Cambridge Healthtech Institute’s 9th Annual

Accessing and Generating RWD

Sources and Strategies for Generating Regulatory Grade RWD

FEBRUARY 19-20, 2020


Real-world evidence solutions have changed the design and execution of clinical trials and post-marketing research. Data generated in real-world data-based studies is essential for multiple stakeholders within and outside pharmaceutical companies, such as regulatory agencies, payers, health care management organizations, formulary inclusion decision makers, healthcare professionals, and patients. CHI’s 9th Annual Accessing and Generating RWD conference is designed to facilitate knowledge exchange around all aspects of real-time, real-world data generation, its quality and applications.

Stay on and attend Part 2 (Thurs-Fri): Leveraging RWD for Clinical and Observational Research

Scientific Advisory Board

Cathy Critchlow, PhD, Vice President, Center for Observational Research, Amgen

Hui Cao, PhD, Executive Director, Real World Evidence, Global Medical Affairs, Novartis Pharmaceuticals

Charles Makin, Global Head, Real World Evidence Strategy, Biogen

Marc Berger, MD, Chair, Real World Evidence Advisory Committee, SHYFT Analytics

Final Agenda

Tuesday, February 18

9:00 am - 7:15 pm Registration Open

2:00 - 5:00 pm User Group Meetings

2:00 - 5:00 pm The NEW SCOPE China Clinical Development Partnering Forum and The NEW SCOPE Scientific Symposium*

*Separate registration required. Must be a Best Value registered attendee.

5:00 - 6:20 pm Evening Kick-Off Plenary Keynote and Participant Engagement Awards

6:20 - 7:30 pm SCOPE’s Kick-Off Networking Happy Hour

(Co-Sponsorship Opportunities Available)

7:30 pm Close of Day

Wednesday, February 19

7:15 am Registration Open and Morning Coffee

8:15 Morning Opening Plenary Keynotes

9:40 Grand Opening Coffee Break in the Exhibit HallHighthink_Med

ROLE OF RWE AND INNOVATION AROUND REGULATORY GRADE RWD

10:40 Chairperson’s Remarks

Cathy Critchlow, PhD, Vice President, Center for Observational Research, Amgen

10:45 Role of Real-World Evidence in Comprehensive Product Evidence Generation Plans

Critchlow_CathyCathy Critchlow, PhD, Vice President, Center for Observational Research, Amgen

Evidence generation is a key deliverable and an important strategic capability to enable efficient drug development and commercialization across the product lifecycle. Real-world evidence (RWE) effectively complements data generated from clinical trial programs and is rapidly growing in importance to evaluate medicine safety, effectiveness and value. A common understanding of RWE approaches to address key questions supporting regulatory and reimbursement agency needs and patient access does not exist. A RWE playbook to guide and support these activities can introduce efficiencies in resource allocation and help insure that the necessary evidence is generated. Growing availability of data and sophistication of analytic tools have transformed RWE generation. The appropriate use of real-world data and analytic tools, and associated challenges impeding the full realization of benefit from RWE, will be discussed.

11:10 Outcomes Ascertainment at the Speed of Digital: The Hugo Experience

Krumholz_HarlanHarlan M. Krumholz, MD, SM, Director, Yale-New Haven Hospital Center for Outcomes Research and Evaluation

The digital transformation holds the possibility of disrupting labor-intensive, expensive and slow research data collection and engaging participants as partners in the process. The 21st Century Cures Act is providing an impetus for change in the research enterprise and there are applications in discovery research, trial recruitment, trial conduct, post-market surveillance and label expansion. Thus, there is a window of opportunity to transform the research landscape and provide real-time clinical, participant-generated and participant-reported information about outcomes that matter.

11:35 Transparent and Replicable ‘Real World’ Evidence from ‘Real World’ Data

Wang_ShirleyShirley Wang, PhD, Assistant Professor, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical School

Regulatory and Health Technology Assessment (HTA) organizations are increasingly looking toward use of ‘real world’ evidence (RWE) from ‘real world’ data sources, such as healthcare utilization databases, to support decision-making. However, they need to be able to effectively and efficiently distinguish between studies of high versus low validity. The REPEAT Initiative is focused on improving transparency, reproducibility and validity of database research. One project involves replication of 150 published database studies.

11:55 Making the (Regulatory) Grade: Approaches to Data Quality in RWE

Castellanos_EmilyEmily Castellanos, MD, Associate Medical Director, Research Oncology, Flatiron Health

The credibility of real-world evidence (RWE) for a specific use case depends upon both the quality of the underlying real-world data (RWD) as well as the analytic methods. This discussion will focus upon evolving approaches to fit for use quality of RWD. Standardized reporting of quality measures can inform the analytic approach, to minimize bias and generate confidence in regulatory decisions supported by RWE. Considerations important for measuring, monitoring, and addressing dimensions of RWD fit for use quality will be described.

