All Times EDT
Legend:
Activity Details
5
Mon, 8/3/2020,
10:00 AM -
11:50 AM
Virtual
Recent Development on Statistical Methods for Precision Medicine — Invited Papers
ENAR , Biometrics Section, Section on Statistics in Epidemiology, Biopharmaceutical Section
Organizer(s): Guanhua Chen, University of Wisconsin-Madison
Chair(s): Yanyao Yi, Eli Lilly and Company
10:05 AM
A Two-Part Model for Individualized Treatment Rule Estimation with Semi-Continuous Outcomes
Guanhua Chen, University of Wisconsin-Madison ; Maureen Smith, University of Wisconsin-Madison; Jared Huling, The Ohio State University
10:30 AM
Bayesian Nonparametric Survival Regression for Optimizing Precision Dosing of Intravenous Busulfan in Allogeneic Stem Cell Transplantation
Presentation
Peter F. Thall, M.D. Anderson Cancer Center ; Yanxun Xu, Johns Hopkins University; William Hua, Johns Hopkins University; Borje Andersson, M.D. Anderson Cancer Center
10:55 AM
Targeted Learning of Causal Impact of Optimal Individualized Treatment Rules Based on Novel Sequentially Adaptive Designs
Ivana Malenica, University of California, Berkeley
11:20 AM
Stochastic Tree Search for Estimating Personalized Treatment Decision Rules
Lu Wang, University of Michigan ; Yilun Sun, University of Michigan Medical School
11:45 AM
Floor Discussion
15 * !
Mon, 8/3/2020,
10:00 AM -
11:50 AM
Virtual
Incorporating Knowledge from Previous Clinical Trials into the Design of New Trials — Invited Papers
Biopharmaceutical Section , Section on Medical Devices and Diagnostics
Organizer(s): Steven J Schwager, Cornell University
Chair(s): Steven J Schwager, Cornell University
10:05 AM
Borrowing from Historical Data in Cancer Drug Development: A Cautionary Tale and Experience with Bayesian Adaptive Platform Designs
James Normington, University of Minnesota ; Connor Jo Lewis, Securian Financial Group, Inc.; Somnath Sarkar, Flatiron, Inc.; Jiawen Zhu, Roche-Genentech; Federico Mattiello, F. Hoffman-La Roche, Inc.
10:30 AM
Statistical Methods and Process for Historical Data Borrowing in Clinical Trials
Lanju zhang, AbbVie Inc ; Zailong Wang, AbbVie Inc; Ivan Chan, AbbVie Inc
10:55 AM
Bayesian Synthetic Control Approaches in Pediatric Drug Development: A Case Study in Acute Lymphoblastic Leukemia
Antara Majumdar, Medidata Solutions ; Jingjing Ye, FDA; Rebecca Rothwell, FDA; Corinne Ahlberg, Acorn AI by Medidata, a Dassault Systèmes company
11:20 AM
Discussant: Pallavi Mishra-Kalyani, US FDA
11:45 AM
Floor Discussion
22 * !
Mon, 8/3/2020,
10:00 AM -
11:50 AM
Virtual
Design and Evaluation of Adaptive Design Clinical Trials: Software Demonstration — Invited Panel
Section for Statistical Programmers and Analysts , Biopharmaceutical Section, Society for Clinical Trials
Organizer(s): Navneet Hakhu, University of California, Irvine
Chair(s): Navneet Hakhu, University of California, Irvine
10:05 AM
Design and Evaluation of Adaptive Design Clinical Trials: Software Demonstration
Panelists:
Scott Berry, Berry Consultants Keaven Anderson, Merck & Co Yannis Jemiai, Cytel Inc Dan Gillen, University of California, Irvine
11:40 AM
Floor Discussion
31 * !
Mon, 8/3/2020,
10:00 AM -
11:50 AM
Virtual
Combination Therapy Dose-Finding in Oncology Drug Development — Topic Contributed Papers
Biopharmaceutical Section , Section on Bayesian Statistical Science, International Chinese Statistical Association
Organizer(s): Rong Liu, BMS
Chair(s): Olga Marchenko, Bayer
10:05 AM
Comparative Review of Phase I Dose-Finding Designs for Drug Combination Trials
Heng Zhou, Merck ; Ruitao LIN, The University of Texas MD Anderson Cancer Center
10:25 AM
Dual-Agent Drug-Combination Designs for Finding Multiple Maximum Tolerated Dose Combinations in Oncology Phase I Trials
Xin Yang, Novartis
10:45 AM
Industry Experience on Combination Therapy Dose-Finding in Oncology Drug Development
Rong Liu, BMS
11:05 AM
Emerging Approaches in Combination Phase I Trials: Utilization of Historical Data and the Need to Consider Model Guided Designs and Endpoints Other Than DLTs
Suman Sen, Novartis Pharmaceuticals Corp
11:25 AM
Discussant: Cindy Gao, FDA
11:45 AM
Floor Discussion
62
Mon, 8/3/2020,
10:00 AM -
2:00 PM
Virtual
Topics in Adaptive Designs: Adaptive-Seamless, Group Sequential and Other Designs — Contributed Papers
Biopharmaceutical Section
Chair(s): Cristiana Mayer, Janssen / Johnson & Johnson
Seamless Adaptive Phase 2-3 Designs Allowing for Mid-Term Modifications Using Multiplicity Strategy
Presentation
Mandy Jin, AbbVie Inc.
