Pharmacovigilance and Risk Management Strategies Conference Short Courses: January 27 | Conference: January 28-30 Omni Shoreham Hotel

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Pharmacovigilance and Risk Management Strategies Conference Short Courses: January 27 | Conference: January 28-30 Omni Shoreham Hotel | Washington, DC

PROGRAM CO-CHAIRS Overview

Stella C. F. Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP Vice President, Global Head of Risk Management DIA’s Pharmacovigilance and Risk Management Strategies Conference provides the IQVIA strongest context, background, updates, new developments, and future direction

William W. Gregory, PhD for regulations and guidance on safety, pharmacovigilance, and risk management Senior Director, Worldwide Safety and Regulatory strategies that cannot be found in any other meeting. This conference will give Pfizer Inc you the foundation for strong strategic planning and practical decision-making in PROGRAM COMMITTEE your pharmacovigilance programs, rather than the cookbook approaches often presented elsewhere. In addition to new developments and updates on regional Mariette Boerstoel-Streefland, MD, MBA, MS Senior Vice President, Global Drug Safety regulations and guidelines, regulators from the US, EU, and other global regions Alexion provide the context and thinking behind evolving requirements

Mick Foy Group Manager, Vigilance Intelligence and Research Group Highlights Medicines and Healthcare products Regulatory Agency (MHRA), European Union • Opening Keynote Address from Katherine A. High, MD, President and Head of E. Stewart Geary, MD R&D, Spark Therapeutics Senior Vice President, Chief Medical Officer, Director, • Four Short Course offerings on January 27, prior to the conference kickoff Corporate Medical Affairs HQ Three full days for maximum learning opportunities Eisai Co., Ltd. • NEW! • NEW! Daily Keynote Speakers Lisa Melanie Harinstein, PharmD, BCPS Safety Evaluator, Division of Pharmacovigilance-I, Office of • Luncheon round table discussions with key thought leaders Surveillance and Epidemiology • NEW! Abstract poster session featuring four intriguing presentations CDER, FDA Stephen Knowles, MB.BS, MRCP Who Should Attend? President, Drug Safety and Pharmacovigilance Halozyme Therapeutics • Professionals involved in:

Barbara A. Morollo • Drug Safety/Pharmacovigilance Pharmacovigilance and Risk Management Consultant • Risk Management, including Risk Evaluation and Mitigation Strategies (REMS) Captain Robert G. Pratt, USPHS • Benefit-risk assessment and communication Consultant, OSE CDER, FDA • Medical Product Safety Assessment

Michael Richardson, MD, FFPM • Regulatory affairs International Head GPV&E and EU Qualified Person for • Pharmaceuticals, biologics, combination products, devices Pharmacovigilance Bristol-Myers Squibb • Clinical Research and Clinical Research Organizations • Pharmacoepidemiology Annette Stemhagen, DrPH Senior Vice President, Safety, Epidemiology, Registries and • Post-market studies and real world evidence generation Risk Management UBC • Customer engagement programs, including patient support programs

FDA LIAISON • Medical information, medical communications • Health outcomes Cheryl L. Campbell, MS • Academic research centers Associate Director, Executive Operations, Outreach and Communications Team, Office of Surveillance and • Regulatory agencies Epidemiology CDER, FDA

800 Enterprise Road #PVRMS19 | DIAglobal.org Suite 200 Horsham, PA 19044 USA As of January 17, 2019 Schedule At-A-Glance SHORT COURSES | SUNDAY, JANUARY 27 ROOM 8:00AM-5:00PM Registration and Continental Breakfast West Registration (Lobby Level) 9:00AM-5:00PM Short Course 1: Introduction to Pharmacoepidemiology Senate Room 9:00AM-5:00PM Short Course 2: Pharmacovigilance and Risk Management Planning Congressional AB 12:30-1:30PM Luncheon for Short Course Attendees Executive Room 9:00AM-12:30PM Short Course 3: Reference Safety Information Cabinet Room 1:30-5:00PM Short Course 4: Combination Products Cabinet Room

