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11: Statistics [clear filter]
Sunday, June 23
 

9:00am

SC24: #24: Data Visualization in the Life Sciences
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-504-L04-P; CME 3.25; IACET 3.25; RN 3.25

Traditional approaches to medical product development rely on generating pages upon pages of analysis results to describe the safety and effectiveness of novel therapies. Study teams struggle to understand and communicate the story hidden within the data to their colleagues. First and foremost, with the high cost of conducting translational clinical research, it is common to collect as much data as possible on as many endpoints as possible. This phenomenon is further reinforced due to our limited understanding of biological mechanisms and pathways, including the potential genomic underpinnings of a disease or treatment response. For example, we may have a clear understanding for how a novel therapy induces an efficacious response, but there is typically limited knowledge into the downstream effects of the drug to other body systems. A second challenge to communication lies in the increased use of sensitivity analyses to assess the consistency and robustness of study results to varying assumptions. Given the volume of data to review and the variety of analyses to perform, it should come as no surprise that clear insight is often out of reach. In this environment, the traditional means of data summary – tables and listings – are ineffective for gaining insight; visualization is the key to effective communication for the modern clinical trialist. Ben Shneiderman stated that “the purpose of visualization is insight.” Therefore, the goal of this short course is to describe data visualization techniques to aid in the understanding and communication of results from applications in clinical trials and genomics research. Numerous practical illustrations and examples from the literature will be presented. To be accessible to a wide audience, this course will focus on principles and interpretation, and limit technical jargon. At least 2/3 of the course will focus on case studies specific to clinical trials, while the remainder will be spent on examples in genomics. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed to be accessible to a wide audience, it will focus on principles, limit technical jargon, and interpret numerous examples of data visualization using data from the life sciences literature. The audience may include any individual interested in developing their skills for more efficient interpretation and communication of various aspects of study design and analysis.


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Describe the transition from traditional methods of data analysis to visual approaches;
  • Interpret life science data using one or more data visualizations;
  • Assess the strengths and limitations of various graphical techniques;
  • Explain the “data story” of numerous clinical research examples using data visualization techniques.



Speakers
avatar for Kelci Miclaus

Kelci Miclaus

Sr Manager, Advanced Analytics R&D, SAS Institute Inc., JMP Division
Dr. Kelci Miclaus is Advanced Analytics Sr. Manager for the JMP Life Sciences division at SAS Institute where she manages the R&D team and develops statistical features for JMP Genomics and JMP Clinical software. She joined SAS in 2006 and holds a PhD in Statistics from North Carolina... Read More →
avatar for Richard C. Zink

Richard C. Zink

Senior Director, Data Management and Statistics, TARGET Pharmasolutions Inc
Richard C. Zink is Senior Director of Data Management and Statistics at TARGET PharmaSolutions. He is the 2019 Chair of the Biopharmaceutical Section of the American Statistical Association, host of the Biopharmaceutical Section Statistics Podcast, and Associate Editor for the DIA... Read More →


Sunday June 23, 2019 9:00am - 12:30pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Real World Evidence
  • Credit Type ACPE, CME, IACET, RN
  • Tags Tutorial

1:30pm

SC36: #36: A Novel Interactive Safety Graphic to Evaluate Potential Drug-Induced Hepatotoxicity
Component Type: Tutorial
CE: ACPE 3.25 Application UAN: 0286-0000-19-515-L04-P; CME 3.25; IACET 3.25; RN 3.25

The DIA-ASA Biopharm Safety Evaluation Working Group is developing a series of novel interactive safety graphic tools to enhance the ability of safety and clinical development professionals to identify and evaluate safety signals. Each will be made available as an open-source, non-proprietary application widely available to anyone interested in drug safety evaluation. An open source project like this will likely result in a longer-lasting software solution. The first tool to be released is designed to explore cases of potential drug-induced hepatotoxicity based on the eDISH plot developed by FDA. Building upon the existing static eDISH plot, the tool allows the user to dynamically adjust laboratory thresholds to account for disease states with elevated transaminase and bilirubin values, modify the time dimension for the occurrence of peak ALT/AST and bilirubin values, account for the extent of alkaline phosphatase elevation, with filters for treatment assignment, gender, race and age group. Cases that appear in the potential Hy’s Law, Temple’s corollary and hyperbilirubinemia quadrants can be individually explored to detail the time course of changes in various analytes, and assess the concurrence with adverse events and the exposure to concomitant medications. In order to assist the safety reviewer, a workflow is provided to guide the user through the recommended analyses, using the features of the tool, for each of the quadrants of interest. The workflow is based on evaluations supported by expert hepatologists and the medical literature. The short course will demonstrate the functions of the tool by way of case examples exploring various hepatotoxicity signals. Prior attendees of the DIA Advanced Signal Detection course will find this course builds upon the concepts presented in that course, but it is not a prerequisite for attending this class. In addition, information will be provided on how attendees can implement this RShiny/JavaScript tool in their organizations. An additional registration fee is required for all preconference short courses. Back to DIA 2019 Short Courses

Who should attend?

