection Bias in an Open-Label Trial
Presentation
Olga Kuznetsova, Merck & Co., Inc.
5:35 PM The Performance of Largest Caliper Matching: An Application to SUPPORT Data
Presentation
Sharif Mahmood
 
 

Register 91
Mon, 7/29/2019, 7:00 AM - 8:15 AM H-Centennial Ballroom G-H
Biopharmaceutical Section A.M. Roundtable Discussion (Added Fee) — Roundtables AM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
ML01: Clinical Trial Design in Alzheimer’s Disease: Innovation, Collaboration, and Bayesian Statistics
Barbara Wendelberger, Berry Consultants, LLC
ML02: Role of Bayesian Methods in Active Control Trial
Samiran Ghosh, Wayne State University; Ram Tiwari, CDRH, FDA
 
 

218848
Mon, 7/29/2019, 7:30 AM - 12:00 PM H-Mineral Hall G
Biopharmaceutical Section Executive Committee Meeting (Closed) — Other Cmte/Business
Biopharmaceutical Section
Chair(s): Richard Zink, TARGET PharmaSolutions
 
 

100 * !
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-709
Pragmatic Randomized Clinical Trials: Challenges and Impact on Clinical Practice and Health Policies — Invited Papers
Health Policy Statistics Section, Biopharmaceutical Section, Section on Statistics in Marketing
Organizer(s): Valentina Bayer, Boehringer Ingelheim
Chair(s): Victoria Gamerman, Boehringer Ingelheim
8:35 AM Key Elements in the Design of Pragmatic Randomized Clinical Trials
Presentation
Valentina Bayer, Boehringer Ingelheim
9:00 AM Estimating the Per-Protocol Effect in Pragmatic Trials
Miguel Hernan, Harvard University
9:25 AM Generalization of Randomized Trial Results with Latent Motivation Effect
Andrea B Troxel, NYU School of Medicine; Chenxiang Li, NYU School of Medicine
9:50 AM Data Sources Used in Pragmatic Clinical Trials: How Do the Puzzle Pieces Fit Together?
Vincent Willey, HealthCore
10:15 AM Floor Discussion
 
 

111 * !
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-102
Evidence Beyond Traditional Clinical Trials — Invited Papers
Section on Medical Devices and Diagnostics, Biopharmaceutical Section, Health Policy Statistics Section
Organizer(s): Martin Ho, FDA
Chair(s): Chava Zibman, FDA Center for Devices and Radiological Health
8:35 AM Recent Statistical Developments in Considering Real World Evidence for Regulatory Decisioin Making
Martin Ho, FDA; Weili He, AbbVie
8:55 AM Propensity Score-Integrated Approaches for Incorporating Real-World Evidence in Clinical Studies
Presentation
Chenguang Wang, John Hopkins University
9:15 AM External Evidence: Latest Developments from the Eponymous Medical Device Innovation Consortium Working Group
Presentation
Theodore Lystig, Medtronic
9:35 AM Use of Past Control Observations Within a Perpetual Platform Trial
Presentation
Kert Viele, Berry Consultants; Scott Berry, Berry Consultants
9:55 AM Discussant: Telba Irony, FDA CBER
10:15 AM Floor Discussion
 
 

114 * !
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-108
Applying the ICH E9(R1) Addendum: Practical Considerations in Choosing Estimands, Estimators, and Sensitivity Analyzes — Topic Contributed Papers
Biopharmaceutical Section, ENAR, WNAR
Organizer(s): Pilar Lim, PhD, Janssen Research & Development, LLC
Chair(s): Pilar Lim, PhD, Janssen Research & Development, LLC
8:35 AM Descending from the Theoretical to the Practical: Our Journey from Clear Definition to Messy Inference and Back
Presentation
Michael O'Kelly, IQVIA
8:55 AM How Should We Select and Define Trial Estimands? – Examples Based on a Disease State
Presentation
Elena Polverejan, Johnson & Johnson-Janssen R&D
9:15 AM Implementation of Estimand Framework in Oncology Clinical Trials
Presentation
Kalyanee Appanna, Novartis Pharmaceutical Corporation; Bharani Dharan, Novartis Pharmaceuticals; Yuanbo Song, Novartis Pharmaceutical Corporation; Ekkehard Glimm, Novartis Pharma AG
9:35 AM Lessons Learned from Implementing ICH E9 in Phase 2 Trials Across Multiple Therapeutic Areas
Presentation
Jared Christensen, Pfizer Research
9:55 AM Discussant: Craig Mallinckrodt, PhD, Biogen
10:15 AM Floor Discussion
 
 

115 * !
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-201
Novel Statistical Methods for Emerging Problems in Modern Clinical Trials and Drug Development — Topic Contributed Papers
Biopharmaceutical Section, International Chinese Statistical Association, Biometrics Section
Organizer(s): Yuan Ji, The University of Chicago
Chair(s): Inna Perevozskaya, GSK
8:35 AM Optimal Selection Procedures and Adaptive Designs for Seamless Phase 2/3 Clinical Trials
Presentation
Vladimir Dragalin, Janssen R&D
8:55 AM Master Protocol and Designs for Setting Where Randomized Controlled Trials Are Not Feasible*
Sue-Jane Wang, Center for Drug Evaluation and Research U.S. Food and Drug Administration
9:15 AM A Unified Framework for Time-To-Toxicity Dose-Finding Designs in Immune and Non-Immune Clinical Trials
Presentation
Yuan Ji, The University of Chicago; Tianjian Zhou, The University of Chicago
9:35 AM Bayesian Models for Precision Oncology Clinical Trials
Presentation
Peter Müller, University of Texas Austin; Yanxun Xu, Johns Hopkins University; Don Berry, MDACC; Apostolia Tsimberidou, MDACC
9:55 AM Robust Clinical Trial Design and Analysis When Non-Proportional Hazards Are Likely
Keaven Anderson, Merck & Company, Inc.
10:15 AM Floor Discussion
 
 

118 * !
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-107
Emerging Challenges in Precision Medicine — Topic Contributed Papers
Biometrics Section, Biopharmaceutical Section, ENAR
Organizer(s): Li Ma, Duke University
Chair(s): David Banks, SAMSI/Duke University
8:35 AM Knockoff Assisted Outcome Adaptive Lasso for Doubly Robust Treatment Effect Estimation
Guanhua Chen, University of Wisconsin-Madison
8:55 AM Variable Selection and Estimation in Causal Inference Using Bayesian Spike and Slab Priors
David Michael Vock, University of Minnesota; Brandon Koch, University of Nevada Reno; Julian Wolfson, University of Minnesota
9:15 AM Sparse Learning and Structure Identification for Ultra-High-Dimensional Image-On-Scalar Regression
Xinyi Li, SAMSI; Li Wang, Iowa State University; Huixia Judy Wang, The George Washington University
9:35 AM Characterizing Outcome Distributions of Dynamic Treatment Regimes
Presentation
Daniel Lizotte, The University of Western Ontario
9:55 AM Single-Cell Analyzes for Developing HIV Vaccine
Lynn Lin, Penn State University
10:15 AM Floor Discussion
 
 

128
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-103
SPEED: Biometrics and Biostatistics Part 1 — Contributed Speed
Biometrics Section, Section on Statistics in Epidemiology, Biopharmaceutical Section, Section on Bayesian Statistical Science
Chair(s): Caroline Ledbetter, University of Colorado
Poster Presentations for this session.
8:35 AM Oversampling and Replacement Strategies in Propensity Score Matching: a Critical Review Focused on Small Samples
Presentation
Daniele Bottigliengo, University of Padova; Ileana Baldi, University of Padova; Corrado Lanera, University of Padova; Jonida Bejko, University of Brescia; Tomaso Bottio, University of Padova; Vincenzo Tarzia, University of Padova; Massimiliano Carrozzini, University of Padova; Gino Gerosa, University of Padova; Paola Berchialla, University of Torino; Dario Gregori, University of Padova
8:40 AM A Concordance Statistic for Survival Analysis with a Censored Predictor
Presentation
Kai Ding, University of Oklahoma Health Sciences Center; Justin Dvorak, University of Oklahoma Health Sciences Center
8:45 AM Meta-Analysis of Binary Outcomes Combining Individual Patient Data and Aggregate Data
Presentation
Neha Agarwala, University of Maryland - Baltimore County; Anindya Roy, University of Maryland - Baltimore County
8:50 AM Multiplicity Adjustment in Clinical Trials
Presentation
Michael Proschan, National Institute of Allergy and Infectious Diseases; Erica Brittain, National Institute of Allergy and Infectious Diseases
8:55 AM Hierarchical Likelihood Approach for Joint Models of Longitudinal Non-Survival Responses and Survival Data: a Semiparametric Model with Gamma Shared Random Effects
Presentation 1 Presentation 2 Presentation 3 Presentation 4
Karl Stessy Bisselou, University of Nebraska Medical Center; Hongying Dai, University of Nebraska Medical Center; Gleb Haynatzki, University of Nebraska Medical Center
9:00 AM A Scalable Algorithm for Joint Modeling of Longitudinal and Competing Risks Time-To-Event Data
Presentation
Shanpeng Li, UCLA Department of Biostatistics; Eric Kawaguchi, UCLA Department of Biostatistics; Gang Li, UCLA
9:05 AM Synthetic Data Method to Incorporate External Information into a Current Study
Presentation
Tian Gu, University of Michigan; Jeremy Taylor, University of Michigan; Bhramar Mukherjee, University of Michigan
9:10 AM Predicting the Cross-Validated Penalty Parameter in Nodewise Lasso Regression
Presentation
Mo Huang, University of Pennsylvania; Nancy Zhang, University of Pennsylvania
9:15 AM Statistical Assessment of Bovine Body Weight via Functional Gait Data
Andrew Raim, US Census Bureau; Nagaraj Neerchal, University of Maryland, Baltimore County; Dan Tasch, Step Analysis LLC; Uri Tasch, Step Analysis LLC
9:20 AM Adaptive Design with Biomarker Population Deselection and Enrichment for Oncology Trials
Pingye Zhang; Yue Shentu, Merck & Co., Inc.; Qi Liu, Merck & Co., Inc.
9:30 AM Unblinded Sample Size Re-Estimation for Ordinal Data
Huaihou Chen, Biogen; Ray Zhang, Biogen; Weihua Tang, Biogen; Li Zhu, Biogen; Chunlei Ke, Biogen
9:35 AM A Natural Lead-In Approach to Response-Adaptive Allocation
Erin Donahue, Virginia Commonwealth University; Roy T Sabo, Virginia Commonwealth University
9:40 AM Bayesian Analysis of Mixed Continuous and Time-To-Event Outcomes with Latent Variables
Presentation
Xinyuan Song, The Chinese University of Hong Kong; Deng Pan, Huazhong University of Science and Technology
9:45 AM Survival Analyzes in the Presence of Unadjudicated Events
Presentation
Rakhi Kilaru, Pharmaceutical Product Development; Andrew Montgomery Hartley, Pharmaceutical Product Development
9:50 AM A Comparison of Stacked and Pooled Multiple Imputation
Presentation
Paul Bernhardt, Villanova University
9:55 AM Design of a Phase 3 Trial for an Acute Treatment of a Rare Disease with Episodic Attacks
Presentation
Sharon Murray
10:00 AM Quantitative Decision Making (QDM) in Phase I/II Studies
Presentation 1 Presentation 2
Kevin Gan, GlaxoSmithKline; Jonathan Haddad , GlaxoSmithKline
10:05 AM Bayesian Modeling of Rare Events with Informative Censoring in Meta-Analysis
Xinyue Qi, UT MD Anderson Cancer Center; Yucai Wang, Mayo Clinic; Chan Shen, College of Medicine, Penn State University; Michael Wang, The University of Texas MD Anderson Cancer Center; Shouhao Zhou, PennState College of Medicine
10:10 AM A Bayesian Approach with Propensity Score for Confounding Control with Case Study in Non-Medical Switch Real World Observational Studies
Zhenyi Xue, AbbVie; Hongwei Wang, AbbVie Inc.
10:15 AM Floor Discussion
 
 

130
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-112
Statistical Methods for Time-To-Event Data and Applications — Contributed Papers
Biopharmaceutical Section
Chair(s): Ruvie Martin, Novartis Pharmaceuticals
8:35 AM Tools to Compare Restricted Mean Survival Times in Randomized Controlled Studies with Small Sample Data
Miki Horiguchi, Kitasato University; Hajime Uno, Dana-Farber Cancer Institute
8:50 AM Survival Analysis in the Absence of Proportional Hazards: Defining the Relevant Null Hypothesis
Presentation
Steven Snapinn, Alder Biopharmaceuticals; Qi Jiang, Seattle Genetics
9:05 AM Modeling the Impact of Dose Intervention on Time-To-Event Outcomes
Amir Nikooienejad, Eli Lilly and Company; Yongming Qu, Eli Lilly and Company
9:20 AM A Flexible Parametric Survival Model for Fitting Time-to-Event Data in Clinical Trials
Presentation
Jason Liao, Merck & Co. Inc.; Frank G Liu, Merck Sharp & Dohme Inc.
9:35 AM Teasing Out the Overall Survival Benefit with Adjustment for Treatment Switching to Other Therapies
Presentation
Meijing Wu, AbbVie; Weili He, AbbVie; Qiming Liao, VIIV Health Care; Yabing Mai, AbbVie
9:50 AM An Approach to Increase Power in Immuno-Oncology Trials When Non-Proportional Hazard Is Present
Presentation
Nan Jia, Sanofi US
10:05 AM Evaluating Methods for Correcting the Impact of Treatment Switching on Overall Survival
Jin Zhang
 
 