DAY ONE | MONDAY, JANUARY 28 ROOM 7:30AM-5:30PM Registration In Front of the Ambassador Ballroom 7:15-8:15AM Continental Breakfast and Networking Ambassador Ballroom 8:00-8:15AM DIA Opening Remarks Regency Ballroom 8:15-8:30AM Welcome Remarks Regency Ballroom 8:30-9:00AM Opening Keynote Regency Ballroom 9:00-10:15AM Session 1: FDA Updates Regency Ballroom 10:15-10:45AM Refreshments, Exhibits, and Networking Break Ambassador Ballroom 10:45AM-12:15PM Session 2: Patient Perspectives on the Risks and Benefits of Opioid Therapy Regency Ballroom 12:15-1:30PM Luncheon, Exhibits, and Round Table Discussions Ambassador Ballroom 1:30-2:15PM Session 3: Rare Disease I Regency Ballroom 2:15-3:00PM Session 4: Rare Disease II: The Patient Story Regency Ballroom 3:00-3:30PM Refreshments, Exhibits, and Networking Break Regency Ballroom 3:30-5:00PM Session 5: Pharmacovigilance and Risk Management Challenges in Advanced Therapeutics Regency Ballroom 5:00-7:00PM DIA 2019 Program Launch and Networking and Exhibits Reception Ambassador Ballroom

DAY TWO | TUESDAY, JANUARY 29 ROOM 7:30AM-4:30PM Registration In The Front of Ambassador Ballroom 7:30-8:30AM Continental Breakfast and Networking Ambassador Ballroom 8:30-8:45AM Welcome Remarks Regency Ballroom 8:45-10:00AM Session 6: EU Updates Regency Ballroom 10:00-10:30AM Refreshments, Exhibits, and Networking Break Ambassador Ballroom 10:30AM-12:00PM Session 7: Generating Real World Evidence (RWE) Fit for Regulatory Decisions: Learning from Safety Regency Ballroom 12:00-1:30PM Luncheon, Exhibits, and Round Table Discussions Ambassador Ballroom 1:30-3:00PM Session 8: Abstract Session Regency Ballroom 3:00-3:30PM Refreshments, Exhibits, and Networking Break Ambassador Ballroom 3:30-4:30PM Session 9: Reference Safety Information Use in Clinical Trials Regency Ballroom

DAY THREE | WEDNESDAY, JANUARY 30 ROOM 7:15AM-3:00PM Registration In Front of Ambassador Ballroom 7:15-8:15AM Continental Breakfast and Networking Ambassador Ballroom 8:15-8:30AM Welcome Remarks Regency Ballroom 8:30-10:00AM Session 10: Emerging Regions Issues Regency Ballroom 10:00-10:30AM Refreshments, Exhibits, and Networking Break Ambassador Ballroom 10:30-11:30AM Session 11: Immuno-Oncology – The Benefits and the Safety Challenges Regency Ballroom 11:30-1:00PM Luncheon, Exhibits, and Networking Break Ambassador Ballroom 1:00-2:30PM Session 12: Drug-Induced Liver Injury: What is New, the Impact of Immuno-Oncology Products on the Liver and Perspectives on Causality Assessments Regency Ballroom 2:30-3:00PM Refreshments and Networking Break Ambassador Ballroom 3:00-4:15PM Session 13: Aspects of Benefit-Risk Assessment Regency Ballroom 4:15-5:00PM Session 14: Hot Topics Regency Ballroom 5:00-5:15PM Closing Remarks Regency Ballroom

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 2 Learning Objectives

At the conclusion of this Conference, participants should be able to: • Apply the current regulatory framework for pharmacovigilance in key markets, including the US and the changing EU • Examine the influence of recent regulatory developments and expectations in China and countries outside of the EU on safety and pharmacovigilance practice • Discuss changes in pharmacovigilance and risk management resulting from the emergence of new and advanced technologies and therapies such as immunogene, and somatic cell therapies • Describe benefit-risk assessment and communication considerations and approaches, including incorporation of patient perspectives, for rare disease therapies, opioids, and established products • Apply good practices and align with regulatory agency expectations for the use of Reference Safety Information (RSI) in clinical trials • Summarize the key scientific criteria for evaluating the quality of RWE for regulatory decision making Continuing Education Credit ACPE CREDIT REQUESTS DIA is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing MUST BE SUBMITTED BY pharmacy education. This program is designated for up to 24 contact hours or 2.4 continuing WEDNESDAY, MARCH 13, 2019 education units (CEU’s). Type of Activity: Knowledge DIA is required by the Accreditation Council for Pharmacy Education (ACPE) to report pharmacy-requested CEUs through the CPE Monitor system. All ACPE-certified activity credit requests need to be submitted through DIA’s My Transcript within 45-days post activity. If ACPE credit is not requested by Wednesday, March 13, 2019, the CEU request will not be transmitted through to the CPE Monitor. Pharmacists will need to provide their National Association of Boards of Pharmacy (NABP) e-Profile ID and date of birth (MMDD) to ensure the data is submitted to the ACPE and NABP properly. If you need to obtain your NABP e-Profile, please visitwww.cpemonitor.net . DIA has been accredited as an Authorized Provider by the International Association for Continuing Education and Training (IACET). As an IACET Authorized Provider, DIA offers CEUs for its programs that qualify under the ANSI/IACET Standard. DIA is authorized by IACET to offer up to 2.4 CEUs for this program. Participants must attend the entire program in order to be able to receive an IACET statement of credit. No partial credit will be awarded. Nursing