This short course is designed for professionals involved in:
  • Clinical safety and pharmacovigilance
  • Pharmacoepidemiology
  • Biostatistics
  • Benefit-risk management
  • Clinical development
  • Data scientists
  • Information technology supporting pharmacovigilance activities


Learning Objectives

At the conclusion of this course, participants should be able to:
  • Evaluate the potential for drug-induced hepatotoxicity with a new open-source, interactive safety graphic based on the eDISH plot;
  • Assess cases of potential Hy’s Law, Temple’s corollary and hyperbilirubinemia with respect to lab changes, clinical symptoms and identify possible confounding elements;
  • Perform analyses with a work-flow procedure based on established medical precedent.



Speakers
avatar for James Buchanan

James Buchanan

Drug Safety Consultant, Covilance LLC
Dr. Buchanan has been in the pharmaceutical industry working in drug safety for over 30 years. He started his career at Genentech and led the drug safety departments at a number of subsequent companies, Gilead Sciences, Tularik and Nuvelo, before serving as the head of the medical... Read More →
avatar for Jeremy Wildfire

Jeremy Wildfire

Senior Data Scientist, Rho, Inc.
I have worked as a biostatistician for the NIAID funded Inner City Asthma Consortium (ICAC) for nearly 10 years. My work with ICAC has included the development of the Composite Asthma Severity Index, the first quantitative measure of asthma severity, and the analysis of multiple late-stage... Read More →


Sunday June 23, 2019 1:30pm - 5:00pm
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Credit Type ACPE, CME, IACET, RN
  • Tags Tutorial
 
Monday, June 24
 

10:10am

#101 CH: Modernizing Data Review in Drug Development with R Shiny
Component Type: Workshop
Level: Intermediate

ASA Biopharm/DIA Safety Working Group, safety clinicians, statisticians and data scientists, developed an R Shiny tool available for all, initially for detection and review of hepatotoxicity. Discuss this new tool and seek input on next steps and support.

Learning Objectives

Describe an R Shiny safety tool developed by the Interactive Safety Graphics taskforce of the ASA Biopharm/DIA Safety Working Group, for review of liver function and Hy’s Law for use during drug development and submission review; Discuss audience feedback on path forward for this new model of agile software development and support, with consideration of compliance.

Chair

Susan Duke, MSc


Speakers
avatar for Susan Duke

Susan Duke

Mathematical Statistician, OB, OTS, CDER, FDA
Susan became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG. She is currently a Mathematical Statistician reviewer in CDER and member of a cross-functional safety monitoring WG co-sponsored by DIA and ASA Biopharm... Read More →


Monday June 24, 2019 10:10am - 10:40am
Content Hub Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

11:00am

#118: Statistical Considerations for Trials Using Surrogate Endpoints for Accelerated Approval
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-531-L04-P; CME 1.00; IACET 1.00; RN 1.00

For slowly progressed diseases, it may take years, if not decades, to observe clinical outcomes, such as mortality. Owing to the regulatory agencies’ accelerated approval pathway, drug developers can now consider applying for a drug approval based on surrogate endpoints. In this session, many surrogate endpoints adopted in various diseases and statistical methods for determining the validity of the surrogacy will be discussed, including their implementation in real case examples.

Learning Objectives

Describe cases that utilize the accelerated approval and what good surrogate endpoints are for common and rare diseases; Identify how to apply appropriate statistical methods for assessing the association between surrogate endpoints and clinical outcomes.

Chair

Yeh-Fong Chen, PhD

Speaker

FDA Update
Aloka Chakravarty, PhD

Statistical Assessment of Potential Surrogate Endpoints: Is the Bar too High?
Marc E. Buyse, DrSc

Using the Biomarker Endpoint for Accelerated Approval
Stephanie O. Omokaro, MD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
avatar for Aloka Chakravarty

Aloka Chakravarty

Acting Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She served as an Adjunct Faculty in Depart. of Statistics, Foundation for Advanced Education in the Sciences, NIH. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development... Read More →
avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician, Office of Translational Sciences, CDER, FDA
Dr. Chen is the Acting Associate Director of the Division of Biometrics II within CDER of FDA, supervising reviewers for the Division of Metabolism and Endocrinology Products and Division of Anesthesia, Analgesia, and Addiction Products. She joined FDA in 2000 after receiving her... Read More →
avatar for Stephanie Omokaro

Stephanie Omokaro

Lead Medical Officer, Office of New Drugs, CDER, FDA
Dr. Omokaro is a Lead Medical Officer in the Division of Gastroenterology and Inborn Errors Products in the Center for Drug Evaluation and Research at FDA. She was educated in Biochemistry at Cornell University, and in medicine at Boston University. She completed a pediatric residency... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#152: Making Data Meaningful: Using Data Visualization to Drive Efficiency in Safety Analysis
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-550-L04-P; CME 1.00; IACET 1.00; RN 1.00

Data visualization can transform and advance clinical trial safety analysis. Discover the latest data visualization use cases for safety and how dynamic data exploration yields meaningful data review, better patient outcomes and trial efficiency.