Overview of Recent Advances in Biomarker Driven Adaptive Designs
Ilya Lipkovich, Eli LIlly and Company ; Sarah E Johnson , University of Oklahoma Health Sciences Center; Alex Dmitrienko , Mediana, Inc; Daniel Zhao , University of Oklahoma Health Sciences Center
Adaptive Simon’s Two-Stage Design with or Without Targeting Biomarker Subpopulation
Weichao Bao, Bayer
An Adaptive-Flavored Group Sequential Design with Connection to Conditional Error
Presentation
Dong Xi, Novartis ; Paul Gallo, Novartis
Automated Design of Adaptive Trials Using Bayesian Optimization
Joe Marion, Berry Consultants
Early Phase Cancer Clinical Trial Design for Dose Finding of Treatment Combinations Across Patient Groups
Bethany Horton, University of Virginia
63 *
Mon, 8/3/2020,
10:00 AM -
2:00 PM
Virtual
Power and Sample Size: Methods and Applications — Contributed Papers
Biopharmaceutical Section
Chair(s): John J. Han, Janssen / Johnson & Johnson
Sample Size and Power Estimation in a Rare Disease Trial Using Mixture of Negative Binomial Distributions
Aparna Raychaudhuri, CSL Behring ; Sergei Leonov, CSL Behring; Harald Iverson, CSL Behring
Calculating Power for the Finkelstein and Schoenfeld Test Statistic for a Composite Endpoint with Two Components
Thomas Zhou, Boston University ; Michael P LaValley, Boston University; Kerrie P Nelson, Boston University; Howard Cabral, Boston University; Joseph M Massaro, Boston University
Sample Size and Power Computations Methods for Two-Stage Randomized Trial, with Focus on Time-to-Event Data
Rouba Chahine, University of Alabama at Birmingham ; Inmaculada Aban, University of Alabama at Birmingham; Dustin Long, University of Alabama at Birmingham
Model-Robust Inference for Clinical Trials That Improve Precision by Stratified Randomization and Adjustment for Additional Baseline Variables
Bingkai Wang, Johns Hopkins Bloomberg School of Public Health ; Michael Rosenblum, Johns Hopkins Univ, Bloomberg School of Public Health
Improved Analyses of Randomized Clinical Trials with Stratified Enrollment
Devan Mehrotra, Merck ; Rachel Marceau West, Merck Research Laboratories; Julie Kobie, Merck & Company Inc
Power Formulas for Mixed Effects Models with Random Slope and Intercept Comparing Rate of Decline Across Groups
Presentation
Yu Zhao, UC-San Diego ; Sarah J Banks, UC San Diego; Mark W Bondi, UC San Diego; Steven D Edland, UC San Diego
Uncover Evidence in Real World Data with Uncertain Genetic Information
Wei Zhuang, NCTR, FDA
64
Mon, 8/3/2020,
10:00 AM -
2:00 PM
Virtual
Statistical Issues Specific to Therapeutic Areas — Contributed Papers
Biopharmaceutical Section
Chair(s): Gajanan Bhat, Spectrum Pharmaceuticals
Simulation Studies of Different Statistical Methods for Time-to-Event Prediction
Yiding Zhang, University of Massachusetts Amherst ; Wenjing Lu, Abbvie; Ying Zhou, Abbvie; Danielle M Sullivan, Abbvie
Borrowing Historical Data for Vaccine Efficacy Trials
Guanghan Frank Liu, Merck Inc. ; Mandy Jin, AbbVie Inc.; Dai Feng, AbbVie
An Extension of Bayesian Principal Stratum Analysis of Clinical Disability Progression in SPMS
Zhe Chen, Biogen ; Katherine Riester, Biogen
Bayesian Approaches for Modeling Repeated Computerized Assessment of Cognitive Function in Alzheimer’s Disease
Nairita Ghosal, Merck & Co., Inc., ; Santosh Sutradhar, Merck & Co., Inc.; Sarah Janicki Hsieh, Merck & Co., Inc.,
Prevalence of Response for Evaluation of Duration of Response with Application to Disability Improvement in Multiple Sclerosis
Zheng Ren, Biogen - RTP, NC ; Li Zhu, Biogen; Chunlei Ke, Biogen
A Critical Appraisal of Statistical Practice in Translational Animal Studies
Olivia Hogue, Cleveland Clinic and Case Western Reserve University ; Eashwar Somasundaram, Case Western Reserve University School of Medicine; Kenneth B Baker, Department of Neurosciences, Cleveland Clinic; Jill S Barnholtz-Sloan, Department of Population and Quantitative Health Sciences, Case Western Reserve University; Abagail Postle, Neurological Institute, Cleveland Clinic; Tucker J Harvey, University of Pittsurgh; Dena Crozier, Cleveland Clinic Lerner College of Medicine; Francis May, Cleveland Clinic Lerner College of Medicine
The Mechanistic Analysis of Founder Virus Data in Challenge Models
Ana Maria Ortega-Villa, National Institutes of Health ; Dean Follmann, NIAID
Treatment Effect in Sequential Parallel Comparison Design Studies
Xiaoyan Liu, Boston University ; Gheorghe Doros, Boston University
77
Mon, 8/3/2020,
10:00 AM -
2:00 PM
Virtual
Contributed Poster Presentations: Biopharmaceutical Section — Contributed Poster Presentations
Biopharmaceutical Section
1:
The Framework of a Three-Stage Design for Allergen Immunotherapy Trials
Xinyu Tang, FDA ; Lihan Yan, Vaccine Evaluation Branch-CBER-FDA
2:
Using Digital Data in an Early Phase Clinical Trial to Quantitate Baroreceptor Sensitivity Variability
Anwesha Mukherjee, Merck & Co Inc ; Geoffrey Walford, Merck& Co., Inc; Leticia Arrington, Merck & Co, Inc; Adrienne DiBenedetto, Merck & Co., Inc; Ruben Declercq, Merck & Co, Inc; Inge DeLepeleire, Merck & Co., Inc; Helia Norouzizadeh, Merck & Co., Inc
3:
Statistical Impact of Delayed Treatment Effect: A Simulation Study
Yuanbo Song, Novartis Pharmaceuticals Corp ; Bharani Bharani-Dharan, Novartis Oncology
4:
The Role of Design and Analysis Methods on Go/No-Go Decision in Proof-of-Concept Trials
Macaulay Okwuokenye
5:
Accounting for Suboptimal Treatment Adherence When Estimating Heterogenous Treatment Effects from Observational Data
Gabrielle Simoneau, Biogen Canada ; Fabio Pellegrini, Biogen International GmbH; Carl de Moor, Biogen Inc; Lu Tian, Department of Biomedical Data Sciences, Stanford University