131
Mon, 7/29/2019, 8:30 AM - 10:20 AM CC-101
Topics in Clinical Trials — Contributed Papers
Biopharmaceutical Section
Chair(s): Guanglei Yu, Eli Lilly and Company
8:35 AM Methods for Evaluating Heterogeneity in Treatment Effects in a Randomized Clinical Trial
Alok Dwivedi, Texas Tech University Health Sciences Center El Paso; Muditha Perera, Texas Tech University Health Sciences Center El Paso; Sada Nand Dwivedi, All India Institute of Medical Sciences; Rakesh Shukla, University of Cincinnati
8:50 AM Estimation of Treatment Effect in a Multi-Regional Clinical Trial with Survival Endpoint
Hsiao-Hui Tsou, National Health Research Institutes; Yu-Chieh Cheng, National Health Research Institutes; Yuh-Jeng Wu, Chung Yuan Christian University; Chin-Fu Hsiao, National Health Research Institutes
9:05 AM Challenges of Filing Externally Conducted Clinical Trials
Presentation 1 Presentation 2
Kenneth Liu, Merck & Co., Inc; Jonathan Hartzel, Merck & Co., Inc
9:20 AM An Integrative Shrinkage Estimator for Random-Effects Meta-Analysis of Rare Binary Events
Presentation
Lie Li, Merck & Co.; Xinlei Wang, Southern Methodist University; Ou Bai, NA
9:35 AM Exploring Heterogeneity of Treatment Response: Assumptions, Logic, Algorithm, Computations
Presentation
Lev Sverdlov, Redmond Analytics, LLC
9:50 AM Dynamic Data Monitoring for On-Going Clinical Trials
Tai Xie, Brightech International; Ping Gao, Brightech International; Peng Zhang, Brightech International; Yue Tu, Brightech International; Joe Shih, Rutgers University
10:05 AM Blinded Safety Monitoring in Clinical Trials and IND Safety Reporting: Challenges and Lessons Learned
Presentation
Barbara Hendrickson, AbbVie
 
 

Register CE_15C
Mon, 7/29/2019, 8:30 AM - 5:00 PM CC-403/404
Analysis of Clinical Trials: Theory and Applications (ADDED FEE) — Professional Development Continuing Education Course
ASA, Biopharmaceutical Section
Instructor(s): Devan Mehrotra, Merck & Co., Inc; Alex Dmitrienko, Mediana Inc; Jeff Maca, Bayer Pharmaceuticals
The course covers seven important topics that commonly face statisticians and research scientists conducting clinical research: stratified trials, longitudinal trials with dropouts, time-to-event trials with small sample sizes, crossover trials, pharmacogenomics studies for personalized medicine, multiple comparisons, and interim decision making with adaptive designs. The course offers a well-balanced mix of theory and applications. It presents practical advice from experts and discusses regulatory considerations. The discussed statistical methods will be implemented using SAS and R software. Clinical trial examples will be used to illustrate the statistical methods. The course is designed for statisticians working in the pharmaceutical or biotechnology industries as well as contract research organizations. It is equally beneficial to statisticians working in institutions that deliver health care, government branches that conduct health-care related research and academics interested in learning about contemporary statistical topics in clinical drug development. The attendees are required to have a basic knowledge of clinical trials. Familiarity with drug development is highly desirable, but not necessary. This course was taught at JSM 2005-2018 and received the Excellence in Continuing Education Award in 2005.
8:30 AM Analysis of Clinical Trials: Theory and Applications (ADDED FEE)
Alex Dmitrienko, Mediana Inc; Devan Mehrotra, Merck & Co., Inc; Jeff Maca, Bayer Pharmaceuticals
 
 

148 * !
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-201
Statistical Methods, Challenges and Impacts on Early Phase Trials — Invited Papers
ENAR, Biopharmaceutical Section, Statistics in Biopharmaceutical Research Journal
Organizer(s): Yu Du, Eli Lilly and Company
Chair(s): Pandurang Kulkarni, Eli Lilly & Company
10:35 AM Model-Based Phase I Designs for Immuno-Oncology
Jun Yin, Mayo Clinic; Yu Du, Eli Lilly and Company; Sumithra Mandrekar, Mayo Clinic
10:55 AM The Use of Bayesian Basket Design in Early Phase Trials
Presentation
Shiling Ruan, Novartis; Matt Whiley, Novartis
11:15 AM Advancing Pharmacogenomics Analysis of Drug Response in Early-Phase Clinical Trials
Presentation
Judong Shen, Merck & Co., Inc.; Hong Zhang, Merck & Co., Inc.; Devan Mehrotra, Merck & Co., Inc
11:35 AM Revolutionizing the Early Drug Development
Yongming Qu, Eli Lilly and Company
11:55 AM Discussant: Ying Yuan, University of Texas M.D. Anderson Cancer Center
12:15 PM Floor Discussion
 
 

155 * !
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-107
Research Reproducibility for Precision Medicine: From Controlled Experiments to Real-World Evidence — Topic Contributed Papers
Biopharmaceutical Section, Biometrics Section, Government Statistics Section
Organizer(s): Dong Wang, FDA National Center for Toxicological Research (NCTR); Wei Vivian Zhuang, U.S. Food and Drug Administration
Chair(s): Dong Wang, FDA National Center for Toxicological Research (NCTR)
10:35 AM Make Genomics Reproducible Again – MAQC and Beyond
Presentation
Weida Tong, FDA National Center for Toxicological Research (NCTR); Zhihua Xu, U.S. Food and Drug Administration
10:55 AM On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Gene Therapy Trials
Presentation
Qing Liu, Quantitative and Regulatory Medical Science, LLC
11:15 AM Reproducible Evidence: Practices to Enhance and Achieve Transparency of “Real World” Evidence from “Real World” Databases
Shirley Wang
11:35 AM A Nonparametric Statistical Method for More Reproducible Biomarker Detection
Presentation
Wei Zhuang, NCTR/U.S. FDA; Luísa Camacho, NCTR/U.S. FDA; Camila Silva, NCTR/U.S. FDA; Huixiao Hong, NCTR/U.S. FDA
11:55 AM R Markdown: a Software Ecosystem for Reproducible Publications
Yihui Xie, RStudio, Inc.
12:15 PM Floor Discussion
 
 

164 * !
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-112
FDA Adaptive Designs and Master Protocols Guidance for Clinical Trials – Reflection and Outlook — Topic Contributed Papers
Biopharmaceutical Section, Section on Bayesian Statistical Science, Biometrics Section
Organizer(s): Aijun Gao, Covance/Chiltern; Fanni Natanegara, Eli Lilly and Company; Shiling Ruan, Novartis
Chair(s): Aijun Gao, Covance/Chiltern
10:35 AM How the 2018 FDA Adaptive Design Draft Guidance Can Help to Increase the Use of Adaptive Designs in Industry
Presentation
David Manner, Eli Lilly & Company
10:55 AM Increasing Efficiency of Oncology POC Studies Using Bayesian Adaptive Approach
Rong Liu, Celgene Co.
11:15 AM Finding a Balance of Synergy and Flexibility in Master Protocols
Presentation
Melanie Quintana, Berry Consultants; Scott Berry, Berry Consultants
11:35 AM Points to Consider in the Design of Adaptive Platform Clinical Trials in Non-Alcoholic Steatohepatitis
Presentation
Peter Mesenbrink, Novartis Pharmaceuticals
11:55 AM Discussant: Telba Irony, FDA CBER
12:15 PM Floor Discussion
 
 

166 * !
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-703
New Developments for Using R in the Biopharmaceutical Industry — Topic Contributed Panel
Section for Statistical Programmers and Analysts, Biopharmaceutical Section, Section on Statistical Learning and Data Science
Organizer(s): Kuolung Hu, Ionis Pharmaceuticals, Inc.
Chair(s): Marianne Miller, Eli Lilly and Company
10:35 AM Open-Source Tools for Monitoring Clinical Trial Safety – Taking it to the Next Level Towards Cross-Functional Collaboration
Presentation 1 Presentation 2 Presentation 3
Panelists: Eric Nantz, Eli Lilly
Jeremy Wildfire, RHO, Inc
Min Lee, Amgen
Paul Schuette, FDA
Satha Thill, AbbVie
12:10 PM Floor Discussion
 
 

170
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-105
SPEED: Biopharmaceutical Methods and Application I, Part 1 — Contributed Speed
Biopharmaceutical Section
Chair(s): Sarah Ryan
Poster Presentations for this session.
10:35 AM Bayesian Leveraging of Historical and Concurrent Data to Assess the Contribution of a New Molecular Entity with a Delayed Effect in a Combination Survival Trial
Samson Ghebremariam, Novartis Pharmaceutical Corporation; Lisa Hampson, Novartis Pharmaceutical Corporation; Amy Racine-Poon, Novartis Pharmaceutical Corporation; Beat Neuenschwander, Novartis Pharmaceutical Corporation; Bharani Dharan, Novartis Pharmaceuticals; Kalyanee Appanna, Novartis Pharmaceutical Corporation
10:40 AM Design of Clinical Trials for Bivariate Endpoints
Presentation 1 Presentation 2
Junxiao Hu, University of Colorado; Patrick Blatchford, University of Colorado; John Kittelson, University of Colorado
10:45 AM Bayesian Modeling in Historical Data Borrowing on Controls in Clinical Trials
Presentation
Zhuqing Yu, AbbVie Inc.; Zailong Wang, AbbVie Inc.; Lanju Zhang
10:50 AM Analysis Methods for Skewed Data Distributions
Annpey Pong
10:55 AM Identification of Potential Predictive Biomarker Candidates Through Strategic Analysis of Cytokine Profiles Across Multiple Anti-PD-1 Clinical Trials
Jeea Choi, Novartis; Ying Amanda Wang, Novartis; John Millholland, Novartis; Albert Reising, Novartis; Jan Christoph Brase, Novartis; Xiaoshan Wang, Novartis; Connie Wong, Novartis; Kitty Wan, Novartis; Yiqun Yang, Novartis; Gullu Gorgun, Novartis; Parul Patel, Novartis; Hemant Patel, Novartis
11:00 AM Precise and Accurate Power of the Rank-Sum Test for a Continuous Variable
Presentation
Katie Rose Mollan, University of North Carolina Chapel Hill; Ilana Trumble, University of Colorado Denver; Sarah Reifeis, University of North Carolina at Chapel Hill; Orlando Ferrer, University of North Carolina Chapel Hill; Camden P Bay, Harvard Medical School; Pedro L. Baldoni, University of North Carolina At Chapel Hill; Michael Hudgens, University of North Carolina at Chapel Hill
11:05 AM Reducing Misclassification Effect on Dynamic Treatment Regimen (DTR) of Sequential Multiple Assignment Randomized Trial Designs (SMART)
Jun He, Virginia Commonwealth University; Roy T Sabo, Virginia Commonwealth University; Donna McClish, VCU
11:10 AM Simple Adjustment for Bias Due to Unobserved Confounding
Yiran (Bonnie) Hu, AbbVie; Hui Xie, University of Illinois at Chicago
11:15 AM Umbrella and Platform Trials: Statistical Considerations on Efficiencies and a Case Study
Presentation
Xiaoyun (Nicole) Li, Merck; Cong Chen, Merck & Co., Inc; Fang Liu, Merck; Wen Li, Merck
11:20 AM Flexible Semiparametric Bayesian Hierarchical Model for Basket Trials
Veronica Bunn, Takeda Pharmaceuticals; Jianchang Lin, Takeda Pharmaceuticals; Rachael Liu, Takeda Pharmaceuticals
11:30 AM Evaluating the “One-Model Fits All” Approach for Modeling Clinical Trial Adverse Events
Presentation
Stephanie Pan
11:35 AM How Many Imputations Are Enough When Reporting Clinical Trials?
Anders Gorst-Rasmussen, Novo Nordisk A/S
11:40 AM Meta-Analysis of Longitudinal Preclinical Efficacy Screens
William Forrest, Genentech, Inc; Bruno Alicke, Genentech; Magdalena Osinska, Genentech; Shannon Ruppert, Genentech; Michal Jakubczak, Roche; Pawel Piatkowski, Roche
11:45 AM The Application of Beta Regression for Modeling a Covariate Adjusted ROC
Presentation
Xing Meng, Baylor University; Jack D. Tubbs, Baylor University
11:50 AM Examining the Replication Crisis: The Effect of Underpowered Studies and Publication Bias
Christine M. Orndahl, Virginia Commonwealth University Dept of Biostatistics; Robert A. Perera, VCU Department of Biostatistics
11:55 AM Comparison of Bayesian Network Meta-Analysis Models for Survival Data
Presentation
Purvi Prajapati, Baylor University; James D Stamey, Baylor University; John Seaman, Baylor University; Michael Sonksen, Eli Lilly & Co.; Min-Hua Jen, Eli Lilly & Co.
12:00 PM Advantages of Parallel Design Over Crossover Design in the Study on Effects of Cannabis on Driving in Healthy Adults
Anya Umlauf, UC San Diego; Barth Wilsey, UC San Diego; Thomas Marcotte, UC San Diego; Florin Vaida, UC San Diego
12:05 PM Probability of Undetectable Error in Independent Dual Programming Validation for Analysis Results in Clinical Trials
Presentation 1 Presentation 2
Long Zheng, Takeda Pharmaceutical
12:10 PM An Extension of Cohen’s Kappa for Clustered Data and Group Sequential Testing
Presentation
Mary Ryan, University of California, Irvine; Daniel L. Gillen, University of California, Irvine
12:15 PM Optimal Design and Analysis of Efficacy Expansion in Phase I Oncology Trials
Presentation
Iris Wu, Merck & Co.; Fang Liu, Merck; Heng Zhou, Merck & Co., Inc; Cong Chen, Merck & Co., Inc
 
 