Learning Objectives

Discuss various data visualization examples that can be applied to the area of clinical trial safety analysis; Outline key benefits and efficiencies statisticians, medical monitors, and others in charge of safety can achieve through data visualizations that are being used in this area of clinical trials today.

Chair

Demi Niforos, MS

Speaker

Summary of Safety Visualization Efforts
William Wang, PhD

Panelist
Nathan Bennett, PhD, MA



Speakers
DN

Demi Niforos

Vice President, Biostatistics and Statistical Programming, eClinical Solutions
Demi Niforos is VP, Biostatistics and Statistical Programming for eClinical Solutions and has more than 20 years of drug development experience in both the CRO and Pharmaceutical/Biotech environments. Prior to joining eClinical Solutions she held leadership roles in biostatistics... Read More →
avatar for William Wang

William Wang

Executive Director, Clinical Safety Statistics, BARDS, Merck Research Laboratories
Dr. William (Bill) Wang is an executive director, clinical safety statistics, Merck Research Laboratories. He has over 25 years of experience in the pharmaceutical industry, with ~18 years with Merck & Co Inc. He is co-chairing the ASA safety working group, and is a deputy topics-leader... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#169: How Statistics Can Help Improve Data Quality: ICH E6 R2
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-564-L04-P; CME 1.00; IACET 1.00; RN 1.00

ICH E6 (R2) guidance stresses the importance of centralized monitoring, especially by leveraging statistical analyses in order to assess data quality. This session will discuss how statistical methodology fits in the overall quality risk management strategy for the trial portfolio.

Learning Objectives

Discuss centralized statistical monitoring approaches and their impacts to data quality and integrity.

Chair

Cheng Su, PhD

Speaker

Using Appropriate Count-Based Statistical Models for KPIs, Risk Metrics, QTLs, etc.
Michael Farnum, PhD, MS

ICH E6 R2: How Statistics Can Help Improve Data Quality
Stephen Young, MSc

Centralized Statistical Monitoring: A Large Pharma's Approach
Taras Carpiac, MBA



Speakers
avatar for Taras Carpiac

Taras Carpiac

Director, Head of Innovation and Process Improvement, Amgen, Inc.
MF

Michael Farnum

Senior Director, Covance
Michael Farnum is a Senior Director for Covance Informatics. In 5+ years working at Covance, he has helped to create the Xcellerate platform, consisting of data integration, analytics, and decision support tools that enable Risk-Based Monitoring, Medical Review, Statistical Review... Read More →
CS

Cheng Su

Executive Director, Data Sciences and Analytics, Biomarin Pharmaceutical
Cheng got his Ph.D. in statistics from NCSU and has 22 years of experiences in both nonclinical and clinical areas. Cheng has a broad interpest in applying statistical thinking and analytics to support drug research and development. His specific areas of expertise include high throughput... Read More →
avatar for Stephen Young

Stephen Young

Chief Operations Officer, CluePoints
As CluePoints COO, Steve is primarily responsible for ensuring successful customer adoption of the CluePoints platform. Steve worked for three bio-pharma companies over a span of 15 years where he assumed leadership positions in data management and led the successful enterprise roll-out... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

6:00pm

Community Meet Up: Statistics Community Meet and Greet
Component Type: Forum

Please join the Statistics Community for a meet and greet. This is an opportunity for members and non-members to learn more about our Community and activities, to share ideas and discuss future direction, and most importantly to volunteer and be part of a dynamic group!

Chair

Joan Buenconsejo, PhD, MPH


Speakers
avatar for Joan Buenconsejo

Joan Buenconsejo

Director and Biometrics Team Leader, AstraZeneca
Dr. Joan Buenconsejo is a Director and Biometrics Team Leader supporting the oncology therapeutic area within the Biometrics and Information Sciences at AstraZeneca. Prior to joining AZ, she was a statistics team leader at CDER, US FDA. She is the chair of DIA Statistics Community... Read More →


Monday June 24, 2019 6:00pm - 7:00pm
Community Area 2 Sails San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Tuesday, June 25
 

8:00am

#218: Real World Data to Real World Evidence
Component Type: Session
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-583-L04-P; CME 1.25; IACET 1.25; RN 1.25

We discuss the advantages and disadvantages of real world data and real world evidence compared to other sources. Join the Clinical Research Community for a follow up Round Table discussion on Wednesday, June 26, 9:30-10:30AM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Describe the importance of real world data for answering regulatory questions; Discuss the benefits and challenges of evidence derived from real world sources compared to clinical trials.