6:
Regional Efficacy Evaluation in Multi-Regional Clinical Trials Using a Discounting Factor Weighted Z Test
XUANXUAN YU, Dept of Epidemiology & Biostatistics, School of Public Health, University of South Carolina ; Jianling Bai, School of Public Health, Nanjing Medical University; Hao Yu, School of Public Health, Nanjing Medical University
7:
Multiplicity in Vaccine Clinical Studies – A Case Study with Gate-Keeping Testing Strategy
Huajun Wang, GSK Vaccines ; Ellen Ypma, GSK Vaccines; Uwe Nicolay, GSK Vaccines
8:
Interim Analysis in Double Blinded Vaccine Trials Using the Semiparametric Density Ratio Model
Jing Qin, National Institute of Allergy and Infectious Diseases
9:
TITE-ToBI: The Time-to-Event Toxicity Burden Interval Design to Accelerate Phase I Trials
Meizi Liu, University of Chicago ; Yuan Ji, University of Chicago
10:
Revisiting Prior Selection for Scale Parameters in Hierarchical Models
Qianqiu Li, Janssen Research & Development ; Jyh-Ming Shoung, Janssen Research & Development; Bill Pikounis, Janssen Research & Development
11:
Bioequivalence Study Design for Multiplicity
Michelle Quinlan, Novartis Oncology ; Ekkehard Glimm, Novartis; Bharani Bharani-Dharan, Novartis Oncology
12:
Bayesian Topic Modeling of Adverse Event Data
Andrew Bean, Novartis ; Thibaud Coroller, Novartis
13:
Statistical Analysis Considerations for Clinical Outcome Assessments Based Endpoints
Chul Ahn, FDA-CDRH
14:
Randomization and Randomization Tests for Multi-Armed Randomized Clinical Trials
Yanying Wang
15:
A Multiomics Statistical Framework for Drug Repositioning
Zhaolong Yu, Yale University ; Kexuan Liang, Peking University; Hongyu Zhao, Yale University
16:
Imputation Strategies Within the Estimand Framework to Evaluate the Overall Likelihood of Patient Improvement in Longitudinal Trails
Lysbeth Floden, Clinical Outcomes Solutions
17:
Cost-Efficient Two Stage Sequential Multiple Assignment Randomized Trial Designs
Grecio Sandoval, The George Washington University ; Ionut Bebu, The George Washington University; John Lachin, The George Washington University
18:
Evaluation of Safety Signals Using Association Rule Mining (ARM)
Jagannath Ghosh, Novartis Pharmaceutical ; Tania Roy, Novartis Pharmaceutical
19:
Evaluation of Improper Analysis of Clinical Trials with Prognostic Factor Not Pre-Specified in Stratified Randomization
Shuyan Wan, Merck ; Pingye Zhang, Merck & Co, Inc; Christine Gause, Merck & Co, Inc
21:
Estimation of Discrete Survival Function Through Modeling Diagnostic Accuracy for Mismeasured Outcome Data
Hee-Koung Joeng, MERCK
22:
Dismantling the Fragility Index: A Demonstration of Statistical Reasoning
Gail Potter, The EMMES Corporation
23:
ATCV: A Shiny Application for Safety Tables Review
Lie Li, Merck & Co., Inc. ; Yiwen Luo, Merck & Co., Inc.; Clare Bai, Merck & Co., Inc.
24:
Development of MSD Multiplex Antigenicity Assay
Yongwei Liu, Sanofi Pasteur ; Lingyi Zheng, Sanofi; Jason Szeto, Immunology Platform, Sanofi Pasteur; Melinda Donovan, Immunology Platform, Sanofi Pasteur; Tricia Chen, Immunology Platform, Sanofi Pasteur
25:
Bayesian Prior Choice in Clinical Trials with Binary Responses Using Historical Data
Yonggang Zhao, I-Mab Biopharma ; Qianqiu Li, Janssen Research & Development
26:
Evaluation of Dependency of Multiple Events
Dateng Li
27:
Just in Time, on the Job Statistics Training for Biologics Process Characterization
Melissa Matzke, Merck & Co., Inc. ; Seth Clark, Merck & Co., Inc.; Richard K. Burdick, Burdick Statistical Consulting, LLC; David Christopher, Merck & Co., Inc.
28:
Implementations and Interim Monitoring for Two-Stage Phase II/III Adaptive Designs with a Case Example
Aijun Gao, Covance ; Lingyun Liu, Cytel
29:
On the Use of the Bootstrap to Test Functions of Correlated Variances in Pre-Post Clinical Trials
Navneet Hakhu, University of California, Irvine ; Dan Gillen, University of California, Irvine
30:
AUTOMATING the ORGANIZATION and PROCESSING of BIOASSAY DATA with the R PACKAGE and R SHINY APP AXL
JENNIFER NGUYEN, Merck ; TOM STEINMETZ, Merck
31:
Curtailed Binomial Sampling Design for Clinical Trials with Two Dependent Endpoints
Chishu Yin, Syracuse University ; Pinyuen Chen, Syracuse University
32:
Bayesian Data Envelopment Analysis for Assessing Drug Benefit-Risk
Guangyi Gao ; Jo A. Wick, University of Kansas Medical Center; Byron J Gajewski, KUMC
33:
A Bayesian Adaptive Phase I/II Clinical Trial Design with Late-Onset Competing Risk Outcomes
YIFEI ZHANG ; YONG ZANG, INDIANA UNIVERSITY
35:
Prediction of Long Term Joint Damage in Placebo Patients with Active Psoriatic Arthritis
Yan Liu, Johnson and Johnson ; Prasheen Agarwal, Johnson and Johnson; Xiwu Lin, Janssen Research and Development, LLC; Bei Zhou, Johnson and Johnson
36:
The Use of Subgroup Meta-Analysis to Support Decision Making of Prescription Label Change
Mehreteab Aregay, Novartis Pharmaceuticals Corporation ; Baldur Magnusson, Novartis Pharmaceuticals Corporation; Gregory Ligozio, Novartis Pharmaceuticals Corporation
37:
Preparation and Qualification of an Internal Rabies Reference Standard for RFFIT: From Present to Future
Mohammad Rahman, Sanofi ; Lingyi Zheng, Sanofi
38:
Using Bootstrap to Verify Normal Assumptions in Statistical Inference for Treatment Difference
Ruji Yao, Merck ; Amarjot Kaur, Merck & Co., Inc; Qing Li, Merck Research Labs; Anjela Tzontcheva, Merck & Co., Inc
39:
Evaluations of Methods for Missing Data Imputation Under Missing Not at Random
Weining Robieson, AbbVie ; Mandy Jin, AbbVie Inc.