12:15 pm Synthetic Control Arms (SCAs): The End of Placebos, or Not Quite a Silver Bullet?

Richards_MegMeg Richards, Executive Director, Real World Solutions, PRA Health Sciences

Synthetic Control Arms (SCAs): The End of Placebos, or Not Quite a Silver Bullet? Considerations in design, conduct, analysis and reporting of SCAs.


12:45 Transition to Lunch

12:50 LUNCHEON PRESENTATION: Going Virtual in Real-World Research: Opportunities & Challenges

Thompson_DavidDavid Thompson, PhD, Senior Vice President, Real World and Late Phase, Syneos Health

Real-world research is proving to be a useful setting to pilot test new technologies and process innovations for use in RCTs. One such area is mobile technologies, which hold promise to enable conduct of so-called “virtual” studies that significantly reduce the need for site-based patient recruitment, enrollment, and data collection. This presentation will highlight the opportunities and challenges for going virtual in real-world research, using an ongoing fully-virtual prospective cohort study as a case example.

1:20 Coffee and Dessert Break in the Exhibit Hall

RWD TO SUPPORT TRIAL DESIGN

2:15 Chairperson’s Remarks

Chair to be Announced, Syneos Health

2:20 Considerations on the Use of Real-World Data in Trial Design

Alemayehu_DemissieDemissie Alemayehu, PhD, Vice President, Biostatistics, and Head of Statistical Research and Data Science, Pfizer

When traditional randomized controlled trials are not feasible for operational or ethical reasons, use of external control arms constructed from historical clinical trial or real-world data may be viable options. However, the validity of the evidence generated from such trials is dependent on several factors, including data quality, disease natural history, study design, analytical approaches and similarity of data sources. In this talk, we highlight some of the issues that arise in the design and analysis of trials with external control arms, with emphasis on trending topics in the statistical and regulatory spaces.

2:50 Leveraging Electronic Health Records to Inform Protocol Design and Accelerate Enrollment in Clinical Trials

Yonchuk_JohnJohn Yonchuk, Manager, Digital Clinical Trials, GlaxoSmithKline

One of the key challenges facing Pharma is the ability to identify and recruit the best participants to enroll and complete clinical trials. As these participants become more highly sought after and harder to find, it becomes critical to be able to quickly and correctly identify and recruit such participants. The recent trend of looking to electronic health record data, and using artificial intelligence to analyze it, holds great promise to help improve identification and recruitment in clinical trials.

3:20 The Use of Real-World Evidence in Clinical Design to Support Regulatory Decision-Making

BaumfeldAndre_ElodieElodie Baumfeld Andre, PhD, Senior Director, Epidemiology Strategy & Policy Lead, Worldwide Safety and Regulatory, Pfizer

Real-World Evidence (RWE) could have a significant impact on how pharmaceutical and biotech companies conduct clinical development and pursue regulatory approval of new treatments. Due to three major drivers: 1) increased regulator acceptance of RWE designs; 2) availability of new data types/sets; and 3) broad availability of advanced data analysis capabilities, RWE approaches are gaining traction industry-wide, which might lead to more efficiencies in drug development and better medical outcomes for patients. The objective of this presentation is to provide the audience with a baseline understanding of key RWE concepts and its application to regulatory decision-making in the life sciences industry, as well as an opportunity to discuss the vision for the future.

3:50 Talk Title to be Announced

Galil_KarimKarim Galil, CEO & Cofounder, Mendel.ai


INTERACTIVE BREAKOUT DISCUSSION GROUPS

4:20 Find Your Table and Meet Your Moderator

4:25 Interactive Breakout Discussion Groups

Concurrent breakout discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss key issues presented earlier in the day’s sessions. Delegates will join a table of interest and become an active part of the discussion. Bring your pharma, biotech, CRO, site, hospital or patient perspective to each of the discussions. To get the most out of this interactive session and format, please come prepared to share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and most importantly, participate in active idea sharing.

5:10 Welcome Reception in the Exhibit Hall (Sponsorship Opportunity Available)

6:45 Close of Day

Thursday, February 20

7:15 am Registration Open

7:45 BREAKFAST PRESENTATION: Navigating the Patient Journey: A Responsibility all Technology Providers Share

Mike Nolte, Chief Executive Officer, Signant Health

8:15 Session Break

DEMYSTIFYING FIT-FOR-PURPOSE DATA

8:20 Chairperson’s Remarks

Chair to be Announced, United BioSource Corporation

8:25 Demystifying Fit-for-Purpose Data

Lieberman_GracieGracie Lieberman, Senior Director, Regulatory Policy, Genentech

Technological advancement has raised expectations that data collected in routine clinical practice could be utilized for regulatory decision-making. Hence, understanding RWD quality requirements for regulatory use became key. In December 2018, the FDA published a Framework for FDA’s Real-World Evidence Program and underscored the need for the data to be “fit for regulatory use.” This talk will examine the individual components contributing to data fitness and discuss key considerations.