As an Accredited Provider by the Accreditation Council for Pharmacy Education (ACPE) the American Nurses Credentialing Center (ANCC) recognizes ACPE Credit(s)™ issued by DIA as acceptable toward license CE requirements for nursing. If you would like to receive a statement of credit, you must attend the entire short course and/or all three days of the conference, sign in each day at the DIA registration desk upon arrival and complete the online credit request process through My Transcript. Participants will be able to download a statement of credit upon successful submission of the credit request. My Transcript will be available for credit requests beginning February 13. Credit Allocation

Short Course 1: Introduction to Pharmacoepidemiology: Pharmacy 6.5 contact hours or .65 CEUs, UAN: 0286-0000-19-007-L04-P; IACET .7 CEUs Short Course 2: Pharmacovigilance and Risk Management Planning: Pharmacy 6.5 contact hours or .65 CEUs, UAN: 0286-0000-19-008-L04-P; IACET .7 CEUs Short Course 3: Reference Safety Information: Pharmacy 3.25 contact hours or .235 CEUs, UAN: 0286-0000-19-008-L04-P; IACET .3 CEUs Short Course 4: Combination Therapies: Pharmacy 3.25 contact hours or .235 CEUs, UAN: 0286-0000-19-010-L04-P; IACET .3 CEUs Conference: Day 1: Pharmacy 6 contact hours or .6 CEUs, UAN: 0286-0000-19-011-L04-P; IACET .6 CEUs Conference: Day 2: Pharmacy 5.25 contact hours or .525 CEUs, UAN: 0286-0000-19-012-L04-P; IACET .5 CEUs Conference: Day 3: Pharmacy 6.25 contact hours or .625 CEUs, UAN: 0286-0000-19-013-L04-P; IACET .6 CEUs

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DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 3 SHORT COURSES | SUNDAY, JANUARY 27

8:00AM-5:00PM Registration* *Short Courses require a separate registration fee.

9:00AM-5:00PM Short Course 1: Introduction to Pharmacoepidemiology Instructors Adebola O Ajao, MPH, PhD, Epidemiologist, CDER, FDA Veronica V Sansing Foster, MS, PhD, Epidemiologist, CDER, FDA Efe Eworuke, MSc, PhD, Epidemiologist, CDER, FDA Danijela Stojanovic, Research Officer, OSE, CDER, FDA Lisa Melanie Harinstein, PharmD, BCPS, Safety Evaluator, Division of Pharmacovigilance-I, Office of Surveillance and Epidemiology, CDER, FDA Annette Stemhagen, DrPH, Senior Vice President, Safety, Epidemiology, Registries and Risk Management UBC This course will provide an overview of epidemiologic principles as they apply to pre- and post-marketing safety surveillance and risk management. Topics to be covered include: study designs for observational (non-interventional) studies, including case-control and cohort studies and studies of disease natural history; database studies and “big data”; and fundamental methods of analysis. Examples of epidemiologic approaches to meeting regulatory requirements for post-marketing studies will be reviewed. At the conclusion of this short course, participants should be able to: • Apply epidemiologic methods to drug/biologic/device safety surveillance • Recognize the role of epidemiology during product development • Explain the advantages and limitations of various study designs, and make recommendations about the best approaches to address safety analyses • Determine how to identify the best data source(s) for answering a regulatory requirement for post- marketing surveillance • Describe sources of “big data” and their value in safety surveillance and risk management

