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Innovative Trial Design [clear filter]
Monday, June 24
 

11:00am

#105: Addressing Heterogeneity of Real World Evidence in Drug Safety
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-522-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session addresses the concern of inconsistent real world evidence in drug safety by presenting 1) epidemiological approaches for assessing inconsistent findings in observational studies, 2) lessons learned from observational studies in a distributed research network and 3) challenges using methods that synthesize findings from observational studies.

Learning Objectives

Identify possible study factors contributing to heterogeneity of real world evidence using claims databases; Discuss lessons learned from observational studies in a distributed research network regarding heterogeneity of RWE; Describe methods for synthesizing findings from observational studies and their limitations.

Chair

Michael Blum, MD, MPH

Speaker

Epidemiological Approaches for Assessing Inconsistent Findings in Observational Studies
Huei-Ting Tsai, PhD, MS

Minimizing Heterogeneity in Distributed Research Networks: Examples from the Canadian Network for Observational Drug Effect Studies
Adrian Levy, PhD, MSc

Challenges with Methods Used to Synthesize Findings from Observational Studies
Alicia Gilsenan



Speakers
avatar for Michael Blum

Michael Blum

Deputy Director, Office of Pharmacovigilance and Epidemiology, CDER, FDA
Michael Blum, MD, MPH is currently Deputy Director, Office of Pharmacovigilance and Epidemiology in FDA CDER. He is a pediatric infectious diseases specialist. Dr. Blum worked as a medical reviewer in the CDER Division of Anti-Infective Drug Products, followed by over 20 years in... Read More →
avatar for Alicia Gilsenan

Alicia Gilsenan

Senior Director and Head, Epidemiology, RTI Health Solutions
Alicia Gilsenan, PhD, is Senior Director and Head, Epidemiology within RTI-HS and a licensed pharmacist. Dr. Gilsenan’s primary area of expertise is pharmacoepidemiology and therapeutic risk management and the structured benefit-risk assessment of medications. Since joining RTI... Read More →
avatar for Huei-Ting Tsai

Huei-Ting Tsai

Epidemiologist, Office of Surveillance and Epidemiology, CDER, FDA
Huei-Ting Tsai, Ph.D., is an Epidemiologist in the Office of Surveillance and Epidemiology in FDA CDER. Prior to joining FDA, she was an Assistant Professor at the Georgetown University Medical Center and served as the principle investigator of multiple grants to assess effectiveness... Read More →
avatar for Adrian Levy

Adrian Levy

Professor, Department of Community Health and Epidemiology, Dalhousie University
Adrian R. Levy PhD is Professor in the Department of Epidemiology and Community Health at Dalhousie University. He is author of over 150 peer reviewed publications (https://bit.ly/2CBWeI5) and is the co-editor of two volumes in the Springer Handbook of Health Services Research entitled... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

11:00am

#109: When Disaster Strikes: Developing a Proactive Plan to Address Challenges Brought on by Large Scale Natural Disasters
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-536-L04-P; CME 1.00; IACET 1.00; RN 1.00

We are faced with many challenges in drug development and clinical trial continuity, one that we must all be prepared for is how to manage your trial and team in the face of a natural disaster. Often we do not think about this until disaster strikes.

Learning Objectives

Discuss the importance of having a natural disaster management plan; Describe factors to consider when developing a natural disaster management plan; Identify key aspects of staff, site and patient support and how to be proactive and reactive in face of a natural disaster.

Chair

Mark Summers

Speaker

Panelist
Jennifer Sheller, MPH

Panelist
Raymond Policare, MPH



Speakers
JS

Jennifer Sheller

Regional Head, NA, Global Clinical Trial Operations, Merck & Co, Inc.
Jennifer Sheller joined Merck in 2017 as an Associate Vice President and the North America Regional Head of Global Clinical Trials Organization. In this role she is responsible for all aspects of Merck Research Laboratories clinical trial operations in the United States and Canada... Read More →
avatar for Mark Summers

Mark Summers

President, Patient Engagement Division, WIRB-Copernicus Group (WCG)
Mark Summers currently serves as President of the Patient Engagement Division at WCG, a division which includes all business units within WCG that provide services that directly interface with patients. Prior to his role at WCG he founded ThreeWire, Inc., a patient recruitment, enrollment... Read More →
RP

Raymond Policare

Senior Director, Clinical Operations, PRA


Monday June 24, 2019 11:00am - 12:00pm
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

11:00am

#110: Automation with Intelligence: Transformation From Human Resource to Artificial Intelligence in Risk Management
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-524-L04-P; CME 1.00; IACET 1.00; RN 1.00

Learn 1) challenges in adapting AI in risk management; 2) approaches in proactively managing clinical operation risk and in streamlining the process for automation; 3) a automation model to transform resource based monitoring into risk based approach

Learning Objectives

Discuss challenges in adapting artificial intelligence in risk management; Discuss innovative approaches in leveraging real-time access to clinical data and analytics in proactively managing clinical operation risk and in streamlining the process for automation in risk assessment and mitigation; Discuss automation maturity model to transform resource based monitoring into risk based approach.

Chair

Hanming H Tu, MSc

Speaker

Using Artificial Intelligence to Centralize, Standardize, and Ensure Data Integrity in Clinical Trial Monitoring
Michelle Marlborough

Artificial Intelligence in Risk Management
Susan Olson, PhD, MS

Utilizing AI to Transform Drug Safety Case Processing
Christina J Wocosky



Speakers
avatar for Susan Olson

Susan Olson

Chief Operating Officer, edj
Susan is Chief Operating Officer at EdjAnalytics and manages operations and innovates advanced analytics solutions in life sciences. Edj is a Louisville, Kentucky company that offers advanced analytics for healthcare providers, pharmaceutical companies, government organizations, payers... Read More →
avatar for Hanming Tu

Hanming Tu

Vice President, IT, Frontage Laboratories, Inc.
* MS, MCRP * VP, Clinical IT & Database Administration (DBA) at Frontage * Manager, DBA in Accenture Life Science Cloud * Director, Clinical IT in Octagon Research * Over 20 years of pharmaceutical and clinical IT experience * Oracle Certified Professional (OCP) and DBA Master from... Read More →
avatar for Christina Wocosky

Christina Wocosky

Industry Principal, Innovation and Though Leadership, Accenture
Chris Wocosky is an Industry Principal in Accenture’s Life Sciences R&D practice, and is based in San Diego, CA. She served as the Product Manager for an AI Platform, PV First, for case intake and pre-case processing, and was instrumental in developing and bringing the platform... Read More →
MM

Michelle Marlborough

Chief Product Officer, AiCure
As Chief Product Officer at AiCure, Michelle Marlborough is responsible for the product direction, definition and delivery of the company’s award-winning artificial intelligence platform. With over 23 years of experience in life sciences and software development, Michelle has been... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 1AB 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

Deputy Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development and has presented and published widely on it. Dr. Chakravarty received the FDA Award of Merit in 2008 and... Read More →
avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, FDA
Dr. Chen is the Team Leader of the Division of Biometrics IX within CDER of FDA, supervising reviewers for the Division of Non-Malignant Hematology Products. She joined FDA in 2000 after receiving her Ph.D. from the University of Iowa. She has had years' experience in reviewing drug... 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

11:00am

#119: Making Value-Based Contracting Stick
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-532-L04-P; CME 1.00; IACET 1.00; RN 1.00

Value-based contracting between payers, providers and manufacturers is an evolving space. Finding approaches that will can both scale and succeed long term have been challenging. The first speaker will describe a set of examples that focus on the payer-pharma relationships. The second two speakers will describe a new approach to using provider clinical outcomes in specialty conditions as the level of measurement.

Learning Objectives

Describe the current state of value-based contracting; Discuss examples of success and failure in value-based contracting; Identify a new approach to using provider clinical outcomes.

