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 (Regency Rotunda)

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 (Regency Ballroom S)

6:20 - 7:30 pm SCOPE’s Kick-Off Networking Happy Hour (Upper Pool Deck, 4th Floor)

(Co-Sponsorship Opportunities Available)

7:30 pm Close of Day

Wednesday, February 19

7:15 am Registration Open (Regency Rotunda) and Morning Coffee (Regency Ballroom S Foyer)

8:15 Morning Opening Plenary Keynotes with Light Refreshments (Regency Ballroom Sprovided by Clinical_Ink_NoTagline

9:40 Grand Opening Coffee Break in the Exhibit Hall (Windermere Ballroom)


Celebration 7-8

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.

11:15 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.

11:45 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.

PRA_New12:15 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 (Windermere Ballroom)Abond


2:15 Chairperson’s Remarks

Andrew Rosner, Vice President, Real World & Late Phase, 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

Locantore_NickNick Locantore, Senior Director, Digital Data Integration and Analytics, Value Evidence & Outcomes, 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 RWD in Oncology and Immuno-Oncology Research

Kelsh_MichaelMichael Kelsh, PhD, Executive 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.

3:50 Organizing EMR data with AI

Galil_KarimKarim Galil, CEO & Co-Founder,

Mendel built the world's first AI machine that can ingest any data type from different EMR systems and decipher it to generate analytics-ready datasets in near realtime. The company will showcase a live demo and a side-to-side comparison to standard manual data extraction methods. 


4:20 Find Your Table and Meet Your Moderator

For more details on the Breakout Discussions:

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 (Windermere Ballroom)d_wise_NEW

(Co-Sponsorship Opportunities Available)

6:45 Close of Day

Thursday, February 20

7:15 am Registration Open (Regency Rotunda)

7:45 BREAKFAST PRESENTATION: Navigating the Patient Journey: A Responsibility all Technology Providers Share (Regency Ballroom T)

Nolte_MikeMike Nolte, Chief Executive Officer, Signant Health

8:15 Session Break


8:20 Chairperson’s Remarks

Rachel Sobel, DrPH, FISPE, Vice President, Epidemiology, United Biosource Corporation (UBC)

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

Larson_LeanneLeanne Larson, Corporate Vice President, WW Head, Real-World Evidence, 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 NEW: Technologies that Enable Fit for Purpose Applications of RWD: Enriching a Rare Disease Registry through Linkage to Secondary Data

Aaron Berger, Senior Director, RWE, Clinical Operations, United Biosource Corporation (UBC)

We will examine technological capabilities that support the assembly of fit for purpose data through the lens of a rare disease, non-interventional study enriched with secondary data through a robust IT architecture, informed by epidemiological expertise.


10:25 Coffee Break in the Exhibit Hall (Windermere Ballroom)Aris_Global_New


11:20 Chairperson’s Remarks

Leanne Larson, Corporate Vice President, WW Head, Real-World Evidence Parexel

11:25 Patient‐Community Perspectives on Real‐World Evidence

Oehrlein_ElisabethElisabeth M. Oehrlein, PhD, MS, Senior Director, Research and Programs, National Health Council

To advance a patient-centered health ecosystem, patient perspectives must be considered as researchers, policy makers, and regulators establish standards for collecting and applying real-world evidence (RWE) in decision-making. The patient community’s role in RWE is not widely understood. This presentation will describe findings from a convening on patient-community views on RWE, related concerns, and the communications, information, and tools needed by patients to understand, trust, contribute to and use RWE.

11:55 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.

12:25 pm Transition to Lunch

Medidata 12:30 BRIDGING LUNCHEON PRESENTATION: Supercharge Study Design and Feasibility: AI with Integrated RWD and Cross-Industry Clinical Trial Data/Metrics

Jef Benbanaste, Senior Director, Product Lead, Acorn AI by Medidata, a Dassault Systèmes company

While RWD can support both clinical and operational trial planning, its value is maximized when analyzed together with data and metrics from a large set of cross-industry clinical trials. This session will share perspectives on how to select fit-for-use RWD sources, manage and transform RWD, and apply analytics in conjunction with clinical trial data for use cases such as Synthetic Controls, protocol optimization, and site feasibility.

1:00 Coffee and Dessert Break in the Exhibit Hall (Windermere Ballroom)BioTelResearch


2:00 Close of Conference

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


Tuesday Evening, Wednesday Morning and Thursday 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:
For more details on the Participant Engagement Award:


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:

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