40:
On the No Free Lunch and Logistic Regression in Healthcare Prediction Problems
Xuan Zhou, Merck Research Lab ; Thomas Jemielita, Merck & Co., Inc; Jie Chen, Merck & Co., Inc.; Xin Chen, Merck Co. & Inc; Gail Fernandes, Merck Co. & Inc; Zifang Guo, Merck Co. & Inc; Zhiwen Liu, Merck Co. & Inc; Richard Baumgartner, Merck
41:
Evaluation of Type I Error Control for Testing Clinical Endpoint with Pre-Testing Surrogate Endpoint
Hsiao-Hui Tsou, National Health Research Institutes ; Yu-Chieh Cheng, National Health Research Institutes; Hsiao-Yu Wu, National Health Research Institutes; Ya-Ting Hsu, National Health Research Institutes; Fang-Jing Lee, National Health Research Institutes; James Hung, Center for Drug Evaluation and Research, FDA
43:
Yet Another Randomization System
Dennis Sweitzer
44:
Quantitative Determination of Threshold in Clinical Outcome Assessments (COA) for a Clinically Meaningful Change
Wenting Cheng, Biogen ; Zijuan Chen, Texas A&M University; Kun Chen, Biogen; Li Zhu, Biogen; Chunlei Ke, Biogen
46:
One- and Two-Sided Hypothesis Tests for a Single Proportion with Clustered Binary Data: Derivation, Type I Error, and Power
Meghan I Short, UT Health San Antonio ; Howard Cabral, Boston University; Janice M Weinberg, Boston University; Michael P LaValley, Boston University; Gina Peloso, Boston University; Joseph M Massaro, Boston University
47:
Modified Factorial Design
Li Yu, Merck & Co., Inc. ; Shu-Chih Su, Merck & Co., Inc.
48:
Utilizing Historical Data in Bayesian Interval Dose-Escalation Designs
Frank Shen
49:
The Effect of Unobserved Covariate in Statistical Inference Under Covariate-Adaptive Randomized Experiment
Yang Liu, George Washington University ; feifang Hu, George Washington University
50:
Impact of Competing Risk on Win Ratio Analysis for Composite Time-to-Event Endpoint
Ran Liao, Eli Lilly and Company ; Bochao Jia; Margaret Gamalo-Siebers, Eli Lilly & Co; Guanglei Yu, Eli Lilly and Company; Sujatro Chakladar, Eli Lilly and Company
51:
Bayesian Design of Clinical Trials for Joint Models of Recurrent Event with a Terminating Event
Jiawei Xu, UNC Chapel Hill ; Matthew A. Psioda, University of North Carolina; Joseph G. Ibrahim, University of North Carolina at Chapel Hill
100 * !
Mon, 8/3/2020,
1:00 PM -
2:50 PM
Virtual
Statistical Innovations for Drug Approval and Reimbursement for Rare Disease — Invited Papers
International Indian Statistical Association , Biopharmaceutical Section, Health Policy Statistics Section
Organizer(s): Amit Bhattacharyya, Alexion Pharmaceuticals
Chair(s): Amit Bhattacharyya, Alexion Pharmaceuticals
1:05 PM
On Analysis of Single Arm Trial with Natural History Controls
Qing Liu, Quantitative and Regulatory Medical Science, LLC
1:25 PM
Challenges in Rare Disease Clinical Trials: The PREVENT Trial for NMOSD
Fanny O'Brien, Alexion
1:45 PM
Predictive Models in Rare Diseases: Risks and Opportunities in Clinical Trials, with Examples in Amyotrophic Lateral Sclerosis (ALS)
Alex Karanevich, EMB Statistical Solutions
2:05 PM
Innovative Designs and Rare Diseases: Bridging Information Through Evidence Synthesis
Satrajit Roychoudhury, Pfizer Inc.
2:25 PM
Use Case Scenario: Indirect Comparisons to Enhance Reimbursement Negotiations in Rare Disease
Jinesh Shah, CSL Behring
2:45 PM
Floor Discussion
114 * !
Mon, 8/3/2020,
1:00 PM -
2:50 PM
Virtual
ICH E9 R1 Addendum: Can We Deliver on the Promise? — Invited Panel
Council of Chapters , Biopharmaceutical Section, ENAR
Organizer(s): Pilar Lim, Janssen Research & Development, LLC; Akiko Okamoto, Janssen Research & Development, LLC
Chair(s): Pilar Lim, Janssen Research & Development, LLC
1:05 PM
ICH E9 R1 Addendum: Can We Deliver on the Promise?
Presentation
Panelists:
Craig Mallinckrodt, Biogen Steve Ruberg, Analytix Thinking, LLC Elena Polverejan, Janssen Pharmaceuticals Scott Emerson, University of Washington H. M. James Hung, CDER at FDA
2:45 PM
Floor Discussion
118 * !