Chair

Richard Gliklich, MD

Speaker

Trends and Practices of Applying Real World Evidence to Market Access and Value-Based Care
Robert A. DiCicco, PharmD

Medical Scores to Enable Value-Based Care
Richard Gliklich, MD

Industry Update
Thomas Michael Renshaw, MBA, RPh



Speakers
avatar for Richard Gliklich

Richard Gliklich

Chief Executive Officer, OM1
Dr. Richard Gliklich is the CEO of OM1, Inc., an outcomes and technology company focused on real world evidence and measuring and predicting outcomes. Previously, he was founder and CEO of Outcome, which he led from inception through its acquisition by Quintiles. A graduate of Yale... Read More →
avatar for Thomas Renshaw

Thomas Renshaw

Senior Director, Business Solutions, Acentrus Specialty (Vizient)
Tom currently functions as the Senior Director of Business Solutions for Apexus with responsibility for innovative business strategies and development. Tom has served as a subject matter expert in the areas of transitional care, population health management, bioinformatics, 340B... Read More →
avatar for Robert DiCicco

Robert DiCicco

Deputy Chief Health Officer, IBM Watson Health
Rob DiCicco is a Deputy Chief Health Officer for IBM Watson Health where he focuses on Life Sciences. Rob joined IBM in 2018 after a long career in Pharmaceutical R&D spanning nearly 30 years. His area of expertise includes clinical pharmacology and experimental medicine, clinical... Read More →


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

3:30pm

#154: Current Initiatives on Patient Involvement in the Medicinal Product Lifecycle: CIOMS XI
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-555-L04-P; CME 1.00; IACET 1.00; RN 1.00

The forum will cover latest developments on CIOMS XI from the perspective of patients, regulators, and industry. Current thinking in more closely involving patients throughout the medicines lifecycle will be presented and audience feedback sought. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Tuesday, June 25, 2:00-3:00PM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Discuss the latest developments in patient involvement and engagement in the medicinal product lifecycle from development to post-marketing, pharmacovigilance and risk management; Describe current progress of the CIOMS XI WG in terms of involving patients and patient groups or associations more closely with medicines addressing medical needs.

Chair

Judith Zander, MD

Speaker

An Introduction to CIOMS and the CIOMS XI Initiative on Patient Involvement in the Medicinal Product Lifecycle
Herve Le Louet, DrMed, DrSc, MD, PhD, MS

FDA Perspective
Theresa Mullin, PhD

The Role of Patients in Drug Development and Lifecycle Use
Marc M. Boutin, JD

An Overview of the CIOMS XI Initiative
Kerry Leeson-Beevers

Using Patient Data to Evaluate the Benefits and Risks of Medicines
Meredith Y. Smith, PhD, MPA



Speakers
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →
KL

Kerry Leeson-Beevers

National Development Manager, Alström Syndrome UK
I joined Alström Syndrome UK (ASUK) in 2003 when my son was diagnosed with the condition. I am now the National Development Manager responsible for overseeing the Family Support Service and I work closely with our medical teams in Birmingham, UK to support the highly specialised... Read More →
avatar for Herve Le Louet

Herve Le Louet

President, Head of PV Coordination, CIOMS and APHP
Herve Le Louet is Professor of Clinical Pharmacology. He is currently head of Pharmacovigilance department of Paris University Hospitals (AP-HP). He is an hepatologist by training and has a PhD in Pharmacogenetic from the Paris University. He is the President of CIOMS and immediate... Read More →
avatar for Theresa Mullin

Theresa Mullin

Associate Director for Strategic Programs, CDER, FDA
Theresa Mullin, Ph.D., serves as CDER’s Associate Director for Strategic Initiatives. She leads Patient-Focused Drug Development which includes implementation of the 21st Century Cures Act. She leads the CDER International program and heads the FDA delegation to ICH, IPRP and other... Read More →
avatar for Meredith Smith

Meredith Smith

Global Risk Management Officer, Global Patient Safety, Amgen Inc.
Meredith Smith is Global Risk Management Officer at Amgen, Inc. where she leads a team of 6 scientists responsible for medicinal product benefit-risk assessment and risk management. She is a behavioral scientist and health services researcher by training with over 15 years of experience... Read More →
avatar for Judith Zander

Judith Zander

Director, Office of Pharmacovigilance and Epidemiology, OSE, CDER, FDA
Judy Zander is the Director of the Office of Pharmacovigilance and Epidemiology (OPE), in the Office of Surveillance and Epidemiology (OSE), in the Center for Drug Evaluation (CDER) at the U.S. Food and Drug Administration (FDA).She oversees the Divisions of Pharmacovigilance 1 and... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#156: Clinical Research in Emerging Regions
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-566-L04-P; CME 1.00; IACET 1.00; RN 1.00

Changing times, technology and a surge in activities in the emerging regions have prompted the global clinical research industry to further enhance capabilities by building capacity and developing talent. Outsourcing services to emerging regions continue to grow especially in the realm of risk based monitoring, data management, clinical trial management and site management. On the scientific arena, innovative research activities in countries like Korea, China, and Singapore to name a few have developed biomedical, biotech and clinical research infra structures with the ultimate goal of attracting big pharma to partner with them to co-develop their discoveries. However, capability, capacity, credibility and overall quality remain to be sticking points.

In this session, current trends, issues, challenges and opportunities will be discussed by experts who work in these emerging regions. Their hope is that by sharing experiences and information the global pharma and biotech industry would have better understanding of the huge opportunities and untapped potential these emerging regions can provide. Join the Good Clinical Practices & Quality Assurance Community for a follow up Round Table discussion on Tuesday, June 25, 3:15-4:15PM in the DIA Community Zone 2, Sails Pavilion.

Learning Objectives

Discuss the current environment of clinical research in emerging regions; Identify strategic advantages in conducting clinical trials in emerging regions; Identify solutions to challenges in conducting clinical research activities and trial management in these emerging regions.

Chair

Nadina Jose, MD

Speaker

What is the Best Collaborative Model to Deliver Pivotal Clinical Trials in Developing Countries
Asita De Silva, MD, PhD, FRCP

Strategic Inclusion of Regions in Multi-Regional Clinical Trials
Eunyoung Kim, PharmD, PhD

An Integrated Mobile eSource-EDC Solution to Streamline Clinical Trial Data Management for Large Global Trials
Avik Kumar Pal, MBA



Speakers
avatar for Nadina Jose

Nadina Jose

Assistant Professor, School of Health Professions, MS Clinical Research Program, Rutgers, The State University of New Jersey
Rejoined academia on Oct 2016 and is currently Assistant Professor at Rutgers University, Department of Health Informatics, Biopharma Education Initiative, School of Health Professions MS in Clinical Research Management. Dr. Jose is the Founder and President of Anidan Group Pte. Ltd... Read More →
EK

Eunyoung Kim

Professor, College of Pharmacy, Chungang University
avatar for Avik Pal

Avik Pal

Chief Executive Officer, CliniOps
Avik is the CEO of CliniOps, a mobile, cloud-based, digital solutions company, for the clinical trial industry & global health research. Prior to that, he worked with two successful startups and is a Founding Board member at iKure, a healthcare startup. He is passionate about Global... Read More →
AD

Asita De Silva

Professor of Pharmacology, University of Kelaniya
Professor Asita de Silva has held many positions in academic medicine over the last 25 years. He is a Clinical Pharmacologist, and is currently Senior Professor of Pharmacology at the Faculty of Medicine, University of Kelaniya, Sri Lanka. He is also Director of the Clinical Trials... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 9 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 Scientific Officer, CluePoints
As Chief Scientific Officer for CluePoints, Steve oversees the research and development of advanced methods for data analytics, data surveillance and risk management, along with providing guidance to customers in RBQM methodology and best practices. Steve worked for three bio-pharmaceutical... 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

3:30pm

#171: The Courage of Career Transitions
Component Type: Session
Level: Intermediate

This session will present examples of career transition considerations to advance growth and development. Speakers who have transitioned from within industry functional areas, from industry to consulting or regulatory agencies; and from health care

Learning Objectives

Discuss critical career decision-making criteria; Describe challenges and opportunities to career transitions; Outline benefits and risks of career transition; Describe best practices in cultivating new skills and experience to inform transitions; Identify strategies to help ensure transitions are successful.

Chair

Matthew Wong, PharmD, MBA

Speaker

Industry Perspective
Dan Fredeman

Industry Perspective
Jasminder Kumar, PharmD, RPh



Speakers
avatar for Dan Fredeman

Dan Fredeman

Associate Director, US Oncology Markegting, AML Franchise, Daiichi Sankyo
My educational background is in materials engineering, biomaterials, and engineering management. After several years of interning at multiple research labs, I transitioned to an office-based commercial position within healthcare. I have been with Daiichi Sankyo (DSI) for almost 6... Read More →
avatar for Jasminder Kumar

Jasminder Kumar

Manager, Global Regulatory Affairs, Oncology, Janssen Research & Development, LLC
Jasminder Kumar, PharmD, is a North America Regulatory Affairs Manager within the Global Regulatory Affairs Oncology group at Janssen Research & Development, LLC. At Janssen, she has contributed to the operational and strategic aspects of regional oncology drug development for early... Read More →
avatar for Matthew Wong

Matthew Wong

Chief of Staff, Global Regulatory Affairs, Celgene Corporation
Matt graduated in 2008 with his Pharm.D. from Rutgers University and subsequently completed a post-doctoral fellowship in Medical Affairs. From 2008 to 2015, he held various responsibilities and roles within the Medical Affairs umbrella at Daiichi Sankyo. In 2016, Matt made a transition... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 14A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Tuesday, June 25
 

8:00am

#204: Protocol Developments of the Future
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-586-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will discuss specific areas in the protocol development process and the use of emerging technologies.

