Educational Symposia
Saturday, May 19, 2018

5:30 PM - 6:30 PM
Room: 309 (Level 300)
DEBATING THE FUTURE OF HEALTHCARE
In 2016, through the 21st Century Cures Act, Congress gave the directive for the FDA to develop a regulatory framework to evaluate how real-world evidence (RWE) should be used to support regulatory filings. However, this opened the door to many complex questions that the FDA is currently considering: How much weight should be given to RWE when making regulatory decisions? Which decisions could be supported with RWE alone? What are the acceptable methodologies? Using a modified Oxford-style debate format, this lively discussion will feature experts deliberating the extent to which the FDA should use RWE in their decision-making process - and the audience will have the ultimate say in which argument was most persuasive.
Moderator: Peter Pitts, PhD
President, Center for Medicine in the Public Interest, New York, NY, USA
Speaker: Nancy Dreyer, MPH, PhD
Chief Scientific Officer, Real-World & Analytic Solutions, IQVIA, Cambridge, MA, USA
Speaker: Marni Hall, PhD, MPH
Vice President of Clinical Evidence, Center of Advanced Evidence Generation, IQVIA, Rockville, MD, USA
Speaker: Gregory Daniel, PHD, MPH
Deputy Director, Policy, Duke-Robert J. Margolis, MD, Center for Health Policy, Washington, DC, USA
Speaker: Marc Berger, MD
Consultant, Self Employed, New York, NY, USA
AbbVie
(Sponsored by AbbVie)
Sunday, May 20, 2018

5:30 PM - 6:30 PM
Room: 309 (Level 300)
HEALTHCARE GRIDLOCK BETWEEN VALUE, AFFORDABILITY, AND ACCESS: THE GOOD, THE BAD AND THE UGLY
The dilemma of managing the affordability of an innovative healthcare intervention while reaping its benefits often becomes the rate-limiting step to access. In this session, the panel will discuss good principles of value frameworks, as well as other forms of economic assessments. These considerations can help balance the value of a product with its affordability and ultimately translates to better patient access and improved outcomes.
Moderator: David Harrison, PhD
Director, Global Health Economics, Amgen Inc, Thousand Oaks, CA, USA
Speaker: Diana Brixner, PhD, RPh, FAMCP
Professor, Department of Pharmacotherapy, University of Utah College of Pharmacy, Executive Director, Pharmacotherapy Outcomes Research Center, and Director of Outcomes, University of Utah, Salt Lake City, UT, USA
Speaker: Sara Carruth, PharmD
Manager in Health Economic and Outcomes, MedImpact Healthcare Systems, San Diego, CA, USA
Speaker: Jeff Dunn, Pharm.D., MBA
Vice President, Magellan RX, Salt Lake City, UT, USA
Amgen
(Sponsored by Amgen)

6:45 PM - 7:45 PM
Room: 309 (Level 300)
ROLES OF BIOSIMILARS ON PATIENT ACCESS
Many studies demonstrate the clinical benefits of biologics in the early phases of autoimmune diseases. However, only a limited number of patients have access to the treatments due to their high-cost. The role of biosimilars is to make necessary biologic treatments more affordable and available to those needing treatment. We will discuss and compare the use of biosimilars in Europe and the US.
Moderator: HoUng Kim, MBA
Head of Strategy and Operation Divisions, Celltrion Healthcare, Incheon, Korea, Republic of (South)
Speaker: Edmund J. Pezalla, MD, PhD, MPH
Former President, Aetna, Wethersfield, CT, USA
Speaker: Jørgen Jahnsen, Ph.D
Professor, Gastroenterology, Akershus University Hospital, Lørenskog, Norway
Speaker: HoUng Kim, MBA
Head of Strategy and Operation Divisions, Celltrion Healthcare, Incheon, Korea, Republic of (South)
Speaker: Laszlo Gulacsi, DSc
Head of Department/Professor, Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary
Celltrion Healthcare
(Sponsored by Celltrion Healthcare)
Monday, May 21, 2018

12:30 PM - 1:30 PM
Room: 309 (Level 300)
21st CENTURY CURES ACT AND THE FUTURE OF REAL-WORLD EVIDENCE
The 21st Century Cures Act calls for the FDA to develop and implement a program to evaluate the potential use of real-world evidence (RWE) for regulatory decisions. Many questions remain regarding recommendations the FDA will make, how industry will react, and what this means for the future of RWE and the HEOR community. This session will be a panel discussion with experts who have been involved in the process and can share insights on the anticipated direction of policy and potential implications for the generation, use, and value of RWE in the coming years. The panel will include representatives from FDA’s Center for Drug Evaluation and Research (CDER), the National Pharmaceutical Council (NPC), and the Amgen Center for Observational Research. An important aim of the session is to involve the audience in the discussion by allowing ample time for questions and answers. This symposium will serve as a valuable extension to the overall ISPOR 2018 theme of Real-World Evidence, Digital Health and the New Landscape for Health Decision Making.
Moderator: David Thompson, PhD
Senior Vice President, Real World & Late Phase, Syneos Health, Boston, MA, USA
Speaker: Jennifer S. Graff, PharmD
Vice President, National Pharmaceutical Council, Washington, DC, USA
Speaker: Cathy W Critchlow, PhD
Vice President, Center for Observational Research, Amgen Inc, Thousand Oaks, CA, USA
Speaker: David Martin, MD, MPH
Captain, U.S. Public Health Service; Associate Director for Real World Evidence Analytics, U.S. Food and Drug Administration, Office of Medical Policy, Center for Drug Evaluation and Research, Silver Spring, MD, USA
Syneos Health
(Sponsored by Syneos Health)
Tuesday, May 22, 2018