9:00AM-5:00PM Short Course 2: Pharmacovigilance and Risk Management Planning Instructors Stella C.F. Blackburn, MD, FRCP, FISPE, FFPM, Vice President, Global Head of Risk Management, IQVIA, United Kingdom William W. Gregory, PhD, Senior Director, Worldwide Safety and Regulatory, Pfizer Inc This full-day short course will focus on basic aspects of the product lifecycle regulatory framework for pharmacovigilance in the context of risk management planning. The practical aspects of managing biopharmaceutical product risks will be emphasized in the context of benefits and the healthcare delivery system. The main discussion will be devoted to the EU and US situations, but this will be supplemented with experience gained in other selected jurisdictions. Q&A will be encouraged. At the conclusion of this short course, participants should be able to: • Discuss similarities and differences in risk management planning in the three ICH regions and other selected jurisdictions • Describe the differences between important identified risks and important potential risks • Outline the basic structure and contents of an EU Risk Management Plan (in the context of a Risk Management System) and a Risk Evaluation and Mitigation Strategy (REMS) • Discuss primary and non-routine tools for managing product risks, how the effectiveness of a selected tool is assessed, and points to consider for the modification, revision, or release of a given non-routine intervention

12:30-1:30PM Luncheon for Short Course Attendees

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 4 9:00AM-12:30PM Short Course 3: Reference Safety Information Instructors Steve Knowles MB.BS, MRCP, Vice President Drug Safety and Pharmacovigilance, Halozyme Ken Lipetz PhD, MBA, HCLD, GPS Medical Business Advisor, Eli Lilly and Company Krisztina L. Debreczeni, MD, Group Medical Director, Medical Safety Review, Global Pharmacovigilance and Epidemiology, Research and Development, Bristol-Myers Squibb Co. This half-day short course will be conducted in two parts. The first part will focus on the basic aspects of the EU regulation and EU guidances (including Q&A documents) that govern the content, placement, use, and management of the Reference Safety Information as used in assessing expectedness of “suspected” serious adverse reactions (SARs) from clinical trials. The second part will focus on the practical aspects of implementing the regulations and guidances, including a review of acceptable and non-acceptable examples of the RSI and examples of regulatory agency inspection findings related to the RSI. At the conclusion of this short course, participants should be able to: • Describe the requirements for the use and management of the RSI in clinical trials • Understand how the requirements for RSI as described in the CTFG Q&A document are enforced by regulatory agencies • Be able to develop compliant RSI processes and documentation

1:30-5:00PM Short Course 4: Combination Products Instructors Khaudeja Bano, MD, MS, Head of Medical Affairs, Abbott Quynh Nguyen, MS, Associate Director for Human Factors, DMEPA, CDER, FDA Captain Melissa Burns, MS, Senior Program Manager, Office of Combination Products, FDA Marayam Mokhtarzadeh, Medical Officer, FDA Combination products are therapeutic and diagnostic products that combine drugs, devices, and/or biological products. Because combination products involve multiple components, they are associated with new regulatory, policy, development, and review challenges. In this short course, we will discuss updates to combination product regulation and explore some emerging challenges with combination products, such as adverse event reporting and principles of human factor studies during the development of combination products. At the conclusion of this short course, participants should be able to: • Explain what comprises a combination product • Describe components of the final rule on postmarketing safety reporting requirements for combination products (referred to as the combination product PMSR final rule) issued by FDA • Identify different methods for signal detection and management for combination products • List technical specifications for preparing and submitting electronic Individual Case Safety Reports (ICSRs) to incorporate the combination product PMSR final rule • Explain principles of human factors studies during the development of combination products • Describe different ways Industry can prepare to incorporate the combination product PMSR final rule into pharmacovigilance activities

DAY ONE | MONDAY, JANUARY 28

7:00AM-5:30PM Registration

7:00-8:00AM Continental Breakfast and Networking

8:00-8:15AM DIA Opening Remarks

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 5 8:15-8:30AM Welcome Remarks Session Co-Chairs Stella C.F. Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP, Vice President, Global Head of Risk Management, IQVIA, United Kingdom William W. Gregory, PhD, Senior Director, Worldwide Safety and Regulatory, Pfizer Inc

8:30-9:00AM Opening Keynote Session Chair Stella C.F. Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP, Vice President, Global Head of Risk Management, IQVIA, United Kingdom Keynote Speaker Katherine A. High, MD, President and Head of R&D, Spark Therapeutics