Learning Objectives

Identify where and how technology can be applied to improve or transform traditional protocol development processes: Describe best practices for creating, managing and implementing templates, and standard re-usable content; Discuss how consistently drafted protocols can lead to better success in a regulatory submission and aligned global transparency: Explain how to succeed developing protocols and managing amendments for complex study designs including master protocols.

Chair

Bob Brindle, MA

Speaker

Enforcing Better Standards in Protocol Development
Bob Brindle, MA

Objectives, Endpoints, and Outcome Measures: Developing Protocols to Achieve Consistency Between Documents and Registrations
Nate Root, MSc

Using Technology Innovations to Navigate Substantial Protocol Amendments for Studies with Complex Innovative Design
Rachael Cui Song, MBA

Protocol Development of the Future
Robert A. DiCicco, PharmD



Speakers
avatar for Bob Brindle

Bob Brindle

Venture Leader and Associate Director, Life Sciences R&D Practice, Cognizant
After graduating in Biochemistry from Cambridge University, Bob spent more than 20 years working at a major pharmaceutical company. Starting his career in Data Management, he progressed to become Head of Technology for Clinical Operations, and has extensive expertise in clinical trials... Read More →
avatar for Robert DiCicco

Robert DiCicco

Deputy Chief Health Officer, IBM Watson Health
Rob DiCicco is a Deputy Chief Health Officer for IBM Watson Health where he focuses on Life Sciences. Rob joined IBM in 2018 after a long career in Pharmaceutical R&D spanning nearly 30 years. His area of expertise includes clinical pharmacology and experimental medicine, clinical... Read More →
avatar for Nate Root

Nate Root

Associate Director, Disclosure and Transparency, Ionis Pharmaceuticals
Nate is a disclosure and transparency professional, with over 6 years of experience in the environment, working for and with CROs, Big Pharma, Non-Profit, and Small Biotech. He is a member of the DIA Disclosure Community Core Team, PhUSE CTD initiatives, and creator of the West Coast... Read More →
avatar for Rachael Song

Rachael Song

Associate Director - Global Project Management, PPD
Rachael is currently global Project Leader for Oncology clinical trials with focus on Immuno-Oncology and master protocol studies. She has experiences in compound-level clinical planning and design, study-level operational planning and strategy development, and program implementation... Read More →


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

8:00am

#205: Driving Enrollment with a Patient-Centric Focus to Artificial Intelligence
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-587-L04-P; CME 1.25; IACET 1.25; RN 1.25

Artificial intelligence, blockchain, natural language processing and real world evidence have been all the rage lately in industry news. Hear perspectives on these new technologies from a panel of experts from sponsor, technology vendor, patient advocacy group, and patients on what's working and what's not.

Learning Objectives

Describe how to drive patient enrollment in clinical studies with a patient-centric approach, allowing patients to pre-screen themselves and retargeting them if excluded; Identify how to address patient recruitment issues associated with pre-screening, screening and dropout to reduce the current average conversion ratio from 10:1; Discuss how to reduce pre-screening burden via mining of EMR records.

Chair

Rob Wynden, PhD

Speaker

Introduction to Patient-Facing Trial Technology
Rob Wynden, PhD

A Bulls-Eye Clinical Trial via Patient's Point- of-View
Abhit Singh, MD, MHA

User-Driven Markets Data that Help Clinicians Assess Treatments and Cure Diseases
Collin Powell, MBA

Turning Clinical Trial Recruitment on its Head Using EHR-Connected Applicants
Scott Cressman

Pragmatism and AI in Research
Mike Nolte, MBA



Speakers
avatar for Scott Cressman

Scott Cressman

Business Development and Strategic Partnerships, Human API
Scott Cressman leads business development & strategic partnerships in clinical research and digital health for Human API. He spends much of his time working at the intersection of life sciences + digital technologies with startups and Fortune 500s alike. His focus is on improving... Read More →
avatar for Mike Nolte

Mike Nolte

Chief Executive Officer, Signant Health
Mike Nolte is the CEO of CRF Bracket. Mike is a seasoned operational leader in healthcare and technology who is passionate about the patient experience. Before CRF Bracket, Mike was CEO at Influence Health, President and COO at MedAssets, and he held several leadership roles in healthcare... Read More →
CP

Collin Powell

Founder, Chief Executive Officer, The Health Exchange Market (THEM)
The Health Exchange Market (T.H.E.M.) is an online marketplace for de-identified healthcare data that allows organizations to realize new revenue streams through data commercialization, while also being able to obtain access to prospective and retrospective data from a variety of... Read More →
avatar for Abhit Singh

Abhit Singh

Chief Medical Advisor, Medical Advisory Board, WeHeal Foundation
Dynamic (Global) Physician Leader, Speaker and Visionary- specializing in the Medical Advisory, Transactional and Transformational management of Healthcare Advisory, Biotech & Biopharma organizations. Among many, a Medical Advisor for WeHeal Inc, a 'CrowdHealing' Therapeutic Options... Read More →
avatar for Rob Wynden

Rob Wynden

Chief Executive Officer, Aloha Health Network
Rob has been a computer scientist and programmer for over 30 years in the scientific programming, e-commerce, biotech, and health care industries. He implemented study startup and CTMS functionality at 9 bay area biotech companies, and invented a method that allows medical and biotech... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

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

Scientific lead, 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 Zink

Richard 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

10:30am

#228: Emerging Safety Challenges in New Oncology Treatments
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-591-L04-P; CME 1.00; IACET 1.00; RN 1.00

Immuno-oncology (IO) agents are emerging as essential tools to treat a wide range of solid tumors and their indications for use continue to expand. However, the antitumor immune activity of these agents can also be a threat to healthy tissue, resulting in novel presentations of adverse reactions, sometimes with highly undesirable outcomes. Early recognition and intervention can have a dramatic effect outcomes, particularly for very rare events that are progressive. However, presentation can mimic other less serious conditions that do not require early intervention. Thus, these immune-mediated phenomena have become the focus of intense exploration.

This session will provide a very brief conceptual overview of these agents, followed by two presentations. the first presentation will include methodological considerations for identifying potential predictors, such as transcriptomic biomarkers or specific interleukins, etc., that might aid prevention, facilitate early detection, or predict mortality. The second presentation will provide regulatory considerations in ensuring that benefits of these products outweigh their risks. Both presentations will include examples to illustrate key points. The session will conclude with an opportunity for Q&A.

Learning Objectives

Outline basic mechanistic aspects related to the efficacy and safety of immuno-oncology agents; Describe methodological considerations when identifying utility of markers that can help in the prevention or early diagnosis of IO-associated adverse events; Discuss considerations in benefit-risk assessment and communication for these agents.

Chair

William Gregory, PhD

Speaker

Checkpoint Inhibitor-Associated Myocarditis: Prevention or Early Detection?
Tarek Hammad, MD, PhD, MS, MSc, FISPE

Regulatory Challenges in Benefit/Risk Assessment and Labeling of Immuno-Oncology Agents
Meredith K. Chuk, MD, MHS



Speakers
MC

Meredith Chuk

Acting Associate Director for Safety, OHOP, CDER, FDA
avatar for William Gregory

William Gregory

Senior Director, Safety and Risk Management, Pfizer Inc
He received formal training in infectious diseases and molecular mechanisms of pathogenesis and has more than 15 years of experience directing global product development and registration programs. He also has extensive experience in pharmacovigilance and health informatics as well... Read More →
avatar for Tarek Hammad

Tarek Hammad

Therapeutic Area Strategy Lead, Global Pharmacovigilance, Sanofi Genzyme
Dr. Hammad joined Sanofi-Genzyme, in 2019, as a Therapeutic Area Strategy Lead in the Global Pharmacovigilance group. Prior to this, he served as the Head of Signal Detection and Benefit-Risk Assessment in Merck KGaA/EMD Serono. Previously, he was an Executive Director of Pharmacoepidemiology... Read More →
MC

Meredith Chuk

Acting Associate Director for Safety, OOD, OND, CDER, FDA
Dr. Meredith Chuk is the Acting Associate Director for Safety in the Office of Oncologic Diseases in OND/CDER at the FDA.


Tuesday June 25, 2019 10:30am - 11:30am
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#229: An Industry Collaboration on Pharmacovigilance Analytics
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-592-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session will discuss an initiative and how it is facilitating biopharmaceutical companies to enable earlier and systematic identification of potential safety issues to help minimize harm to patients, improve quality of analytic methods to improve product development, and deliver accelerated method development through collaboration and piloting. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Wednesday, June 26, 12:45-1:45PM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Discuss methods for collaborating and innovating in analytics to improve patient safety; Identify how to build awareness of opportunities for greater alignment across the industry in signal detection and management; Describe industry best practices of current and future signal detection and management.