172
Mon, 7/29/2019, 10:30 AM - 12:20 PM CC-109
Quantitative Decision Making in Clinical Trials — Contributed Papers
Biopharmaceutical Section
Chair(s): Xiao Fang, Merck
10:35 AM Defensive Efficacy Interim Design: Dynamic Benefit/Risk Ratio View Using Probability of Success
Zhongwen Tang, Abbvie
10:50 AM In Silico Clinical Trials : a Way to Improve Clinical Development?
Presentation
Nicolas SAVY, Toulouse Institute of Mathematics; Philippe SAINT-PIERRE, Toulouse Institute of Mathematics ; Stephanie SAVY, ESTRIALS; Emmanuel PHAM, IPSEN Innovation SAS
11:05 AM Single-Arm Two- and Three-Stage Phase 2 Clinical Trials with Go/No-Go/Inconclusive Outcomes with Handling of Overrunning/Under-Running
Presentation
Bob Zhong, Johnson and Johnson; Wenchuan Guo, Bristol-Myers Squibb Company; Jianan Hui, Boehringer Ingelheim Pharmaceuticals Inc.
11:20 AM Quantitative Decision-Making for Single Arm POC Studies in Early Phase Oncology
Zhuqing Tina Liu, Eli Lilly and Company; Jingyi Liu, Eli Lilly and Company
11:35 AM Quantitative Decision Making in Early Clinical Development – Some Statistical Considerations
Weidong Zhang, Pfizer
11:50 AM Bayesian Interim Prediction of Probability of Clinical Trial Success
Ying Grace Li, Eli Lilly and Company
12:05 PM Infusing Bayesian Strategies for Pharmaceutical Manufacturing and Development
Presentation
Bill Pikounis, Johnson & Johnson; Dwaine Banton, Janssen R&D; John Oleynick, Johnson & Johnson; Jyh-Ming Shoung, Janssen R&D
 
 

196
Mon, 7/29/2019, 11:35 AM - 12:20 PM CC-Hall C
SPEED: Biometrics and Biostatistics Part 2 — Contributed Poster Presentations
Biometrics Section, Section on Statistics in Epidemiology, Biopharmaceutical Section, Section on Bayesian Statistical Science
Chair(s): Wendy Meiring, University of California At Santa Barbara
Oral Presentations for this session.
21: Oversampling and Replacement Strategies in Propensity Score Matching: a Critical Review Focused on Small Samples
Daniele Bottigliengo, University of Padova; Ileana Baldi, University of Padova; Corrado Lanera, University of Padova; Jonida Bejko, University of Brescia; Tomaso Bottio, University of Padova; Vincenzo Tarzia, University of Padova; Massimiliano Carrozzini, University of Padova; Gino Gerosa, University of Padova; Paola Berchialla, University of Torino; Dario Gregori, University of Padova
22: A Concordance Statistic for Survival Analysis with a Censored Predictor
Kai Ding, University of Oklahoma Health Sciences Center; Justin Dvorak, University of Oklahoma Health Sciences Center
23: Meta-Analysis of Binary Outcomes Combining Individual Patient Data and Aggregate Data
Neha Agarwala, University of Maryland - Baltimore County; Anindya Roy, University of Maryland - Baltimore County
24: Multiplicity Adjustment in Clinical Trials
Michael Proschan, National Institute of Allergy and Infectious Diseases; Erica Brittain, National Institute of Allergy and Infectious Diseases
25: A Comparison of Stacked and Pooled Multiple Imputation
Paul Bernhardt, Villanova University
26: Hierarchical Likelihood Approach for Joint Models of Longitudinal Non-Survival Responses and Survival Data: a Semiparametric Model with Gamma Shared Random Effects
Karl Stessy Bisselou, University of Nebraska Medical Center; Hongying Dai, University of Nebraska Medical Center; Gleb Haynatzki, University of Nebraska Medical Center
27: A Scalable Algorithm for Joint Modeling of Longitudinal and Competing Risks Time-To-Event Data
Shanpeng Li, UCLA Department of Biostatistics; Eric Kawaguchi, UCLA Department of Biostatistics; Gang Li, UCLA
28: Synthetic Data Method to Incorporate External Information into a Current Study
Tian Gu, University of Michigan; Jeremy Taylor, University of Michigan; Bhramar Mukherjee, University of Michigan
29: Predicting the Cross-Validated Penalty Parameter in Nodewise Lasso Regression
Mo Huang, University of Pennsylvania; Nancy Zhang, University of Pennsylvania
30: Statistical Assessment of Bovine Body Weight via Functional Gait Data
Andrew Raim, US Census Bureau; Nagaraj Neerchal, University of Maryland, Baltimore County; Dan Tasch, Step Analysis LLC; Uri Tasch, Step Analysis LLC
31: Adaptive Design with Biomarker Population Deselection and Enrichment for Oncology Trials
Pingye Zhang; Yue Shentu, Merck & Co., Inc.; Qi Liu, Merck & Co., Inc.
32: Unblinded Sample Size Re-Estimation for Ordinal Data
Huaihou Chen, Biogen; Ray Zhang, Biogen; Weihua Tang, Biogen; Li Zhu, Biogen; Chunlei Ke, Biogen
34: A Natural Lead-In Approach to Response-Adaptive Allocation
Erin Donahue, Virginia Commonwealth University; Roy T Sabo, Virginia Commonwealth University
35: Survival Analyzes in the Presence of Unadjudicated Events
Rakhi Kilaru, Pharmaceutical Product Development; Andrew Montgomery Hartley, Pharmaceutical Product Development
36: Design of a Phase 3 Trial for an Acute Treatment of a Rare Disease with Episodic Attacks
Sharon Murray
37: Bayesian Modeling of Rare Events with Informative Censoring in Meta-Analysis
Xinyue Qi, UT MD Anderson Cancer Center; Yucai Wang, Mayo Clinic; Chan Shen, College of Medicine, Penn State University; Michael Wang, The University of Texas MD Anderson Cancer Center; Shouhao Zhou, PennState College of Medicine
38: Bayesian Analysis of Mixed Continuous and Time-To-Event Outcomes with Latent Variables
Xinyuan Song, The Chinese University of Hong Kong; Deng Pan, Huazhong University of Science and Technology
39: A Bayesian Approach with Propensity Score for Confounding Control with Case Study in Non-Medical Switch Real World Observational Studies
Zhenyi Xue, AbbVie; Hongwei Wang, AbbVie Inc.
40: Quantitative Decision Making (QDM) in Phase I/II Studies
Kevin Gan, GlaxoSmithKline; Jonathan Haddad , GlaxoSmithKline
Oral Presentations for this session.
 
 

Register 198
Mon, 7/29/2019, 12:30 PM - 1:50 PM
WITHDRAWN: Biopharmaceutical Section P.M. Roundtable Discussion (Added Fee) — Roundtables PM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
ML09: WITHDRAWN: Prediction Models and Data Visualization in Electronic Health Records
Jagadish Gogate, Johnson & Johnson-Janssen R&D; CV Damaraju, Johnson & Johnson - Janssen R&D
 
 

Register CE_19C
Mon, 7/29/2019, 1:00 PM - 5:00 PM CC-405
Futility Analyzes in Confirmatory Clinical Trials – Methods and Procedures (ADDED FEE) — Professional Development Continuing Education Course
ASA, Biopharmaceutical Section
Instructor(s): Satrajit Roychoudhury, Pfizer Inc ; Paul Gallo, Novartis Pharmaceutical
Futility analyses (FA) are increasingly utilized in clinical trials. FA generally involves interim evaluation of a trial’s primary hypothesis to determine if trial success or clinically meaningful effect seems unlikely. FA can improve resource efficiency by halting trials with ineffective intervention. They also offer ethical advantages by exposing fewer trial participants to ineffective and possibly toxic interventions, and public health advantages in that trial results may be conveyed to the medical community in a more timely fashion. FA should be carefully planned during trial design phase, as there are important statistical and operational consequences. Concerns include the control of statistical error rates and the potential for operational bias resulting from interim evaluations. Challenging questions arise during trial design regarding how FA should be conducted, when futility should be assessed, role of Data Monitoring Committees and thresholds at which futility may be established. Non-constancy of effect size and other limitations of accruing interim data can raise further challenges. In this course, we will describe current practices and recent advancement in methodological approaches of FA with case studies. We describe what FA are, why they are conducted, where and when they should be considered, and how they should be methodologically and operationally performed.
1:00 PM Futility Analyzes in Confirmatory Clinical Trials – Methods and Procedures (ADDED FEE)
Paul Gallo, Novartis Pharmaceutical; Satrajit Roychoudhury, Pfizer Inc
 
 

223 * !
Mon, 7/29/2019, 2:00 PM - 3:50 PM CC-703
The P-Value Controversy: Where Do We Go from Here? — Invited Panel
Biopharmaceutical Section, Biometrics Section, ENAR
Organizer(s): Pranab K Mitra, Merck
Chair(s): Pranab K Mitra, Merck
2:05 PM The P-Value Controversy: Where Do We Go from Here?
Presentation 1 Presentation 2 Presentation 3 Presentation 4
Panelists: David Gal, University of Illinois at Chicago
Jeffrey Blume, Vanderbilt University
Lisa Strug
Michael Lavine, University of Massachusetts, Amherst
Philip B. Stark, UC Berkeley
Xihong Lin, Harvard
3:40 PM Floor Discussion
 
 

240
Mon, 7/29/2019, 2:00 PM - 3:50 PM CC-705
Topics in Multiplicity and Control of False Discovery Rate — Contributed Papers
Biopharmaceutical Section
Chair(s): Huichao Chen, Harvard University
2:05 PM Optimizing Graphical Procedures for Multiplicity Control in a Confirmatory Clinical Trial via Deep Learning
Tianyu Zhan, Immunology, DSS, AbbVie; Alan Hartford, Takeda Pharmaceutical Company; Jian Kang, University of Michigan; Walt Offen, Retired
2:20 PM Application of Discrete False Discovery Rate Controlling Procedures in Clinical Safety Evaluations
Li He, Merck Research Laboratories; Joe Heyse, Merck
2:35 PM Credible Subgroups for Identifying Benefiting Populations with Time-To-Event Data
Presentation
Duy Ngo; Richard Baumgartner, Merck Research Laboratories; Shahrul Mt-Isa, MSD; Dai Feng, AbbVie; Jie Chen, Merck Research Laboratories; Joe Heyse, Merck; Patrick Schnell, Ohio State University
2:50 PM A General Solution to Multiple Hypothesis Testing Problem with Constraints
Presentation
Huajiang Li, Allergan; Hong Zhou, Arkansas State University
3:05 PM Incorporating the Sample Correlation Between Two Test Statistics to Adjust the Critical Points for the Control of Type-1 Error
Presentation
Dror Rom, Prosoft Clinical; Jaclyn Ashley McTague, Prosoft Clinical
3:20 PM MULTIPLE TESTING METHODS for A-PRIORI ORDERED HYPOTHESES
Anjana Grandhi, Merck & Co.
3:35 PM An Extended Simes Test Procedure for Multiple Testing
Presentation
Matthew Hudson, Prosoft Clinical; Dr. Joshua Naranjo, Western Michigan University; Dror Rom, Prosoft Clinical
 
 

251
Mon, 7/29/2019, 2:00 PM - 2:45 PM CC-Hall C
SPEED: Biopharmaceutical Methods and Application I, Part 2 — Contributed Poster Presentations
Biopharmaceutical Section
Chair(s): Sarah Ryan
Oral Presentations for this session.
1: Bayesian Leveraging of Historical and Concurrent Data to Assess the Contribution of a New Molecular Entity with a Delayed Effect in a Combination Survival Trial
Samson Ghebremariam, Novartis Pharmaceutical Corporation; Lisa Hampson, Novartis Pharmaceutical Corporation; Amy Racine-Poon, Novartis Pharmaceutical Corporation; Beat Neuenschwander, Novartis Pharmaceutical Corporation; Bharani Dharan, Novartis Pharmaceuticals; Kalyanee Appanna, Novartis Pharmaceutical Corporation
2: Design of Clinical Trials for Bivariate Endpoints
Junxiao Hu, University of Colorado; Patrick Blatchford, University of Colorado; John Kittelson, University of Colorado
3: Bayesian Modeling in Historical Data Borrowing on Controls in Clinical Trials
Zhuqing Yu, AbbVie Inc.; Zailong Wang, AbbVie Inc.; Lanju Zhang
4: Analysis Methods for Skewed Data Distributions
Annpey Pong
5: Identification of Potential Predictive Biomarker Candidates Through Strategic Analysis of Cytokine Profiles Across Multiple Anti-PD-1 Clinical Trials
Jeea Choi, Novartis; Ying Amanda Wang, Novartis; John Millholland, Novartis; Albert Reising, Novartis; Jan Christoph Brase, Novartis; Xiaoshan Wang, Novartis; Connie Wong, Novartis; Kitty Wan, Novartis; Yiqun Yang, Novartis; Gullu Gorgun, Novartis; Parul Patel, Novartis; Hemant Patel, Novartis
6: Precise and Accurate Power of the Rank-Sum Test for a Continuous Variable
Katie Rose Mollan, University of North Carolina Chapel Hill; Ilana Trumble, University of Colorado Denver; Sarah Reifeis, University of North Carolina at Chapel Hill; Orlando Ferrer, University of North Carolina Chapel Hill; Camden P Bay, Harvard Medical School; Pedro L. Baldoni, University of North Carolina At Chapel Hill; Michael Hudgens, University of North Carolina at Chapel Hill
7: Reducing Misclassification Effect on Dynamic Treatment Regimen (DTR) of Sequential Multiple Assignment Randomized Trial Designs (SMART)
Jun He, Virginia Commonwealth University; Roy T Sabo, Virginia Commonwealth University; Donna McClish, VCU
8: Simple Adjustment for Bias Due to Unobserved Confounding
Yiran (Bonnie) Hu, AbbVie; Hui Xie, University of Illinois at Chicago
9: Umbrella and Platform Trials: Statistical Considerations on Efficiencies and a Case Study
Xiaoyun (Nicole) Li, Merck; Cong Chen, Merck & Co., Inc; Fang Liu, Merck; Wen Li, Merck
10: Optimal Design and Analysis of Efficacy Expansion in Phase I Oncology Trials
Iris Wu, Merck & Co.; Fang Liu, Merck; Heng Zhou, Merck & Co., Inc; Cong Chen, Merck & Co., Inc
11: Evaluating the “One-Model Fits All” Approach for Modeling Clinical Trial Adverse Events
Stephanie Pan
12: How Many Imputations Are Enough When Reporting Clinical Trials?
Anders Gorst-Rasmussen, Novo Nordisk A/S
13: Meta-Analysis of Longitudinal Preclinical Efficacy Screens
William Forrest, Genentech, Inc; Bruno Alicke, Genentech; Magdalena Osinska, Genentech; Shannon Ruppert, Genentech; Michal Jakubczak, Roche; Pawel Piatkowski, Roche
14: The Application of Beta Regression for Modeling a Covariate Adjusted ROC
Xing Meng, Baylor University; Jack D. Tubbs, Baylor University
15: Examining the Replication Crisis: The Effect of Underpowered Studies and Publication Bias
Christine M. Orndahl, Virginia Commonwealth University Dept of Biostatistics; Robert A. Perera, VCU Department of Biostatistics
16: Comparison of Bayesian Network Meta-Analysis Models for Survival Data
Purvi Prajapati, Baylor University; James D Stamey, Baylor University; John Seaman, Baylor University; Michael Sonksen, Eli Lilly & Co.; Min-Hua Jen, Eli Lilly & Co.
17: Advantages of Parallel Design Over Crossover Design in the Study on Effects of Cannabis on Driving in Healthy Adults
Anya Umlauf, UC San Diego; Barth Wilsey, UC San Diego; Thomas Marcotte, UC San Diego; Florin Vaida, UC San Diego
18: Probability of Undetectable Error in Independent Dual Programming Validation for Analysis Results in Clinical Trials
Long Zheng, Takeda Pharmaceutical
19: An Extension of Cohen’s Kappa for Clustered Data and Group Sequential Testing
Mary Ryan, University of California, Irvine; Daniel L. Gillen, University of California, Irvine
20: Flexible Semiparametric Bayesian Hierarchical Model for Basket Trials
Veronica Bunn, Takeda Pharmaceuticals; Jianchang Lin, Takeda Pharmaceuticals; Rachael Liu, Takeda Pharmaceuticals
Oral Presentations for this session.
 