9:00-10:15AM Session 1: FDA Updates Session Chair Gerald J. Dal Pan, MD, MHS, Director, Office of Surveillance and Epidemiology, CDER, FDA FDA representatives will provide updates from the Office of Surveillance and Epidemiology (OSE) within CDER and the Office of Biostatistics and Epidemiology in CBER. Topics will include post-marketing safety monitoring, an overview of pharmacoepidemiology, pharmaceutical risk management, medication error prevention, and updates on safety surveillance from the Office of Generic Drugs. An FDA-CBER Update on Surveillance, Epidemiology, and Risk Management Approaches for Biologics Steve Anderson PhD, Director, Office of Biostatistics and Epidemiology, CBER Updates in Generic Drug Pharmacovigilance Karen B. Feibus MD, Lead Medical Officer, CSSS, Office of Generic Drugs, CDER, FDA James Osterhout PhD, Scientist, FDA

10:15-10:45AM Refreshments, Exhibits, and Networking Break

10:45AM-12:15PM Session 2: Patient Perspectives on the Risks and Benefits of Opioid Therapy Session Chair Captain Robert G. Pratt, USPHS, Consultant, OSE, CDER, FDA Balancing the risks of opioid therapy with the benefits is an ongoing challenge as prescription opioids continue to contribute to the US opioid crisis. This session examines the patient’s understanding, attitudes, and experiences with opioids including concepts of misuse and abuse, communication of risk, and the benefit- risk perspective. Panelists will also discuss strategies to minimize risk as well as challenges with patient engagement in this setting. Measuring Safety and Success: The Consumer Perspective Nicole Kelly, American Chronic Pain Association Exploring Patient Knowledge, Attitudes, and Behaviors to Aid Development of the Information They Need and Want About Opioid Analgesics Paula Rausch, PhD, Director, Division of Health Communications, Office of Communications, CDER, FDA Exploring Patient Knowledge, Attitudes, and Behaviors to Aid Development of the Information They Need and Want About Opioid Analgesics Karin Coyne, MPH, PhD, Vice President, Research, Evidera Opioid Therapy in Patients with Chronic Pain: Present and Future of Risk Assessment and Mitigation Martin Cheatle, PhD, Associate Professor of Psychology in Psychiatry, Perelman School of Medicine, University of Pennsylvania

12:15-1:30PM Luncheon, Exhibits, and Round Table Discussions

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 6 1:30-2:15PM Session 3: Rare Disease I Session Chair Mariette Boerstoel-Streefland, MD, MBA, MS, Senior Vice President, Global Drug Safety Alexion There is considerable unmet medical need for rare disease treatment options. Clinical development and regulatory hurdles are difficult to navigate. The mandate to assess the benefit-risk balance in order to make a decision about any new product before market approval is challenging when data to assess the safety of a new product is very limited. The traditional benefit-risk assessment does not necessarily apply, in case of fatal, or severely debilitating conditions with no alternative treatment options. This session explores the regulatory, ethical, and patient perspective aspects of benefit-risk assessment and managing remaining uncertainty about safety for orphan drugs. Larry Bauer, Consultant Safety Management and Benefit-Risk Assessment for Rare Disease William Haddock, Head of Safety, Ovid Rx

2:15-3:00PM Session 4: Rare Disease II: The Patient Story Session Chair Mariette Boerstoel-Streefland, MD, MBA, MS, Senior Vice President, Global Drug Safety, Alexion In this follow-on session from the previous one we are looking at rare disease from the patient’s perspective. A patient and their caregiver will each share their perspectives on the journey of living with a rare disease, and provide insights in the challenges of getting diagnosed, finding and getting access to the right treatment, accepting uncertainty about benefit and risk, and dealing with potential side effects. This session should allow the audience to switch lenses and step outside the shoes of the safety professional into that of a patient and their caregiver. Donna Kolp, CPht, Social Media Director and Board Member, The Atypical HUS Foundation Bethany Lovegrove, The Atypical HUS Foundation