Chair

Andres Gomez, PhD, MPH

Speaker

Panelist
John Van Stekelenborg, PhD, MBA, MSc

Panelist
Neal Grabowski, MS



Speakers
AG

Andres Gomez

Vice President, Head of Epidemiology, Safety Science, and Analytics, Bristol-Myers Squibb
Dr. Gomez joined at Bristol Myers Squibb Company as an Associate Director of Pharmacoepidemiology in the Global Pharmacovigilance and Epidemiology department in 2003. He currently heads the epidemiology, safety science and analytics groups which is accountable for the real world research... Read More →
NG

Neal Grabowski

Director, Pharmacovigilance & Patient Safety, AbbVie
Prior to joining AbbVie, Neal held multiple roles in Patient Safety and Clinical Operations at Amgen, Genzyme and Sanofi. He holds a MS in biomedical engineering and MBA from Pepperdine University.
avatar for John Van Stekelenborg

John Van Stekelenborg

Director, Group Lead, Methods and Analysis, Johnson & Johnson
John van Stekelenborg is the Head of the Methods & Analysis group in the Global Medical Safety department of Janssen, the Pharmaceutical Companies of Johnson & Johnson. John has been with J&J since 2002 and assumed leadership of the M&A group in 2011 where he leads a group of data... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#242: Use of Real World Evidence to Support Regulatory Decision-Making: First-Year Findings From the RCT-DUPLICATE Project
Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-601-L04-P; CME 1.00; IACET 1.00; RN 1.00

The Cures Act and the PDUFA IV mandated that the US FDA develop rules to guide industry on the use of real world evidence (RWE) for drug approvals. The session will bring together researchers and regulators to discuss the role of real world evidence.

Learning Objectives

Describe the DUPLICATE project replicating RCTs with real world data (RWD) analyses and provide an interim update on the findings from the first year; Discuss key considerations in implementing non-randomized studies using real world data to ensure valid and unbiased findings; Facilitate discussion how real world evidence may be used in regulatory decision-making.

Chair

Jeremy Rassen, DrSc, MS

Speaker

Initial Learnings From the RCT DUPLICATE Project
Jessica Franklin, PhD

Industry Perspective
Brande Yaist, MHS

Overview of the DUPLICATE Real World Evidence Demonstration Project
David Martin, MD, MPH



Speakers
avatar for Jessica Franklin

Jessica Franklin

Biostatistician, Div of Pharmacoepidemiology & Pharmacoeconomics;Asst Professor, Brigham and Women's Hospital and Harvard Medical School
Jessica Franklin, PhD, is an Assistant Professor of Medicine at Harvard Medical School and biostatistician in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. Her research focuses on developing and applying statistical methods for the study... Read More →
avatar for David Martin

David Martin

Associate Director for Real World Evidence Analytics, OMP, CDER, FDA
David Martin is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, FDA Center for Drug Evaluation and Research. He oversees demonstration projects intended to support the agency’s evaluation of real world evidence, reviews real world evidence submissions... Read More →
avatar for Jeremy Rassen

Jeremy Rassen

President and Chief Science Officer, Aetion
Jeremy A. Rassen, ScD is a pharmacoepidemiologist with 25 years of academic and industry experience. He is co-founder, president, and chief science officer at Aetion, a health care technology company that delivers real-world evidence for life sciences companies, payers, and regulatory... Read More →
avatar for Jessica Franklin

Jessica Franklin

Assistant Professor of Medicine, Div of Pharmacoepidemiology and Pharmacoeconomi, Brigham and Women's Hospital and Harvard Medical School
Jessica Franklin, PhD, is an Assistant Professor of Medicine at Harvard Medical School and biostatistician in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. Her research focuses on developing and applying statistical methods for the study... Read More →
avatar for Brande Yaist

Brande Yaist

Senior Director, Global Patient Outcomes & Real World Evidence, Eli Lilly and Company
Mrs. Yaist is the Senior Director of the Center of Expertise in Global Patient Outcomes and Real World Evidence at Eli Lilly and Company. She leads and develops the research talent and capabilities needed to provide scientific services/expertise and support across an array of core... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#247: Pregnancy, Breastfeeding, Childcare, Oh My! Finding a Balance for New Moms
Component Type: Forum
Level: Intermediate

Women face unique challenges in the workplace when returning to work after maternity leave. This panel will discuss how to navigate the period of time after maternity leave when working with a new baby at home and trying to balance breastfeeding, postpartum issues and lack of sleep with wanting to succeed in the workplace. This forum will also be useful for managers who want to create policies to accommodate working moms in the months when they return to work as well as colleagues who want to support women during this time.

Learning Objectives

Discuss the challenges faced by working mothers in the current work environment; Describe techniques and approaches from working mothers to balance career and family; Discuss challenges such as backup child care, breastfeeding in the workplace, and baby sleep issues.

Chair

Rachel SW Turow, JD, MPH

Speaker

Panelist
Robin Kaplan, MEd

Panelist
Shana Bruno, MPH

Panelist
Jen Lynn Varela



Speakers
SB

Shana Bruno

Director, Lactation Supportive Environments, University of California San Diego, Center For Community Health
Shana Wright Bruno is a highly motivated and dedicated public health professional with experience in leadership, program coordination, strategic planning, behavior change and health promotion. As the Director for the Lactation Supportive Environments (LSE) at UC San Diego, Center... Read More →
avatar for Robin Kaplan

Robin Kaplan

President and Owner, San Diego Breastfeeding Center, LLC
Robin Kaplan is an International Board-Certified Lactation Consultant (IBCLC), frequent media commentator on the topic of breastfeeding, author of Latch: a Handbook for Breastfeeding with Confidence at Every Stage, and founding host of The Boob Group podcast. She launched the San... Read More →
avatar for Rachel Turow

Rachel Turow

Executive Counsel, Regulatory Law, Teva Pharmaceutical Industries Ltd.
Rachel Turow is Executive Counsel – Regulatory Law at Teva Pharmaceutical Industries Ltd. In this role, Rachel provides regulatory legal support to Teva’s specialty and generic pharmaceutical businesses, and supports Teva’s drug-device combination products and digital health... Read More →
avatar for Jen Varela

Jen Varela

President, Postpartum Health Alliance and Pediatric Sleep Consultant, Sugar Night Night
Jen Varela, a Certified Gentle Sleep Coach®, co-author of “Loved to Sleep”, founder of Sugar Night Night, a pediatric sleep consulting services and current President for Postpartum Health Alliance. Jen has helped more than 3,750 sleep-deprived families have sweet sleep since... Read More →


Tuesday June 25, 2019 10:30am - 11:30am
Room 14A San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#255: Pharma and Regulatory Perspectives on Machine Learning Applications in Pharmacovigilance
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-610-L04-P; CME 1.25; IACET 1.25; RN 1.25

Machine Learning is one approach in the fast-paced field of artificial intelligence that is positioned to address many key issues in pharmacovigilance. However, it presents several challenges which must be addressed by both industry and regulators. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Wednesday, June 26, 3:15-4:15PM in the DIA Community Zone 2, Sails Pavilion.

Learning Objectives

Review updated developments in machine learning in PV; Discuss risks and challenges; Describe updates on regulatory landscape and areas of future development and collaboration.

Chair

Bruce Donzanti, PhD

Speaker

Chapter Two: Implementing Automation, Machine Learning, and Analytics in Pharmacovigilance - Case Study
Lisa George, RPh

FDA Perspective
Robert Ball, MD, MPH, MSc

MHRA Perspective
Mick Foy



Speakers
avatar for Robert Ball

Robert Ball

Deputy Director, Office of Surveillance and Epidemiology, CDER, FDA
Robert Ball MD, MPH, ScM is Deputy Director, Office of Surveillance and Epidemiology (OSE), Center for Drug Evaluation and Research (CDER), FDA. Dr. Ball shares in the responsibilities for leading OSE staff evaluating drug risks and promoting the safe use of drugs by the American... Read More →
avatar for Bruce Donzanti

Bruce Donzanti

Senior Group Director, Global Pharmacovigilance Innovation Policy, Genentech, A Member of the Roche Group
Bruce has a PhD in pharmacology/neuroscience with almost 30 years of experience in the pharma/biotech industry. Prior to industry, he performed research on mechanisms of neuronal degeneration and neurotoxicology and lectured in neuropharmacology to graduate and medical students while... Read More →
avatar for Mick Foy

Mick Foy

Head of Pharmacovigilance Strategy, Vigilance Intelligence, and Research Group, Medicines and Healthcare Products Regulatory Agency (MHRA)
Mick Foy is Head of Pharmacovigilance Strategy at the MHRA. Among is responsibilities is the running of the UK ADR reporting system and signal detection activities. Mick is also leads the MHRA's work with WHO and the Bill and Melinda Gates Foundation on capacity building in low and... Read More →
avatar for Lisa George

Lisa George

Advisor, Global Patient Safety, Case Management, Eli Lilly and Company
Lisa George has a B.S. in Pharmacy from Butler University, Indianapolis IN. Lisa started in the pharmaceutical industry in 1998 with Eli Lilly and Company's Global Patient Safety (GPS) department. During her time in GPS, she has worked with case management in various capacities. Lisa... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#256: Wearables and Patient Technologies Utilized in Clinical Trials
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-623-L04-P; CME 1.25; IACET 1.25; RN 1.25

Explore an initiative which seeks to facilitate and accelerate progression towards a future where patients have access to innovative technologies that enhance the patient experience and reduce their burden in clinical trials. Describe lessons learned from 3 "smart" trials executed for top 5 pharma companies. Explore the use of technology, devices, and virtual visits in a pediatric clinical trial setting and help to alleviate some of the questions and concerns sponsors and regulators have when endpoints or key outcome measures come from personal devices, wearables, or other patient controlled technology.