Chair

Richard Zink, PhD

Speaker

Real World Data to Real World Evidence: A Case Study of Direct-Acting Antivirals for the Treatment of Hepatitis C Infection
Richard Zink, PhD

Machine Learning 2.0: Causal Models and Big Data in the Era of Real World Evidence
Andrew Wilson, PhD, MS

Matching with Replacement: The COLLECTION of ‘Personal’ EFFECTs Should be Handled with Care
Sarah Anselm Short, MPH



Speakers
SS

Sarah Short

Sr. Statistical Mgr, RWE - Biostats & Medical Writing, ICON plc.
Sarah has over ten years’ experience in health related statistical analysis, primarily in disease or product registries and other prospective observational studies. Her experience encompasses a wide array of therapeutic areas and study designs. As a Senior Statistical Manager, she... Read More →
avatar for Andrew Wilson

Andrew Wilson

Director, Pharmacoepidemiology and Statistics RWDS, PAREXEL
I lead the pharmacoepidemiology & statistics initiatives within Real World Data Services at PAREXEL. To be part of the real world evidence movement, it is essential we embrace the data generation process, and keep data context as all-important. Real world data typically comes from... Read More →
avatar for Richard C. Zink

Richard C. Zink

Senior Director, Data Management and Statistics, TARGET Pharmasolutions Inc
Richard C. Zink is Senior Director of Data Management and Statistics at TARGET PharmaSolutions. He is the 2019 Chair of the Biopharmaceutical Section of the American Statistical Association, host of the Biopharmaceutical Section Statistics Podcast, and Associate Editor for the DIA... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

9:15am

#221 CH: Getting the Question Right (GTQR) with Interdisciplinary Collaboration
Component Type: Workshop
Level: Intermediate

Inspired by the E9 R1 estimand framework, the DIA Getting the Question Right (GTQR) webinar series was organized in 2018 via intercommunity collaboration. This hub will provide a synthetic overview of the 6 popular webinars, followed by open Q&As.

Learning Objectives

Discuss the estimand framework described in the draft ICH E9 addendum; Recognize how the estimand framework may help get the question right and can be applied in various therapeutic and safety/benefit-risk contexts; Define how the estimand framework may advance the interpretation of study results.

Chair

William Wang, PhD


Speakers
avatar for William Wang

William Wang

Executive Director, Clinical Safety Statistics, BARDS, Merck Research Laboratories
Dr. William (Bill) Wang is an executive director, clinical safety statistics, Merck Research Laboratories. He has over 25 years of experience in the pharmaceutical industry, with ~18 years with Merck & Co Inc. He is co-chairing the ASA safety working group, and is a deputy topics-leader... Read More →


Tuesday June 25, 2019 9:15am - 9:45am
Content Hub Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#245: Artificial Intelligence Enhanced Data Analytics for Clinical Trials
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-602-L04-P; CME 1.00; IACET 1.00; RN 1.00

Artificial Intelligence (AI) enhanced drug development is becoming increasingly important in the era of precision medicine.This session illustrates with examples how AI can impact and facilitate modern drug development.

Learning Objectives

Recognize the benefit of using artificial intelligence (AI) approaches in modern drug development; Identify the regulatory paths using biomarker-based, AI-enhanced strategies in clinical trials; Outline how to incorporate advanced analytics into trial analyses and future trial designs.

Chair

Felix Frueh, PhD

Speaker

Panelist
Mohammad Afshar, MD, PhD

Panelist
Federico Manuel Goodsaid, PhD



Speakers
avatar for Mohammad Afshar

Mohammad Afshar

Chief Executive Officer, Ariana Pharma
Mohammad founded Ariana® to accelerate the development of novel drugs with the help of computational decision support technologies. It has developed KEM®, for rapid & exhaustive analysis of multi-parametric/multi-objective data for biomarkers, drug discovery, clinical and early... Read More →
FF

Felix Frueh

Executive Partner, Opus Three LLC
avatar for Federico Goodsaid

Federico Goodsaid

Associate Partner, Biomarkers and Regulatory Affairs, Opus Three LLC
B.A. was in Biochemistry and Biophysics from the University of California at Berkeley and his Ph.D from Yale University, New Haven, in Molecular Biophysics and Biochemistry. He was a Postdoctoral Fellow at Cornell University in Ithaca, New York and at Washington University in St... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#271: Master Protocols: Applications in Oncology
Component Type: Session
Level: Advanced
CE: ACPE 1.25 Application UAN: 0286-0000-19-621-L04-P; CME 1.25; IACET 1.25; RN 1.25

Future advances in medicine depend on our ability to leverage complex innovative designs of clinical trials. Master protocols, including basket, umbrella, and platform trials, as one type of complex innovative designs, provide a method to answer multiple questions in one overall trial structure. In this session, the concept of master protocols, some case studies using real world examples in oncology, and the advantages and challenges of designing and implementing master protocols will be discussed. Join the Statistics Community for a follow up Round Table discussion on Wednesday, June 26, 3:15-4:15PM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Explain the concept of master protocols; Discuss case studies in master protocols with applications in oncology; Describe opportunities and challenges in applying master protocols as an innovative approach in oncology drug development.