 

Register 263
Tue, 7/30/2019, 7:00 AM - 8:15 AM H-Centennial Ballroom G-H
Biopharmaceutical Section A.M. Roundtable Discussion (Added Fee) — Roundtables AM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
TL01: How to Mitigate Bias in Single-Arm Trials in Oncology Drug Development?
Xuejing Wang, Eli Lilly and Company
TL02: WITHDRAWN: Statistical Considerations in HIV PrEP Trial Design
Jiejun Du, Merck & Co, Inc.; Peggy Hwang, Merck & Co, Inc.
 
 

273 * !
Tue, 7/30/2019, 8:30 AM - 10:20 AM CC-109
How Advanced Analytic Tools Deliver Insights for Clinical Investigations Through Real World Data — Invited Papers
Biopharmaceutical Section, Biometrics Section, ENAR
Organizer(s): Junjing Lin, AbbVie
Chair(s): Margaret Gamalo-Siebers, Eli Lilly
8:35 AM Incorporating Prior Knowledge on Phenotyping Accuracy for Association Studies Using Electronic Health Records Data
Yong Chen, University of Pennsylvania; Jing Huang, University of Pennsylvania
8:55 AM Challenges When Applying Advanced Analytics to Multiple Data Sources
David Ohlssen, Novartis
9:15 AM Predict Phase 3 Clinical Trial Results Using Phase 2 Data and Electronic Health Records
Qi Tang, Sanofi; Youran Qi, University of Wisconsin
9:35 AM Analytic Strategies of Using Propensity Scores in Clinical Data Augmentation
Junjing Lin, AbbVie; Margaret Gamalo-Siebers, Eli Lilly; Ram Tiwari, CDRH, FDA
9:55 AM Discussant: Yunling Xu, FDA/CDRH
10:15 AM Floor Discussion
 
 

299
Tue, 7/30/2019, 8:30 AM - 10:20 AM CC-111
Estimands and Imputations Methods — Contributed Papers
Biopharmaceutical Section
Chair(s): Weichao Bao, GlaxoSmithKline
8:35 AM An Approach to Multiple Imputation That Avoids the Inclusion of an Outcome in the Imputation Model
Monelle Tamegnon, Janssen R&D
8:50 AM Missing Data Imputation with Baseline Information in Longitudinal Clinical Trials
Presentation
Yilong Zhang, Merck; Zachary Zimmer, Merck; Lei Xu, Merck; Gregory Golm, Merck; Raymond Lam, Merck; Susan Huyck, Merck; Frank G Liu, Merck Sharp & Dohme Inc.
9:05 AM Using the Retrieved Dropout Approach for Estimating a Treatment Policy Estimand
Presentation
Ruvie Martin, Novartis Pharmaceuticals; Bjoern Bornkamp, Novartis Pharmaceuticals
9:20 AM Missing Data Approaches for Estimating Treatment Effect for Binary Data
Presentation
Anindita Banerjee, Pfizer; Vivek Pradhan, Pfizer; Arnab Maity, Pfizer
9:35 AM Considerations for the Use of Multiple Imputation in a Noninferiority Trial Setting
Presentation
Kimberly Walters, Statistics Collaborative, Inc.; Jie Zhou, Statistics Collaborative, Inc.; Janet Wittes, Statistics Collaborative, Inc; Lisa Weissfeld, Stats Collaborative
9:50 AM Identifying Treatment Effects Using Trimmed Means When Data Are Missing Not at Random
Alex Ocampo, Harvard University
10:05 AM Imputation Strategies When a Continuous Outcome Is to Be Dichotomized for Responder Analysis: a Simulation Study
Presentation 1 Presentation 2
Lysbeth Floden, University of Arizona; Melanie Bell, University of Arizona
 
 

315 *
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-301
Innovative Bayesian Approaches in Clinical Trials and Practical Considerations — Invited Papers
Section on Bayesian Statistical Science, Biopharmaceutical Section, Society for Clinical Trials
Organizer(s): Mandy Jin, Merck & Co., Inc.
Chair(s): Mandy Jin, Merck & Co., Inc.
10:35 AM Revisiting Test-Then-Pool Methods and Some Practical Considerations
Frank G Liu, Merck Sharp & Dohme Inc.; Wen Li, Merck
10:55 AM Nonparametric Bayesian Estimation of Heterogeneous Causal Effects Using Real-World Data
Xinyi Xu, The Ohio State University; Bo Lu, The Ohio State University; Steve MacEachern, The Ohio State University; Ling Wang, Michigan State University
11:15 AM Design of Drug Combination Early Phase Cancer Trials Under the Setting of Partial Toxicity Attribution
Presentation
Mourad Tighiouart, Cedars-Sinai Medical Center
11:35 AM Bayesian Framework for Pediatric Drug Development
Amarjot Kaur, Merck & Co.; Mandy Jin, Merck & Co., Inc.; Qing Li, Merck Research Labs
11:55 AM Discussant: Gregory Campbell, GCStat Consulting
12:15 AM Floor Discussion
 
 

322 !
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-106
Time-To-Event Models in Complex Observational Studies — Invited Papers
Biometrics Section, ENAR, Biopharmaceutical Section
Organizer(s): Soutrik Mandal, National Cancer Institute
Chair(s): Ana Maria Ortega-Villa, National Institutes of Health
10:35 AM A Copula Model Approach for Regression Analysis of Informatively Interval-Censored Failure Time Data
Presentation
(Tony) Jianguo Sun, University of Missouri
11:00 AM Validating Risk Prediction Models with Sub-Samples of Cohorts
Ruth Pfeiffer, National Cancer Institute; Mitchell Henry Gail, National Cancer Institute, Division of Cancer Epidemiology and Genetics; Yei Eun Shin, National Cancer Institute
11:25 AM Cure Rate Frailty Models for Clustered Current Status Data with Informative Cluster Size
Kejun He, Renmin University; Wei Ma, Renmin University; Tong Wang, Texas A&M University; Dipankar Bandyopadhyay, Virginia Commonwealth University; Samiran Sinha, Texas A&M University
11:50 AM Goodness-of-Fit Tests for the Linear Transformation Models with Interval-Censored Data
Presentation
Soutrik Mandal, National Cancer Institute; Suojin Wang, Texas A&M University; Samiran Sinha, Texas A&M University
12:15 PM Floor Discussion
 
 

326 * !
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-108
Use of Concurrent and Non-Concurrent Control Data in Basket and Platform Trials — Topic Contributed Papers
Biopharmaceutical Section
Organizer(s): Weichao Bao, GlaxoSmithKline
Chair(s): Ying Grace Li, Eli Lilly and Company
10:35 AM Use of Historical Control Information in Platform Trials
Satrajit Roychoudhury, Pfizer Inc
10:55 AM Biomarkers and Use of Non-Concurrent Controls: Experiences of the Children’s Oncology Group
Presentation
Lindsay Renfro, University of Southern California and Children's Oncology Group
11:15 AM Statistical Innovations for Complex Diseases: a Multi-Arm Adaptive Platform Trial for Cystic Fibrosis
Benjamin Saville, Berry Consultants
11:55 AM Discussant: Laura Lee Johnson, U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER)
12:15 PM Floor Discussion
 
 

327 * !
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-110
Probabilistic Decision-Making in Clinical Research — Topic Contributed Papers
Biopharmaceutical Section, Section on Bayesian Statistical Science
Organizer(s): Alan Hartford, Takeda Pharmaceutical Company
Chair(s): Qi Tang, Sanofi
10:35 AM Predicting Technical Success of a Phase III Program Using Bayesian Latent Relationship Modeling
Saurabh Mukhopadhyay, AbbVie
10:55 AM Evidence Based Decision Making in Clinical Trials
Presentation
Erik Pulkstenis, AbbVie
11:15 AM Time-To-Event Bayesian Optimal Interval Design to Accelerate Dose-Finding Based on Both Efficacy and Toxicity Outcomes
Kentaro Takeda, Astellas Pharma Global Development, Inc.
11:35 AM Utilizing Bayesian Analysis for Probabilistic Decision Making in a Platform Clinical Trial
Presentation
J. Kyle Wathen, Janssen R&D
11:55 AM Floor Discussion
 
 

339
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-105
SPEED: Biopharmaceutical and General Health Studies: Statistical Methods and Applications, Part 1 — Contributed Speed
Biopharmaceutical Section, Section on Statistics in Epidemiology, Section on Bayesian Statistical Science, Health Policy Statistics Section, ENAR
Chair(s): Sedigheh Mirzaei Salehabadi, St. Jude Children's Research Hospital
Poster Presentations for this session.
10:35 AM Mediation Analysis for Longitudinal Data with Applications to Clinical Trial Data
Yun Zhang
10:40 AM Adjusting Response Adaptive Allocation for Subject Dropout
Presentation
Katharine Stromberg, Virginia Commonwealth University; Adam Sima, Virginia Commonwealth University
10:45 AM The Use of a New Classifier to Maximize the Classification Performance
Presentation
Hua Ma, Merck; Joe Heyse, Merck
10:50 AM Reproducibility of Living Data - Validation of Published Research Using the Parkinson’s Progression Marker Initiative Living Database
Elliot Burghardt, University of Iowa; Christopher Coffey, University of Iowa; Chelsea Caspell-Garcia, University of Iowa; Eric Foster, Ferring Pharmaceuticals
10:55 AM Blinding in Open Label Study with Adaptive Design
Presentation
Bo Xu, Boston Biomedical Inc; Bo Jin, Boston Biomedical Inc; Alex Dmitrienko, Mediana Inc
11:00 AM Estimating the Relative Risk for Response-Biased Samples: Calibration and Conditional Likelihood
Presentation 1 Presentation 2 Presentation 3 Presentation 4
Claudia Rivera-Rodriguez, University of Auckland
11:05 AM Another Estimation Method Besides MMRM for Treatment Effects in Diabetes Clinical Trials
Presentation
Yu Du, Eli Lilly and Company
11:10 AM Criteria for Choosing a Futility Method for Clinical Studies
Presentation
Richard McNally, Covance-Chiltern
11:15 AM Random Forests for Exploring Factors Driving Opioid Prescribing in National Outpatient Health Care Data Using Complex Survey Design
Presentation
Yong Ma, FDA; JaeJoon Song, FDA
11:20 AM An Adaptive Phase II Dose Finding Study Using Sample Size Re-Estimation Design
Qingyang Liu, University of Connecticut; Guanyu Hu, University of Connecticut; Yaoshi Wu, Boehringer-Ingelheim ; Binqi Ye, Boehringer-Ingelheim; Susan Wang, Boehringer-Ingelheim
11:30 AM Sieve Maximum Likelihood Method for Interval-Censored Data with Missing Covariates Under Proportional Hazards Model
Ruiwen Zhou, University of Missouri-Columbia; Huiqiong Li, Yunnan University; (Tony) Jianguo Sun, University of Missouri
11:35 AM Quantifying the Number of Events Borrowed from External Data in Hybrid Control Arms
Presentation
Brian Segal, Flatiron Health; Carrie Bennette, Flatiron Health; Somnath Sarkar, Flatiron Health
11:40 AM Characterizing Irreproducibility in Drug Sensitivity Data from a Large Pharmacogenomic Study
Zoe Rehnberg, University of Michigan; Johann A Gagnon-Bartsch, University of Michigan
11:45 AM Closest Similar Subset Imputation
Macaulay Okwuokenye, Brio Dexteri Pharmaceutical Consultant & UNE; Karl E Peace, Georgia Southern University
11:50 AM Planning and Analyzing Clinical Trials with Competing Risks: Recommendations for Choosing Appropriate Statistical Methodology
Presentation
Misun Yu Lee, Astellas Pharma; Joseph Poythress, University of Georgia; James Young, Astellas Pharma
11:55 AM Estimating and Using the Attained Power Distribution to Ensure We Get the Trial Power We Expect
Yongdong Ouyang, University of British Columbia; Hubert Wong, University of British Columbia; Ehsan Karim, University of British Columbia; Paul Gustafson, University of British Columbia
12:00 PM Bayesian Semiparametric Joint Modeling of Longitudinal Predictors and a Binary Outcome
Presentation 1 Presentation 2 Presentation 3 Presentation 4 Presentation 5 Presentation 6
Woobeen Lim, The Ohio State University; Michael Pennell, Ohio State University
12:05 PM Clustering of Multivariate Data with Varying Dimensions
Presentation
Xiaoqi Lu, Columbia University; Bin Cheng, Columbia University; Ying Kuen Ken Cheung, Columbia University
12:10 PM Floor Discussion
 
