ISPOR Summit 2019
October 11, 2019
Baltimore, MD, USA
Program
ISPOR Summit 2019
Real-World Evidence Transparency Initiative
8:00 AM – 3:30 PM
October 11, 2019
Baltimore Marriott Inner Harbor at Camden Yards
Baltimore, MD, USA
Building Trust in RWE – The Role of Study Registration
This Summit will be a forum for discussion of the work of the Real World Transparency Partnership, led by ISPOR, ISPE, Duke-Margolis, and NPC, and involving a number of other organizations and stakeholders. It will focus on the key elements needed for the creation of a common registration site for these real-world studies that will be oriented for regular use by researchers and seen as a credible registry by decision-makers. The work already completed in this effort will be presented and the future steps envisioned will be discussed with Summit attendees.
Agenda*
| Friday, Oct. 11, 2019 | |
|---|---|
| 8:00AM | Summit Registration |
| 8:45AM | General Welcome - Nancy S. Berg, CEO, Executive Director, ISPOR |
| 9:00AM | Session 1: Transparency in RWE - Time for a Unified Approach This session will provide an overview of the history and goals of the RWE Transparency Initiative and why it is timely, as well as its work to date. This will include a summary of the white paper and its responses. It will cover fundamental aspects of this issue including credibility vs. transparency, characteristics of studies recommended for registration (ie, HETE studies), the importance of reporting, and replicability and the totality of evidence.
Lucinda Orsini, DPM, MPH, Associate Chief Science Officer, ISPOR Nirosha Mahendraratnam-Lederer, PHD, MSPH, Managing Associate, Real World Evidence, Duke-Robert J. Margolis Center for Health Policy Marc Berger, MD, Consultant Sebastian Schneeweiss, MD, ScD, Professor of Medicine and Epidemiology, BWH & Harvard Medical School |
| 10:00AM | Break |
| 10:15AM | Session 2: Registration site(s) - Opportunities to Optimize A sine qua non for pre-registration is having one or more suitable registration sites. During this session, we will hear from representatives of three potential sites - clinicaltrials.gov, ENCePP EU-PAS Register, and Open Science Framework, regarding their current capabilities for registering retrospective observational studies and their plans in this area going forward. The potential to align on a common protocol template and what that might entail will also be discussed.
Brigitta Monz, MD, MPH, MA, Global Head Real World Data Immunology, Infectious Diseases, Ophthalmology & Neuroscience, Roche Rebecca Williams, PharmD, MPH, Assistant Director, ClinicalTrials.gov, National Library of Medicine, National Institutes of Health Massoud Toussi, MD, PhD, MBA, Pharmacoepidemiology & Safety Lead, IQVIA David Mellor, PhD, Director of Policy Initiatives, Center for Open Science |
| 11:15AM | Session 3: Nuts and Bolts of Fit-for-Purpose This session will include discussion of a number of key characteristics of a registration site needed to achieve its desired purposes. These include: a "lockbox" capability, allowing time-stamped pre-registration without making details immediately available to the public, allowing for a period of confidentiality; protocol elements needed for pre-registration to sufficiently specify the hypothesis, its supporting rationale, and how it will be tested, in enough detail to allow transparency of intended vs. actual analysis performed; the level of detail of post-analysis reporting needed to enable replication of the analysis; and potentially other considerations.
Bill Crown, PhD, Chief Science Officer, OptumLabs Shirley Wang, PhD, ScM, Assistant Professor, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, BWH & Harvard Medical School Ebere Onukwugha, MS, PhD, Associate Professor, Pharmaceutical Health Services Research, University of Maryland School of Pharmacy Elise Berliner, PhD, Director, Technology Assessment Program, Agency for Healthcare Research and Quality |
| 1:30PM | Session 4: Behavior Modification - Boosting and Nudging In this session, aspects of registration related to the "behavioral" elements of pre-registration will be examined. What level of detail and attestation about "pre-looks" at the data are appropriate? What is the right balance between information required and overall feasibility - is some pilot testing called for? What external conditions on pre-registration would be the best incentives for encouraging its use?
Richard J. Willke, PhD, Chief Science Officer, ISPOR Laura Happe, PharmD, MPH, Editor-in-Chief, Journal of Managed Care and Specialty Pharmacy David van Brunt, PhD, Senior Director and Head, Division of Evidence and Analytics, AbbVie Stacy Holdsworth, PharmD, Senior Advisor, U.S. Regulatory Policy & Strategy, Eli Lilly & Company |
| 2:30PM | Transparency in RWE - Moving Forward This panel will reflect on the day's discussion and review the next steps needed to move towards making pre-registration common practice for RWD hypothesis-testing studies in light of both the opportunities and the challenges. Substantial time for audience interaction will be allowed.
Lucinda Orsini, DPM, MPH, Associate Chief Science Officer, ISPOR Sally Okun, RN, MMHS, Vice President Policy & Ethics, PatientsLikeMe Jennifer Graff, PharmD, Vice President, Comparative Effectiveness Research, National Pharmaceutical Council Jacqueline Corrigan-Curay, JD, MD, Director, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration |
| 3:30PM | Summit Adjourned |
*Subject to change