Chair

Amy Xia, PhD

Speaker

FDA Update
Yuan-Li Shen, DrPH

Industry Update
Scott M Berry, PhD, MS

Industry Perrspective About Platform Trials
Karen Lynn Price, PhD, MA



Speakers
avatar for Scott Berry

Scott Berry

President and Senior Statistical Scientist, Berry Consultants LLC
Scott Berry is President and a Senior Statistical Scientist at Berry Consultants, LLC. He earned his MS and PhD in statistics from Carnegie Mellon University and was an Assistant Professor at Texas A&M University before co-founding Berry Consultants in 2000. His primary interests... Read More →
avatar for Karen Price

Karen Price

Senior Research Advisor, Statistical Innovation Center, Eli Lilly and Company
Karen received her Ph.D. in Statistics from Baylor University in 2001, and joined Eli Lilly and Company at that time. She is currently Senior Research Advisor at Eli Lilly and Company where she leads the Statistical Innovation Center, a team that focuses on innovative design and analysis... Read More →
avatar for Amy Xia

Amy Xia

Vice President, Biostatistics, Design & Innovation, Amgen Inc.
Amy Xia is Vice President, Biostatistics, Design & Innovation at Amgen. Amy has worked on designing, implementing, and analyzing Phase I-IV clinical trials as well as observational studies in the past 22+ years. Currently, she heads up the Biostatistics and Design & Innovation organizations... Read More →
YS

Yuan-Li Shen

Acting Associate Director, Office of Biostatistics, OTS, CDER, FDA
Yuan-Li is the acting Associate Director in Division of Biometrics V, Office of Biostatistics, Office of Translational Science, CDER, FDA. The teams that she is associated with support all statistical aspects of regulatory submissions in Gastrointestinal, Lung/Head and Neck, Neuro-oncology/Rare... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#298: Clinical Safety Assessment: What’s a Statistician Got to Do with It?
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-637-L04-P; CME 1.25; IACET 1.25; RN 1.25

Statistical attention to safety data is often times left to routine, basic calculations and tabulations. Absent statistical input, the data run the risk of being presented in ways that are unintuitive, uninformative, or in the worst case, deceptive. Speakers in this session will present examples where statisticians are collaborating with clinical colleagues and data scientists to more effectively analyze and clinically interpret safety data.

Learning Objectives

Recognize the importance of statistical engagement in the assessment of clinical trial safety data; Apply appropriate statistical methods in the analysis of safety data; Identify the need to work collaboratively with clinicians and data scientists.

Chair

Mat Soukup, PhD

Speaker

Improving Analyses and Displays for Clinical Trial Safety Data
Mary Nilsson, MS

ASA/DIA Safety WG Presents: Finding Safety Signals During Clinical Development with R Shiny
Susan Duke, MSc

FDA Perspective
Matthew Guerra, PhD



Speakers
avatar for Susan Duke

Susan Duke

Mathematical Statistician, OB, OTS, CDER, FDA
Susan became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG. She is currently a Mathematical Statistician reviewer in CDER and member of a cross-functional safety monitoring WG co-sponsored by DIA and ASA Biopharm... Read More →
MG

Matthew Guerra

Mathematical Statistician, OB, OTS, CDER, FDA
Matthew is a senior statistical reviewer supporting the Division of Dermatology and Dental Products (DDDP) in the Center for Drug Evaluation and Research (CDER) at the FDA. Matthew joined the FDA in 2011 immediately after receiving his Ph.D. in Biostatistics from the University of... Read More →
avatar for Mary Nilsson

Mary Nilsson

Statistician, Eli Lilly and Company
Mary received a MS degree in statistics from Iowa State University in 1989. She has been employed at Lilly since 1989 and is currently a research advisor in the Safety Analytics group within the Statistical Sciences function. She consults with compound teams on safety analysis planning... Read More →
MS

Mat Soukup

Deputy Div. Director, Div. of Biometrics VII, Office of Biostatistics, OTS CDER, FDA
Following his educational endeavors in statistics at the University of Minnesota-Morris and the University of Virginia, Mat joined FDA/CDER in September 2004. His first 6 years were spent reviewing dermatology and dental products before joining the Division of Biometrics 7 as Team... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking,Innovative Trial Design
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session
 
Wednesday, June 26
 

8:00am

#313: Model Integrated Evidence as Pivotal Information for Drug Regulatory Decision Making: When, Where, and Why
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-654-L04-P; CME 1.25; IACET 1.25; RN 1.25

The use of advanced quantitative methods and computational modeling has become part of modern drug development and assessment. Model Integrated Evidence (MIE) is an approach for generating evidence of bioequivalence via modeling and simulation.

Learning Objectives

Identify the core modeling and simulation capabilities (PBPK and QCP) that a generic drug manufacturer must posses; Evaluate lessons learned from the use of a model informed drug development process for new drugs, and apply common processes to generic drug development programs; Define and prepare for the future use of model informed evidence (MIE) in a generic drug submission and identify the value.