Learning Objectives

Define opportunities and challenges with use of patient-facing technology in clinical trials; Discuss how to complement eClinical platform with new technologies; Demonstrate how technology and virtual visits are being utilized to ease burden of participation and increase compliance.

Chair

Margareth Dimitrov-Kuhl, MA

Speaker

Patient-Driven Digital Platform: The Need to Integrate eClinical Technologies, Wearables, RPA, and AI/Machine Learning
Isabelle M de Zegher, DrMed, MD, MS, MSc

Making Pediatric Clinical Trials Fun (and Easy?): Virtualization, Wearables, and Other Technologies Utilized in Clinical Trials
Gina Calarco, BSN, MPH

Implementing Patient-Facing Technology in Clinical Trials: How to Overcome the Industry’s Greatest Challenges
Margareth Dimitrov-Kuhl, MA



Speakers
avatar for Gina Calarco

Gina Calarco

Director of Pediatric Strategy and Planning, Covance
Gina Calarco is Director of Operational Strategy and Planning for the Rare Disease and Pediatric Team at Covance. She is a nurse and received her MPH from the University of Kansas. She is an affiliate member of the American Academy of Pediatrics Section on Advances in Therapeutics... Read More →
MD

Margareth Dimitrov-Kuhl

Clinical Development Function Head, Novartis Pharmaceutical
Margareth Dimitrov-Kuhl is a Clinical Director Function Head at Novartis Pharmaceuticals. She is also a Sponsor-company representative serving on TransCelerate BioPharma’s Patient Technology Initiative focused on developing tools for study teams utilizing patient-facing technologies... Read More →
avatar for Isabelle de Zegher

Isabelle de Zegher

Vice President, PAREXEL Informatics
Isabelle de Zegher is Vice President, Integrated Solutions at PAREXEL Informatics where she is responsible for engineering strategy around Big Data, BI and Advanced Analytics. She has 25 years’ experience in Life Sciences, including extensive work on cross-industry forum on integration... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#268: Current Status of FDA Framework for the Evaluation of Real World Evidence
Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-619-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA issued a framework for a program that will evaluate the use of real world evidence (RWE) to help support the approval of new indications for an approved drug or to help support or satisfy drug post approval study requirements. During this session, FDA will share their takeaways from public comments about the RWE framework and provide participants with an update on the current status of the FDA RWE Program.

Learning Objectives

Identify three topic areas included in the FDA real world evidence program; Identify and discuss two FDA takeaways from the analysis of public comments to the real world evidence framework; Identify one area of interest for real world evidence guidance development.

Chair

M. Khair ElZarrad, PhD, MPH

Speaker

FDA Update
Jacqueline A. Corrigan-Curay, JD, MD

Update from CBER
Steven A. Anderson, PhD

FDA Update
David Martin, MD, MPH



Speakers
avatar for M. Khair ElZarrad

M. Khair ElZarrad

Deputy Director, Office of Medical Policy, CDER, FDA
Dr. ElZarrad is the Deputy Director of the Office of Medical Policy (OMP) at FDA’s Center for Drug Evaluation and Research (CDER), where he leads the development, coordination, and implementation of medical policy programs and strategic initiatives. Dr. ElZarrad currently leads... Read More →
avatar for Steven Anderson

Steven Anderson

Director, Office of Biostatistics and Epidemiology, CBER, FDA
Steven Anderson, Ph.D., M.P.P., is currently the Director of the Office of Biostatistics and Epidemiology (OBE) at the FDA Center for Biologics Evaluation and Research (CBER). He provides leadership for all CBER statistical, epidemiological and risk assessment programs. He was previously... Read More →
avatar for Jacqueline Corrigan-Curay

Jacqueline Corrigan-Curay

Director, Office of Medical Policy, CDER, FDA
Jacqueline Corrigan-Curay, J.D., M.D., serves as Director of the Office of Medical Policy (OMP) in the Center for Drug Evaluation and Research, FDA. OMP is comprised of the Office of Prescription Drug Promotion (OPDP) and the Office of Medical Policy Initiatives (OMPI). Dr. Corrigan-Curay... Read More →
avatar for David Martin

David Martin

Associate Director for Real World Evidence Analytics, OMP, CDER, FDA
David Martin is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, FDA Center for Drug Evaluation and Research. He oversees demonstration projects intended to support the agency’s evaluation of real world evidence, reviews real world evidence submissions... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#281: Structured Evidence Planning, Production, and Evaluation (SEPPE): A “Quality-Based” Framework for Drug Development
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-627-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will convene experts from the regulatory authority, payer, industry, and patient communities to discuss how a new type of strategic “quality by design” framework for drug development.

Learning Objectives

Describe a comprehensive framework for evidence generation to support optimal drug development and more efficient pathways to product approval; Identify pragmatic approaches for integrating this framework into regulatory authority and pharmaceutical company organizational structures.

Chair

Meredith Y. Smith, PhD, MPA

Speaker

SEPPE Framework and Implications for Regulators
Robyn R. Lim, PhD

Implications for Patients
Marc M. Boutin, JD

Pragmatic Lessons Learned from Implenting Within a Pharmaceutical Company
Meredith Y. Smith, PhD, MPA

EMA Perspective
Anthony Humphreys, MPharm



Speakers
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →
avatar for Anthony Humphreys

Anthony Humphreys

Head of Scientific Committees Regulatory Science Strategy, European Medicines Agency (EMA)
He is the Head of Scientific Committees Regulatory Science Strategy Division (SciRS). He is responsible for providing leadership in the Agency in the areas of Scientific Committees Coordination and Regulatory Science Strategy in support of delivering the EMRN 2020 Strategy. He chairs... Read More →
avatar for Robyn Lim

Robyn Lim

Senior Science Advisor, Health Products and Food Branch, Health Canada
Dr Lim’s focus is modernizing Canada’s federal drug regulatory system, developing the benefit-harm-uncertainty evidence standard for Canada’s amended Food and Drugs Act. Her interests are scientifically and socially-responsible patient-focussed and life-cycle approaches for... Read More →
avatar for Meredith Smith

Meredith Smith

Global Risk Management Officer, Global Patient Safety, Amgen Inc.
Meredith Smith is Global Risk Management Officer at Amgen, Inc. where she leads a team of 6 scientists responsible for medicinal product benefit-risk assessment and risk management. She is a behavioral scientist and health services researcher by training with over 15 years of experience... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#283: Let's Talk Risk-Based Monitoring
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: Let's Talk Risk-Based Monitoring; CME 1.25; IACET 1.25; RN 1.25

Let’s face it, Risk Based Monitoring (RBM) has been in the forefront of our industry for more than 10 years. From the beginning, RBM challenged the belief that 100% source document verification (SDV) would yield quality data in clinical trials. Regulators responsible for making decisions based on reliable data were also challenging industry as to the utility of this monitoring methodology as a way to identify and manage issues in clinical trials. It took some time for the focus of RBM to shift risks in a clinical trial that would include identification and assessment, ongoing monitoring, mitigation as well as even prevention. We are now in an epoch of understanding that it’s about the management of risks in clinical trials that could impact the reliability of patient data, patient safety and patient rights and welfare. In March of 2019, FDA has provided a draft guidance to augment the 2013 guidance relating to risk based-monitoring whereby FDA is making recommendations for planning a monitoring approach, developing content for monitoring plans, and the monitoring of results. Since regulations establish the expectations, it remains up to the sponsor to determine the how in order to achieve the expectation.

