Event Sponsors
Featured Speakers
Amila Bewtra
VP of Strategic Development
Voxx Analytics
Carl de Moor
Executive Director, Global Head Medical Data Sciences and Analytics
Biogen
Christoph Glaetzer
VP Global Market Access & RWE
Janssen
Cynthia J Girman
Founder and President
CERobs Consulting, LLC
Dan Riskin
CEO, Adjunct Professor of Surgery and Biomedical Informatics Research, Stanford University
Verantos
David Miller
Executive Director, Epidemiology
Sarepta
Ed Silverman
Chief Analytics Officer
Voxx Analytics
Hayley Belli
Assistant Professor, Biostatistics, Department of Population Health
NYU Grossman School of Medicine
Harini Gopalakrishnan
Evidence Generation & Insights, Technology Product Lead
Sanofi
Jasmanda Wu
Real-World Evidence Head
Insmed
Juliette Chen
Director, Global Evidence and Outcomes
Takeda
Kelly McNeil-Posey
Associate Director, HEOR
Alexion Pharmaceuticals
Mary Tran
VP, Data Insights
Syapse
Melissa Hagan
Director, Medical Affairs HEOR/RWE/Publications
Mitsubishi Tanabe Pharma America, Inc.
Nneka Onwudiwe
former PRO/PE Regulatory Review Officer, CDER/OMP/OPDP; Founder and CEO
FDA; Pharmaceutical Economics Consultants of America (PECA)
Ravinder Dhawan
VP and Head of Oncology, Center for Observational and Real World Evidence
Merck
Sanjay Gupta
Vice President, Head of Global Real World Evidence, OBG and U.S. HEOR
Eisai Inc
Sheena Thakkar
Manager, Global HEOR
Pfizer
Shoshie Resef
Director, Global Epidemiology and Real World Evidence
Teva
Shuvayu Sen
VP and Head Value Evidence and Real World Evidence
Otsuka Pharmaceutical Companies
Sonal Bhatia
VP, North America, North America Medical Lead, Rare Disease
Pfizer
Tom Watson
Executive Vice President – Early Access Programs
Bionical Emas
Event Schedule
ALL TIMES ARE IN EST
Discuss the role of RWE in understanding and confronting the pandemic including virus tracking and containment measures and the development of treatments and vaccines. There is a potential to broaden our disease understanding through RWE in a real time “24/7” on a global scale.
• Focus on the role of data collection, visualization and communication
• Understand and predict how the virus would impact different populations and define appropriate efforts to protect the most vulnerable populations
• Survey the impact of RWE in defining government strategies to contain the virus spread and hence on everybody’s everyday life
• Track the growth of data generation and communication approaches “RWE 24/7 by everyone and everywhere”
• Understand differing data generation expectations across various stakeholders
• Envision opportunities for post launch evidence generation and value substantiation
• Strategize new data collection opportunities in a virtual/remote world
April 14, 2021
11:00 – 11:45 AM
Gain Regulatory Insight on FDA Initiatives to Optimize RWE in Labeling
The FDA's initiatives on advancing the use of RWE to inform regulatory decisions are widely anticipated. There is existing FDA guidance, and lessons learned, which shed light on potential future steps and inform regulatory strategy.
• Review challenges and opportunities for communicating meaningful conclusions about treatment benefit
• Explore best practices and regulatory standards for collecting and analyzing RWD
• Consider approaches for working closely with FDA on use of RWE in regulatory submissions
RWE is well accepted for satisfying post-approval safety monitoring requirements and companies have gained FDA approval for new indications and label extensions. This session details how rigorous the data must be and how these applications can help you with your own post-approval stages in the product lifecycle.
• Review some key case studies for using RWE either for product approval, for new indications or expanded labeling
• Discuss the evidence FDA needs to evaluate whether RWE can support new indications or labeling
• Visualize a potential process for gaining concurrence on using RWE for regulatory purposes, based on another paradigm shift at FDA
Utilize RWE to better inform clinical trial design and execution plans to further improve research and development.
• Deploy RWE for more precise patient targeting/screening and site selection
• Employ innovative solutions via RWE and AI (natural language processing, machine learning) to enhance RCT
• Integrate RWE strategy to realize more patient-centric clinical development for better patient care
• Gauge the impact of RWE on decreasing racial disparities in clinical trials
Due to a growing pressure to increase the efficiency and effectiveness of clinical trials, one solution is the use of real-world data as a synthetic control arm in clinical trials to support regulatory decision. It is particularly important where available populations in a disease are very limited. This session intends to cover the followings:
• What is a synthetic control arm?