Mon, 8/3/2020,
1:00 PM -
2:50 PM
Virtual
Recent Advances in the Design and Analysis of Platform Trials — Topic Contributed Papers
Section on Statistical Graphics , Biopharmaceutical Section, Section on Statistical Computing
Organizer(s): Bo Huang, Pfizer Inc.
Chair(s): Enayet Talukder, Pfizer Inc.
1:05 PM
Basket Design Vs Umbrella Design for Exploratory Trials
Cong Chen, Merck and Company, Inc.
1:25 PM
Finding a Balance of Synergy and Flexibility in Master Protocols
Melanie Quintana, Berry Consultants
1:45 PM
RoBoT: A Robust Bayesian Hypothesis Testing Method for Basket Trials
Yuan Ji, University of Chicago ; Tianjian Zhou, University of Chicago
2:05 PM
Implementation of a Platform Trial in Early Development
Dmitri Pavlov, Pfizer
2:25 PM
Discussant: Erik Bloomquist, FDA
2:45 PM
Floor Discussion
120 * !
Mon, 8/3/2020,
1:00 PM -
2:50 PM
Virtual
Recent Advances in Vaccine Dose and Regimen Finding — Topic Contributed Papers
Biopharmaceutical Section , Biometrics Section
Organizer(s): Robert D Small, Dr.
Chair(s): Ayca Ozol-Godfrey, Sunovion Pharmaceuticals
1:05 PM
SOME ADAPTIVE METHODS for VACCINE DOSE-FINDING
Presentation
James Bolognese, Cytel Inc.
1:25 PM
Considerations in Vaccine Dose-Finding Studies
Aiying Chen, sanofi Pasteur ; Scott Patterson, Sanofi Pasteur ; Fabrice Bailleux, Sanofi Pasteur; Tifany Machabert, Sanofi Pasteur
1:45 PM
Model Informed Development in Vaccines Immunizes Against Choosing Poor Vaccine Candidates and/or Non-Informative Dose-Levels
Presentation
Radha Railkar, Merck & Co., Inc. ; Jeff Sachs, Merck; Jos Lommerse, Certara; Nele Mueller-Plock, Certara; S.Y. Amy Cheung, Certara; Brian Maas, Merck; Luzelena Caro, Merck; Antonios Aliprantis, Merck; Kalpit Vora, Merck; Amy Espeseth, Merck; Andrew Lee, Merck
2:05 PM
Discussant: Lihan Yan, Vaccine Evaluation Branch-CBER-FDA
2:25 PM
Discussant: Debra Yeskey, CEPI
2:45 PM
Floor Discussion
146 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Methods to Evaluate and Correct for Bias in Patient-Reported Outcomes in Clinical Trials: Advancing the Validity of Patient-Centric Drug Development — Invited Papers
Biopharmaceutical Section , Health Policy Statistics Section, Biometrics Section
Organizer(s): Joseph C. Cappelleri, Pfizer Inc.; Jessica Roydhouse, Menzies Institute for Medical Research, University of Tasmania
Chair(s): Demissie Alemayehu, Pfizer Inc.
10:05 AM
Addressing Bias in Responder Analyses of Patient-Reported Outcomes
Presentation
Joseph C. Cappelleri, Pfizer Inc.
10:30 AM
Evaluating the Effect of Interventions on Patient Function in Trials of Severely Ill Patients
Presentation
Elizabeth Colantuoni, Johns Hopkins Bloomberg School of Public Health ; Daniel O Scharfstein, Johns Hopkins University; Ximin Li, Johns Hopkins University
10:55 AM
Assessing Open-Label Bias in Patient-Reported Outcomes in Cancer Trials
Jonathon Vallejo, FDA
11:20 AM
Discussant: Jessica Roydhouse, Menzies Institute for Medical Research, University of Tasmania
11:45 AM
Floor Discussion
148 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Master Protocols in Oncology Drug Development — Invited Papers
ENAR , Biometrics Section, Biopharmaceutical Section
Organizer(s): Xiaoyun (Nicole) Li, Merck
Chair(s): Xiaoyun (Nicole) Li, Merck
10:05 AM
Master Protocols in Oncology: A Regulatory Perspective
Min (Annie) Lin, FDA
10:30 AM
ASA Oncology Working Group Sub-Team Report on Oncology Master Protocols
Chengxing (Cindy) Lu, Biogen Inc. ; Xiaoyun (Nicole) Li, Merck
10:55 AM
Bayesian Platform Trials for Drug Registration
Don Berry, Berry Consultants
11:20 AM
Discussant: Erik Bloomquist, FDA
11:45 AM
Floor Discussion
153 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Utilization of Historical Data for Confirmatory Trials — Topic Contributed Papers
Biopharmaceutical Section , Health Policy Statistics Section, Society for Clinical Trials
Organizer(s): Freda Cooner, Amgen Inc.
Chair(s): Freda Cooner, Amgen Inc.