8:55 Demonstrating and Communicating Real World Data (RWD) Reliability to Support Fitness-for-Use for Regulatory Decision-Making

Silcox_ChristinaChristina Silcox, PhD, Managing Associate, Duke-Margolis Center for Health Policy, Duke University

In December 2018, FDA released the framework on its Real-World Evidence Program including whether RWD are fit-for-use. Fit-for-use RWD is multifaceted concept, including reliability. One way to systematically assess and communicate RWD reliability is to use a standardized set of verification checks. However, it may not be feasible to identify a standardized set of checks to assess all aspects of reliability due to heterogeneity within and between RWD. Instead, a minimum set of standardized verification checks used to assess some aspects of reliability across all data sources could be identified and adopted as a first step. Because data curation is a dynamic process, this minimum set of verification checks should be assessed continuously based on initial review and on findings during analysis.

9:25 CASE STUDY: Successes, Challenges and Lessons Learned Conducting a Multi-Country RWD-Based External Control Arm Study

Kamauu_AaronWAaron W.C. Kamauu, MD, MS, MPH, Vice President, Scientific Data Assets, Parexel

As indicated by regulatory agencies, RWD is recognized as a source to be used as an external control arm in clinical trials. To ensure success, many considerations must be addressed, including scientific, clinical, informatics, data management, etc. We present a case study of an FDA-approved, multi-country, external RWD control arm used for a rare disease. We discuss lessons learned related to the assessment of RWD sources, technological solutions and translation to a common data model.

9:55 Presentation to be Announced

 

10:25 Coffee Break in the Exhibit HallAris-Global

SUPPLEMENTING CANCER RESEARCH WITH RWD

11:20 Chairperson’s Remarks

Aaron W.C. Kamauu, MD, MS, MPH, Vice President, Scientific Data Assets, Parexel

11:25 RWD in Oncology and Immuno-Oncology Research

Kelsh_MichaelMichael Kelsh, PhD, Director, Center for Observational Research, Amgen

This talk will discuss RWD applications in early- and late-stage cancer research. Case studies and examples, as well as relationships with the companies owning the data will be discussed.


11:55 Risk-Based Safety Monitoring for Oncology Clinical Trials

Zhao_SeanSean Zhao, MD, PhD, Head of US Patient Safety, AstraZeneca Pharmaceuticals LP, US Medical Affairs

There are great differences between oncology and non-oncology clinical trials. For example, the nature of the illness of study subjects in oncology clinical trials are more likely severe, life-threatening, and have relatively short survival time periods than those in non-oncology trials. The investigational medicinal product (IMP) in oncology trials are more likely new, innovated treatments and their safety profile may not be fully assessed. Many of these IMPs have specific toxicities, such as general cytotoxic effect, on- or off-target toxicity effects. Combination treatment of two or more oncology products, which are commonly used in oncology clinical trials, may express different levels of toxicity in frequency, severity and duration. A risk-based safety monitoring approach will allow patient safety professionals to proactively protect the rights, safety and well-being of human subjects involved in oncology clinical trials and assure the quality, reliability and integrity of the safety data collected.

12:25 pm Transition to Lunch

12:30 Bridging Luncheon Presentation to be Announced

1:00 Coffee and Dessert Break in the Exhibit HallBioTelResearch

 

2:00 Close of Conference


Stay on and attend Part 2: Leveraging RWD for Clinical and Observational Research

PLENARY KEYNOTES

Tuesday Evening, Wednesday Morning, Wednesday Afternoon Plenary Keynotes Featuring:

SCOPE’s 2020 Participant Engagement Award, in Memory of Jerry Matczak
Patient Perspectives as an Input to Feasibility and Clinical Trial Design
Digital Trends that Are Changing Clinical Research
Health Literacy Throughout Drug Development – Why It Matters to Pharma and to Patients
Implementing an Innovation Methodology to Accelerate Clinical Trial Innovation within Your Organization

For more details on the Plenary Keynotes: www.scopesummit.com/keynotes
For more details on the Participant Engagement Award: www.scopesummit.com/participant-engagement-award

INTERACTIVE BREAKOUT DISCUSSION GROUPS

Concurrent breakout discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss some of the key issues presented earlier in the day’s sessions. Delegates will join a table of interest and become an active part of the discussion at hand. Bring your pharma, biotech, CRO, site, hospital or patient perspective to each of the discussions below. To get the most out of this interactive session and format please come prepared to share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and, most importantly, participate in active idea sharing: www.scopesummit.com/breakouts