3:00-3:30PM Refreshments, Exhibits, and Networking Break

3:30-5:00PM Session 5: Pharmacovigilance and Risk Management Challenges in Advanced Therapeutics Session Chair Barbara A. Morollo, Pharmacovigilance and Risk Management Consultant Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes, tissues, or cells. As such, ATMPs can be classified into three main types: • Gene therapy medicines that contain genes that have a therapeutic, prophylactic, or diagnostic effect • Somatic-cell therapy medicines that contain cells or tissues that have been manipulated to change their biological characteristics such that they can be used to cure, diagnose, or prevent diseases • Tissue-engineered medicines that contain cells or tissues that have been modified so they can be used to repair, regenerate, or replace human tissue This session will examine the evolving Pharmacovigilance and Risk Management landscape concerning ATMPS, and will examine the challenges of conducting meaningful product surveillance and developing patient-central risk minimization plans in the setting of advanced therapies. This session will also explore the concept of conducting benefit-risk assessments in the gene therapy setting and will also spend time discussing the potential impact of the EMA’s draft guidance on safety and efficacy follow-up and risk management of Advanced Therapeutics which was released in February 2018. Adamma Mba-Jonas, MD, Chief Medical Officer, CBER, FDA Barbara A. Morollo, Pharmacovigilance and Risk Management Consultant

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 7 Benefit-Risk Management of Recently Authorized ATMPs in the EU – Lessons Learned Jan Petracek, MD, CEO, PrimeVigilance, Czech Republic David Chonzi, MD, MSc, Vice President of Patient Safety and Risk Management, Kite, A Gilead Company

5:00-7:00PM DIA 2019 Program Launch and Networking and Exhibits Reception AI in the Real World: Advancing Capabilities Across Biopharma DIA is hosting a series of content previews around the US counting down to the DIA 2019 Global Annual Meeting, and we’re bringing the next event in the series to Washington, DC! Enjoy a complimentary evening of food, drinks, and networking with a multidisciplinary group of colleagues who share your passion to drive innovation from the lab to patients. Get a special glimpse of the exciting content offerings via a fireside chat on the newest applications of Artificial Intelligence (AI) in medical product development, from business and process improvement to the field of precision medicine. Panelists Mohammad Afshar, PhD, MD, CEO, Ariana Pharma Susant Mallick, MBA, Leader and Evangelist, Healthcare and Life Sciences, EMEA, Amazon Dave Meyers, National Director, US Life Sciences, Microsoft Madhavi Gidh-Jain, PhD, Head, Medical Writing/Clinical Documentation, Sanofi US

DAY TWO | TUESDAY, JANUARY 29

7:30AM-6:00PM Registration

7:30-8:30AM Continental Breakfast and Networking

8:30-8:45AM Welcome Remarks

8:45-10:00AM Session 6: EU Updates Session Chair Michael Richardson, MD, FFPM, International Head, GPV&E and EU Qualified Person for Pharmacovigilance, Bristol-Myers Squibb Requirements and expectations for pharmacovigilance in the EEA continue to evolve with the objective of improving patient safety. This session will explore recent developments related to pharmacovigilance. The focus will be on the evolving situation in the EEA, set in a global context. In addition, this session will include strategic updates on Module VI and E2D (ICH) and data from PSPs; an update on “Brexit” an industry perspective from an EU perspective and a regulator perspective from the MHRA. Additionally an overview of Rev 2 of Module V (RMP) and removal of risks from RMPs. Mick Foy, Head of Pharmacovigilance Strategy, Vigilance Intelligence and Research Group Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom Vicki Edwards, RPh, Vice President, Pharmacovigilance Excellence and QPPV, AbbVie, Inc., United Kingdom Valerie Simmons, FFPM, MD, Global Patient Safety, Eli Lilly and Company Ltd, United Kingdom

10:00-10:30AM Refreshments, Exhibits, and Networking Break

10:30AM-12:00PM Session 7: Generating Real World Evidence (RWE) Fit for Regulatory Decisions: Learning from Safety

Session Co-Chairs Annette Stemhagen, DrPH, FISPE, Senior Vice President, Safety, Epidemiology, Registries, and Risk Management, UBC Robert Reynolds, DrSc, MSc, FISPE, Vice President, Epidemiology, Worldwide Regulatory and Safety, Pfizer, Inc