 

342 *
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-109
Topics in Adaptive-Seamless and Group Sequential Designs — Contributed Papers
Biopharmaceutical Section
Chair(s): Qi Jiang, Seattle Genetics
10:35 AM Adaptive Sequential Design for Seamless Phase 2/3 Combination
Presentation
Ping Gao, Brightech International; Tai Xie, Brightech International; Peng Zhang, Brightech International; Yue Tu, Brightech International; Lingyun Liu, Cytel; Cyrus Mehta, Cytel
10:50 AM Selecting Critical Boundaries in Group-Sequential Trials with Multiple Endpoints
Toshimitsu Hamasaki, National Cerebral and Cardiovascular Center; Hsien-Ming James Hung, PhD, Food and Drug Administration; Chin-Fu Hsiao, National Health Research Institutes; Scott R Evans, George Washington University
11:05 AM Covariate Adaptive Randomization in Seamless Phase II/III Clinical Trials
Presentation
Hongjian Zhu, University of Texas Health Science Center at Houston; Wei Ma, Renmin University; Mengxi Wang, University of Texas Health Science Center at Houston
11:20 AM Evaluation of Type 1 Error in a 2-In-1 Adaptive Phase 2/3 Design with Dual-Primary Endpoints in Oncology Studies
Presentation
Li Fan, Merck; Jing Zhao, Merck Research Labs
11:35 AM Implementation of an Adaptive Early Phase Trial Design for Drug Combinations
Bethany Horton, University of Virginia; Nolan Wages, University of Virginia
11:50 AM Developing Innovative Group Sequential Design Trials That Account for the Correlation Between Test Statistics
Presentation
JonDavid Sparks, Eli Lilly & Company; Bill Prucka, Eli Lilly & Company; Brian Millen, Eli Lilly & Company
12:05 PM Unblinded Sample Size Re-Estimation in Clinical Trials with Count Outcomes
Yeting Du, Cytel Inc; Lingyun Liu, Cytel
 
 

343
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-111
Innovative Trial Designs and Analytics — Contributed Papers
Biopharmaceutical Section
Chair(s): Darcy Hille, Merck & Company Inc
10:35 AM On the Robustification of MAP Prior in Bayesian Historical Data Borrowing
Hongtao Zhang, AbbVie Inc.
10:50 AM Optimal Two-Stage Designs for Exploratory Basket Trials
Presentation
Heng Zhou, Merck & Co., Inc; Fang Liu, Merck; Cai (Iris) Wu, Merck & Co., Inc; Cong Chen, Merck & Co., Inc
11:05 AM A Case Study of a Complex Design for a Clinical Trial with Features of Randomized Withdrawal and Parallel Randomization in a Rare Disease Area
Junxiang Luo, Sanofi-Aventis; Qi Zhang, Sanofi; Hui Quan, Sanofi US
11:20 AM A Bayesian Design with Conditional Borrowing of Historical Data in a Rare Disease Setting
Presentation
Peng Sun; Ming-Hui Chen, University of Connecticut; Yiwei Zhang, Biogen; John Zhong, Biogen; Charlie Cao, Biogen; Guochen Song, Biogen; Zhenxun Wang, University of Minnesota,
11:35 AM Evaluation of False Discovery Rate in Platform Studies
Qiusheng Chen, Merck; Xiaoyun (Nicole) Li, Merck; Cong Chen, Merck & Co., Inc
11:50 AM Platform Trial Design with Incorporating Historical Data Dynamically
Weichao Bao, GlaxoSmithKline; Ohad Amit, GlaxoSmithKline; Sofia Paul, GlaxoSmithKline; Teri Ashton, GlaxoSmithKline; Karrie Wang, GlaxoSmithKline; Leah Suttner, GlaxoSmithKline
12:05 PM Adjustment of Subgroups Reversal Effect via Bayesian Borrowing Approach in Oncology Regulatory Submission
Rachael Liu, Takeda Pharmaceuticals ; Jianchang Lin, Takeda Pharmaceuticals; Veronica Bunn, Takeda Pharmaceuticals
 
 

355
Tue, 7/30/2019, 10:30 AM - 12:20 PM CC-Hall C
Contributed Poster Presentations: Biopharmaceutical Section — Contributed Poster Presentations
Biopharmaceutical Section
Chair(s): Wendy Meiring, University of California At Santa Barbara
1: Some Tests for the Assessment of Univariate and Multivariate Bioequivalence
Rabab Elnaiem, University of Maryland, Baltimore County; Thomas Mathew, University of Maryland, Baltimore County
2: A Comparison of Methods to Estimate the Event Rate Based on Longitudinal Data
Bo Fu, Astellas Pharma Inc.; Xuan Liu, Astellas Pharma Inc.; Jun Zhao, Astellas Pharma Inc.
3: A Bayesian Answers "Should This Drug Be Approved?"
Konstantinos Vamvourellis, London School of Economics and Politcal Science
5: The Statistics of Synthetically-Controlled Clinical Trials
Aaron Smith, Unlearn.AI; Charles K. Fisher, Unlearn.AI
7: Nonparametric Bayesian Method for Drug Combination with Discrete Doses
Zahra Razaee, Cedars-Sinai Medical Center; Galen Cook-Wiens, Cedars Sinai Medical Center; Mourad Tighiouart, Cedars-Sinai Medical Center
8: Estimand and Analysis Consideration in a Phase III Study of CAR-T with Delayed Treatment Effect - a Case Study of Lymphoma
Wen Gu, Novartis Pharmaceutical Inc.
9: A Bayesian Adaptive Design in Cancer Phase I Trials Using Dose Combinations with Quasi-Continuous Toxicity Index
Sungjin Kim, Cedars-Sinai Medical Center; Zahra Razaee, Cedars-Sinai Medical Center; Andre Rogatko, Cedars-Sinai Medical Center; Mourad Tighiouart, Cedars-Sinai Medical Center
10: Assessing the Performance of Different Outcomes for Tumor Growth Studies with Animal Models
Luke William Patten, Center for Innovative Design and Analysis, University of Colorado, Anschutz Medical Campus; Alexander Kaizer, University of Colorado Anschutz Medical Campus; Patrick Blatchford, University of Colorado
11: Equivalency Test Based on Combinations of Mean and Variance Components in the One-Way Random Effects Model with Application to Device Comparison Study
Yun Bai, Medtronic; BAOLIN WU, University of Minnesota; Zengri Wang, Medtronic plc; Theodore Lystig, Medtronic
12: A DECAY MODEL for HANDLING MISSING DATA in CLINICAL TRIALS
Tao Sheng
13: A Comparison of Migraine Prevention Therapies in the Adult Versus Pediatric Populations Using a Joint Bayesian Network Meta-Analysis Model
Zachary Thomas, Eli Lilly and Company; Phebe Kemmer, Eli Lilly and Company; Tianle Hu, Eli Lilly and Company; Fanni Natanegara, Eli Lilly and Company; Himanshu Upadhyaya, Eli Lilly and Company
14: Dose-Finding Designs Using Time-To-Event Toxicity Data and Multiple Constraints
Meizi Liu, University of Chicago
16: Wearable Devices in Clinical Trials: Making an Impact in the Cardiovascular Space
Vanja Vlajnic; Chrysanthi Dori, Bayer; Mercedeh Ghadessi, Bayer; Stephan Cichos, Bayer; Maike Ahrens, Bayer; Matthias Sachs, Bayer / SAMSI; Paolo Piraino, Bayer
17: Combining Tabular Data with Visual Display to Enhance Interpretation of Clinical Trial Data
Teresa Curto, Cytel; Ashish Aggarwal , Cytel; Angelo Tinazzi , Cytel
18: Tumor-Growth Modeling for Informed Go/No-Go Decisions
Wei Wei, Yale University School of Public Health; Daniel Zelterman, Yale University School of Public Health; Elizabeth Garrett-Mayer, American Society of Clinical Oncology
19: Less Is More – Adaptive Seamless Phase II/III Design
Helen Chen, GSK; Jonathan Haddad , GlaxoSmithKline; Xiaowei Wang, GlaxoSmithKline
20: Estimate of Treatment Difference for Non-Normally Distributed Data in Clinical Trials – Comparison of Hodges-Lehmann Method and Quantile Regression
Youlan Rao, United Therapeutics Corporation; Yonggang Yao, SAS Institute Inc; Lisa Edwards, United Therapeutics Corporation; Chunqin Deng, United Therapeutics Corporation
21: Sequential Multiple Assignment Randomized Trials with Continuous Intermediate Outcome
Holly Elizabeth Hartman, University of Michigan; Kelley Kidwell, University of Michigan; Matthew J. Schipper, University of Michigan
22: Statistical Considerations for Analytical Method Transfer Equivalence Margin
Oluyemi Oyeniran, JNJ; Jyh-Ming Shoung, Janssen R&D
23: Quantifying Impact of Enrichment in Randomized Clinical Trials
Navneet Hakhu, University of California, Irvine; Daniel L. Gillen, University of California, Irvine
24: Predicting Unmeasured Outcomes in the Real-World Data: Bayesian and Frequentist Approaches - a Simulation Study
Wenyu Ye, Eli Lilly and Company; Douglas Faries, Eli Lilly & Company; Xiang Zhang, Eli Lilly and Company; janet ford, Eli Lilly and compnay; zbigniew kadziola, Eli Lilly and company; Xiaojuan Mi, TechData Service Company, LLC; Ilya Lipkovich, Eli Lilly and Company
26: High-Throughput Screening of Features Which Moderate Treatment Effect on Clinical Outcome
Kushal Shah, University of North Carolina (UNC); Michael Kosorok, University of North Carolina at Chapel Hill
27: Non-Collapsibility of Hazard Ratio
Busola Sanusi, The University of North Carolina; Godwin Yung, Takeda Pharmaceuticals; Yi Liu, Takeda Pharmaceuticals International Co.
28: Using BLRM to Find MTDs for Loading Dose and Maintenance Dose in Oncology Trials
Kejian Liu, Sanofi; Yinge Sun, University of Virginia
29: Machine Learning for Protein Design
Yuting Xu, Merck Sharp & Dohme Corp.; Andy Liaw, Merck Sharp & Dohme Corp.
30: Mediation by Progression of Treatment-Related Differences in Patient Reported Outcomes (PROs) in Oncology
Michael Blackowicz, Clinical Outcomes Solutions; Alicyn Campbell, Patient Relevant Evidence; Lysbeth Floden, Clinical Outcomes Solutions; Stacie Hudgens, Clinical Outcomes Solutions; Ethan Basch, University of North Carolina Lineberger Comprehensive Cancer Center
31: The Role of HLA-Class-II (HLAcII) Molecules in Determining the Immunogenicity Potential of Therapeutic Factor VIII Proteins (TFVIIIs) in Hemophilia a (HA): Assessing the Gate Keepe
Henry Mead, Walden University
32: Continuous Glucose Monitoring Technology and a Workflow for Its Data Analysis
Dandan Wang, Faculty of Health Sciences, Univerity of Macau; Xiaohua Douglas Zhang, University of Macau; Zhaozhi Zek Zhang, Washington University
34: Integrative Variable Selection Method for Subgroup Analyzes in Longitudinal Data
Xiaochen Li, Indiana University; Sujuan Gao, Indiana University
35: Bias-Correction in Estimating Treatment Effect in Fallback Analysis: An Approach Based on Randomized Test with Smooth Rejection Functions
Kiichiro Toyoizumi, Shionogi Inc.; Shigeyuki Matsui, Nagoya University Graduate School of Medicine
36: Identifying Two-Stage Optimal Dynamic Treatment Regimes: Compare Performances of Different Methods Under Model Misspecification
Sooyeong Lim, Miami University; Chen Chen, Cincinnati Children's Hospital; Rhonda Szczesniak, Cincinnati Children's Hospital; Gary Lewis McPhail, Cincinnati Children's Hospital; Bin Huang, Cincinnati Children's Hospital
37: Stepwise Progressive Parametric Multiple Testing Procedure with Correlated Normal Test Statistics
Xuan Deng, Merck; Mark Chang, Veristat
38: Quantitative Reproducibility Analysis for Identifying Reproducible Targets from High-Throughput Experiments
Wenfei Zhang, Sanofi (United States)
39: An Event/Trial Binomial Model for Meaningful Change Inference in Randomized Clinical Trials
Daniel Serrano, Pharmerit International
40: Model Averaging of Bayesian Additive Regression Trees via Approximate Gaussian Processes
Kijoeng Nam, Merck; Nicholas Henderson, Johns Hopkins University; Dai Feng, AbbVie
41: Cancer Immunotherapy Trial Design with Delayed Treatment Effect
Jing Wei, no
42: A Personalized Medicine Approach for Comparative Evidence in Non-Randomized Studies
Carl De Moor, Biogen; Lu Tian, Stanford University School of Medicine; Fabio Pellegrini, Biogen International GmbH
43: Sample Size Calculation for the Andersen-Gill Model Comparing Rates of Recurrent Events
Ronan Fitzpatrick, Statsols; Yongqiang Tang, Tesaro
44: Mining Longitudinal Real-World Data to Identify Risk Factors for Cardiovascular Events Related to Anti-Dementia Medications
Meiqi He, University of Pittsburgh School of Pharmacy; Yuting Zhang, University of Melbourne Institute of Applied Economic and Social Research; Inmaculada Hernandez, University of Pittsburgh School of Pharmacy
45: Bootstrap Calibration for Parametric Tolerance Intervals to Improve Coverage Probabilities
Yixuan Zou, University of Kentucky; Derek Young, University of Kentucky
46: Evaluating the Effects of Design Parameters on the Performances of Phase I Trial Designs
Yaqian Zhu, University of Pennsylvania; Wei-Ting Hwang, University of Pennsylvania; Yimei Li, University of Pennsylvania
 
 

Register 364
Tue, 7/30/2019, 12:30 PM - 1:50 PM H-Centennial Ballroom G-H
Biopharmaceutical Section P.M. Roundtable Discussion (Added Fee) — Roundtables PM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
TL08: WITHDRAWN: Lunch with the Incoming 2020 Chair of the ASA Biopharmaceutical Section
Bruce Binkowitz, Shionogi & Co., Ltd.
 