RBM has come a long way however sponsors may still struggle with certain aspects in the multi-prong processes needed to be successful with RBM. This forum will present various approaches to RBM, and give the opportunity to learn from experts, so Let’s Talk Risk-Based Monitoring!

Learning Objectives

Describe what is comprehensive RBM , with an understanding of key components to achieve goals of Risk-Based Monitoring in Clinical Trials; Develop a risk-based approach to monitoring of clinical trials that will achieve the expectations for quality clinical data; Identify how to determine best approach based on risks within the clinical trial and consider how to best monitor ongoing risks.

Chair

Susan Callery-D'Amico, BSN

Speaker

A Comparison of SDV to MANA Method: A Risk-Based Monitoring Approach
Penelope K. Manasco, MD, MS

Holistic Approach to Risk-Based Management of Studies and Monitoring of Investigators
Gregg Larson, PhD



Speakers
avatar for Susan Callery-D'Amico

Susan Callery-D'Amico

Vice President, R&D Quality Assurance, AbbVie, Inc.
Susan Callery D’Amico is a pharma leader with extensive experience in quality assurance and clinical development. She is the VP, R&D Quality Assurance, AbbVie, with the mission to drive quality excellence and continuous improvement as a strategic advantage across the lifecycle of... Read More →
avatar for Penelope Manasco

Penelope Manasco

Chief Executive Officer, MANA RBM
Dr. Manasco has conducted clinical research for over 30 years at the NIH and as an executive at GSK. She has spent 10 years in the area of clinical trial technology. Since starting MANA RBM, her focus has been on enhancing the processes of clinical research to rapidly identify and... Read More →
avatar for Gregg Larson

Gregg Larson

Vice President, Development Operations, AbbVie
Gregg’s career over 25+ years has spanned medical affairs, clinical research, and clinical operations. Gregg assumed his current role as VP, Development Operations in October 2017, which includes Site Management and Monitoring, Clinical Program Development, Strategic Clinical Operations... Read More →


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#290: The Responsibility Industry, Agencies, and Early Education own in Cure-Model Based Therapeutics
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-632-L04-P; CME 1.25; IACET 1.25; RN 1.25

In today’s pharmaceutical and biotech industry we are at an inflection point with regard to the future of therapeutic development. In this forum the focus will be to explore how industry, agencies, and academic institutions collaborate to drive innovative technologies to speed therapeutic development. Specifically, the forum will explore how we are leveraging STEM (Science, Technology, Engineering, Mathematics) programs both independently and interactively to enable current and future entrepreneurial opportunities. The goal is to foster an environment where we best take advantage of the opportunities presented by advances in life sciences and technology.

Learning Objectives

Evaluate the quality of interaction among various stakeholders in cure-based drug development; Assess the quality of those interactions in their own environments; Recognize the importance of fostering an environment where creativity and an entrepreneurial spirit is nurtured. Determine how STEM (Science, Technology, Engineering, Mathematics) programs are put to use to address the aforementioned objectives.

Chair

Dan Tierno, MA, MBA

Speaker

Industry Perspective
Yaron Drucker, MBA

Student Perspective
Advaita Chandramohan

Panelist
Melanie Matheu, PhD



Speakers
AC

Advaita Chandramohan

Undergraduate Research Associate, University of Southern California
Advaita Chandramohan is a second year student at the University of Southern California studying Biomedical Engineering with an Electrical emphasis. Throughout her academic career, Advaita has participated in and led various STEM activities, leading to her continued interest in the... Read More →
avatar for Yaron Drucker

Yaron Drucker

Staffing Lead, Cloud Partners, Google
Yaron has a Bachelor's Degree in Electrical Engineering, and an MBA from Baruch College. In his early career, Yaron worked in aerospace and defense as an EMC/EMI Engineer, and at SAP for a year. He then worked at Microsoft for 7 years in various roles in digital media on the advertising... Read More →
avatar for Dan Tierno

Dan Tierno

Associate Director, Global Clinical Data Sciences and Analytics, Bayer
Mr. Dan Tierno has over 20 years of experience in the pharmaceutical and biotech industry, specializing in drug development, data sciences and analytics for clinical trials, and commercialization of novel small molecule and cell based therapeutics. Mr. Tierno has spoken on training... Read More →
MM

Melanie Matheu

Founder and Chief Executive Officer, Prellis Biologics, Inc.


Tuesday June 25, 2019 4:15pm - 5:30pm
Room 16AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Wednesday, June 26
 

8:00am

#301: So Much Data, So Little Time: Hot Topics in Benefit-Risk Assessment
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-646-L04-P; CME 1.25; IACET 1.25; RN 1.25

Both regulators and sponsors are increasingly using structured frameworks to assess the benefit-risk profile of medicinal products. Such frameworks can accommodate a variety of data sources and can be supplemented by an array of graphical presentations. As experience with using structured benefit-risk assessment (SBRA) frameworks in both the pre- and post-market context accrues, a number of important questions remain, including: 1) What has been learned by FDA and EMA in regard to using a structured benefit-risk assessment framework for new drug application assessments? 2) How are these regulatory authorities continuing to train and support their reviewers in conducting such assessments? 3) What are the challenges involved in incorporating real world data into structured benefit-risk assessment analyses? 4) What are good practices in terms of evaluating the impact of different types of uncertainty on the quality of the benefit-risk assessment decision?

Learning Objectives

Describe the FDA’s and EMA’s experiences to date in implementing structured approaches to benefit-risk assessment, including the use of the Effects Tables and other tools; Discuss the key considerations in evaluating the impact of uncertainty on the quality of the benefit-risk assessment decision; Identify the challenges involved in incorporating real world evidence into a structured benefit-risk assessment.

Chair

Meredith Y. Smith, PhD, MPA

Speaker

FDA's Experience to Date Using the Structured Benefit-Risk Assessment Framework to Support Regulatory Decision-Making
Graham Thompson

Use of Real World Evidence in Personalized Benefit-Risk Assessment: Closing the Knowledge Gap
Tarek Hammad, MD, PhD, MS, MSc, FISPE

EMA Perspective
Douwe Postmus, PhD, MSc



Speakers
avatar for Tarek Hammad

Tarek Hammad

Therapeutic Area Strategy Lead, Global Pharmacovigilance, Sanofi Genzyme
Dr. Hammad joined Sanofi-Genzyme, in 2019, as a Therapeutic Area Strategy Lead in the Global Pharmacovigilance group. Prior to this, he served as the Head of Signal Detection and Benefit-Risk Assessment in Merck KGaA/EMD Serono. Previously, he was an Executive Director of Pharmacoepidemiology... Read More →
avatar for Douwe Postmus

Douwe Postmus

Researcher, University Medical Center Groningen
Dr Douwe Postmus is employed as a researcher at the Department of Epidemiology of the University Medical Center Groningen, The Netherlands. His research focuses on applying quantitative modeling techniques to decision making problems in health care, including regulatory, reimbursement... Read More →
avatar for Meredith Smith

Meredith Smith

Global Risk Management Officer, Global Patient Safety, Amgen Inc.
Meredith Smith is Global Risk Management Officer at Amgen, Inc. where she leads a team of 6 scientists responsible for medicinal product benefit-risk assessment and risk management. She is a behavioral scientist and health services researcher by training with over 15 years of experience... Read More →
GT

Graham Thompson

Operations Research Analyst, Decision Support and Analysis Team, OSP, CDER, FDA
Graham Thompson joined FDA in 2012 and currently serves as an operations research analyst for the Decision Support and Analysis Team (DSAT) in FDA’s Center for Drug Evaluation and Research (CDER). He works on CDER projects and initiatives related to benefit-risk assessment and supporting... Read More →


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

8:00am

#310: Project Planning 101: Turning Strategy into Execution
Component Type: Workshop
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-651-L04-P; CME 1.25; IACET 1.25; PDU 1.25 PMI 2166RVVC1X; RN 1.25

A successful project starts with good planning. This session will provide tool agnostic best practices for creating a credible, realistic timeline that will help your project team achieve its deliverables on time.

Learning Objectives

Describe fundamental components of at least one project management methodology for managing a project; Compare and contrast differences between project management approaches; Describe the various roles and responsibilities of project managers either in non-drug development projects or in junior roles.

Chair

Matthew Curin, PharmD

Speaker

Best Practices for Project Planning
Ken Hays, MBA



Speakers
avatar for Ken Hays

Ken Hays

Senior Project Management Consultant, Independent Consultant
Ken has been engaged in Project Management since 1992, with experience in IT, Data Communications, Medical Device R&D and manufacturing, Drug Development and Process Improvement initiatives. He has driven key changes to large and small Project Organizations globally. As a PMP and... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 15AB 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
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

Deputy Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development and has presented and published widely on it. Dr. Chakravarty received the FDA Award of Merit in 2008 and... 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

#318: Opportunities and Challenges of Collecting Data in a Pre-Approval Access Setting: A Multi-Stakeholder Perspective
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-658-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session reviews the benefits and risks of collecting real world data through single-patient requests (SPRs)/expanded access programs (EAPs) in a pre-approval access (PAA) setting and discusses what may confer the highest value to an organization from a multi-stakeholder perspective.