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 8 Tools and expertise exist in pharmacovigilance to ensure that real world data meet the necessary quality standards for regulatory decisions. Studies are already being designed in accordance with current best practices to reduce bias and produce valid results. This session builds on the longstanding use of RWE in PV to provide lessons, case studies, and guidances relevant to the goal of developing a broader framework for integrating RWE in regulatory decisionmaking. When and How can RWD Analyses Support Regulator Decision-Making Sebastian Schneeweiss, MD, ScD, Chief, Professor of Medicine and Epidemiology; Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical School, Harvard T.H. Chan School of Public Health RWE for Drug Development and Regulatory Approvals Cathy W. Critchlow, PhD, Vice President, Amgen Inc., Center for Observational Research 21st Century Cures and the Evaluation of RWE at FDA David Martin, MD, MPH, Captain, U.S. Public Health Service, Office of Medical Policy, CDER, FDA; Associate Director for Real World Evidence Analytics Panelists Cathy W. Critchlow, PhD, Vice President, Amgen Inc., Center for Observational Research Andrew Robertson, PhD, JD, AVP, Head of Regulatory Science and Policy, NA, Sanofi US

12:00-1:30PM Luncheon, Exhibits, and Round Table Discussions 1:30-3:00PM Session 8: Abstract Session Session Chair Lisa Melanie Harinstein, PharmD, BCPS, Safety Evaluator, Division of Pharmacovigilance-I, Office of Surveillance and Epidemiology, CDER, FDA For the first time at DIA’s Pharmacovigilance and Risk Management Strategies Conference, this new session will feature four presentations selected from an abstract submission process. This session will explore the innovative use of pharmacoepidemiology studies, real world evidence, social media, and signal management tools in pharmacovigilance. The European IV Iron PASS Consortium: Experiences from Coordinating a Multi-Company, Multi-Registry PASS Michael Forstner, PhD, Senior Vice President - Head of Risk Management and Pharmacoepidemiology, PrimeVigilance Innovative Pharmacovigilance (PV) Solution in Social Media: Compliance, Signaling, and Social Media Footprint Jennifer Hannan, MS, Director, Pharmacovigilance Business Liaison, Merck & Co., Inc. Evaluation of Different Signal Management Tools: New Regulatory Perspectives Sanjeev Miglani, MD, Founder and Director, AWINSA Life Sciences The Value of RWD in Safety Signal Refinement Irene Cosmatos, MS, Senior Research Scientist, UBC

3:00-3:30PM Refreshments, Exhibits, and Networking Break 3:30-4:30PM Session 9: Reference Safety Information Use in Clinical Trials Session Co-Chairs Michael Richardson, MD, FFPM, International Head, GPV&E and EU Qualified Person for Pharmacovigilance, Bristol-Myers Squibb Stephen Knowles, MB.BS, MRCP, President, Drug Safety and Pharmacovigilance, Halozyme Therapeutics The regulatory expectations in Europe on the use of the RSI to assess expectedness in clinical trial cases has led to challenges for companies to implement the guidances and has led to critical and major findings in EMA regulatory inspections. The updated Clinical Trial Facilitation Group Q&A will be reviewed along with publicly available inspection findings. This will be followed by a review of the challenges for PV departments and examples of good practice. Krisztina L. Debreczeni, MD, Group Medical Director, Medical Safety Review, Global Pharmacovigilance and Epidemiology, Bristol-Myers Squibb Co. Research and Development Andrea Best, MD, MPH, Executive Medical Director, AbbVie, Inc.

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 9 DAY THREE | WEDNESDAY, JANUARY 30

7:15AM-3:00PM Registration

7:15-8:15AM Continental Breakfast and Networking

8:15-8:30AM Welcome Remarks

8:30-10:00AM Session 10: Emerging Regions Issues Session Chair E. Stewart Geary, MD, Senior Vice President, Chief Medical Officer, Director, Corporate Medical Affairs HQ, Eisai Co., Ltd., Japan As China has joined the ICH community it has leaped ahead in published requirements for electronic reporting (E2B(R3)) during clinical trials and requests for Risk Management Plans sometimes without detailed guidance or the infrastructure for clinical trial sponsors and marketing authorization holders to be confident in their path to compliance. The current, rapidly evolving situation in China for these requirements will be presented along with suggestions on how industry can meet the expectations of the Chinese regulatory authorities. Since the adoption of the Pharmacovigilance System Master File in the EU, a number of non-EU countries have indicated interest in adopting formal or informal requirements for a local PSMF. A presentation will be given on how a coordinated approach to generating and maintaining a PSMF for regulators outside the EU. Dionne Usher, Office of the EU QPPV, Merck Sharp & Dohme Limited, United Kingdom Han Ma, PhD, Asia Pacific Site Head, Roche, China Naomi Morris, MS, Head, Global Compliance Office, Global QPPV Office, Pfizer, Italia