 

383 * !
Tue, 7/30/2019, 2:00 PM - 3:50 PM CC-505
Experimental Design Applications in the Pharmaceutical Industry — Invited Papers
Section on Physical and Engineering Sciences, Biopharmaceutical Section, Quality and Productivity Section
Organizer(s): Stan Altan, Janssen R&D
Chair(s): Areti Manola, Janssen R&D
2:05 PM Experimental Design in the Pharmaceutical Industry
Presentation
Brad Evans, Pfizer, Inc
2:25 PM Central Composite Designs for Process Characterization, Why?
Jose Ramirez, Amgen, Inc.
2:45 PM Overview of DoEs and Applications in the Pharmaceutical Industry
Presentation
Jyh-Ming Shoung, Janssen R&D; Dwaine Banton, Janssen R&D; Areti Manola, Janssen R&D
3:05 PM An Extended Youden Design for Biological Assays
Presentation
Yi Hua, University of Illinois at Chicago; Samad Hedayat, University of Illinois at Chicago; Min Yang, University of Illinois at Chicago; Stan Altan, Janssen R&D
3:25 PM Discussant: Stan Altan, Janssen R&D
3:45 PM Floor Discussion
 
 

398 * !
Tue, 7/30/2019, 2:00 PM - 3:50 PM CC-102
Considerations in Optimization of Pediatric Drug Development — Topic Contributed Panel
Biopharmaceutical Section, Society for Clinical Trials, Academy for Health Services Research and Health Policy
Organizer(s): Freda Cooner, Amgen Inc.
Chair(s): Freda Cooner, Amgen Inc.
2:05 PM Statistical Considerations in Optimization of Pediatric Drug Development
Presentation
Panelists: Amy Xia, Amgen Inc
Lynne Yao, FDA/CDER
Fanni Natanegara, Eli Lilly and Company
Gary Noel, Johnson & Johnson
3:40 PM Floor Discussion
 
 

405
Tue, 7/30/2019, 2:00 PM - 3:50 PM CC-101
Statistical Issues Specific to Therapeutic Areas — Contributed Papers
Biopharmaceutical Section
Chair(s): Bochao Jia, Eli Lilly and Company
2:05 PM Dynamic Prediction of Alzheimer's Disease Progression Using Features of Multiple Longitudinal Outcomes
Kan Li, Merck & Co.; Sheng Luo, Duke University Medical Center; Richard Entsuah, Merck & Co.
2:20 PM Estimating Knots in Bilinear Spline Growth Models with Time-Invariant Covariates in the Framework of Individual Measurement Occasions
Jin Liu; Robert A. Perera, VCU Department of Biostatistics; Robert M. Kirkpatrick, Virginia Institute for Psychiatric & Behavioral Genetics
2:35 PM Assessing Correlates of Protection in Vaccine Trials: Statistical Solutions in the Context of High Vaccine Efficacy
Fabian Tibaldi, GSK Vaccines; Andrea Callegaro, GSK Vaccines
2:50 PM Statistical Modeling Strategies for Medication Adherence Research
Presentation
Josh DeClercq, Vanderbilt University Medical Center; Leena Choi, Vanderbilt University Medical Center
3:05 PM Characteristics of Meta-Analyzes Used for Assessment of Vaccine Safety
Rositsa Dimova, FDA
3:20 PM Nonparametric Estimation of Enriched Crossover Design with High Placebo Response Rate
Siying Li, IQVIA; Gary Koch, University of North Carolina at Chapel Hill
3:35 PM Two New Dual-Agent Dose Escalation Methods
Presentation
Yue Yang, North Carolina State University; Wentao Feng, Seattle Genetics; Qianwen Tan, Seattle Genetics; Lisa Brown, Seattle Genetics
 
 

426
Tue, 7/30/2019, 3:05 PM - 3:50 PM CC-Hall C
SPEED: Biopharmaceutical and General Health Studies: Statistical Methods and Applications, Part 2 — Contributed Poster Presentations
Biopharmaceutical Section, Health Policy Statistics Section, ENAR
Chair(s): Sedigheh Mirzaei Salehabadi, St. Jude Children's Research Hospital
Oral Presentations for this session.
1: Mediation Analysis for Longitudinal Data with Applications to Clinical Trial Data
Yun Zhang
2: An Adaptive Phase II Dose Finding Study Using Sample Size Re-Estimation Design
Qingyang Liu, University of Connecticut; Guanyu Hu, University of Connecticut; Yaoshi Wu, Boehringer-Ingelheim ; Binqi Ye, Boehringer-Ingelheim; Susan Wang, Boehringer-Ingelheim
4: Quantifying the Number of Events Borrowed from External Data in Hybrid Control Arms
Brian Segal, Flatiron Health; Carrie Bennette, Flatiron Health; Somnath Sarkar, Flatiron Health
5: Characterizing Irreproducibility in Drug Sensitivity Data from a Large Pharmacogenomic Study
Zoe Rehnberg, University of Michigan; Johann A Gagnon-Bartsch, University of Michigan
6: Closest Similar Subset Imputation
Macaulay Okwuokenye, Brio Dexteri Pharmaceutical Consultant & UNE; Karl E Peace, Georgia Southern University
7: Planning and Analyzing Clinical Trials with Competing Risks: Recommendations for Choosing Appropriate Statistical Methodology
Misun Yu Lee, Astellas Pharma; Joseph Poythress, University of Georgia; James Young, Astellas Pharma
8: Estimating and Using the Attained Power Distribution to Ensure We Get the Trial Power We Expect
Yongdong Ouyang, University of British Columbia; Hubert Wong, University of British Columbia; Ehsan Karim, University of British Columbia; Paul Gustafson, University of British Columbia
9: Bayesian Semiparametric Joint Modeling of Longitudinal Predictors and a Binary Outcome
Woobeen Lim, The Ohio State University; Michael Pennell, Ohio State University
10: Clustering of Multivariate Data with Varying Dimensions
Xiaoqi Lu, Columbia University; Bin Cheng, Columbia University; Ying Kuen Ken Cheung, Columbia University
11: Sieve Maximum Likelihood Method for Interval-Censored Data with Missing Covariates Under Proportional Hazards Model
Ruiwen Zhou, University of Missouri-Columbia; Huiqiong Li, Yunnan University; (Tony) Jianguo Sun, University of Missouri
12: Adjusting Response Adaptive Allocation for Subject Dropout
Katharine Stromberg, Virginia Commonwealth University; Adam Sima, Virginia Commonwealth University
13: Estimating the Relative Risk for Response-Biased Samples: Calibration and Conditional Likelihood
Claudia Rivera-Rodriguez, University of Auckland
14: The Use of a New Classifier to Maximize the Classification Performance
Hua Ma, Merck; Joe Heyse, Merck
15: Reproducibility of Living Data - Validation of Published Research Using the Parkinson’s Progression Marker Initiative Living Database
Elliot Burghardt, University of Iowa; Christopher Coffey, University of Iowa; Chelsea Caspell-Garcia, University of Iowa; Eric Foster, Ferring Pharmaceuticals
16: Blinding in Open Label Study with Adaptive Design
Bo Xu, Boston Biomedical Inc; Bo Jin, Boston Biomedical Inc; Alex Dmitrienko, Mediana Inc
17: Another Estimation Method Besides MMRM for Treatment Effects in Diabetes Clinical Trials
Yu Du, Eli Lilly and Company
18: Criteria for Choosing a Futility Method for Clinical Studies
Richard McNally, Covance-Chiltern
19: Random Forests for Exploring Factors Driving Opioid Prescribing in National Outpatient Health Care Data Using Complex Survey Design
Yong Ma, FDA; JaeJoon Song, FDA
Oral Presentations for this session.
 
 

218849
Tue, 7/30/2019, 5:30 PM - 7:30 PM H-Centennial Ballroom A
Biopharmaceutical Section Business Meeting — Other Cmte/Business
Biopharmaceutical Section
Chair(s): Richard Zink, TARGET PharmaSolutions
 
 

Register 430
Wed, 7/31/2019, 7:00 AM - 8:15 AM H-Centennial Ballroom G-H
Biopharmaceutical Section A.M. Roundtable Discussion (Added Fee) — Roundtables AM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
WL01: Information Borrowing in Pediatric and Small Population Drug Development
Ziliang Li, Vertex Pharmaceuticals
WL02: Success Recipe for a CRO Biostatistician: Building a Collaborative Environment
Sourav Santra, Cytel
 
 

219203
Wed, 7/31/2019, 7:30 AM - 9:00 AM H-Mineral Hall B
ASA BIOP Real World Data and Real World Evidence Scientific Working Group Meeting (Closed) — Other Cmte/Business
Biopharmaceutical Section
Chair(s): Weili He, AbbVie
 
 

447 * !
Wed, 7/31/2019, 8:30 AM - 10:20 AM CC-201
Recent Advances in Propensity Score Methods for Observational Studies with Multiple Treatments — Invited Papers
Health Policy Statistics Section, Biopharmaceutical Section, Section on Statistics in Epidemiology
Organizer(s): Elande Baro, US Food and Drug Administration; Jessica Kim, US Food and Drug Administration
Chair(s): Jessica Kim, US Food and Drug Administration
8:35 AM Generalized Propensity Score Matching: Updates and Challenges Toward Establishing Best Practices
Douglas Faries, Eli Lilly & Company; Zhanglin Cui, Eli Lilly & Company; Li Li, Eli Lilly & Company; Shu Yang, North Carolina State University; Shuhan Tang, The Ohio State University
9:00 AM Approximate Bayesian Bootstrap Procedures to Estimate Multilevel Treatment in Observational Studies with Application to Type 2 Diabetes Treatment Regimens
Presentation
Roee Gutman, Brown University; Anthony D. Scotina, Simmons University; Robert J Smith, Brown University; Andrew R Zullo, Brown University
9:25 AM Utility of Regression Splines for Propensity Score Adjustment in Post Market Safety Analyzes with Multiple Treatments
Elande Baro, US Food and Drug Administration; Yuxi Tian, University of California Los Angeles; Rongmei Zhang, Food and Drug Administration; Yuqin Wei, Acumen LLC; Mao Hu, Acumen LLC; Jiemin Liao, Acumen LLC; Sandia Akhtar, Acumen LLC; Michael Wernecke, Acumen LLC; Jeffrey Kelman, Centers for Medicare & Medicaid Services; David Graham, Acumen LLC
9:50 AM Discussant: Jessica M Franklin, Brigham and Women’s Hospital and Harvard Medical School
10:00 AM Discussant: Yi Huang, University of Maryland Baltimore Country
10:10 AM Floor Discussion
 
 

456 * !
Wed, 7/31/2019, 8:30 AM - 10:20 AM CC-207
Design and Analysis of Cancer Immunotherapy Trials with Complex Survival Patterns — Topic Contributed Papers
Biopharmaceutical Section, International Chinese Statistical Association, Biometrics Section
Organizer(s): Zhenzhen Xu , FDA
Chair(s): Bifeng Ding, Amgen
8:35 AM Survival Analysis Using a 5-STAR Approach in Randomized Clinical Trials
Devan Mehrotra, Merck & Co., Inc; Rachel Marceau West, Merck & Co., Inc.
8:55 AM Designing Cancer Immunotherapy Trials with Complex Survival Patterns
Zhenzhen Xu , FDA; BIN ZHU, NIH/NCI; YONGSOEK PARK, University of Pittsburg, Department of Biostatistics
9:15 AM Robust Group Sequential Designs for Immunotherapy Trials
Pranab Ghosh, Cytel Inc.; Cyrus Mehta, Cytel
9:35 AM A Flexible Test/Estimation Coherent Approach to Evaluate the Treatment Effect of Immunotherapy on Time-To-Event Outcomes
Hajime Uno, Dana-Farber Cancer Institute; Miki Horiguchi, Kitasato University; Lu Tian, Stanford University School of Medicine
9:55 AM Discussant: Shiowjen Lee, FDA
10:15 AM Floor Discussion
 
 