10:05 AM
Use of Historical Data in Regulatory Applications – Advantages and Challenges
Shiowjen Lee, FDA/CBER/OBE/DB
10:25 AM
Statistical Considerations for Borrowing Historical Data in Small Populations
Fanni Natanegara, Eli Lilly and Company
10:45 AM
Historical Data Borrowing with Informative Inferential Prior for Bayesian Hypothesis Testing
Presentation
Hui Quan, Sanofi ; Bingzhi Zhang, Sanofi; Yu Lan, Sanofi; Xiaodong Luo, Sanofi; Xun Chen, Sanofi
11:05 AM
Improving Clinical Trial Efficiency Using Natural History Study Data and Innovative Trial Design
Presentation
Barbara Wendelberger, Berry Consultants, LLC ; Melanie Quintana, Berry Consultants; Scott Berry, Berry Consultants
11:25 AM
Discussant: Haijun Ma, NeKtar Therapeutics
11:45 AM
Floor Discussion
154 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Integrating Real World Data with Clinical Trials: Opportunities and Challenges — Topic Contributed Papers
Caucus for Women in Statistics , ENAR, Biopharmaceutical Section
Organizer(s): Yimei Li, University of Pennsylvania
Chair(s): Yimei Li, University of Pennsylvania
10:05 AM
An Integrated Analysis of Randomized Clinical Trials and Real World Evidence Studies: Theory and Application
Xiaofei Wang, Duke University ; Shu Yang, North Carolina State University
10:25 AM
Synthesizing External Aggregated Information in the Presence of Population Heterogeneity: A Penalized Empirical Likelihood Approach
Mi-Ok Kim, UCSF
10:45 AM
Multiple Imputation Strategies for Handling Missing Data When Transporting Randomized Clinical Trial Findings Using Propensity Score-Based Methodologies
Albee Ling, Stanford University ; Maria Montez-Rath, Stanford University; Kris Kapphahn, Stanford University; Maya Mathur, Stanford University; Manisha Desai, Stanford University
11:05 AM
Evaluating the Use of Real-World Data as a Source for Full or Hybrid Control Arms for Clinical Trials
Douglas Faries, Lilly Research Laboratories ; Zhanglin Cui, Eli Lilly and Company
11:25 AM
COMET: An Active Surveillance Trial in Low Risk DCIS, Challenges and Integration with National Cancer Database
Terry Hyslop, Biostat and Bioinformatics
11:45 AM
Floor Discussion
157 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Translational Statistics: Problems in Preclinical Studies and a Call for Change — Topic Contributed Papers
Section on Teaching of Statistics in the Health Sciences , Biopharmaceutical Section, Committee on Applied Statisticians
Organizer(s): Penny Reynolds, University of Florida School of Medicine
Chair(s): Sujata M Patil, Memorial Sloan Kettering Cancer Center
10:05 AM
Non-Informative Preclinical Research: What Statisticians Need to Know.
Penny Reynolds, University of Florida School of Medicine
10:25 AM
Common Problems in the Design of Preclinical Animal Experiments
Presentation
Stanley Lazic, Prioris.ai Inc.
10:45 AM
Statistical Reporting Must Improve in Preclinical Research
Presentation
Romain-Daniel Gosselin, Lausanne University Hospital (CHUV)
11:05 AM
Discussant: Cynthia W Garvan, University of Florida School of Medicine
11:25 AM
Discussant: Nancy Flournoy, University of Missouri-Columbia
11:45 AM
Floor Discussion
164 * !
Tue, 8/4/2020,
10:00 AM -
11:50 AM
Virtual
Leveraging Real-World Data in the Drug Development Process — Topic Contributed Papers
Biopharmaceutical Section , Biometrics Section, Health Policy Statistics Section
Organizer(s): Rebecca A Hubbard, University of Pennsylvania
Chair(s): Nandita Mitra, University of Pennsylvania
10:05 AM
Hybrid Controlled Trials with EHR-Derived Data for Cancer Trials: What, Why, When, and How
Brian Segal, Flatiron Health ; Melissa Curtis, Flatiron Health; Meghna Samant, Flatiron Health; Katherine Tan, Flatiron Health; Shrujal Baxi, Flatiron Health; Somnath Sarkar, Flatiron, Inc.
10:25 AM
Data-Adaptive Weighting of Real-World and Randomized Controls Using Propensity Scores
Joanna Harton, University of Pennsylvania ; Nandita Mitra, University of Pennsylvania; Rebecca A Hubbard, University of Pennsylvania
10:45 AM
Leveraging RWD and Historical Data in Design and Analysis of Clinical Trials
Samson Ghebremariam, Novartis Pharmaceuticals ; Lisa Hampson, Novartis Pharmaceuticals; Bharani Bharani-Dharan, Novartis Oncology; Amy Racine-Poon, Novartis Pharma AG; Beat Neuenschwander, Novartis pharmaceuticals
11:05 AM
Calibrated Survival Curve and Treatment Comparison with Imperfect Survival Outcomes from Electronic Health Records
Chuan Hong, Harvard Medical School ; Liang Liang, Harvard T. H. Chan School of Public Health; Tianxi Cai, Harvard University
11:25 AM
Discussant: Elizabeth Teeple, US Food and Drug Administration
11:45 AM
Floor Discussion
193
Tue, 8/4/2020,
10:00 AM -
2:00 PM
Virtual
Modeling, Design Strategies and Assessments of Biomarkers — Contributed Papers
Biopharmaceutical Section
Chair(s): Gitanjali Paul, GlaxoSmithKline plc
Visualizing Biomarkers and Their Association with Clinical Outcomes: A Machine Learning Approach
Hui Zheng, Harvard Medical School
Modeling Count Data with Extreme High Counts
Ping Xu, Merck & Co Inc ; Qing Li, Merck Research Labs; Anjela Tzontcheva, Merck & Co., Inc; Ruji Yao, Merck; Guanghan Frank Liu, Merck Inc.
A Three-Stage Deep Learning Method for Biomarker Identification from Gene Expression Data and Baseline Disease Activities
Bochao Jia
Effect of Tumor Size Change and Duration of Response on Overall Survival in Immunotherapy Trials
Meihua Wang, Merck & Co. ; Jing Yang, Merck & Co.; Iris Wu, Merck; Heng Zhou, Merck; Cong Chen, Merck and Company, Inc.
Issues and Solutions in Biomarker Evaluation When Sub-Classes Are Involved Under Binary Classification
Yingdong Feng, Eli Lilly & Company ; Lili Tian, University at Buffalo
A Linear Mixed Effects Model with Censored Response for Longitudinal Biomarker Data Subject to Lower Limits of Detection
Xueli Liu, AbbVie Inc. ; Chun Zhang, AbbVie Inc.