10:00-10:30AM Refreshments, Exhibits, and Networking Break

10:30-11:30AM Session 11: Immuno-Oncology – The Benefits and the Safety Challenges Session Co-Chairs Stephen Knowles, MB.BS, MRCP, President, Drug Safety and Pharmacovigilance, Halozyme Therapeutics William W. Gregory, PhD, Senior Director, Worldwide Safety and Regulatory, Pfizer Inc The introduction of immuno-oncology (IO) agents (e.g., checkpoint inhibitors) into the clinic has led to huge improvements in the treatment of solid tumors. With these benefits come significant concerns related to immune-mediated organ toxicity which can be severe or fatal and requiring prompt recognition and treatment. An overview of the underlying biology of checkpoint inhibitors will be presented, along with how this translates into safety concerns and how these risks are monitored, assessed, and managed from an industry perspective. This will be followed by a discussion of the challenges facing regulatory agencies in assessing the benefit-risk balance of these agents, how this is evolving as the experience with these agents has increased in the post-approval setting, broader prescriber base and different tumor types, and how this translates into ensuring a positive benefit-risk through continued safety surveillance and risk mitigation measures. Oswaldo L. Bracco, Associate Vice President, Oncology Early Phase and External Collaborations – Clinical Safety and Risk Management, Merck Margaret Thompson, MD, Medical Officer, CDER, FDA Ashley Ward, MD, Medical Officer, CDER, FDA

11:30AM-1:00PM Luncheon, Exhibits, and Networking Break

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 10 1:00-2:30PM Session 12: Drug-Induced Liver Injury: What is New, the Impact of Immuno-Oncology Products on the Liver and Perspectives on Causality Assessments

Session Co-Chairs Stephen Knowles, MB.BS, MRCP, President, Drug Safety and Pharmacovigilance, Halozyme Therapeutics William W. Gregory, PhD, Senior Director, Worldwide Safety and Regulatory, Pfizer Inc It is critical to detect the hepatotoxic potential of drugs and to identify susceptible individuals at risk for both predictive and idiosyncratic liver toxicities. This is particularly important as immuno-oncology (IO) agents, e.g., checkpoint inhibitors, known to cause immune-mediated hepatitis, are used in increasingly broad populations as first-line treatment of malignancies and there is increasing understanding of drug-induced liver injury (DILI) in the setting of chronic liver disease. Recent advances in knowledge of immune-mediated hepatotoxicity secondary to IO agents and DILI application to classical will be reviewed. This will be followed by a discussion on DILI in patients with underlying liver disease, focusing on NASH. A panel discussion will conclude the session addressing points to consider in the pragmatic causality assessment of liver injury in the setting of exposure to IO agents. David Paar, MD, Associate Medical Director, Medical Safety Assessment, Bristol-Myers Squibb Arie Regev, MD, Head, Safety Advisory Hub, Chair, Liver and GI Safety Committee, Global Patient Safety, Eli Lilly and Company Mark I. Avigan, MD, CM, Associate Director, Office of Surveillance and Epidemiology, OPE, CDER, FDA

2:30-3:00PM Refreshments and Networking Break

3:00-4:15PM Session 13: Aspects of Benefit-Risk Assessments Session Chair Stella C. F. Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP, Vice President, Global Head of Risk Management, IQVIA, United Kingdom This session will look at three different aspects of benefit-risk assessment: benefit-risk for regulatory decisionmaking; a practical example of how to do benefit-risk estimations for a well-established medicine; and finally on patient preferences and how to communicate risk to patients. Deborah Ashby, OBE, MedSci, Director of the School of Public Health, Imperial College London, United Kingdom Rosa Picirillo, MD, Senior Director and Global Head, Medical Safety, Core Safety Services, IQVIA Bennett Levitan, MD, PhD, Senior Director, Benefit-Risk Assessment, Department of Epidemiology, Janssen Research & Development, LLC.

4:15-5:00PM Session 14: Hot Topics Session Chair: Mick Foy, Group Manager, Vigilance Intelligence and Research Group, Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom Raj Long MBPs, MSc, Deputy Director - Integrated Development, Global Health, Bill & Melinda Gates Foundation, United Kingdom

5:00-5:15PM Closing Remarks

DIAglobal.org Follow us @DrugInfoAssn #PVRMS19 for real-time updates 11