459 !
Wed, 7/31/2019, 8:30 AM - 10:20 AM CC-111
Rethinking Intercurrent Events and Estimators Within the ICH E9(R1) Estimand Framework — Topic Contributed Papers
Biopharmaceutical Section, Biometrics Section, ENAR
Organizer(s): Dong Xi, Novartis
Chair(s): Forrest Williamson, Eli Lilly
8:35 AM A Constructive Critique of the Draft ICH E9 Addendum
Presentation
Daniel Scharfstein, Johns Hopkins School of Hygiene & Public Health
8:55 AM Estimands in Clinical Trials with Intercurrent Events
Shanthi Sethuraman, Eli Lilly and Company; Yongming Qu, Eli Lilly and Company; Linda Shurzinske, Eli Lilly and Company
9:15 AM Comparison of Assumptions Required for Estimating Different Parameters in the Presence of Intercurrent Events
Presentation
Michael Rosenblum, Johns Hopkins Bloomberg School of Public Health
9:35 AM Some Thoughts on Recurrent Event Estimands and Estimators
Dong Xi, Novartis; Jiawei Wei, Novartis; Tobias Muetze, Novartis
9:55 AM Discussant: Thomas Permutt, U.S. Food and Drug Administration
10:15 AM Floor Discussion
 
 

467
Wed, 7/31/2019, 8:30 AM - 10:20 AM CC-113
Modeling, Design Strategies and Assessment of Biomarkers — Contributed Papers
Biopharmaceutical Section
Chair(s): Yodit Seifu, Merck
8:35 AM Modeling the Prediction Classifier of Overall Survival with Clinical and Gene Expression Data of Leukemia Patients – a Case Study
Presentation
Kao-Tai Tsai, Celgene
8:50 AM A New Method for the Analysis of Categorical Data with Repeated Measurements - Demonstrated by Precision Data Analysis for Clinical Diagnostics
Presentation
Tinghui Yu, AstraZeneca
9:05 AM Learning Moral Graphs in Construction of High-Dimensional Bayesian Networks for Mixed Data
Presentation
Bochao Jia, Eli Lilly and Company; Suwa Xu, University of Florida; Faming Liang, Purdue University
9:20 AM Biomarker Enrichment Subgroup Analysis - a Case Study
Rui Qin, Johnson & Johnson; Steven Sun, J&J; Grace Liu, Johnson & Johnson
9:35 AM Design Strategies to Assess Benefit for Biomarker Sub-Populations in Phase III Clinical Trials
Presentation
Bharani Dharan, Novartis Pharmaceuticals; Ekkehard Glimm, Novartis Pharma AG
9:50 AM Exact Bayesian Screening for Rapidly Identifying Uninformative Features from High-Dimensional Biomedical Arrays
Presentation
A Lawrence Gould, Merck Research Laboratories; Richard Baumgartner, Merck Research Laboratories
10:05 AM Prognostic Models from Data Integration of Clinical Characteristics and Gene Expression Data Using Bayesian Networks
Duncan Rotich, University of Kansas Medical Center; Jeffrey A. Thompson, University of Kansas Medical Center
 
 

468
Wed, 7/31/2019, 8:30 AM - 10:20 AM CC-112
Statistical Methods in Clinical Trials — Contributed Papers
Biopharmaceutical Section
Chair(s): Geng Chen, Alnylam
8:35 AM Percent Change from Baseline as an Endpoint in Clinical Trials
Presentation
Jitendra Ganju, Ganju Clinical Trials, LLC; Kefei Zhou, Jazz Pharma
8:50 AM Network Meta-Analysis for Benefit-Risk Assessment
Sammy Yuan, Merck; Chang Liu, North Carolina State University
9:05 AM Baseline-Covariate Adjusted Confidence Interval for Proportional Difference Between Two Treatment Groups in Clinical Trials
Presentation
JINGJING CHEN, Takeda Pharmaceuticals; Fang Liu, Merck
9:35 AM A Flexible Bayesian Method to Individualized Treatment Allocation
Saptarshi Chatterjee, Northern Illinois University; Sanjib Basu, University of Illinois at Chicago
9:50 AM Practical Determining the Late Effect Parameter in Fleming-Harrington Test Using Asymptotic Relative Efficiencies with Prototypical Lag Models Under Delayed Treatment Effect
Yuichiro Kaneko, Astellas Pharma; Satoshi Morita, Kyoto University
 
 

479 * !
Wed, 7/31/2019, 10:30 AM - 12:20 PM CC-207
Complex Innovative Designs in Practice of Early Phase Drug Development — Invited Papers
Biopharmaceutical Section, ENAR, Society for Clinical Trials
Organizer(s): Vladimir Dragalin, Janssen R&D
Chair(s): Sue-Jane Wang, Center for Drug Evaluation and Research U.S. Food and Drug Administration
10:35 AM Incorporating Time-To-Event Total Toxicity Burden into Dose-Finding Trials
Ji Lin, Sanofi US; Yuan Ji, The University of Chicago; Meizi Liu, University of Chicago
10:50 AM Novel Designs to Accelerate Phase I Oncology Trials
Daniel Li, Juno Therapeutics, A Celgene Company
11:05 AM Adaptive Designs for Drug Combination Informed by Longitudinal Model for the Response
Presentation
Tobias Mielke, Janssen
11:20 AM Bayesian Optimal Interval (BOIN) Design in Phase 1 Oncology Dose-Finding Trials: An Industry Experience
Presentation
Wijith Prasantha Munasinghe, AbbVie Inc
11:35 AM Phase 1/2 Seamless Design
Inna Perevozskaya, GSK; Rosemary Schroyer, GSK; Helen Chen, GSK
11:50 AM Discussant: Yuan Ji, The University of Chicago
12:05 PM Floor Discussion
 
 

491 *
Wed, 7/31/2019, 10:30 AM - 12:20 PM CC-703
Database Lock to Data Safety Monitoring Board Meeting – More Than a Click of a Button — Invited Panel
Section for Statistical Programmers and Analysts, Biopharmaceutical Section, Section on Statistical Consulting
Organizer(s): Vipin Arora, Eli Lilly and Company
Chair(s): Vipin Arora, Eli Lilly and Company
10:35 AM Database Lock to Data Safety Monitoring Board Meeting – More Than a Click of a Button?
Presentation 1 Presentation 2 Presentation 3 Presentation 4
Panelists: David Prince, Axio Research
Kevin Buhr, University of Wisconsin
Lisa Weissfeld, Stats Collaborative
Natasa Rajicic, Cytel Inc
12:15 PM Floor Discussion
 
 

493 * !
Wed, 7/31/2019, 10:30 AM - 12:20 PM CC-506
Leveraging Historical Data and Real World Evidence in Drug Development Program Evaluation — Topic Contributed Papers
Health Policy Statistics Section, Biopharmaceutical Section, International Chinese Statistical Association
Organizer(s): Freda Cooner, Amgen Inc.
Chair(s): Yang Wang, Amgen
10:35 AM From Quantitative Drug Safety to Real-World Evidence: Activities at the US FDA
Hana Lee, U.S Food and Drug Administration; Mark Levenson, FDA CDER
10:55 AM Leveraging Historical Data in Diabetes Cardiovascular Outcome Trials
Shuang Li, Southern Methodist University; Freda Cooner, Amgen
11:15 AM Sources of Data and Statistical Strategies for Design and Analysis: Real World Insights
Presentation
Olga Marchenko, Bayer
11:35 AM Real World Evidence Use in CBER
Jennifer Kirk, FDA, Center for Biologics Evaluation and Research (CBER)
11:55 AM Discussant: Freda Cooner, Amgen Inc.
12:15 PM Floor Discussion
 
 

496 * !
Wed, 7/31/2019, 10:30 AM - 12:20 PM CC-603
Estimand Framework and Its Impact on Drug Development in Oncology — Topic Contributed Papers
Lifetime Data Science Section, Biopharmaceutical Section, Biometrics Section
Organizer(s): Evgeny Degtyarev, Novartis ; Kaspar Rufibach, F. Hoffmann-La Roche
Chair(s): Rui Tang, Servier
10:35 AM Estimand Framework in Oncology Drug Development – Impact and Opportunities
Presentation
Evgeny Degtyarev, Novartis ; Kaspar Rufibach, F. Hoffmann-La Roche; Jonathan Siegel, Bayer HealthCare Pharmaceuticals Inc.; Viktoriya Stalbovskaya, Merus; Steven Sun, J&J
10:55 AM Importance of Censoring Mechanisms in Selecting Appropriate Estimands
Presentation
Jonathan Siegel, Bayer HealthCare Pharmaceuticals Inc.; Michelle Casey , Pfizer; Hans-Jochen Weber, Novartis; Anja Schiel, EMA BSWP/SAWP; Stefan Englert, AbbVie Deutschland GmbH & Co KG; Steven Sun, J&J; Kaspar Rufibach, F. Hoffmann-La Roche
11:15 AM Sensitivity Analysis Vs Supportive Analysis Under Estimand Framework: a Case Study in Hematological Malignancies
Presentation
Steven Sun, J&J; Hans-Jochen Weber, Novartis; Marie-Laure Casadebaig, Celgene; Emily Butler, GlaxoSmithKline; Satrajit Roychoudhury, Pfizer Inc ; Kaspar Rufibach, F. Hoffmann-La Roche; Viktoriya Stalbovskaya, Merus
11:35 AM Estimand Framework – Are We Asking the Right Questions? a Case Study in the Solid Tumor Setting
Presentation
Michelle Casey , Pfizer; Evgeny Degtyarev, Novartis ; Maria Jose Lechuga, Pfizer, Inc.; Paola Aimone, Novartis Pharma AG; Feng Feng Liu, AstraZeneca; Viktoriya Stalbovskaya, Merus; Rui Tang, Servier; Emily Butler, GlaxoSmithKline; Oliver Sailer, Boehringer Ingelheim Pharma GmbH & Co
11:55 AM Discussant: Kunthel By, Division of Biometrics V, OB/OTS/CDER FDA
12:15 PM Floor Discussion
 
 

507
Wed, 7/31/2019, 10:30 AM - 12:20 PM CC-210/212
Non-Inferiority, Biosimilarity and Related Topics — Contributed Papers
Biopharmaceutical Section
Chair(s): Junjing Lin, AbbVie
10:35 AM Use of Tolerance Intervals for Assessing Biosimilarity
Chian Chen, Institute of Population Health Sciences, National Health Research Institutes; Chin-Fu Hsiao, National Health Research Institutes
10:50 AM Incomplete Data Analysis of Non-Inferiority Clinical Trials: Difference in Binomial Proportions Case
Yulia Sidi, University of Connecticut; Ofer Harel, Dept of Statistics, U of Connecticut
11:05 AM Theory and Practice of Equivalence and Non-Inferority Analyzes
Presentation
Kallappa M. Koti, FDA (Retired)
11:20 AM Assessing the Ratio of Means as a Causal Estimand in Clinical Endpoint Bioequivalence Studies in the Presence of Intercurrent Events
Yiyue Lou, University of Iowa College of Public Health; Michael P. Jones, University of Iowa College of Public Health; Wanjie Sun, FDA
11:35 AM New Approaches for Testing Non-Inferiority for Three-Arm Trials with Poisson Distributed Outcomes
Erina Paul, Merck & Co Inc.; Samiran Ghosh, Wayne State University; Shrabanti Chowdhury, Icahn School of Medicine at Mount Sinai
11:50 AM Statistician’s Perspective of Meta-Analysis to Establish Non-Inferiority Margin for Phase 3 Study
Presentation
Aparna Raychaudhuri, CSL Behring; Fanny Mitrani-Gold, GlaxoSmithKline
12:05 PM A Framework for Considering the Risk-Benefit Trade-Off in Designing Trials Using Non-Inferiority or Composite Outcome Approaches
Presentation
Ritesh Ramchandani, Harvard University; Grace Montepiedra, Harvard University; Soyeon Kim, Harvard University; Sachiko Miyahara, Harvard University
 
 

Register 533
Wed, 7/31/2019, 12:30 PM - 1:50 PM H-Centennial Ballroom G-H
Biopharmaceutical Section P.M. Roundtable Discussion (Added Fee) — Roundtables PM Roundtable Discussion
Biopharmaceutical Section
Organizer(s): Stephine L. Keeton, PPD, Inc.
WL08: WITHDRAWN: Statistical and Regulatory Issues in Combining Potency Estimates from Multiple Bioassay Runs
Areti Manola, Janssen R&D
 
 

557 * !
Wed, 7/31/2019, 2:00 PM - 3:50 PM CC-108
Data Monitoring Committees – the Multi-Disciplinary Approach to Drug Safety Assessment — Topic Contributed Papers
Biopharmaceutical Section, International Indian Statistical Association, Section for Statistical Programmers and Analysts
Organizer(s): Amit Bhattacharyya, Alexion Pharmaceuticals
Chair(s): William (Bill) Wang, Merck Research Lab
2:05 PM EMERGING CHANGES in DMC OVERSIGHT
Susan S. Ellenberg, University of Pennsylvania
2:25 PM A Journey Through Guidelines for DMC in Addressing Evolving Paradigm Changes – What Really Matters
Presentation
Estelle Russek-Cohen, FDA CDER
2:45 PM The Perfect DMC – a Multi-Disciplinary Approach to Monitor Patient Safety:
Presentation
Jonathan Seltzer, ACI Clinical
3:05 PM Implementing Effective DMC Decision-Making in Complex Clinical Trial Designs
Presentation
Paul Gallo, Novartis Pharmaceutical
3:25 PM Are Interactive Graphics in a DMC Ready for Prime-Time, for Better Safety Reviews?
Presentation
James Buchanan, Covilance LLC
3:45 PM Floor Discussion
 
 