Estimation and Construction of Confidence Intervals for the Cutoff-Points of Continuous Biomarkers in Trichotomous Settings
Brian Mosier, University of Kansas Medical Center ; Leonidas Bantis, University of Kansas Medical Center
Should We Check the Normality? The Impact of Model Assumptions in Early Phase Clinical Studies
Presentation
Qianyu Dang, FDA Center for Drug Evaluation and Research (CDER) ; Ran Bi, FDA/HHS
Assessment of Predictive Biomarkers for Vaccine Development
Ivan David Ordonez, Sanofi Pasteur ; Robert D Small, Dr.; Maïna L’Azou, Sanofi Pasteur; Edith Langevin, Sanofi Pasteur; Pauline Jurvilliers, Sanofi; Darin Edwards, Moderna Therapeutics
194
Tue, 8/4/2020,
10:00 AM -
2:00 PM
Virtual
Topics in Clinical Trials - I — Contributed Papers
Biopharmaceutical Section
Chair(s): Sophie Sun, Novartis
Study Design for Non-Oncology Combination Therapies
Abigail Sloan, Pfizer Inc.
Randomized Discontinuation Trials: Modified Amery-Dony Designs
Presentation
Valerii Fedorov, ICONplc ; Parvin Fardipour, ICONplc
OncCOVID: Integrated Survival Estimates from Cancer Treatment Delay During the Covid-19 Pandemic
Presentation
Holly Hartman, University of Michigan ; Kelley Kidwell, University of Michigan; Matthew Schipper, University of Michigan
A Simulation-Free Group Sequential Design Using Max-Combo Tests in the Presence of Non-Proportional Hazards
Cheng Zheng, Sanofi ; Lili Wang, University of Michigan; Xiaodong Luo, Sanofi
Do Placebo Doses Vary Hot Flash Placebo Effects?
Jun He, Mayo Clinic ; Jennifer Le-Rademacher, Mayo Clinic; Charles L. Loprinzi, Mayo Clinic; David Zahrieh, Mayo Clinic
A Two-Stage Stratified Clinical Trial Design with Survival Outcomes and Adjustment for Misclassification in Predictive Biomarkers
Shou-En Lu, Rutgers School of Public Health ; Yanping Chen, Rutgers School of Public Health; Yong Lin, Rutgers School of Public Health; Weichung Joe Shih, Rutgers School of Public Health; Hui Quan, Sanofi
Assurance in Clinical Development Planning and Decision Making
Michael Fries, CSL Behring
Improving Precision and Power in Randomized Trials for COVID-19 Treatments Using Covariate Adjustment, for Binary, Ordinal, and Time-to-Event Outcomes
Michael Rosenblum, Johns Hopkins Univ, Bloomberg School of Public Health
195
Tue, 8/4/2020,
10:00 AM -
2:00 PM
Virtual
Topics in Personalized/Precision Medicine - II — Contributed Papers
Biopharmaceutical Section
Chair(s): Jennifer Bogert, Janssen / Johnson & Johnson
From Illogical Efficacy Measures to Mistakes in Current Computer Packages – All That Is Wrong in Biomarker Subgroup Identification with Real Example and Proposed Solution
Jason Hsu, Ohio State University ; Yi Liu, Nektar Therapeutics; Bushi Wang, Boehringer Ingelheim Pharmaceuticals, Inc.
One-Step Value Difference Test for the Existence of a Subgroup with a Beneficial Treatment Effect Using Random Forests
Dana Johnson, North Carolina State University ; Wenbin Lu , North Carolina State University; Marie Davidian, North Carolina State University
Boosting Algorithms for Estimating Optimal Individualized Treatment Rules
Duzhe Wang, University of Wisconsin-Madison ; Haoda Fu, Eli Lilly and Company; Po-Ling Loh, UW-Madison
Inference on Selected Subgroups in Clinical Trials
Xinzhou Guo, Harvard University ; Xuming He, University of Michigan
A Benchmark Framework for Subgroup Identification Methods in Clinical Trials
Hui Sun, Novartis Parmaceuticals ; Ardalan Mirshani, Novartis; Bjoern Bornkamp, Novartis
Matching-Based Classification Tree for Subgroup Effect Identification in Observational Data
Bo Lu, The Ohio State University
Three mistakes in current stratified analyses and confident patient subgroup identification for randomized controlled trials, using a Subgroup Mixable Estimation app
Siyoen Kil, LSK Global PS ; Jason Hsu, Ohio State University
244 *
Tue, 8/4/2020,
1:00 PM -
2:50 PM
Virtual
Recent Advances in Causal Inference with Applications for the Public Good — Invited Papers
Section on Statistics in Epidemiology , Biometrics Section, Health Policy Statistics Section, Biopharmaceutical Section
Organizer(s): Fan Yang, University of Colorado Anschutz Medical Campus
Chair(s): Fan Yang, University of Colorado Anschutz Medical Campus
1:05 PM
Assessing Surrogate Paradox Risk in the Meta-Analytic Causal Association Framework
Yun Li, University of Pennsylvania Perelman School of Medicine & The Children’s Hospital of Philadelphia ; Anna Conlon, University of Michigan; Nico Kaciroti, University of Michigan
1:30 PM
Tobacco Use Epidemic: Understanding Its Public Impact in the New Era
Jing Cheng, University of California, San Francisco
1:55 PM
Experimental Evaluation of Computer-Assisted Human Decision Making: Application to Pretrial Risk Assessment Instrument
Kosuke Imai, Harvard University ; Zhichao Jiang, University of Massachusetts, Amherst; James Greiner, Harvard Law School; Ryan Halen, Harvard Law School; Sooahn Shin, Harvard University
2:20 PM
Causal Ball Screening: Outcome Model-Free Causal Inference with Ultra-High-Dimensional Covariates
Linbo Wang, University of Toronto
2:45 PM
Floor Discussion
247 *
Tue, 8/4/2020,
1:00 PM -
2:50 PM
Virtual
Wearable/Implantable Device Data in Clinical Trials — Invited Papers
Section on Medical Devices and Diagnostics , Biopharmaceutical Section
Organizer(s): Tracy Bergemann, Medtronic
Chair(s): Tyson Rogers, NAMSA
1:05 PM
Digital Health Technology: Use in Clinical Investigations to Evaluate Clinical Benefit in Patients
Ebony Dashiell-Aje, BioMarin Pharmaceutical Inc.
