Real-world data (RWD) is playing an increasing role in health care decisions, especially with diseases where progression and treatment pathways may differ drastically and healthcare costs are high. Applying advanced analytics to RWD can improve our understanding of the history of these diseases, better demonstrate treatment effectiveness, and provide insights into more personalized benefit:risk profiles and treatment pathways. This session will explore:

• What are advanced analytics and opportunities with applying them to RWD
• Common mistakes, such as missing data, and strategies for overcoming
• Using predictive models for identifying underdiagnoses, understanding risk of events, such as hospital admissions, and more

Speakers:

Gary Curhan
Chief Medical Officer
OM1

Zhaohui Su, PhD
VP of Biostatistics
OM1

Attendees will receive an overview of recent regulations focusing on how to leverage patient registries to navigate success with German authorities. Kantar will share results from a recent survey with global life science companies focused on what they think and how they’ll react to new regulations.

Speaker:

Michael Fronstin
Global Head of Innovation
Kantar Health

Dr. Michael Schultze
Senior Project Manager, Real-World Evidence
Kantar Health

Real-world data (RWD) and real-world evidence (RWE) are playing an increasing role in health care decisions. In addition to the use of RWE/RWD to support reimbursement decisions, FDA/EMA and other regulatory agencies use RWD and RWE to monitor post-market safety and adverse events and to make regulatory decisions. Medical product developers are using RWD and RWE to support clinical trial designs (e.g., large simple trials, pragmatic clinical trials) and observational studies to generate innovative, new treatment approaches. The health care community is using these data to support coverage decisions and to develop guidelines and decision support tools for use in clinical practice.

This session will discuss:

• New development of using RWE/RWD in supporting clinical development and regulatory decisions.
• Examples of using RWE/RWD in clinical development, and cases of using RWE to obtain regulatory approvals.
• Opportunities, challenges, and new trends of the RWE/RWD industry

Speaker:

Dr Boxiong Tang
Head of Health Economics and Outcomes Research (HEOR), U.S., Europe & New Markets Medical Affairs,
BeiGene, Ltd.

From accelerating clinical trials and gaining regulatory approval to expanding access and understanding market needs, this session will cover the basics of what makes real-world data regulatory and research grade and how it can transform your R&D and market access programs for chronic diseases, including autoimmune and cardiovascular conditions. Join the session to explore:

1. How new technologies can collect and process data for RWE applications
2. Data quality criteria and getting to regulatory-grade results
3. RWD utility across research and development
4. Case examples in immunology, heart failure, and more

Speaker:

Richard Gliklich
CEO
OM1

With factors as various as COVID-19, telemedicine, gene therapy outcomes, regulatory requirements, and value-based pricing, it is becoming imperative to engage as many patients as possible into a registry or long term follow up studies without requiring a clinical visit to a site. In this session, we will discuss the opportunities and challenges for adopting study designs, virtual research visit methods, and the data sources for operating a registry beyond site visits.

Speaker:

Christopher Jones
CEO
iTakeControl Health

From engaging patients in RWD collection to utilizing RWE to increase patient engagement, putting the patients at the center will optimize your impact and improve your project’s methodology, recruitment, and outcomes. This session will explore:

• The patients’ perspectives on RWD and RWE
• Patient engagement in research to improve RWD collection, interpretation, and application
• The potential of RWE to engage patients in care and decision-making
• Building trust through information, education and transparency

Speaker:

Dr Rebekah Angove
Vice President, Patient Experience and Program Evaluation
Patient Advocate Foundation

Traditional patient registries continue to be critical for evaluating and demonstrating real-world outcomes. But, with greater access to real-world datasets and innovative tools for linking, standardizing, and analyzing the data, there is another way. This session challenges the status quo and explores innovative approaches to building and leveraging registries and custom cohorts for succeeding in the new world of real-world data and multi-stakeholder requirements. Join this session to explore:

• Defining and understanding patient registries
• How they are changing and what that means for research
• Best practices and new guidelines
• Use case examples

Speaker:

Richard Gliklich
CEO
OM1

Much of the recent development of RWE strategy and analytics has been with EHR-based data due to the availability of larger sample sizes and greater longitudinal follow-up than seen in clinical trials. However, there is another type of data source that can and should be considered in any RWE analysis: The prospective, observational data collected in a patient registry. To maximize the utility of patient registry data it’s important to know what its strengths are:

• Understanding the difference between primary (patient registries) and secondary (EHR-based) data sources.
• Benefit of using registry data in comparator cohorts for single arm trials.
• Usefulness of registry data for validation of other real-world data sources.

Speaker:

Susan Fish
Associate Director, Portfolio Data Strategy & Internal Collaborations,
Strategic Evidence Integration & Collaborations
Bristol Myers Squibb

• SH has a LTFU model that will provide massive operational and cost efficiencies
• This model will increase patient engagement and retention
• This model is flexible and scalable (can be considered as a platform approach incorporating multiple products, for clinical trial and de novo patients before and after commercialization)

Speaker:

Mike D’Ambrosio
Executive Director
Syneos Health

Mimi Fenton MPH PhD
Executive Director
Syneos Health

• Overview of regulatory perspectives on epidemiology and RWE
• Rare disease epidemiology and RWE
• Methodological challenges and approaches
• Case examples
• Future directions

Speakers:

J. Alexander Cole
Executive Director, Global Head Epidemiology
Alexion Pharmaceuticals