Learning Objectives

Recognize relevance and value for an organization of collecting real world data: Discuss the value of real world data from a pre-approval access (PAA) setting as it relates to regulatory and access strategies; Describe benefits and risks of collecting real world data in an single-patient requests (SPR)/named-patient program (NPP) and its potential implications on regulatory and access strategies.

Chair

Beverly L Harrison

Speaker

Pre-Approval Access and Real World Evidence Landscape Set-up
Sasha Richardson, MBA, MSc

Panelist
Arnaud Foucher, MBA, MS

Panelist
Alison Bateman-House, PhD, MA, MPH

Panelist
Michael Fine, MD



Speakers
avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, NYU Langone Health
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Langone Health's School of Medicine. She is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies ethical issues concerning... Read More →
avatar for Michael Fine

Michael Fine

Medical Director, Health Net
Dr. Michael Fine is a physician executive with over forty years of experience in all aspects of health care delivery. He is board certified in Internal Medicine and was among the first group of physicians who received additional certification in Geriatric Medicine. Doctor Fine began... Read More →
avatar for Arnaud Foucher

Arnaud Foucher

EMEA Medical Program Director, Medical Affairs, Europe, Middle-East and Africa, Janssen
Arnaud Foucher is EMEA Medical Program Director at Janssen, where he is responsible for the Company Regional Evidence Generation & Pre-Approval Access Programs, leading the EMEA Medical Program Management team. With a 20 years career in the pharmaceutical industry, Arnaud is focused... Read More →
avatar for Beverly Harrison

Beverly Harrison

Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson
Leading the Patient Support Group @ Janssen R&D (a J&J Co.), reporting directly to the Chief Medical Officer, Beverly focuses on developing strategies for patients’ needs by working with internal/external stakeholders & engaging patient advocacy organizations. She is a R&D & nonprofit... Read More →
avatar for Sasha Richardson

Sasha Richardson

Managing Director, EY
Sasha is an Executive Director in EY's Commercial Life Science Advisory practice and brings over 20 years of global experience in the healthcare / life science sector. Prior to EY, she headed up the US (SF/NY based) and EU (London based) Commercial & Market Access Consulting Practice... Read More →


Wednesday June 26, 2019 8:00am - 9:15am
Room 14B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

10:30am

#328: Risk-Based Monitoring: Best Practice Today and Technology for Tomorrow
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-673-L04-P; CME 1.00; IACET 1.00; RN 1.00

Organizations have faced challenges when implementing risk-based monitoring (RBM) and there is no one size fits all approach to overcome these challenges. Sharing best practices and implementing new facets of technology are opportunities to take RBM from the present to the future.

Learning Objectives

Discuss experiences and best practices on risk-based monitoring implementation including central monitoring; Demonstrate how technology enablement such as process automation and artificial intelligence can enhance the risk-based monitoring experience.

Chair

Suzanne Lukac

Speaker

The Keys to Successful Risk-Based Monitoring Implementation
Suzanne Lukac

Ahead of Curve in Risk-Based Monitoring Through Process Automation and Artificial Intelligence
Zabir Macci, MBA

Central Monitoring: Best Practices
Hadley Stinson



Speakers
avatar for Suzanne Lukac

Suzanne Lukac

Senior Account Director, CluePoints
Suzanne has nearly 20 years experience in the pharmaceutical industry including hands-on experience with RBM implementation. She is currently Senior Account Director at CluePoints where she is responsible for managing enterprise-level accounts and working with customers to lay the... Read More →
avatar for Hadley Stinson

Hadley Stinson

Associate Director, Clinical Monitoring Services, Premier Research
I am currently the Associate Director of Clinical Monitoring Services at Premier and oversee the NA CRA division. Prior to that I have worked in CRA Line Management and CRA positions and have global experience in multiple therapeutic areas including Oncology, Cardiovascular and R... Read More →
ZM

Zabir Macci

Centralized Monitoring Manager, IQVIA
Zabir is a Process Design & Analytics Manager for IQVIA focused on their approach to risk-based monitoring (RBM), Data-driven Trial Execution. He brings expertise spanning risk-based monitoring, business analysis, continuous process improvement, and analytics for clinical trials... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 9 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 →
avatar for Michelle Campbell

Michelle Campbell

Senior Clinical Analyst for Stakeholder Engagement, DNP, OND, 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 Science, IQVIA
Jean Paty is a leader in effective strategies and practices of capturing patient experience data for use in clinical development and commercial success of new and existing medical products. Jean has been published extensively in the areas of Clinical Outcome Assessments (COA) and... 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

#358: Improving the Trial Experience for Rare Disease Patients: Identifying and Overcoming Obstacles
Component Type: Workshop
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-697-L04-P; CME 1.25; IACET 1.25; RN 1.25

Participants will engage in an “overcoming obstacles” activity designed to encourage collaboration among rare disease patients, trial sponsors, and advocates. You will identify obstacles to trial participation and work to develop creative solutions.

Learning Objectives

Describe valuable and detailed input from patients on clinical trial design; Design patient interactions that encourage collaboration among patients, sponsors, and advocacy group; Explain how to optimize clinical trials to ensure they meet the needs of rare disease patients.

Chair

Kelly Franchetti, RN

Speaker

Patient Perspective
Kim Stephens, PhD, MA

Industry Perspective
Kelly Franchetti, RN

Academic Perspective
Ellyn Getz



Speakers
avatar for Kelly Franchetti

Kelly Franchetti

Vice President, Global Head Patient Insights and Engagement, ICON plc
Kelly Franchetti has a diverse background comprised of nursing, patient advocacy and clinical research. This broad range of experiences gives her a unique view of patients’ and caregiver’s perspectives in the realm of disease management and treatment. As a critical care nurse... Read More →
avatar for Ellyn Getz

Ellyn Getz

Associate Director, Development and Community Engagement, CISCRP
Ellyn Getz is the Associate Director of Development and Community Engagement at CISCRP. She is responsible for directing CISCRP’s educational and outreach initiatives, including AWARE for All: Clinical Research Education Days, MT Pharmacy and Journey to Better Health mobile experiential... Read More →
avatar for Kim Stephens

Kim Stephens

President, Project Alive - Hunter Syndrome
Dr. Kim Stephens is the President of Project Alive, a nonprofit organization whose mission is to cure Hunter Syndrome/Mucopolysaccharidosis II through research and advocacy. Having a son with Hunter Syndrome herself, this is also a personal mission for Dr. Stephens. Dr. Kim Stephens... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#359: Training for the Electronic Capture of PRO Data in Clinical Trials: Views from ePRO Vendors, Sponsors, Sites, and Patients
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-698-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum presents the ePRO Consortium’s best practices recommendations for training subjects and sites on technology use in clinical trials. Sponsor, ePRO vendor, site, and patient perspectives will be shared on training challenges and solutions.

Learning Objectives

Discuss best practice recommendations from the Consortium for training on the use of technology to collect PRO data in clinical trials; Identify challenges associated with training subjects and study sites from a vendor, sponsor, site, and patient perspective.

Chair

Serge Bodart, MS

Speaker

Panelist
Jenny Ly, PhD

Panelist
Patricia DeLong, MS

Panelist
T.J. Sharpe, PMP

Panelist
Jessica Branning



Speakers
avatar for Serge Bodart

Serge Bodart

Senior Advisor, Outcomes Science, CRF Bracket
Serge has extensive experience working in patient outcomes and new technologies. With Dr Bruno Pornel, he founded SYMFO, a European based ePRO provider in 2000. After he sold his company, he acted as the eCOA Subject Matter Expert at Biomedical Systems. Serge is currently Senior Advisor... Read More →
avatar for Patricia DeLong

Patricia DeLong

Manager, Patient-Reported Outcomes, Janssen Global Services, LLC
Patricia (Trish) Shepherd Delong has worked in the PRO and eCOA fields for more than ten years in both contract research and pharmaceutical organizations. She currently holds the role of PRO Process expert within the PRO team at Janssen. In this role Trish represents the PRO functional... Read More →
avatar for Jenny Ly

Jenny Ly

Clinical Science Advisor, ERT
Jenny Ly is currently a Clinical Science Advisor at ERT. She is a neuropsychologist with over 10 years of experience in the management and design of clinical trials. She has extensive experience developing training content for site staff, study participants, and caregivers that align... Read More →
avatar for T.J. Sharpe

T.J. Sharpe

Patient Advocate, Starfish Harbor LLC
T.J. Sharpe is a Stage IV melanoma patient who shares his journey through cancer in his Patient #1 Blog.  He was diagnosed in August 2012 with metastatic melanoma, weeks after his second child was born. He has undergone six surgeries and four immunotherapy treatments over two different... Read More →
avatar for Jessica Branning

Jessica Branning

Owner and Co-Founder, ClinCloud
Jessica Branning is the Co-Founder and Owner of ClinCloud, LLC, a Florida based clinical research site. She has nearly 5 years of clinical research experience in multiple therapeutic areas including Alzheimer's disease, Parkinson's disease, migraine, osteoarthritis, diabetes, and... Read More →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 10 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#379: From Trials to Real World: How Safety Protocols Impact REMS
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-700-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will present the approach for evaluation of interventions and/or protocol amendments in trials, and how these interventions impact the determination of the need for a REMS, and its design. Presentation will use an approved REMS.

