31st International Conference on Pharmacoepidemiology
& Therapeutic Risk Management
Hynes Convention Center
August 22-26, 2015


(As of August 12, 2015)

Final Program - At a Glance
Final Program - Pages 1-60
Final Program - Pages 61-End
Final Program - Session Tracks


August 21, 2015

4:00pm-6:00pm Registration
  • We recommend individuals who are attending courses on Saturday morning register on Friday night in order to avoid long lines on Saturday morning, or arrive by 7:30am on Saturday morning to ensure you register in time for your course.

August 22, 2015
Hynes Convention Center
Boston, MA USA

7:30-6:00pm Registration
  • We recommend individuals who are attending courses on Saturday morning register on Friday night in order to avoid long lines on Saturday morning, or arrive by 7:30am on Saturday morning to ensure you register in time for your course.
7:30-5:00pm Speaker Ready Room
9:00-5:00pm ISPE Board of Directors Meeting
Commonwealth Ballroom, Sheraton Boston Hotel
(Open to ISPE members; notify ISPE staff if you would like to attend.)
(*Registration required)
8:00-6:00pm Pharmacogenetics for Pharmacoepidemiologists
Includes Basic and Advanced Sessions & Lunch
  • Course Description

    Brief overview of course:
    This highly interactive course is intended to involve participants in:

    • A dynamic didactic experience of the foundational principles of pharmacogenomics
    • An appreciation for the different methods used in pharmacogenomics research.
    • Examination of real world examples of pharmacogenomic epidemiology.
    • Application of methodology to designing pharmacogenomic epidemiology studies.
    • Interpretation of the data found in the pharmacogenomic epidemiology literature, and
    • Understanding wider implications of the results.

    Educational Objectives:

    • To understand the genetic and pharmacogenomic terminology and how cellular and molecular biology informs pharmacogenomic studies at the level of analysis.
    • To better understand the study design and methodological approaches, their strengths and weaknesses used in pharmacogenomic epidemiology.
    • To understand the latest statistical approaches used in pharmacogenomics studies.
    • To understand the role that pharmacogenomics can play in pharmacovigilance.
    • To understand how pharmacogenomics relates to the current focus of Personalized Medicine
    • To have an overview of clinical utility of pharmacogenomics and associated regulatory issues as well as policy-relevant research, including policy relevant to Big Data


    8:00-8:10 am Welcome and Introduction to the Course [Amalia Issa]
    8:15-8:45 am The Convergence of Pharmacoepidemiology and Pharmacogenomics [Gillian Bartlett]
    8:45-9:15am Genetic Research in Drug Discovery & Development [David Pulford]
    9:15-9:45 am The Lore of Databases, the Agony of Proper Outcomes Collection [Bruce Carleton]

    BASIC SESSION Moderator: Anke-Hilse Maitland van der Zee
    10:00-10:30 am Genetics for Pharmacoepidemiologists [Anke-Hilse Maitland van der Zee]
    10:30-11:00 am "Pharmacogenomics Translation: From Discovery to Confirmation to Clinical Utility" [Andy Freedman & Kelly Filipski]

    ADVANCED SESSION Moderator: Amalia M. Issa
    10:00-10:30 am "Omics Research and the Pharmacoepidemiologist: What you need to know." [Geoff Liu]
    10:30-11:00 am Pharmacogenomic Research: From Biospecimens and Data to Improvements in Patient Care [Bruce Carleton]

    11:15--11:45pm Leveraging Real World Data to Impact Drug Discovery and Development!!!!![Margaret Ehm]
    12:00-1:30 pm Lunch & Debate Presentation (Moderator: Anke-Hilse Maitland Van der Zee): Big Data, Big Promises and Big Policy Challenges [Amalia Issa & Gillian Bartlett]
    1:30 -2:00pm Clinical Utility and Clinical Adoption: A Real World Example 2:00-4:30pm Q&A Roundtables and Meet and Greet

8:30-12:30pm MORNING COURSES
  • Introduction to Pharmacoepidemiology

    Brief overview of course:
    This half-day course will provide participants with a short introduction to the basic principles and concepts of pharmacoepidemiology. The course includes lectures on cohort studies, case-control studies, and bias & confounding.

    Educational Objectives:

    • Explain common study designs used in pharmacoepidemiology and appreciate their strengths and weaknesses.
    • Describe the key steps in the design and analysis of cohort and case-control studies.
    • Understand the major types of biases that threaten the validity of pharmacoepidemiologic research (confounding, selection bias, measurement bias).

    Target Audience:

    • Attendees who are new to pharmacoepidemiology or those who want a succinct refresher of the field's core concepts.

    Course Faculty/Presentations:

    Almut Winterstein, FISPE, University of Florida College of Pharmacy,
    Cohort Studies

    Jennifer Lund, Gillings School of Global Public Health, University of North Carolina at Chapel Hill,
    Case-Control Studies

    Tobias Gerhard, FISPE, Ernest Mario School of Pharmacy, Rutgers University,
    Bias & Confounding

  • Practical Skills in Protocol Writing and Computer Programming Relevant to Pharmacoepidemiology

    Brief overview of course:
    Pharmacoepidemiologic studies that involve the use of electronic healthcare data have complex and unique characteristics that must be taken into consideration when executing the studies. This highly interactive course will engage participants in the following topics:

    1. Pre-specifying study components in a protocol, including design, analysis, conduct, and reporting of the study, along with a science-based rationale for the choices pertaining to these components.
    2. Translating elements of the study protocol into SAS programming 

    Educational Objectives:

    • To introduce a framework for translating study questions into a clear and detailed protocol supported by a rational thought process that is tailored to available data for pharmacoepidemiologic studies using electronic healthcare data.
    • To execute the protocols of pharmacoepidemiologic studies into SAS programming.
    • To develop the skills to document and report pharmacoepidemiologic studies using electronic healthcare data in a transparent and reproducible way.

    Target Audience:

    • People who are interested in conducting pharmacoepidemiologic studies using electronic healthcare data, in learning about protocol writing and/or implementing the protocol components into SAS programming.
    • An understanding of basic SAS programming language is required for this course.

    Course Faculty/Presentations:

    • Chih-Ying (Natasha) Chen, Epidemiologist, U. S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of Pharmacovigilance and Epidemiology, Office of Surveillance and Epidemiology, Division of Epidemiology II
      Presentation: Comparator Selection, Power and Feasibility of the Study, Analytical Plans and Handling of Confounders and Effect Modifications
    • Christian Hampp, Epidemiologist, U. S. Food and Drug Administration, Center for Drug Evaluation and Research, Office of Pharmacovigilance and Epidemiology, Office of Surveillance and Epidemiology, Division of Epidemiology II
      Presentation: Study Design, Data Sources Selection, Inclusion/Exclusion Criteria of Study Population
    • Jessica Jalbert, Laser Analytica, Weill Cornell Medical College,
      Presentation: Exposure Definition and Ascertainment, Outcome Definition, Ascertainment and Validity
    • Nicole Pratt, Senior Research Fellow, Quality Use of Medicines and Pharmacy Research Centre, Sansom Institute, School of Pharmacy and Medical Sciences, University of South Australia
      Presentation: From Protocol to SAS Programming
    • Soko Setoguchi, FISPE, Duke Clinical Research Institute
  • Pediatric Pharmacoepidemiology

    Brief Overview of Course:
    The increasing use of medications by children and the history of excluding children from clinical trials have created the need for pediatric pharmacoepidemiology, a sub-specialty within pharmacoepidemiology. Unique challenges in studying children, accessing data, defining outcomes, and designing studies require specialized methodologic skills and operational approaches. This half-day course will introduce participants who have a good understanding of pharmacoepidemiology to the specialized methodologic and operational approaches used to study medications in children.

    Educational Objectives:
    The course will be taught in an interactive format with ample opportunities for questions and discussion. Regulatory issues around use of medications in children will be highlighted throughout the course. Participants will gain an understanding of key issues in pediatric pharmacoepidemiology including:

    • An overview of pediatric pharmacoepidemiology and how it differs from pharmacoepidemiology in older age groups
    • Databases available for research in pediatric pharmacoepidemiology
    • Research designs currently used to study pediatric pharmacoepidemiology
    • Definition and measurement of outcomes in pediatric pharmacoepidemiology
    • Two to three case studies highlighting the variety of critical challenges in pediatric pharmacoepidemiology

    Target Audience:
    ICPE attendees interested in gaining a basic understanding of the need for and approaches to pediatric pharmacoepidemiology.

    Course Faculty/Presentations:
    Tamar Lasky, President and Owner, MIE Resources
    Overview, Defining age sub-groups

    Ann W. McMahon, Supervisory Medical Officer US Food and Drug Administration
    Databases available for pediatric pharmacoepidemiology

    Rachel E. Sobel, Senior Director, Epidemiology, Worldwide Research and Development  / Pfizer Inc.
    Study designs and case study: Challenges in the design and execution of a prospective registry to monitor the safety of celecoxib in Juvenile Idiopathic Arthritis

    Michael Goodman, System Area Lead Global Patient Safety Informatics   Astra Zeneca
    Defining, measuring and analyzing pediatric outcomes

    Susan dosReis,  Associate Professor, University of Maryland School of Pharmacy
    Department of Pharmaceutical Health Services Research
    Case Study: Early initiation of psychotropic medication

    Suggested Reading:
    Improving Medicines for Children in Canada
    The Expert Panel on Therapeutic Products for Infants, Children and Youth
    Council of Canadian Academies, 0ttawa, Canada 2014

12:30-2:00pm LUNCH ON YOUR OWN
1:30-3:30pm Local Host Committee presents a Boston Duck Tour. Click here for more information.
(*Registration required)
  • Comparative Effectiveness Research: Challenges When Studying Orphan and Targeted Therapies

    Brief Overview of the Course:
    Comparative effectiveness research (CER) is increasingly important in situations where randomized trials conducted for regulatory purposesdo not fully inform decisions that other policy makers need to make. This year's course will start with an overview of methodological aspects related to CER and then will narrow the focus to study settings in which few patients are exposed either because the condition is rare, the treatment indication is highly targeted, or the medication is new to the market.

    Educational objectives
    Upon completing this course, participants will be able to:

    • Recognize the need for comparative effectiveness evidence for niche medications, such as orphan drugs and other highly targeted therapies
    • Understand biases that commonly occur in comparative effectiveness evaluations and strategies for overcoming these biases
    • Describe particular challenges to assessing the comparative effectiveness of niche medications and the strategies best suited for addressing them

    Target audience

    • This course is designed for pharmacoepidemiologists conducting comparative effectiveness research and others interested in gaining an appreciation for the challenges to conducting comparative effectiveness studies for niche medications
    • This beginner-to-intermediate-level course assumes knowledge of basic pharmacoepidemiologic methods

    Course faculty/presentations:

    Frequently observed biases and investigator errors that are avoidable in CER
    Sebastian Schneeweiss, FISPE
    Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, USA

    Dr. Schneeweiss will provide insights into typical pharmacoepidemiological strategies to reduce the risk of bias in studies using secondary databases and registries and how to make the conclusions of CER studies more meaningful for decisions makers.

    Evaluating the effectiveness of orphan drugs
    Joshua Gagne
    Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, USA

    Medications that treat rare conditions are challenging to evaluate before and after market authorization. Dr. Gagne will review current strategies to evaluate the comparative effectiveness and safety of orphan medications and provide advice on how to plan such studies.

    Evidence development for highly targeted medications
    Gregory Daniel
    Center for Health Policy, Brookings Institution, Washington, DC, USA

    As medications are increasingly targeted based on biological markers including gene expression patterns, fewer and fewer patients will be exposed to specific medications. Dr. Daniel will review the challenges of conducting CER in such settings and provide case studies of practical experiences. He will summarize the current state of the art and provide recommendations for planning CER studies in this space.

  • Medical Device Epidemiology

    Brief Overview of the Course:
    This course will comprise a 4-hour session that will incorporate didactic lectures and interactive case studies that highlight novel medical devices and encourage rich discussion. This course provides a foundation for medical device epidemiology and its application to the real world.

    Educational Objectives:

    • Explore methodologic considerations of research involving medical devices and learn about medical device safety and effectiveness
    • Examine the dynamic regulatory environment for medical devices and how it differs from drugs/medications
    • Learn about real-world medical devices in an interactive forum

    Target Audience:

    • ISPE (academia, government, industry) membership
    • ISPE student membership

    Course Faculty/Presentations:

    Danica Marinac-Dabic, Director, Division of Epidemiology, CDRH, FDA

    Art Sedrakyan, Director of Comparative Effectiveness, Hospital for Special Surgery, Weill Cornell Medical College
    Introduction to Medical Device safety and effectiveness, and brief description of international initiatives for improving the safety of medical devices

    Jessica Jalbert, Director of Epidemiology, LAASER
    Methodological considerations for conducting device epidemiological research

    Bill Horton, VP of New Technologies and Surgical Affairs, DePuySynthes Spine, Johnson & Johnson

    Colin Anderson-Smits, Branch Chief, Division of Epidemiology, CDRH, FDA
    Overview of Class III Degenerative Disc Devices and Key Challenges

    Veronica Sansing, Lead Epidemiologist, Division of Epidemiology, CDER, FDA

    Mary E. Ritchey, Senior Epidemiologist, Procter & Gamble
    Comparison of drugs/medicines and devices - global regulations and overall value proposition (including benefit-risk)

    Jonathan Schelfhout, Associate Director, Outcomes Research, Merck
    Devices Supporting Surgery and Anesthesia, Overview of Considerations in Monitoring the Level of Neuromuscular Blockade (NMB) and Key Challenges

    Myoung Kim, Johnson & Johnson

  • Database Utilization Workshop: Using Pharmacoepidemiology Databases in Drug Safety Research

    Brief Overview of Course:
    This highly interactive workshop will involve participants in the following topics:

    • Examine the use of database resources in pharmacoepidemiology research
    • Define the different types of data resources available for pharmacoepidemiology and the selection criteria to select the most appropriate resource, workshop will strengthen the concepts discussed through participant interaction
    • Review the details of data available from the Centers for Medicaid and Medicare Services (CMS)
    • Assess the quality of electronic medical record data
    • Examine characteristics of available resources for data linkage between pharmacoepidemiology resources

    Educational objectives:

    • To review the Guidelines for Good Database Selection and use in Pharmacoepidemiology Research.
    • To gain an understanding of available pharmacoepidemiology resources with a focus on US data resources
    • To develop the skills to select an appropriate resource to conduct pharmacoepidemiology research.
    • To examine tools for assessing the quality of electronic medical record data
    • To provide an overview of data linkage of available pharmacoepidemiology resources

    Target audience:

    • Intermediate level of epidemiology background preferred
    • New members of the Database SIG
    • Those interested in exploring pharmacoepidemiology resources
    • Those working in harmonizing data resources
    • Those working in creating analytic datasets
    • Clinical pharmacoepidemiologists
    • Clinical guideline developers
    • Health practitioners utilizing research from observational data

    Course Faculty/Presentations:

    1. Gillian Hall
      Overview of Database use in Pharmacoepdiemiology {45 minutes}

    2. Beth L. Nordstrom, Senior Research Scientist and Executive Director, Epidemiology, Evidera
      Selection of Databases for Pharmacoepidemiology Research {45 minutes}

    3. Interactive coffee break
      Database Selection Workshop Activity {15 minutes}

    4. Charles Leonard, Senior Research Investigator, Center for Pharmacoepidemiology Research and Training, Perelman School of Medicine, University of Pennsylvania
      Available Data Resources from the Centers for Medicaid and Medicare Services {45 minutes}

    5. Arlene Gallagher, Senior Researcher, and Rachael Williams, Senior Researcher, Clinical Practice Research Datalink
      Data linkage of Pharmacoepidemiology Resources {60 minutes}
      • Method of linkage
      • Representativeness of the population covered
      • Impact on sample size / study period (and getting this right)
      • Agreement - are datasets measuring the same thing?
      • Suitability (would you study hospital procedures in a primary care database?)
      • Practicalities - reviews, costs, time impact, governance

    6. Group Discussion {30 minutes}
      • Build on audience experience with pharmacoepidemiology database resources
      • Address topics raised in the education session to facilitate interaction with ISPE membership
      • Highlight database related tracks within the ICPE meeting

  • Pharmacoepi Without Large Databases: Study Design and Data Collection

    Brief overview of the course:

    • Basic theory and practical tips in conducting pharmacoepidemiologic studies when information is required that is not captured in databases or when large databases are not available,
    • Common study design used to collect data through observation, medical records or questionnaires
    • Strengths and limitations of working without large databases
    • Case studies illustrating the use of different study designs to answer relevant questions about safety and utilization of medicines in primary and secondary care settings

    Educational objectives:

    • To develop the skills to develop pharmacoepidemiologic studies without large databases
    • To discuss strengths and limitations of different study designs and data collection methods
    • To identify strategies to improve quality and reliability of pharmacoepidemiological studies conducted without large databases

    Target audience:

    • Professionals working with medicines regulatory authorities, health insurances or are involved in quality of care assurance programs, health care professionals interested in pharmacoepidemiology
    • Faculty and Postgraduate students of Medical, Dental, Pharmacy institutions and Pharm D students interested in the area of Pharmacoepidemiology
    • Aspirants and newer recruits in Pharmacosurveillanceand DUR programs

    Course Faculty/Presentations:

    Annie Fourrier-Reglat, Pharmacology - Inserm Unit 657, University Bordeaux, France

    Carol Louik, Slone Epidemiology Center, Boston University, Boston, USA

    Lisa Pont, FISPE, Senior Lecturer, Pharmacology and Clinical Pharmacy, The University of Sydney, Australia

    Supporting faculty:

    Kiyoshi Kubota, FISPE, President, NPO Drug Safety Research Unit Japan

    Veronika Wirtz, Associate Professor, Department of Global Health, School of Public Health, Boston University

  • Establishing a Successful Early Stage Career in Pharmacoepidemiology

    Brief overview of course: This ICPE Pre-Conference Course is aimed at early stage pharmacoepidemiologists who have been in the field for less than 3 years.  Participation will lead to enhancement in skills (i.e., development of mentor-mentee relationships, research presentation, and manuscript writing) that are crucial to early success in pharmacoepidemiology. We will also discuss important aspects of early stage professional development in careers in government/regulatory science, academia, and industry. This course is jointly sponsored by the ISPE Academic Council, ISPE Student Council and ISPE Membership Committee.

    Educational Objectives:

    • Establish more effective relationships with mentors for research and career development
    • Prepare and present effective research poster presentations
    • Improve delivery of oral research presentations
    • Improve writing skills for research manuscripts
    • Develop productive strategies for success in the early stages of careers in pharmacoepidemiology within government/regulatory science, academia, or industry

    Target Audience: Early stage pharmacoepidemiologists (i.e., undergraduate/graduate students, postdoctoral fellows, and early career pharmacoepidemiologists) who have been conducting active research in the field of pharmacoepidemiology for less than 3 years

    Course Faculty/Presentations:

    Vincent Lo Re III,  FISPE
    Assistant Professor of Medicine and Epidemiology, Center for Pharmacoepidemiology Research & Training, University of Pennsylvania
    Establishing productive mentor-mentee relationships

    Caitlin Knox
    Manager, Epidemiology, Value & Outcomes Advisory, Quintiles
    Creating an effective scientific poster

    Tarek Hammad, FISPE
    Executive Director, Epidemiology, Merck Research Laboratories
    Preparing an effective oral research presentation

    Sengwee Darren Toh, FISPE
    Assistant Professor of Population Medicine, Harvard Medical School & Harvard Pilgrim Health Care Institute
    Tips for successful manuscript writing

    Stella Blackburn, FISPE
    Vice President & Global Head of Risk Management, Real-World, and Late Phase Research, Quintiles
    How to get the most out of the early stages of a career in pharmacoepidemiology working within government/regulatory science

    Til Stürmer, FISPE
    Professor of Epidemiology, Gillings School of Global Public Health, University of North Carolina
    How to get the most out of the early stages of a career in pharmacoepidemiology working within academia

    Quazi Ataher
    Senior Director, GEP Team Lead, Epidemiology, Pfizer
    How to get the most out of the early stages of a career in pharmacoepidemiology working within industry


Student/Young Professionals Meet & Greet

- Open To All Students & Young Professionals

- Organized by the ISPE Student Council

- Meet in the Sheraton Boston Hotel Lobby at 6:15pm

August 23, 2015
Hynes Convention Center
Boston, MA USA

6:00-7:00am Morning Group Run/Walk with ISPE President
Join your ISPE colleagues, including ISPE's President, at the Sheraton Boston Hotel Lobby and enjoy a run, or walk, around Boston. Enjoy the weather and get energized before starting the day's programming. Running/walking routes will be available. NOTE: This is not an ISPE sponsored event. Any run that you participate in you do so at your own risk. ISPE takes no responsibility for personal injuries or new or existing medical conditions that may be aggravated by running.
7:30am-6:00pm Registration
  • We recommend individuals who are attending courses on Sunday morning register on Friday in order to avoid long lines on Sunday morning, or arrive by 7:30am on Sunday morning to ensure you register in time for your course.
7:30am-5:00pm Speakers' Ready Room
(*Registration required)
8:30am-12:30pm MORNING COURSES
  • Intermediate Pharmacoepidemiology - Unmeasured Covariates

    Covariates that may confound or modify a result are often poorly measured or not measured at all. The result is pharmacoepidemiologic studies that seem to contradict one another. Most critiques of observational research rest on unmeasured covariates. This course will classify the options for dealing with unmeasured covariates in study design and analysis, and present examples of self-controlled case series and matched designs, propensity-balancing and proxy-variable designs, and instrumental variable analyses.

    Educational Objectives:

    • Recognize when missing covariate data threatens the inferences from an observational study.
    • Know study designs and analytic strategies that reduce distortions due to missing covariates.
    • Develop an intellectual framework for understanding novel problems that involving missing covariates.

    Course Faculty/Presentations:
    Alexander M Walker, FISPE, World Health Information Science Consultants

    • Confounding by Indication as a Motivating Example of Missing Covariates
    • Self-Controlled Case Series and Matching
    • Propensity Scores as a Technique for Controlling for Confounder Through Proxy Measures
    • Instrumental Variables
  • Introduction to Drug Utilization Research

    Brief overview of course:
    This educational session provides an overview of drug utilization research and presents essential methods used. The session includes interaction with participants, question and answer sessions, and discussion during and at the end of each presentation. At the end of the session, the participant will be exposed to:

    • Description of the theoretical framework and practical applications of different methods illustrated using selected examples
    • Classification systems used in drug utilization monitoring and research
    • Limitations of data sources and methods
    • Interpretation of aggregate and individual-based data, variation and change
    • Importance of drug utilization research for public health and implications for policy decisions

    Educational Objectives:

    • To provide an overview of Drug utilization Research within the context of Pharmacoepidemiology, Health Services Research and Public Health
    • To describe fundamental principles for classifying and quantifying drug use and to provide an understanding of the methodological challenges
    • To provide the basic knowledge and understanding of epidemiological measures of drug use and studies based on individual patient data
    • To describe methodological and policy issues in Drug utilization Research in the United States

    Level of course: Intermediate

    Target Audience

    • New members of the DUR/HSR SIG and graduate students new to DUR/HSR
    • Those interested in drug utilization research who need an overview of the area
    • Those interested in monitoring of medicine use and quality development
    • Those working in health care organizations involved in payment and reimbursement of medicines, in medicines policy areas, or planning of health services

    Course Instructors/Presentations:

    Lisa Pont, Senior Research Fellow, Australian Institute of Health Innovation, Macquarie University, Australia
    Methodological Framework and Skills Needed in Drug utilization Research

    Solveig Sakshaug, Senior Adviser, WHO Collaborating Centre for Drug Statistics Methodology, Norwegian Institute of Public Health, Oslo, Norway
    The Anatomical Therapeutic Chemical Classification and the Defined Daily Dose Methodology: Classifying and Quantifying Drug Use.

    Bjorn Wettermark, Associate Professor, Centre for Pharmacoepidemiology, Karolinska Institute, Stockholm, Sweden
    Drug utilization Research and Monitoring in Individual Based Registers - Study Designs and Epidemiological Measures of Drug Use.

    Andrew Gilbert, Emeritus Professor, University of South Australia, Adelaide, Australia
    Bridging the evidence-practice gap using theoretical models of behavioural and organisational change

  • Propensity Scores in Pharmacoepidemiology

    Brief overview of course:
    Issues of bias and confounding relate to study design and analysis in the setting of non-random treatment assignment where compared subjects might differ substantially with respect to comorbidities. Failing to address a lack of balance in the covariates between treated and comparison groups can produce confounded estimates of treatment effect. Faculty will explain how propensity scores can be used to mitigate confounding through standard observational approaches (restriction, stratification, matching, regression, or weighting). The advantages and disadvantages of standard adjustment relative to propensity score-based methods will be discussed. Details of propensity score methodology (variable selection, use, and diagnostics) will also be discussed.

    Educational Objectives:

    • Discuss how propensity scores are useful for observational research;
    • Recognize research conditions where propensity scores offer advantages; and
    • Explain how propensity scores may be applied in research (restriction, stratification, matching, modeling, and weighting), and the effect of each application on inference.

    Target Audience:

    • Those who are planning to use propensity scores
    • Those who have used propensity scores
    • Those who would like to learn more about propensity scores

    Course Instructors/Presentations:

    John Seeger, FISPE, Assistant Professor, Brigham & Women's Hospital
    Presenter and discussant

    Jeremy Rassen, Chief Scientific Officer, Aetion
    Presenter and discussant

  • Pharmacovigilance & Signal Detection

    This half-day course will introduce participants to the principles of surveillance in pharmacovigilance with a focus on quantitative aspects. The course will cover classical pharmacovigilance signal detection using spontaneous adverse event reports, quantitative signal detection methods for electronic medical record and claims data, and international initiatives, including the EU-ADR project, and the US FDA’s Mini-Sentinel program, aimed at using routinely collected electronic healthcare data to conduct signal detection and prospective medical product safety monitoring. The course will involve lectures with ample opportunities for questions and discussion.

    Educational Objectives:
    Upon completing this course, participants will be able to:

    • Explain the need for pharmacovigilance and its component activities with a focus on safety surveillance.
    • Describe the potential data sources (e.g., spontaneous reports, claims, electronic medical records, etc.) for   safety surveillance in pharmacovigilance.
    • Understand the array of basic analytic methods for pharmacovigilance in spontaneous adverse events reports and signal detection and active monitoring in electronic healthcare databases.
    • Recognize the limitations of each data source and approach and how they complement each other.

    Course Faculty/Presentations
    Andrew Bate, Pfizer Ltd, UK, Safety surveillance across multiple data streams: current status and future horizons
    Gianluca Trifirò, Erasmus University Medical Center & University of Messina, Italy, Electronic Medical Records and Claims Data for Drug Safety Signal Detection: Lessons Learned from the EU-ADR Project
    Joshua Gagne, Department of Medicine, Brigham and Women’s Hospital & Harvard Medical School, Semi-Automated, Distributed, Prospective Medical Product Safety Monitoring

  • Regulatory Pharmacoepidemiology

    Educational Objectives:

    • Understand the intent and structure of regulation to protect the Public Health
    • Appreciate the challenges of decision making with weak data
    • Know about practical approaches typically chosen by regulators
    • Recognize the challenges epidemiology faces to provide sufficient information for public health decision making
    • Gain an overview of worldwide challenges for drug regulatory agencies and variation in approaches
    • Know about current strategies to strengthen regulation in the face of these challenges

    Course Faculty/Presentations:

    Gerald Dal Pan, Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, Food and Drug Administration
    Challenges in Regulatory Epidemiology

    June Raine, Vigilance and Risk Management of Medicines Division, Medicines and Healthcare Regulatory Products Agency
    Challenges in Regulatory Epidemiology

  • Registries/Prospective Cohort Studies

    Brief overview of course:
    This course will provide an overview of operational considerations for prospective studies and registries, including regulatory, methodological and logistical challenges.Topics will include data protection, ethics, informed consent, safety reporting, enrollment generalizability, dealing with new treatments and issues relating to missing data. Special applications relating to pregnancy registries will be reviewed, as well as current guides to quality for prospective studies. An interactive case study about risk management will be presented.

    Educational Objectives:

    • To understand how registries can be designed and used to support safety, effectiveness and value
    • To be able to explain some special challenges for pregnancy registries and for studying the effects of dynamic therapies
    • To be familiar with the guides available for quality in observational studies

    Target Audience:

    • Those interested in designing and conducting prospective observational cohort studies, including registries/registers, for comparative effectiveness and safety
    • Researchers wishing to learn how prospective studies, including registries, can be used to support post-marketing safety commitments and provide evidence for health technology authorities
    • Anyone interested in gaining an understanding of the methodological and practical considerations for conducting pregnancy registries.

    Course Faculty/Presentations:

    Nancy A. Dreyer, FISPE , Global Chief of Scientific Affairs & Senior Vice President
    Quintiles Real-world & Late Phase Research, Cambridge, MA USA
    Overview of Registries and Operational Considerations, Guides to Quality

    Christina D. Mack, Sr. Director, Epidemiology and Health Outcomes
    Quintiles Real-world & Late Phase Research, Research Triangle Park, NC USA
    Methodological Considerations: Bias and Missing data

    Sonia Hernández-Díaz, FISPE , Professor of Epidemiology&Director Pharmacoepidemiology Program
    Department of Epidemiology, Harvard T.H. Chan School of Public Health, Boston, MA
    Special Application to Pregnancy Registries

    Deborah Layton, Principal Research Fellow, Drug Safety Research Unit
    Honorary Senior Lecturer, Portsmouth University
    Interactive case study on Risk Management

  • Modern Pregnancy Pharmacoepidemiology: General Aspects, Newer Data Sources and Methods for Study Design, Conduct, and Analysis

    Brief overview of course:
    After reviewing some fundamentals of pharmacoepidemiologic research in pregnant women, this half-day course will present various data sources that have become available for perinatal epidemiologic research over the years, along with the implications of using each type of data source for optimal study design, conduct and analysis. In addition, the course will critically evaluate the opportunities that some of the newer epidemiologic methods offer to pregnancy research. The course format will include both formal lectures and an active learning approach. To allow for effective group discussions and hands-on exercises, participants will be asked to read three articles and will be provided some key questions to think about in preparation for the course.

    Educational Objectives:
    Upon completion of the course, participants will:

    • Have gained knowledge and skills necessary to critically evaluate scientific evidence in this area.
    • Be better equipped to select the best data source, study design, and analytic approach(es) from the rich armamentarium available to address their specific perinatal research question.

    Target Audience:

    • Researchers, policy makers, and other healthcare professionals involved in the conduct, evaluation, or interpretation of pharmacoepidemiologic pregnancy studies.
    • Professionals involved in the regulation of drugs in pregnant women.
    • Students interested in pursuing a career in perinatal epidemiology.

    Course Faculty/Presentations:

    Andrea Margulis, RTI Health Solutions, Spain
    Basics of drug safety research in pregnancy, selected aspects

    Helle Kieler, Associate professor, Director CPE (Centre for Pharmacoepidemiology), Karolinska Institutet, Stockholm, Sweden
    European Databases

    Brian Bateman, Brigham and Women's Hospital / Massachusetts General Hospital / Harvard Medical School, USA

    Krista Huybrechts, Brigham and Women's Hospital / Harvard Medical School, USA
    US databases and advanced methods for confounding control and bias quantification

    Pre-course reading materials:

    • Chambers C, Hernández-Díaz S, Van Marter L, et al. Selective Serotonin-Reuptake Inhibitors and Risk of Persistent Pulmonary Hypertension of the Newborn. New England Journal of Medicine 2006;354:579-87

    • Kieler H, Artama M, Engeland A, et al. Selective serotonin reuptake inhibitors during pregnancy and risk of persistent pulmonary hypertension in the newborn: population based cohort study from the five Nordic countries. BMJ 2012;344:d8012.

    • Huybrechts KF, Bateman BT, Palmsten K, Desai RJ, Patorno E, Gopalakrishnan C, Levin R, Mogun H, Hernandez-Diaz S. Antidepressant use late in pregnancy and risk of persistent pulmonary hypertension of the newborn. JAMA. 2015 Jun 2;313(21):2142-51.

    Questions for discussion:

    • What are the strengths and weaknesses of each of the approaches and data source used to address this question? In responding, please consider the following:
      • Which is the source population?
      • How was exposure defined?
      • How was the outcome defined?
      • Which comparisons were made and between whom?
      • Which statistical analyses were performed? Were sensitivity analyses performed?
      • Are the conclusions in accordance with the findings?
    • What factors might account for the divergent findings regarding this risk in these three studies?
    • After examining these articles, what do you believe to be the risk of PPHN associated with SSRI use?
    • If you were a clinician treating pregnant patients with depression, how would you council them regarding this potential risk?
    • Does the available data support a warning on the label for SSRIs regarding this potential risk?

    Supplemental reading (not required):

    • Grigoriadis S, Vonderporten EH, Mamisashvili L, et al. Prenatal exposure to antidepressants and persistent pulmonary hypertension of the newborn: systematic review and meta-analysis. BMJ 2014;348:f6932.

12:30-2:00pm LUNCH ON YOUR OWN
12:30-2:00pm New Member/New Investigator Luncheon (By Invitation)
(*Registration required)
  • Advanced Topics in Pharmacoepidemiology

    Course Instructors/Presentations:

    Timothy L. Lash (Rollins School of Public Health, Emory University): bias analysis to evaluate the influence of genotyping errors in an overview of tamoxifen pharmacogenetics

    Darren Toh (Harvard Medical School/Harvard Pilgrim Health Care Institute): Distributed analytics: privacy-protecting methods for multi-center studies

    Michele Jonsson Funk (Gillings School of Global Public Health, University of North Carolina at Chapel Hill): Assessing treatment effect heterogeneity in observational studies

    Miguel Hernan (Harvard T.H. Chan School of Public Health): Comparative effectiveness and safety of dynamic treatment strategies: the renaissance of the g-formula?

  • Advanced Drug Utilization

    Brief overview of course:
    This interactive educational program includes presentations, question and answer sessions and a final discussion with the faculty panel. The session will involve participants in the:

    • Use of drug utilization information from administrative health data to improve medicines use
    • Development of prescribing quality indicators
    • Application of principles derived from Health Services and Implementation Research to the design of a clinical intervention
    • Methods for evaluating the effect of clinical or policy interventions
    • Interpretation of the evaluation data and dissemination of results

    Educational Objectives:

    • To gain an advanced understanding of planning, implementation and evaluation requirements for an effective quality use of medicines intervention
    • To develop a sophisticated understanding of the importance of project planning processes to the effectiveness of interventions
    • To develop an appreciation of the significance of this work to knowledge transfer at the patient care practice and policy change levels of the health system

    Level of the course: Advanced

    Target Audience:

    • Members of the DUR/HSR SIG
    • Graduate students interested in advanced methodologies
    • Those interested in quality development and implementation research, knowledge transfer, evaluation of interventions, health and medicines policy areas, health funds and third-party payers
    • Clinical pharmacoepidemiologists, health service planners, clinical guidelines developers, health practitioners

    Course Instructors/Presentations:

    Petra Denig, Professor, Faculty of Medical Sciences, University of Groningen, Groningen, Netherlands
    Developing Valid Prescribing Quality Indicators

    Eimir Hurley, Program Director, Alosa Foundation, Inc., Boston, USA
    Evaluating Interventions on Drug utilization: Advanced methods

    Libby Roughead FISPE, Research Professor, Division of Health Sciences, School of Pharmacy and Medical Sciences, University of South Australia, Adelaide, Australia
    Methods for Identifying and Managing Problems with Medicines Use in Practice

    Jerry Avorn FISPE, Professor of Medicine, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital, Harvard Medical School, USA
    Evaluating Practice or Policy

  • Benefit Risk Assessment: Understanding and Using Patient Preference Methodologies

    Benefit-risk assessment and communication is an important, interdisciplinary field within the pharmaceutical and regulatory sciences. In addition to exploring requirements, challenges and opportunities in benefit-risk assessment and communication, this pre-conference tutorial focuses on state of the art methodologies and tools to assess and communicate the benefit-risk profile and related patient preferences for the use of pharmaceutical products.

    Introduction to the topic of patient preferences

    1. What is "patient-centered benefit risk assessment"?
    2. Why use a patient-centered approach?
    3. What does it entail?
    4. Examples of previous use

    Methods for obtaining Patient Preference Data

    1. Quantitative Methods
      1. Conjoint Analysis
      2. Direct elicitation methods
    2. Qualitative Methods
      1. Focus groups
      2. Individual interviews- these methods can stand on their own and can provide a way to develop the questionnaires for the quantitative methods

    What are the best practice standards in regard to using these approaches?

    When would you conduct a patient preference study?

    How to use patient preference data in a filing submission?

    Where's the field going?

    Course Instructors/Presentations:
    Brett Hauber, RTI Health Solutions
    Alicia Gilsenan, RTI Health Solutions
    Juhaeri Juhaeri, Sanofi
    Yuan Zhong,  Johnson and Johnson

    Becky Noel, Eli Lilly

  • Introduction to the Evaluation of Therapeutic Risk Management Programs

    Brief overview of course:
    This half-day course presents an overview of EU and US risk management principles, with a focus on methods for evaluating the effectiveness of risk minimization measures. The didactic section includes speakers from the regulatory and research communities to present background, regulatory requirements, and examples of effective evaluation techniques.  The workshop section asks participants to work in groups to develop an evaluation plan for one of two health outcomes of interest for a hypothetical product.  The groups present their plans from this case study to the “regulatory panel (faculty)” for feedback and discussion.

    Educational Objectives:

    • To provide basic overview/framework for biopharmaceutical risk management including:
      • EU GVP requirements and examples – the thread of risk management, communication and evaluation
      • An update on  US risk evaluation & mitigation strategies (REMS)
    • To describe the current requirements for evaluation studies and types of results that have been seen in the past few years
    • To illustrate key concepts of evaluation research with examples of RMP/REMS assessment studies
    • To provide an opportunity for interactive session in evaluating Risk Minimisation Measures in US and EU

    Target Audience:

    • Epidemiologists with little prior experience developing RMPs and REMS, and in conducting assessments of such programs
    • Pharmacovigilance and regulatory professionals  who are involved with reviewing, and negotiating risk RMPs/REMS and their assessments

    Course Faculty/Presentations:

    Elizabeth Andrews FISPE, VP Pharmacoepidemiology and Risk Management, RTI Health Solutions
    Course introduction and objectives
    Overview of Risk Management and the Role of Evaluation

    Priya Bahri, Pharmacovigilance Lead for Guidelines and Risk Communication, European Medicines Agency
    EU GVP requirements and examples – the thread of risk management, communication and evaluation

    Gerald Dal Pan, Director, Office of Surveillance and Epidemiology,
    Center for Drug Evaluation and Research, US Food and Drug Administration
    Requirements and Lessons Learned from Evaluating REMS in the US – Update from FDA

    Carla Van Bennekom,  Epidemiologist, Slone Epidemiology Center, Boston University  
    Examples and Evaluations

    Judith Jones, FISPE, President and CEO, The Degge Group, Ltd.
    Workshop for Participants to Develop an Evaluation Program for a  Risk Minimization Plan

  • Pharmacoepidemiologic Considerations for Biosimilars

    Brief overview of course:
    This highly interactive workshop will involve participants in the following topics:

    • Define biosimilars, bio-betters and synonyms
    • Review regulatory pathways and legislation
    • Discuss uptake considerations and coverage decisions
    • Develop skills to craft a feasible study plan

    Educational Objectives:

    • To gain an understanding of biosimilars
    • To understand regulatory pathways
    • To examine uptake considerations and coverage decisions
    • To learn how to craft a feasible study plan to generate real-world evidence on biosimilars

    Target Audience:

    • Those interested in conducting safety and effectiveness research on biosimilars
    • Those interested in understanding how to provide data for health technology assessments

    Course Faculty/Presentations:

    Nancy A. Dreyer, FISPE, Global Chief of Scientific Affairs, Quintiles Real-World & Late Phase Research, Cambridge, MA; Adjunct Professor of Epidemiology, University of North Carolina, Raleigh, NC.
    Overview of Biosimilars

    Aaron S. Kesselheim, Associate Professor of Medicine at Harvard Medical School;
    Director, Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics; Brigham and Women's Hospital
    Current regulatory pathways and legal issues affecting biosimilar approval and use, with implications for postmarket safety analysis

    Gregory W. Daniel, Managing Director for Evidence Development & Innovation, Center for Health Policy; Fellow, Economic Studies, The Brookings Institution
    Postmarket safety surveillance data considerations; payer coverage, naming, and coding considerations; biosimilar uptake considerations

    Jaclyn L. F. Bosco, Director of Epidemiology and Outcomes Research, Real-World & Late Phase Research, Quintiles; Adjunct Assistant Professor of Epidemiology, Boston University School of Public Health, Boston, MA
    Considerations in generating real-world evidence for biosimilars for multiple stakeholders

  • Pharmacoepidemiology of Vaccine Safety

    Brief overview of course:

    Vaccines are and will hopefully remain key tools for global public health.  Increasingly, pharmacoepidemiologic studies are providing critical evidence needed for risk-benefit decision making.  This is true both in maturing immunization programs with successful control/elimination of the target vaccine-preventable diseases (VPD) as well as low and middle income countries (LMIC) where new technology vaccines against challenging diseases (e.g., AIDS, Dengue, Ebola, malaria, etc.) are under development.  The annual ICPE has had a vaccine-specific session for >10 years but a Vaccine Special Interest Group (VAXSIG) was just formed in 2014.  Among the goals of the new VAXSIG is offering educational activities (with the ISPE Education Committee) on epidemiology of vaccines.  The VAXSIG proposes to start with a pre-conference course on Epidemiology of Vaccine Safety at the 2015 ICPE in Boston. 

    This half-day (~4 hours with break) course aims to establish an understanding of key issues surrounding epidemiology of vaccine safety. This course will introduce participants to issues such as: Why is there a need for vaccine safety; how does vaccine safety differ from drug safety; the public health impact of vaccine safety scares; what are the methodological challenges and solutions associated with vaccines safety studies,  in LMIC’s which have no databases; and in countries which have large electronic databases. The first half of the course will introduce participants to the basic vaccine safety issues. The second half of the course will cover specific case studies in vaccine safety research to illustrate the methods and theory. The course will involve lectures with ample opportunities for audience participation (questions and discussion).

    Educational objectives:

    Upon completing this course, participants will be able to:

    • Explain the need for vaccine safety/surveillance studies.
    • Describe the potential data sources (e.g., spontaneous reports, claims, electronic medical records, etc) for vaccine safety/surveillance studies in developed and resource limited settings.
    • Describe the use of various self-control study designs for vaccine safety studies.
    • Describe the common methodological challenges in conducting vaccine safety studies.

    Target audience:

    • ICPE attendees interested in gaining an understanding of the epidemiology of vaccine safety.
    • This beginner-to-intermediate level course assumes knowledge of basic epidemiologic concepts.

    Course Instructors/Presentations:

    Moderator S. Rizwan Ahmad, FISPE, Pharmacovigilance Consultant/Assistant Professor (adjunct) Georgetown University, Washington, DC, USA

    Robert T Chen, USA Introduction to Vaccine Preventable Diseases and Vaccine Safety

    Daniel Weibel, Erasmus, the Netherlands Vaccine Safety Studies in Low and Middle Income Countries

    Martin Kulldorff, Harvard Pilgrim Health Institute, Boston, MA, USA.  Methodological Issues in Vaccine Safety

    Case Studies in Vaccine Safety:

    1. Patricia Saddier, MD, PhD, Merck:  Lessons Learned from Post-licensure Evaluation of the Safety of MMRV Vaccine
    2. Jonathan Duffy, MD, CDC: Febrile seizure risk following vaccination in young children.
    3. Miriam Sturkenboom, PhD, Erasmus, the Netherlands Narcolepsy-H1N1 Pandemic Influenza Vaccine story: will we ever unravel the truth?
    4. W. Katherine Yih, PhD, MPH, Harvard Pilgrim Health Care Institute and Harvard Medical School: Studying the Association between Rotavirus Vaccines and Intussusception using a Self-Controlled Design: a Case Study from the FDA Post-Licensure Rapid Immunization Safety Monitoring (PRISM) Program
    5. Alison Tse Kawai,  ScD, Harvard Pilgrim Health Care Institute and Harvard Medical School: Approaches to Examining the Safety of Vaccines During Pregnancy in the FDA Post-Licensure Rapid Immunization Safety Monitoring (PRISM) Program

Welcome Reception/Academic Showcase/Chapter Showcase
Co-sponsored by Optum
Music provided by Crowes Pasture (Monique Byrne and Andy Rogovin