Chair

Liang Zhao, PhD

Speaker

Industry Update
Michael A. Eldon, PhD, RPh

FDA Update From New Drugs Perspective
Yaning Wang, PhD

FDA Update from Generic Drugs Perspective
Liang Zhao, PhD



Speakers
avatar for Yaning Wang

Yaning Wang

Director, Division of Pharmacometrics, OCP, OTS, CDER, FDA
Dr. Yaning Wang is the Director of the Division of Pharmacometrics in the Office of Clinical Pharmacology at FDA. Before joining FDA, Dr. Wang received his Ph.D. in Pharmaceutics and master’s degree in Statistics from the University of Florida from 1999 to 2003. He also obtained... Read More →
LZ

Liang Zhao

Director, Division of Quantitative Methods and Modeling, OGD, CDER, FDA
Dr. Liang Zhao has been serving as Director of the Division of Quantitative Methods and Modeling (DQMM), Office of Research and Standards (ORS) in Office of Generic Drugs, CDER since 2015. He initially joined FDA as a clinical pharmacology reviewer in the Office of Clinical Pharmacology... Read More →
avatar for Michael Eldon

Michael Eldon

Expert Consultant, NDA Partners LLC
Dr. Eldon has more than 37 years of experience in clinical pharmacology, pharmacokinetics/ pharmacodynamics and pharmacometrics in the pharmaceutical industry. He is an Expert Consultant at NDA Partners and as VP of Clinical Pharmacology at Nektar Therapeutics he was responsible for... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 6E San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#316: Implementation of Innovative and Adaptive Designs in Clinical Trials
Component Type: Session
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-656-L04-P; CME 1.25; IACET 1.25; RN 1.25

The use of innovative designs and possible adaptations can help reduce the number of patients required for trial recruitment, thereby shortening drug development time. Experts from FDA and the industry will provide feedback on the complex innovative designs and how they inform regulatory decision making.

Learning Objectives

Outline the advances in adaptive designs; Utilize newly developed innovative designs in clinical trials.

Chair

Aloka Chakravarty, PhD

Speaker

Recent Advances in Group Sequential and Adaptive Designs for Unblinded Sample Size Re-estimation and Seamless Phase 2/3 Pivotal Trials
Sandra Bihary-Waltz, BSN, MSN

EMA Update
Andrew Thomson, PhD, MA, MS

FDA Update
Meiyu Shen, PhD



Speakers
MS

Meiyu Shen

Expert Mathematical Statistician, Office of Translational Sciences, CDER, FDA
A team leader and a senior statistical reviewer at the Center of Drug Evaluation and Research, Food and Drug Administration (FDA). She obtained her Ph.D. in statistics from Statistics Program of Department of Mathematics at University of Maryland at College Park in 2015 and her Ph.D... Read More →
AT

Andrew Thomson

Statistician, Biostatistics and Methodology Support Office, European Medicines Agency (EMA)
Andrew Thomson is a statistician in the Office of Biostatistics & Methodology at the European Medicines Agency. He provides methodological advice and guidance across all stages of development, and across all therapeutic areas. He also is the lead of the Statistics subgroup of ICH... Read More →
SB

Sandra Bihary-Waltz

Senior Director, Global Regulatory Affairs, PRA Health Sciences
Sandra joined Idis as the Global Head of REgulatory Affairs in 2012, after spending over 24 years in the pharmaceutical industry, predominantly in regulatory affairs. She has extensive experience in all phases of drug development, through post-approval activities, including EAP p... Read More →
SB

Sandra Bihary-Waltz

Senior Director, Global Regulatory Affairs, PRA Health Sciences
Sandra joined Idis as the Global Head of REgulatory Affairs in 2012, after spending over 24 years in the pharmaceutical industry, predominantly in regulatory affairs. She has extensive experience in all phases of drug development, through post-approval activities, including EAP p... Read More →
avatar for Aloka Chakravarty

Aloka Chakravarty

Acting Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She served as an Adjunct Faculty in Depart. of Statistics, Foundation for Advanced Education in the Sciences, NIH. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

8:00am

#317: Designing Clinical Trials with the Right Endpoints: Applying ICH-E9(R1) - Getting the Questions Right (GTQR),Estimands and Handling Missing Data
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-657-L04-P; CME 1.25; IACET 1.25; RN 1.25

Industry and FDA experts will share how to operationalize ICH E9(R1) to design trials with clinically meaningful endpoints. Forum will include a summary from the DIA Stats Community GTQR Series, the Oncology Drug Development Working group on Estimands, and Tipping Point analysis to account for missing data.

Learning Objectives

Discuss key elements of ICH E9(R1) and GTQR; Define estimands and ongoing efforts for implementation; Identify how to apply estimands in oncology with time-to-event endpoint; Describe an overview and examples of applying tipping point analysis to explore the impact of missing data assumptions on study results, along with key considerations during the planning stage of these sensitivity analyses.

Chair

Munish Mehra

Speaker

Estimand Framework and its Impact on Oncology Drug Development: Findings From An Industry-Wide Working Group
Yi Liu, PhD

Assessment of Tipping Point Analysis for Handling Various Types of Missing Data
Martin Clancy, MSc

Estimands for Efficacy Analyses
Thomas J. Permutt, PhD



Speakers
MC

Martin Clancy

Senior Statistician, PHASTAR
I'm a Senior Statistician at PHASTAR, a specialist CRO offering statistical consultancy, clinical trial reporting and data management services by providing expert consultants and delivering in-house projects. Working in a Contract Research Organisation (CRO) for the past 4 years... Read More →
avatar for Yi Liu

Yi Liu

Director Biostatistics, Nektar Therapeutics
Yi Liu got her Ph.D. degree in Statistics from the department of Statistics at The Ohio State University. She is currently a director at Nektar Therapeutics responsible for clinical trials in collaborations with external partners and CMC and biomarker related statistical analyses... Read More →
avatar for Munish Mehra

Munish Mehra

Executive Director, Tigermed
Dr. Munish Mehra, is a drug development expert with Tigermed, providing services to Pharmaceutical and Biotechnology companies to design, analyze and report on Phase 2 and 3 studies. He has represented several sponsors at pre-IND, End of Phase 2 and pre-NDA meetings. He is a frequent... Read More →
avatar for Thomas Permutt

Thomas Permutt

Associate Director for Statistical Science and Policy, OB, OTS, CDER, FDA
Tom Permutt is Associate Director for Statistical Science and Policy, Office of Biostatistics, Center for Drug Evaluation and Research at FDA. Tom’s contributions to causal inference in clinical trials date from 1989 when he proposed an instrumental-variable estimator of the effect... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum

9:30am

#322 RT: Round Table Discussion: Real World Data to Real World Evidence
Component Type: Session
Level: Basic

Join DIA Clinical Research Community for a round table discussion tied to session #218: Real World Data to Real World Evidence (Tuesday, June 25, 8:00-9:15AM). Seating is limited. To include special guests from the session: Andy Wilson, Sarah Short

Chair

Richard Zink, PhD


Wednesday June 26, 2019 9:30am - 10:30am
Community Zone Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#344: Meaningful Patient-Focused Drug Development for Rare Disease and Personalized Medicine
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-672-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will provide a platform to discuss measurement challenges and practical considerations for selection of fit-for-purpose clinical outcome assessments (COAs) to support endpoints in rare disease drug development programs.

Learning Objectives

Discuss practical considerations for utilizing clinical outcome assessments (COAs) to support clinical trial endpoint(s) in drug development; Identify the measurement challenges and recommendations for the selection of COAs for rare disease drug development.

Chair

Michelle Campbell, PhD

Speaker

Generalized Pairwise Comparisons for Benefit/Risk Assessment in Personalized Medicine
Marc E. Buyse, DrSc

Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease Drug Development: A Regulatory Perspective
Lili Garrard, PhD

Selection of Fit-for-Purpose COAs to Support Endpoints in Rare Disease
Jean Paty, PhD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
MC

Michelle Campbell

Division of Neurology Products, Office of New Drugs, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
LG

Lili Garrard

Mathematical Statistician, Office of Biostatistics, OTS, CDER, FDA
Lili Garrard is a statistical reviewer on the Clinical Outcome Assessment (COA) Statistical Support Team at the Division of Biometrics III, Office of Biostatistics (OB), Office of Translational Sciences (OTS), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration... Read More →
avatar for Jean Paty

Jean Paty

Vice President, Patient-Centered Endpoints, IQVIA
Dr. Paty is an acknowledged leader in the effective strategies and practices of capturing patient perspective data for use in the clinical development of new medical products. He has published extensively in the areas of Patient Reported Outcomes (PRO) and electronic PRO (ePRO), as... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#371: Efficient Pediatric Drug Development: Incorporating Innovative Techniques Using Extrapolation and Historical Information
Component Type: Session
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-694-L04-P; CME 1.25; IACET 1.25; RN 1.25

Pediatric drug development for adult indications is mandated. Yet children should be protected from unnecessary risks. This session discusses innovative techniques using extrapolation from adult data as an efficient way to study drugs in children.

Learning Objectives

Explain the regulatory and ethical context of pediatric drug development; Discuss the role, assumptions, and data needed for using extrapolation from adult data; Identify innovative approaches using Bayesian methodologies to demonstrate the efficacy of drugs in pediatrics; Discuss ways to integrate pediatric into adult drug development.

Chair

Robert Meland Nelson, MD, PhD

Speaker

The Use of Extrapolation in Pediatric Drug Development
Robert Meland Nelson, MD, PhD

Using Historical Data to Establish More Efficient Clinical Trials
Jeen Liu, PhD

Design and Analytic Strategies for Incorporating External Data in a Global Pediatric Trial
Margaret Gamalo-Stiebers, PhD



Speakers
MG

Margaret Gamalo-Stiebers

Principal Research Scientist, Eli Lilly and Company
JL

Jeen Liu

Associate VP, Biostatistics, Allergan Inc.
I have worked in the Pharma industry for 20+ years as a statistician, currently responsible for biostatistics support of all the ophthalmology products for the company. Prior to joining Allergan, I have worked for Novartis, Astellas and Eisai. My experience in clinical trials covers... Read More →
avatar for Robert Nelson

Robert Nelson

Senior Director, Pediatric Drug Development, Johnson & Johnson
Robert “Skip” Nelson, MD, MDiv, PhD. is currently Senior Director, Pediatric Product Development at Johnson & Johnson. Previously, he was the Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics, Office of the Commissioner at the U.S. Food and... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Beginner
  • Featured Topics Patient Focused,Innovative Trial Design
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session

3:15pm

#376.1 RT: Round Table Discussion: Master Protocols: Applications in Oncology
Component Type: Session
Level: Basic

Join DIA Statistics Community for a round table discussion tied to the session #271: Master Protocols: Applications in Oncology (Tuesday, June 25, 2:00-3:15PM). Seating is limited. To include special guests from the session: Amy Xia

Chair

Freda W. Cooner, PhD


Speakers
FC

Freda Cooner

Director, Statistical Innovation, Amgen
Dr. Freda Cooner is currently a Director, statistical innovation in Amgen. She is leading the exploration of innovative design options for several oncology and non-oncology products, including master protocols and seamless trials. Before joining Amgen late last year, she was an associate... Read More →


Wednesday June 26, 2019 3:15pm - 4:15pm
Community Area Sails Pav San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#394: Demystifying Basic Statistical Concepts for Anyone Involved with Clinical Trials
Component Type: Workshop
Level: Basic
CE: ACPE 1.25 Application UAN: 0286-0000-19-710-L04-P; CME 1.25; IACET 1.25; RN 1.25

Not a statistician? Then this workshop is for you!

Group activities and slide-based learning will be used to introduce commonly used statistical concepts using real clinical trial results from publicly posted FDA reviews.

Learning Objectives

Explain basic statistical concepts such as randomization, blinding, hypothesis testing, p-values, the challenges of false positives and adjusting for multiplicity, and approaches to handling missing data; Identify the appropriate statistical analysis for a given outcome; Discuss assumptions and limitations of analysis methods with a study team; Assess the clinical importance of statistical results.

Chair

Matt Baldwin, MS

Speaker

Facilitator
Stephen Corson, PhD

Facilitator
Munish Mehra



Speakers
avatar for Matt Baldwin

Matt Baldwin

Senior Biostatistician, Advance Research Associates, Inc (ARA)
Matt Baldwin is a Senior Biostatistician who works with Advance Research Associates to advance clinical solutions through analysis. Matt’s various positions have provided experience in randomization, generic and new drugs, statistical clinical development, DMC support, and analysis... Read More →
avatar for Stephen Corson

Stephen Corson

Senior Statistician, Phastar
I am a Senior Statistician working at Phastar, a specialist CRO offering statistical consultancy, clinical trial reporting and data management services to pharmaceutical and biotech companies. Prior to this I spent 3 years working as a Consultant Statistician in the Department of... Read More →
avatar for Munish Mehra

Munish Mehra

Executive Director, Tigermed
Dr. Munish Mehra, is a drug development expert with Tigermed, providing services to Pharmaceutical and Biotechnology companies to design, analyze and report on Phase 2 and 3 studies. He has represented several sponsors at pre-IND, End of Phase 2 and pre-NDA meetings. He is a frequent... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 11B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#395: Utilization and Evaluation of Innovative Approaches for Efficient Drug Development
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-716-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will discuss how to promote utilization of innovative methodologies such as M&S and Bayesian approach for efficient drug development. Regulators will present their recent activities for reviewing/qualifying innovative methodologies and share their experiences.

Learning Objectives

Identify how to recognize innovative methodologies recently applied for efficient new drug development and related activities in regulatory agencies aiming at accelerating drug development; Discuss how to consider the use of such methodologies in drug development through better communication between industry and regulatory agencies.

Chair

Yuki Ando, PhD

Speaker

Utilization and Evaluation of Innovative Methodologies: PMDA Perspective
Yuki Ando, PhD

Extrapolation: Recent European Regulatory Considerations
Andrew Thomson, PhD, MA, MS

Model-Based Bayesian Methods in Pharmaceutical Clinical Trials
Neal Thomas, PhD



Speakers
YA

Yuki Ando

Senior Scientist for Biostatistics, Office of New Drug I, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Yuki Ando is a Senior Scientist for Biostatistics of the Pharmaceuticals and Medical Devices Agency (PMDA), Japan. She received a master’s degree in Engineering from Tokyo Science University, and PhD in Health Science from Osaka University. Currently she is responsible for the... Read More →
avatar for Neal Thomas

Neal Thomas

Executive Director, Biostatistics, Pfizer Inc
Neal Thomas joined Pfizer in 2002, and has been a member of the Statistics Research and Innovation center since 2004 where he has worked on clinical and non-clinical applications in many therapeutic areas. He received a PhD in Statistics from the University of Chicago, and he was... Read More →
AT

Andrew Thomson

Statistician, Biostatistics and Methodology Support Office, European Medicines Agency (EMA)
Andrew Thomson is a statistician in the Office of Biostatistics & Methodology at the European Medicines Agency. He provides methodological advice and guidance across all stages of development, and across all therapeutic areas. He also is the lead of the Statistics subgroup of ICH... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  • Level Intermediate
  • Featured Topics Regulator Thinking
  • Credit Type ACPE, CME, IACET, RN
  • Tags Session