Learning Objectives

Describe how safety interventions/trial protocol modifications are evaluated to ensure pre- and post-market continuity in safety management planning and the need for a REMS; Discuss evaluation of safety interventions in trials and their impact on REMS design; Discuss an example of the evaluation of trial protocol modifications and safety intervention considerations effecting an approved REMS.

Chair

Jamie Wilkins, PharmD

Speaker

Panelist
James Nickas, PharmD

Panelist
Christine Brown, MS

Moderator
Elaine Morrato, DrPH, MPH



Speakers
avatar for Christine Brown

Christine Brown

Executive Director, National PKU Alliance
Christine Brown is the first Executive Director of the National PKU Alliance, a non-profit organization working to improve the lives of individuals with PKU and pursue a cure. Christine has more than 30 years of experience in building and leading non-profit organizations at the local... Read More →
JN

James Nickas

Vice President, Pharmacovigilance and Clinical Medical Writing, Biomarin
James (Jim) Nickas is currently Vice President of Pharmacovigilance & Clinical Medical Writing at BioMarin in San Rafael, California. He joined the company in October 2010 bringing with him 12 years of academic experience at UCLA Medical Center and 18 years of drug development and... Read More →
avatar for Jamie Wilkins

Jamie Wilkins

Deputy Director, Division of Risk Management, OSE, CDER, FDA
Jamie Wilkins, Pharm.D. is the Deputy Director for the Division of Risk Management (DRISK) in the Office of Surveillance and Epidemiology (OSE) in the Center for Drug Evaluation (CDER) at the U.S. Food and Drug Administration (FDA). DRISK provides risk management expertise on development... Read More →
avatar for Elaine Morrato

Elaine Morrato

Professor Health Systems, Management and Policy, Colorado School of Public Health
Elaine Morrato, DrPH MPH is a Professor in Health Systems, Management and Policy and Associate Dean for Public Health Practice at the Colorado School of Public Health. Her research focuses on accelerating the translation of drug warnings into practice, and she has contributed her... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#382: Real World Data Quality for Regulatory Decision-Making
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-701-L04-P; CME 1.25; IACET 1.25; RN 1.25

Demonstrating the quality of real world data (RWD) to support regulatory decision-making is a topic of great interest. This session will explore what is being done to demonstrate that RWD is of sufficient quality to support regulatory assertions.

Learning Objectives

Describe how sponsors and data companies can demonstrate data quality, how FDA defines data quality, and how FDA reviewers assess real world evidence data quality; Discuss challenges with demonstrating data quality and what can be done to make advances in this area.

Chair

Rebecca Lipsitz, PhD

Speaker

Evaluating Real World Data and Evidence
David Martin, MD, MPH

Considerations for Selecting an Appropriate Real World Data Source
Brande Yaist, MHS

Aspects of Data Relevancy and Quality for Regulatory-Grade Real World Data
David Thompson, PhD

Real World Evidence Regulatory Grade Quality: Lessons from Recent Use Cases
Rebecca A Miksad, MD, MPH



Speakers
avatar for Rebecca Lipsitz

Rebecca Lipsitz

Director, Janssen
Rebecca Lipsitz is a Director in Janssen’s Global Regulatory Policy and Intelligence office. She advises and develops policy on a broad range of FDA-related regulatory issues. Her portfolio areas include Immunology, Infectious Disease, Vaccines, Precision Medicine, Combination Products... Read More →
avatar for David Martin

David Martin

Associate Director for Real World Evidence Analytics, OMP, CDER, FDA
David Martin is the Associate Director for Real World Evidence Analytics, Office of Medical Policy, FDA Center for Drug Evaluation and Research. He oversees demonstration projects intended to support the agency’s evaluation of real world evidence, reviews real world evidence submissions... Read More →
avatar for Rebecca Miksad

Rebecca Miksad

Senior Medical Director, Research Oncology, Flatiron Health
Rebecca Miksad is a health outcomes and clinical trial researcher who serves as senior medical director at Flatiron Health. In this role, Rebecca focuses on generating real-world evidence, establishing regulatory-grade quality and contributing to the development of clinically relevant... Read More →
avatar for David Thompson

David Thompson

Senior Vice President, Real World and Late Phase, Syneos Health
David Thompson, PhD is a health economist with 25+ years of experience in HEOR consulting and real-world research design. He currently holds the position of Senior Vice President for Real-World & Late Phase Research at Syneos Health, where he provides RWE design solutions to study... Read More →
avatar for Brande Yaist

Brande Yaist

Senior Director, Global Patient Outcomes & Real World Evidence, Eli Lilly and Company
Mrs. Yaist is the Senior Director of the Center of Expertise in Global Patient Outcomes and Real World Evidence at Eli Lilly and Company. She leads and develops the research talent and capabilities needed to provide scientific services/expertise and support across an array of core... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 1AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Thursday, June 27
 

9:00am

#410: Advancing Benefit-Risk Assessment to Support FDA’s Regulatory Review of Human Drugs and Biologics
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-724-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA currently uses a structured, qualitative framework for benefit-risk assessment of human drugs and biologics. This session will cover FDA’s efforts to enhance this qualitative framework and explore more advanced benefit-risk assessment methods.

Learning Objectives

Describe FDA’s approach to benefit-risk assessment under PDUFA V and VI; Discuss efforts to continue enhancing FDA’s methodological approach to benefit-risk assessment; Summarize findings from analysis of BRFs in 2017 novel drug approvals.

Chair

Graham Thompson

Speaker

Implementation of a Structured Benefit-Risk Framework into FDA's Human Drug Review
Leila Grace Lackey, MHS, PhD

CBER’s Experience with Benefit-Risk
Hong Yang, PhD

Planning and Assessing Benefit-Risk Quantitatively in the Next Decade
Susan Duke, MSc

Best Practices for Quantitative Benefit-Risk Assessments
Tommi Tervonen



Speakers
avatar for Susan Duke

Susan Duke

Mathematical Statistician, OB, OTS, CDER, FDA
Susanis is a Mathematical Statistician reviewer in CDER, assigned to pulmonary, allergy and critical care indications. She became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG, which lead to subsequent interests... Read More →
avatar for Leila Lackey

Leila Lackey

Operations Research Analyst, OPSA, OSP, CDER, FDA
Dr. Lackey has a Master of Health Science from Johns Hopkins in Environmental Health Sciences and a doctorate from UCLA in Environmental Science and Engineering. Professionally, she has worked for the Environmental Protection Agency and for the Food and Drug Administration leading... Read More →
avatar for Tommi Tervonen

Tommi Tervonen

Research Scientist, Evidera, PPD
Tommi Tervonen, PhD, is an Associate Director of Patient Preferences and Research Scientist at the Patient-Centered Research team of Evidera in London, UK. Dr. Tervonen joined Evidera in September 2015 after leaving his position of Assistant Professor at the Erasmus School of Economics... Read More →
GT

Graham Thompson

Operations Research Analyst, Decision Support and Analysis Team, OSP, CDER, FDA
Graham Thompson joined FDA in 2012 and currently serves as an operations research analyst for the Decision Support and Analysis Team (DSAT) in FDA’s Center for Drug Evaluation and Research (CDER). He works on CDER projects and initiatives related to benefit-risk assessment and supporting... Read More →
avatar for Hong Yang

Hong Yang

Biologist, Office of Biostatistics and Epidemiology, CBER, FDA
Dr. Hong Yang is a senior regulatory scientist in the Office of Biostatistics and Epidemiology, CBER, FDA. She holds Ph.D. degree in Biological Engineering. Dr. Yang has expertise in benefit-risk assessment of biological products. She has been devoted in regulatory review, as well... Read More →


Thursday June 27, 2019 9:00am - 10:15am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA