Modern Pregnancy Pharmacoepidemiology: Threats to the Validity of Studies on Drug Safety in Pregnancy and Approaches to Address Them.
Speakers: Andrea Margulis, Brian Bateman, Krista Huybrechts
Description: In this course, faculty will present and discuss considerations related to threats to the validity of research on pregnancy pharmacoepidemiology.
The course will consist of three recorded presentations by faculty, each of them 30’-40’ long, and a live 30’-60’ Q&A session. This is a new format for this course, in line with all skill courses offered as a part of ICPE All Access 2020. In 2015, the course provided an overview of issues that are particular to drug safety research in pregnancy. In 2016 the course focused on challenges related to exposure measurement, in 2017 the focus was on maternal and neonatal outcomes, in 2018 the course presented pros and cons of various study designs. In 2019, the course discussed significance testing and analytical approaches to address biases. The content of the 2020 edition of this course will cover some of the main themes discussed in previous years.
The course will start with a general overview on drug safety in pregnancy, planned to provide context to the course and to be especially useful for first-time course attendees; followed by more focused talks on unmeasured confounding, selection bias, random error and external validity.
The live Q&A session will take place on October 14, 2020, starting at 9 am EST.
Speakers: Tamar Lasky, Daniel Horton, Steven Bird, Mehemt Burcu
Description: 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 pediatric studies will be discussed. This course will introduce participants who have a good understanding of pharmacoepidemiology to the specific methodologic issues arising in pediatric pharmacoepidemiology and operational approaches used to study medications in children
Participants will gain an understanding of how key issues apply in pediatric pharmacoepidemiology including:
Pharmacoepidemiologic Considerations for Biologics and Biosimilars
Speakers: Jaclyn Bosco, Thijs Giezen, Gianluca Trifirò, Rohini Hernandez
Description: This workshop will provide participants with information on the following topics:
Pharmacovigilance and Signal Detection
Speakers: Andrew Bate, Joshua Gagne, Gianluca Trifirò
Description: This course will introduce participants to the principles of surveillance in pharmacovigilance with a focus on quantitative aspects and recent advances in the field. 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 work in the US FDA’s Sentinel program aimed at using routinely collected electronic healthcare data to conduct signal detection. Real-world examples will be used across the lecture sessions to illustrate and connect different concepts. Participants will engage in interactive discussions about contemporary issues in and challenges facing pharmacovigilance, such as the role of social media data and the use of the same data for both signal detection and subsequent evaluation.
Pragmatic Trials: Design, Methods, and Implementation
Speakers: Jennifer Christian, Cynthia Girman, Anne Trontell
Description: Pragmatic randomized clinical trials (pRCTs) require the expertise of pharmacoepidemiologists who understand considerations and methodologic challenges that arise with more “pragmatic” RCTs compared with traditional “explanatory” RCTs. This course will highlight issues such as real-world site and patient identification, enrollment and retention, ethical review issues, approach to randomization, selection bias, and defining the appropriate comparator. In addition, choice of the study design including cluster randomization, step wedge design or individual randomization, and associated analytic considerations will be highlighted. Randomizing patients to “standard of care (SOC)”, accounting for changes in therapies over time, and other methodological considerations will also be discussed. Operational challenges and lessons learned from conducting pRCTs will be shared along with case examples.
Propensity Scores in Pharmacoepidemiology
Speakers: John Seeger, Jeremy Rassen
Description: 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.
Prospective Research: Tools and Tips for 21st Century
Speakers: Nancy Dreyer, Christina Mack
Description: This course will provide an overview of the methods and applications for studies that use primary data collection for effectiveness and safety research, including regulatory submissions of real-world data for label approvals or expansions. Topics include data collection from clinicians, electronic health records, or directly from patients without the use of clinicians, and will address methodological and logistical challenges with a focus on recruitment and retention. Examples will include working with patient communities, data collection through clinicians, call centers, and digital technologies, and integrating electronic health data, with applications in rheumatology, pregnancy and injury research, among others.
Real World Evidence (RWE) generation for rare diseases drug development: data sources and innovated methods.
Speakers: Jasmanda Wu, Federica Pisa, Rima Izem, Janet Sultana, Cynthia Jones, Xabier García-Albéniz, Emily Bratton
Description: This course presents an overview of approaches to generate Real World Evidence (RWE) for Rare Diseases (RD) drug development, with a focus on identifying adequate data source/s and innovative methods. RWE generation is required at various stages of RD drug development, yet it faces specific challenges related to the very small number of patients, disease heterogeneity, disease coding, and frequent lack of treatment standards. The course will start with an overview of data sources and data collection approaches to capture relevant RD patient populations and information, describe data and methods to measure the prevalence and incidence of RD, and present methods for generating evidence to evaluate effectiveness and safety of RD drugs. These include methods based on target trial emulation, Natural History Studies and statistical methods for borrowing evidence. Course faculty will present real-world case studies to facilitate discussion.
Syllabus Outline: In the first presentation, the students will be introduced to strengths and limitations of different study design and methods of data collections in pharmacoepidemiology. The following presentations highlight examples of research studies that use original data collection to augment the information availability via electronic database. The presentations are followed by a workshop in which students in small group design a study using original data collection to answer to given research question. All presentations are pre-recorded.
Katja Taxis, MSc, PhD, Department of Pharmacy, Unit of PharmacoTherapy-Epidemiology & -Economics, University of Groningen, The Netherlands, Common study designs used to collect data through observation, medical records or questionnaires.
Lisa Pont, BSc, BPharm, PhD, Australian Institute of Health Innovation, Macquarie University, Australia, Field studies to value add in pharmacoepi: focus on efficiency in study design
Veronika Wirtz, MSc, PhD, Boston University School of Public Health, USA, Course moderator and facilitator
Using Pharmacoepidemiology Database Resources to Address Drug Safety Research
Speakers: Kevin Haynes, Gillian Hall, Matthew Reynolds, Keith Marsolo, Daniel Beachler, Sudha Raman
Description: Pharmacoepidemiology requires the utilization of database resources to evaluate drug safety. This course will define the different types of data resources available for pharmacoepidemiology and the selection criteria to select the most appropriate resource. The course focuses on the Guidelines for good database selection and use in pharmacoepidemiology research provides an overview of available resources, addresses the need for outcome validation, and highlights data linkage considerations.
Weighing the Benefits & Risks: The Science and Methods of Benefit-Risk Assessment in Drug Development
Speakers: Rebecca Noel, Tarek Hammad, Juhaeri Jhuaeri, Brett Hauber
Description: Benefit-risk assessment is now a fundamental science for understanding and evaluating the development, approval, and continued marketing of drugs and devices. This course is aimed at introducing attendees to core concepts and tools of both the science and practice of benefit-risk assessment.