7:15 AM - 8:15 AM
Room: 309 (Level 300)
SEEING THE FOREST FOR THE TREES: DEVELOPING ROBUST RWE USING DATA LINKAGE
Robust real-world evidence (RWE) must be supported by appropriate real-world data (RWD). As a result of the fractured nature of the US healthcare system, RWD reside in silos (e.g., administrative claims, electronic medical records, laboratory results, hospital administrative), each with its own strengths and limitations. Thus, evidence generated from any one data source may be insufficient to build a robust value story. Historically, HIPAA privacy rules have made it challenging to combine data sources that address their respective limitations. However, new technology has emerged that enables HIPAA-compliant data linkages aimed at achieving a 360 degree view of a patient’s care to support evidence development.
Speaker: Peter M. Wahl, MLA, MS, ScD
Director, Pharmacoepidemiologist, Covance Market Access Services Inc., Gaithersburg, MD, USA
Speaker: Andrew Kress, BA
CEO, HealthVerity, Philadelphia, PA, USA
Covance Market Access
(Sponsored by Covance Market Access)

12:30 PM - 1:30 PM
Room: 309 (Level 300)
ADVANCEMENTS IN METHODS OF SURVIVAL BENEFIT ESTIMATION FOR NOVEL ONCOLOGY DRUGS AND THEIR APPLICATIONS IN ICER REVIEWS
Clinical trials for a number of emerging cancer therapies, such as immuno-oncology treatments, have identified patients who achieve long-term remission following therapy. In situations where a significant proportion of individuals achieve durable remission, traditional survival models may not be appropriate to represent the complex hazard functions. Furthermore, they have been found to introduce bias into survival assessments and influence results in cost-effectiveness analysis. In this educational symposium, Dr. Wu will moderate and introduce common methodological challenges in modeling survival benefits for novel cancer therapies. Dr. Ramsey will present mixture cure modeling as an alternative approach to address the issues. He will briefly discuss the method and then provide an example to illustrate the differences in outcomes between the mixture cure approach and alternative modeling approaches. Dr. Ramsey will also provide guidance on when to consider mixture cure models, and then discuss some limitations in survival estimation that should be considered for novel therapies with limited follow-up data. Dr. Xie will present other alternative methods to predict survival outcomes for treatments that can "cure" diseases. Using survival data with long-term follow-up, she will compare results using different extrapolation methods and discuss the validation of long-term survival projections. Dr. Ollendorf will discuss the challenges facing health technology assessment (HTA) organizations in evaluating what is often immature and uncontrolled evidence on survival. In addition, he will present a number of approaches to address this uncertainty from both the evidence synthesis and decision analysis perspectives. Finally, Dr. Ollendorf will discuss the application of novel methods in a number of recent HTA reviews, including CAR-T treatments for a variety of cancers.
Moderator: Eric Q. Wu, PhD
Managing Principal, Analysis Group, Inc., Boston, MA, USA
Speaker: Scott D. Ramsey, MD, PhD
Director, Public Health Sciences, Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA
Speaker: Daniel A. Ollendorf, PhD
Director of Health Economics, Institute for Clinical and Economic Review, Boston, MA, USA
Speaker: Jenny Zhou, PHD, MS
Manager, Analysis Group, Boston, MA, USA
Analysis Group
(Sponsored by Analysis Group)
Wednesday, May 23, 2018

7:15 AM - 8:15 AM
Room: 309 (Level 300)
BRIDGING THE GAP BETWEEN RETROSPECTIVE DATA AND REGISTRIES: WHERE DO WE GO FROM HERE?
Real-world data (RWD) and real-world evidence (RWE) are playing an increasing role in healthcare decision making for both regulators dealing with safety and approval, and payers dealing with access. While automated healthcare databases (electronic health records, claims, etc) are good ow-budget resources, they often lack some of the key elements required for evidence generation. Patient registries can be expensive, requiring extensive resources and time, which delay critical decision making. In this session, our panel of stakeholders will address the: (1) current approaches utilized for generating RWE, and (2) what gaps can be addressed using methodologies that are reasonable with regard to cost, time, and resources, without compromising the robustness of evidence.
Moderator: Christopher M Blanchette, PhD, MBA
Senior Vice President, Global Evidence, Value and Access, Ipsos Healthcare, New York, NY, USA
Speaker: Jeffrey Vernice, PhD, MBA, MS
Senior Director, Analytics Business Partner and Data Science, Horizon Blue Cross Blue Shield of New Jersey, Newark, NJ, USA
Speaker: Elaine B. Yu, PharmD, MS
Head of Evidence for Access BioOncology, Genentech, Inc., South San Francisco, CA, USA
Speaker: Joshua M Noone, PhD
Director, Global Evidence, Value and Access, Ipsos Healthcare, New York, NY, USA
Speaker: Emily Zacherle, MS
Senior Research Manager, Global Evidence, Value and Access, Ipsos Healthcare, New York, NY, USA
Ipsos Healthcare
(Sponsored by Ipsos Healthcare)
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