1:25 PM
Statistical Challenges in Incorporating Data from Mobile Technology into Clinical Trials
Manisha Desai, Stanford University
1:45 PM
Normalization of Minute-Level Activity Counts from Chest- and Wrist-Worn Accelerometers: An Example of Actiheart, Actiwatch, and Actigraph
Vadim Zipunnikov, Johns Hopkins University, Bloomberg School of Public Health ; Jiawei Bai, Johns Hopkins Bloomberg School of Public Health
2:05 PM
Use of Accelerometer Data to Evaluate Physical Activity as a Surrogate Endpoint in Heart Failure Clinical Trials
Presentation
Tracy Bergemann, Medtronic
2:25 PM
Data-Driven Chronotype Discovery Using Functional Data Methods
Jeff Goldsmith, Columbia University Mailman School of Public Health
2:45 PM
Floor Discussion
262 *
Tue, 8/4/2020,
1:00 PM -
2:50 PM
Virtual
The FDA Complex Innovative Trial Design Pilot Program: Learning from Case Examples and Simulations for the Public Good — Topic Contributed Papers
Biopharmaceutical Section , ENAR, Section on Bayesian Statistical Science
Organizer(s): Dionne L Price, Food and Drug Administration
Chair(s): Dionne L Price, Food and Drug Administration
1:05 PM
An Overview of Innovative Designs and Statistical Considerations: Lessons Learned
Laura Lee Johnson, FDA
1:25 PM
A Bayesian Adaptive Trial in Duchenne Muscular Dystrophy
Stephen Lake, WAVE Life Sciences ; Melanie Quintana, Berry Consultants; Scott Berry, Berry Consultants; Jennifer Panagoulias, Wave Life Sciences; Michael A. Panzara, Wave Life Sciences
1:45 PM
The Design, Challenges, and Benefits of Evaluating Multiple Assets and Disease Types in a Master Protocol
JonDavid Sparks, Eli Lilly and Company ; Saptarshi Chatterjee, Eli Lilly and Co; Karen L Price, Eli Lilly and Company
2:05 PM
Floor Discussion
272 * !
Tue, 8/4/2020,
1:00 PM -
2:50 PM
Virtual
Statistical Innovations in Regulatory Science — Topic Contributed Papers
Biometrics Section , Biopharmaceutical Section, Health Policy Statistics Section
Organizer(s): Alejandra Avalos Pacheco, Harvard Medical School
Chair(s): Andrea Arfe, Harvard Medical School
1:05 PM
The Impact of Early Trials in Drug Development in Oncology: Design and Interpretation
Alexia Iasonos, Memorial Sloan-Kettering Cancer Center
1:25 PM
A Semi-Mechanistic Dose-Finding Design in Oncology Using Pharmacokinetic/Pharmacodynamic Modeling
Yisheng Li, University of Texas-MD Anderson Cancer Center ; Xiao Su, PlayStation; Peter Müller, University of Texas Austin; Kim-Anh Do, The University of Texas MD Anderson Cancer Center
1:45 PM
Inference in response-adaptive clinical trials when the enrolled population varies over time
Massimiliano Russo, Harvard Medical School ; Lorenzo Trippa, Harvard School of Public Health; Steffen Ventz, Harvard School of Public Health; Victoria Wang, Harvard School of Public Health
2:05 PM
Leveraging External Data in Bayesian Adaptive Designs
Alejandra Avalos Pacheco, Harvard Medical School ; Steffen Ventz, Harvard School of Public Health; Lorenzo Trippa, Harvard School of Public Health; Brian Alexander, Foundation medicine; Andrea Arfe, Harvard Medical School
2:45 PM
Floor Discussion
220192
Tue, 8/4/2020,
7:30 PM -
8:30 PM
Virtual
Biopharmaceutical Section Open Business Meeting — Other Cmte/Business
Section on Statistics in Epidemiology , ENAR, Biometrics Section
Organizer(s): Ekaterina Smirnova, Virginia Commonwealth University
Chair(s): Ekaterina Smirnova, Virginia Commonwealth University
10:05 AM
Unraveling the Relationship Between Physical Activity and Health Using Wearable Sensors
Charles Matthews, National Cancer Institute Shady Grove
10:25 AM
Fast Univariate Inference for Longitudinal Functional Models
Erjia Cui, Johns Hopkins Bloomberg School of Public Health ; Andrew Leroux, University of Colorado Anschutz Medical Campus; Ciprian Crainiceanu, Johns Hopkins University; Ekaterina Smirnova, Virginia Commonwealth University
10:45 AM
The Digital Biomarker Discovery Project: An Open Source Toolbox Biosignal Analysis
Jessilyn Dunn, Duke University ; Brinnae P Bent, Duke University; Emilia Grzesiak, Duke University; Chentian Jiang, Duke University; Yuankai P Qi, Duke University; Yihang P Jiang, Duke University; Peter P Cho, Duke University
11:05 AM
Genome-Wide Association Studies of 27 Accelerometry-Derived Physical Activity Measurements Identifies 6 Novel Loci and Links with Blood and Immune Mechanisms
Guanghao Qi, Johns Hopkins University ; Diptavo Dutta, Johns Hopkins University; Andrew Leroux, University of Colorado Anschutz Medical Campus; Debashree Ray, Johns Hopkins University; Ciprian Crainiceanu, Johns Hopkins University; Nilanjan Chatterjee, Johns Hopkins University
11:25 AM
New Methodology for Characterizing Circadian Rhythm in Actigraphy Data Collected from a Wearable Device
Sungduk Kim, National Cancer Institute ; Paul S. Albert, National Cancer Institute
This Biopharmaceutical Section will host our annual open business meeting through Zoom. Zoom is graciously provided by Abbvie, Inc. https://abbvie.zoom.us/j/95972313003?pwd=UXh6TGM5MTI1VVFWQU1KTU9YZ3R1Zz09