• When to use a synthetic control arm
• External RWE control for non-randomized single arm trials under FDA RWE Framework
• Synthetic control methods
• What constitutes a good synthetic control data set?
• Successful examples where RWE has been used as external controls to support regulatory decision
Improving health outcomes through better decision making and more targeted innovation is one of the most exciting opportunities for the RWE ecosystem. Advances in AI and machine learning have tremendous potential for predicting, diagnosing, treating and preventing disease.
• Spotlight benefits of machine learning and natural language processing for curating unstructured data
• Build predictive models to reveal disease patterns and improve outcomes through early detection
• Leverage unstructured data to unlock richness and identify clinically relevant data points
• Understand value prediction in clinical development and clinical trial feasibility
• Compare treatment effectiveness
• Digital technologies and cloud based platforms for ensuring privacy / traceability on patient data while doing AI/ML
Regulators, payers, providers, and patients represent an ever-growing demand for data richness, evidence validity and “Fit for Purpose” application of real world data
• Understand RWD data sources, the strength and limitations from the perspective of the stakeholders (patient, payer, regulator, provider)
• Build an infrastructure to facilitate real-time data availability, ensure data consistency, quality and evidence validity.
• Build ease of integration across data sources and adaptability to meet evolving data needs.
- Integrate scientific network, social listening, and real world data (claims) to understand how these data assets can work together to find hidden insights (unicorns).
- Exploring how business decisions can be optimized based on new ways of understanding what is actually going on
ALL TIMES ARE IN EST
Regulators, payers, providers, and patients are increasingly more receptive to expanding the use of RWE beyond traditional safety related applications. A strategic plan for 2025, that involves a cross-functional team, is required to meet the need for regulatory grade evidence that improves health care decision making, innovation and outcomes.
• Build internal strategy and capability for meeting future regulatory and payer needs
• Evaluate your infrastructure and spot gaps and areas for improvement
• Benchmark your RWE strategy with key learnings from successful use cases as well as pitfalls on what to avoid when working cross functionally
• Identify opportunities to evaluate therapeutic areas for prioritizing RWE initiatives
• Overcome difficulties related to who uses RWE – and how
There are over 7,000 rare diseases affecting 30 million people and many of these do not have treatments. There were an estimated 19 million new cancer cases in 2020. RWE can be used to identify unmet patient needs and facilitate earlier diagnosis and intervention.
- Facilitate earlier diagnosis and intervention
- Build predictive models to detect disease patterns
- Determine which endpoints will be most valuable
- Identify sub-populations that will be most likely to respond to therapy
- How to engage early with patients and patient organizations
Learn more about the critical elements involved in using RWE to support product effectiveness for payers and prescribers. Such insights and statistically significant cost/value analysis are being used to advance reimbursement models.
• Identify patient subgroups who will benefit most from your drugs
• Demonstrate the value of a therapy for payers and prescribers
• Assess decision support tools for physicians
• Harness RWE in precision medicine
• Discuss the role of RWE in the future of value based care
Real world data can supplement clinical trial data, or in the case of pre-approval access in especially conditions like rare disease, can be a source for drawing evidence. How do we best determine the role of RWE and RWD in extracting some conclusions regarding EAPS.
• Digital health tools used to collect real world data
• Methods for extracting evidence from RWD for EAPs
• Update on best practices for ethical use of RWE by industry, regulators, payers in non trial pre-approval access to investigational interventions
• Setting up to draw evidence and make meaningful conclusions from data
HEOR is critical to informing decisions along the lifecycle of a medicine. Robust HEOR planning is required to successfully communicate product value to a range of stakeholders.
• Communicate clinical, economic and quality of life endpoints of various treatments
• Set publication strategies of scientific presentations and evidence generation
• Create high impact evidence for linking reimbursement to clinical outcomes
• Harness RWE for predictive analytics and outcomes-based models
• Discuss best practices for gathering RWE and what types of data are needed
Addressing the demands for information at the launch stage requires early product lifecycle planning for how to strategically leverage RWE. Understand the role and value of RWE for product launch.
• Discuss potential timings of RWE strategy and development
• Evaluate potential different types of evidence and how they can potentially fill data gaps
• Explore how impactful RWE can be when assessed in a cross-functional platform
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