558 * !
Wed, 7/31/2019, 2:00 PM - 3:50 PM CC-207
The Big Data Revolution in Health Care: Promise and Potential — Topic Contributed Papers
Biopharmaceutical Section, Health Policy Statistics Section, Society for Clinical Trials
Organizer(s): Satrajit Roychoudhury, Pfizer Inc
Chair(s): Satrajit Roychoudhury, Pfizer Inc
2:05 PM Perspectives on Use of Real-World Evidence in Drug Development
Demissie Alemayehu, Pfizer, Inc.; Satrajit Roychoudhury, Pfizer Inc
2:25 PM Reliable Healthcare Evidence from the Large-Scale Evidence Generation Across a Network of Databases (LEGEND) Study
Marc Suchard, UCLA
2:45 PM Longitudinal Causal Inference Using EHRs
Presentation
Roy Adams, Johns Hopkins University; Katharine E Henry, Johns Hopkins University; Hossein Soleimani, University of California - San Fransisco; Michael Rosenblum, Johns Hopkins Bloomberg School of Public Health; Suchi Saria, Johns Hopkins University
3:05 PM Discussant: Aloka Chakravarty, Office of Biostatistics of CDER/FDA
3:25 PM Discussant: Ram Tiwari, CDRH, FDA
3:45 PM Floor Discussion
 
 

571
Wed, 7/31/2019, 2:00 PM - 3:50 PM CC-210/212
Special Topics and Case Studies in Clinical Trials — Contributed Papers
Biopharmaceutical Section
Chair(s): Oluyemi Oyeniran, JNJ
2:05 PM Analysis of Multiple Outcome Measures with Applications to Disability Improvement in Multiple Sclerosis
Wenting Cheng, Biogen; Yangqing Deng, University of Minnesota; Lili Yang, Biogen; Shifang Liu, Biogen; Chunlei Ke, Biogen
2:20 PM Use of Extended Kaplan-Meier and Time-Dependent Cox Model in EU Submission of Kymriah
Jufen Chu
2:35 PM Assessing Similarity of Curves: An Application in Assessing Similarity Between Pediatric and Adult Exposure-Response Curves
Presentation
Yodit Seifu, Merck; Mathangi Gopalakrishnan, University of Maryland; Junshan Qiu, FDA/CDER; Junjing Lin, AbbVie; Margaret Gamalo-Siebers, Eli Lilly
2:50 PM Making an Impact: the Filing Story of ZINPLAVA
Alison Pedley
3:05 PM Assessing Similarity to Support Pediatric Extrapolation
Presentation
Forrest Williamson, Eli Lilly
3:20 PM Dealing with Issues of Pediatric Clinical Trials
Aobo Wang, Merck
3:35 PM Evaluation of Impacts of Concomitant Use of Acetylcholinesterase Inhibitors and Memantine on Cognitive Decline in ADNI Data
Hui Zheng, AbbVie Inc.; Weining Robieson, AbbVie Inc.; Deli Wang, AbbVie; Hana Florian, AbbVie
 
 

605 * !
Thu, 8/1/2019, 8:30 AM - 10:20 AM CC-603
Innovations in Use of Historical Control Data in Clinical Trials — Topic Contributed Papers
Biopharmaceutical Section, Section on Medical Devices and Diagnostics, Biometrics Section, Section on Statistical Consulting, Statistics and Pharmacometrics Interest Group
Organizer(s): Steven Schwager, Cornell University
Chair(s): Steven Schwager, Cornell University
8:35 AM Exploring External Controls Using Prior Clinical Trial Data
Presentation
Pallavi Mishra-Kalyani, US Food and Drug Administration
8:55 AM Composite Likelihood Approach for Incorporating the Entropy Balance Weighting of Real World Data (RWD) in Uncontrolled and Randomized-Controlled Trials (RCTs)
Guanglei Yu, Eli Lilly and Company; Margaret Gamalo-Siebers, Eli Lilly; Yuanyuan Bian, Eli Lilly and Company
9:15 AM Incorporation of Historical Information in Pediatric Trials
Presentation
James Travis, FDA
9:35 AM Real World Data for Oncology Drug Development: Promise and Pitfalls
Presentation
Catherine Tuglus, Amgen; Qui Tran, Amgen; Chris Holland, Immunocore
9:55 AM Strengthening Clinical Trials Through Synthetic Control Arms
Antara Majumdar, Medidata Solutions; Ruthie Davi, Medidata Solutions; Steven Schwager, Medidata Solutions
10:15 AM Floor Discussion
 
 

608
Thu, 8/1/2019, 8:30 AM - 10:20 AM CC-704
Patient-Focused Clinical Trials: Challenges and Considerations for Trial Design, Endpoints, and Analysis — Topic Contributed Panel
Biopharmaceutical Section, Health Policy Statistics Section
Organizer(s): Pallavi Mishra-Kalyani, US Food and Drug Administration; Jessica Roydhouse, US Food and Drug Administration
Chair(s): Jessica Roydhouse, US Food and Drug Administration
8:35 AM Patient-Focused Clinical Trials: Challenges and Considerations for Trial Design, Endpoints, and Analysis
Presentation 1 Presentation 2
Panelists: Mallorie H Fiero, US Food and Drug Administration
Melanie Bell, University of Arizona
Stacie Hudgens, Clinical Outcomes Solutions
Stephanie Manson, Novartis Oncology
10:10 AM Floor Discussion
 
 

610
Thu, 8/1/2019, 8:30 AM - 10:20 AM CC-706
Power, Sample Size, and Applications to Time-To-Event — Contributed Papers
Biopharmaceutical Section
Chair(s): Wenting Cheng, Biogen
8:50 AM Joint Modeling of Longitudinal and Time-To-Event Data with Application to Multiple Myeloma
Liangcai Zhang, Johnson & Johnson; Hong Tian, Janssen Pharmaceutical
9:05 AM A Unified Approach to Sample Size Determination for Common Nonlinear Regression Models
Presentation
Michael J. Martens, The Emmes Corporation; Brent R. Logan, Medical College of Wisconsin
9:20 AM Prediction of Number of Events Based on Blinded or Partially Blinded Survival Data
Presentation
Youyi Shu, Janssen R&D
9:35 AM Analysis of Covariance (ANCOVA) in Randomized Trials: More Precision and Valid Confidence Intervals, Without Model Assumptions
Presentation
Bingkai Wang, Johns Hopkins Bloomberg School of Public Health; Michael Rosenblum, Johns Hopkins Bloomberg School of Public Health; Elizabeth Ogburn, Johns Hopkins Bloomberg School of Public Health
9:50 AM Power Calculations for Common, Nonparametric Tests in Survival
Godwin Yung, Takeda Pharmaceuticals; Yi Liu, Nektar Therapeutics
10:05 AM Floor Discussion
 
 

628 * !
Thu, 8/1/2019, 10:30 AM - 12:20 PM CC-710
Advances in Clinical Outcome Assessments — Invited Papers
Biopharmaceutical Section, Health Policy Statistics Section, Biometrics Section
Organizer(s): Joseph C Cappelleri, Pfizer Inc
Chair(s): Bellinda King-Kallimanis, U.S. Food and Drug Administration
10:35 AM Methods for Identifying Treatment-Emergent Symptomatic Adverse Events from the Patient Perspective with Application to the PRO-CTCAE
Presentation
Gina Lynn Mazza, Mayo Clinic; Ethan Basch, University of North Carolina Lineberger Comprehensive Cancer Center; Lauren J. Rogak, Memorial Sloan Kettering Cancer Center; Amylou C. Dueck, Mayo Clinic
11:00 AM Mixed Location Scale Hidden Markov Model with an Application to Ecological Momentary Assessment Data
Presentation
Xiaolei Lin, Fudan University; Robin Mermelstein, University of Illinois at Chicago; Donald Hedeker, University of Chicago
11:25 AM Identification of Distinct Disease-Activity Trajectories in Patients with Rheumatoid Arthritis
Presentation
David Gruben, Pfizer, Inc.
11:50 AM Discussant: Joseph C Cappelleri, Pfizer Inc
12:10 PM Floor Discussion
 
 

644 * !
Thu, 8/1/2019, 10:30 AM - 12:20 PM CC-607
Statistical Methods for the Co-Development of Drug and Companion Diagnostic in Oncology — Topic Contributed Papers
Section on Medical Devices and Diagnostics, Biopharmaceutical Section, Society for Medical Decision Making
Organizer(s): Rong Liu, Celgene
Chair(s): Frank Shen, Celgene Co.
10:35 AM Evaluation of Biomarker Threshold Designs in Cancer Therapy
Kui Shen, Bayer U.S. LLC; Xiaowen Tian, University of Washington; Jonathan Siegel, Bayer HealthCare Pharmaceuticals Inc.
10:55 AM A Case Study in Bridging for Companion Diagnostic Development: Pembrolizumab and PD-L1 Selected 2nd Line NSCLC Patients
Jared Lunceford, Merck & Co., Inc.; Ellie Corigliano, Merck & Co., Inc.; Siddhartha Mathur, Merck & Co., Inc.; Ziwen Wei, Merck & Co., Inc.; Yue Shentu, Merck & Co., Inc.
11:15 AM Drug-Device Co-Development in the Era of Precision Medicine: Industry Perspectives on Statistical Challenges
Shunguang Wang, Novartis Institutes for BioMedical Research Inc.
11:35 AM Logical Inference on Treatment Efficacy When Subgroups Exist
Presentation
Ying Ding, University of Pittsburgh
11:55 AM Innovative Designs for Drug-Device Co-Development in the Area of Precision Medicine
Rui Tang, Servier
12:15 PM Floor Discussion
 
 

646 * !
Thu, 8/1/2019, 10:30 AM - 12:20 PM CC-708
Applications of Deep Learning in Pharmaceutical Development — Topic Contributed Papers
Biopharmaceutical Section, Section on Statistical Learning and Data Science, Biometrics Section, Text Analysis Interest Group
Organizer(s): Xin Huang, AbbVie Inc.
Chair(s): Weili He, AbbVie
10:35 AM Deep Learning-Based Histology Image Analysis for Patient Diagnosis and Selection
Presentation
Xin Huang, AbbVie Inc.; Liuqing Yang, AbbVie; Yan Sun, AbbVie; Mufeng Hu, AbbVie
10:55 AM Leveraging Free Text Data for Decision Making in Drug Development
Presentation
Yan Sun, AbbVie; Jiyeong Jang, University of Illinois at Chicago; Xin Huang, AbbVie Inc.; Hongwei Wang, AbbVie Inc.; Weili He, AbbVie
11:15 AM Diagnosis of Diabetic Retinopathy Using Medical Images and Deep Learning Method
Xuanyao He, Eli Lilly and Company
11:35 AM Discussant: Hongwei Wang, AbbVie Inc.
11:55 AM Discussant: Mandy Jin, Merck & Co., Inc.
12:15 PM Floor Discussion
 
 

650 * !
Thu, 8/1/2019, 10:30 AM - 12:20 PM CC-501
Quantum Computing: Optimization Algorithms and Applications — Topic Contributed Papers
Section on Statistical Computing, Biometrics Section, Biopharmaceutical Section
Organizer(s): Sergei Leonov, CSL Behring
Chair(s): James Wendelberger, Los Alamos National Laboratory and University of New Mexico
10:35 AM Quantum Computing in the Life Sciences
Mark Fingerhuth, ProteinQure
10:55 AM Treasure Hunt for Computational Problems That Can Be Solved Faster by Quantum Annealing
Presentation
Barry Sanders, University of Calgary; Archismita Dalal, University of Calgary; Radhakrishnan Balu, United States Army Research Laboratory
11:15 AM Quantum Computing at Lockheed Martin
Kristen Pudenz
11:35 AM Optimization Algorithms of Model-Based Design: Simulated Vs Quantum Annealing
Presentation 1 Presentation 2
Valerii Fedorov, ICONplc
11:55 AM Discussant: Sergei Leonov, CSL Behring
12:15 PM Floor Discussion
 
 

653
Thu, 8/1/2019, 10:30 AM - 12:20 PM CC-712
Machine Learning and Other Statistical Methods in Clinical Trials — Contributed Papers
Biopharmaceutical Section
Chair(s): Sherlly Xie, Boston Scientific
10:35 AM Deep Neural Networks for Survival Analysis Using Pseudo Values
Presentation
Dai Feng, AbbVie; Lili Zhao, University of Michigan
10:50 AM Alternatives to Logistic Regression for Detecting Treatment by Covariate Interactions with Binary Endpoints
Presentation
Radha Railkar, Merck & Co., Inc.; Devan Mehrotra, Merck & Co., Inc
11:05 AM Comparison of Data Mining Methods for Signal Detection of Targeted Therapy Related Adverse Events in Breast Cancer Patients
Presentation
Efstathia Polychronopoulou, UTMB; Sharon Giordano, MD Anderson Cancer Center; Lin-Na Chou, The University of Texas Medical Branch; Xiaoying Yu, UTMB; Yong-Fang Kuo, The University of Texas Medical Branch
11:20 AM Application of CART Regression in Early Discovery Efforts to Better Understand Proinsulin as Possible Therapeutic Target
Presentation
Santosh Sutradhar, Merck & Co., Inc.; Geoffrey Walford, Merck & Co., Inc.; Tami Crumley, Merck & Co., Inc.; Anita Lee, Merck & Co., Inc.; Jennifer Abrams, Merck & Co., Inc.
11:35 AM Machine Learning Methods Evaluation for Small-Size Overlapping Data with Class Imbalance Issue
Presentation
Guolin Zhao, Biogen Inc.; Shuo Li, Boston University,; Feng Gao, Biogen Inc.
11:50 AM Statistical Analysis and Machine Learning Using Data from Continuous Glucose Monitoring in Clinical Trials
Presentation
Chen Gao, MedImmune; Yi-Ting Chang, MedImmune; Jay Zhang, MedImmune
12:05 PM Floor Discussion