Cambridge Healthtech Institute’s 4th Annual

Leveraging RWD for Clinical and Observational Research

Data Integration and Real-Time RWE Generation

February 20-21, 2019

The abundance of data generated during routine health care is growing in significance and should be used for clinical and observational research. Patient electronic records, registries, data from pharmacy and social media, and wearable sensors have been increasingly used as eSources. This process requires strategizing, utilizing novel data technologies, as well as close collaboration between pharmaceutical companies and organizations that possess the data. Cambridge Healthtech Institute’s 4th Annual Leveraging RWD for Clinical and Observational Research conference will discuss challenges and solutions with secondary use of existing healthcare data to support the medicine lifecycle.

Final Agenda

Arrive early and attend Part 1 (Tues-Wed): Late Stage Research Strategy and Operations

Wednesday, February 20

11:30 am Registration Open


Jane Quigley, Senior Vice President, Medical Informatics, PRA Health Sciences

1:10 Coffee and Dessert Break in the Exhibit Hall

2:10 Plenary Keynotes

3:20 Booth Crawl & Refreshment Break in the Exhibit Hall, Last Chance for Exhibit Viewing


4:05 Chairperson’s Remarks

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

4:10 Presentation to be Announced

4:40 Challenges and Opportunities with Use of Real World Evidence in Drug Development

Alemayehu_DemissieDemissie Alemayehu, PhD, Vice President, Biostatistics, Pfizer

With the growing demand for new medicines to meet critical healthcare needs with speed and efficiency, it has become essential to explore novel approaches and data sources. Thanks to the prevailing digital revolution, and advances in predictive analytics and computing platforms, a new frontier has emerged to enhance the drug development process. We elucidate pertinent aspects of the use of real world evidence in regulatory settings, with emphasis on study design, analytical strategies, data quality and regulatory requirements.

5:40 RWD Strategies for Improving Development and Access to Innovative Therapeutics

Daniel_gregGregory Daniel, PhD, Deputy Center Director, Duke-Margolis Center for Health Policy, Duke University

This presentation will include an overview of the requirements on FDA, established by the sixth reauthorization of PDUFA and the 21st Century Cures Act, to consider the use of RWE. This session will also include emerging recommendations by stakeholders regarding a framework and approaches for evaluating data quality, improving observational studies, and how broader RWE might be considered along with other factors to reach regulatory evidentiary requirements.

6:107:10 Networking Reception (Sponsorship Opportunity Available) or Close of Day

Thursday, February 21

7:15 am Registration Open

7:45 BREAKFAST PRESENTATION (Sponsorship Opportunity Available) or Morning Coffee

8:15 Session Break


8:20 Chairperson’s Remarks

Chairperson to be Announced

8:25 CO-PRESENTATION: Delivering the Unicorn of Novel Trial Methodologies in the UK

Collier_SueSue Collier, UK National Physician Lead, Respiratory, GlaxoSmithKline

ChadhaManek_DivyaDivya Chadha Manek, Head, Business Development and Marketing, National Institute for Health Research (NIHR)

During this presentation, you will hear from GlaxoSmithKline (GSK) on delivering the Salford Lung Study: a pioneering real-world, community-based clinical trial, and gain an understanding of the benefits and challenges of setting up and delivering a real world trial which bridges the gap between, and combines the strength of the traditional RCT model and an observational trial. Join it to learn how the National Institute for Health Research (NIHR), a globally unique government-funded integrated health research system, supported the delivery of this unique trial.


9:00 CO-PRESENTATION: Real-World Data & Analytics Empowered Clinical Trials Design

Wang_XiaXia Wang, PhD, Director, Health Informatics & Global Medicines Development, AstraZeneca

Fang_JaneJane Fang, MD, Head, Clinical Business Management & Analytics, MEDI Biologics Unit, AstraZeneca

Patient-centric drug product development starts with a characterization of the targeted patient population. Real world evidence data (RWD) provides a new way to gain such insights. Incorporating RWD-use into current study planning process will advance and innovate trial design, conduct and patient recruitment. The talk will feature a RWD-empowered trial feasibility and patient recruitment process that has joined the expertise from different fields such as feasibility function, informatics and data scientists, study teams, technologists and sites.

9:35 Data-Driven Patient Recruitment with Real World Data

Jin_LipingLiping Jin, Data-Driven Patient Recruitment Lead, Pharma Research and Early Development, pREDi Early Development Workflows, Roche

With the increasing use of Real World Data (RWD) in the pharma industry, Data-Driven Recruitment (DDR) team at Roche Pharm Research & Early Development (pRED) would like to share our experience of integrating RWD (e.g. insurance claims, EMR) with trial metrics data to optimize study protocol design and target patient recruitment strategy. While the team has received positive feedback from our business partners (translational medicine, clinical program teams, and study leaders), we would also like to share the challenges to expanding the effort in broader US, Europe and international settings.

9:55 Talk Title to be Announced

Mandziuk_Kathleen_PH42Kathleen Mandziuk, Vice President, Patient Strategy, PRA Health Sciences

10:25 Networking Coffee Break (Sponsorship Opportunity Available)


11:10 Chairperson’s Remarks

Chairperson to be Announced

11:15 Supplementing Clinical Trials in Oncology with Real World Evidence

Sail_KavitaKavita Sail, PhD, Associate Director, Health Economics and Outcomes Research, AbbVie

Real world evidence is growing in importance and can be used effectively in oncology to test sub-populations, identify risk groups and even inform the right population prior to conducting a clinical trial. Studies using big data require expertise in handling and pose unique challenges related to potential bias and complex data management strategies. It’s important that these studies are conducted with expert data scientists alongside medical and clinical reviewers for correct data interpretation.

11:45 Presentation to be Announced

12:15 pm Transition to Shared Sessions


Chairperson’s Remarks

Ramzi Najm, Senior Associate, Waife & Associates

12:20 Blockchain Opportunities for Patient Data Donation & Clinical Research

Baara_MuntherMunther Baara, MS, Head, New Clinical Paradigm, Pfizer

Imagine a solution that makes it easy to aggregate health data in a secure, trusted, automated, and error-free way. A solution which enforces rules, privacy, and regulations in a mutually agreed upon manner, resulting in a smart-contract between patient and healthcare stakeholders. This enables patients to aggregate their data from diverse health sources and share what they choose to with their physicians and researchers.

12:35 Blockchain’s Opportunity Today and Potential for the Future

Postings_MalMal Postings, Vice President, Head, Innovation/Emerging Technologies & Chief Architect, Research & Development Solutions, IQVIA

Today, blockchain is about a data workflow using general ledger type sharing of information. Here, it is important to understand the role of a governing body to engage the stakeholders and own the smart contract rules of working. The future is moving more broadly into trusted distributed data sharing. This will start with enablement of distributed queries and then move into the ability to construct virtual data stacks.

12:50 Blockchain and Pragmatism: A Necessary Marriage

Waife_RonaldRonald Waife, MPH, President, Waife & Associates, Inc.

Biopharma is improving its track record in adopting advances in software and work process. However, the use of blockchain technologies may be too immature and unproven to expect rapid incorporation into clinical research. A productive approach for biopharma may be to select a focused business problem. For instance, the “mining” of data from RWD sources could be more feasible with blockchain security. But biopharma will need to follow best practices for technology evaluation, process impact, compliance assurance, vendor management and user acceptance.

1:05 INTERACTIVE PANEL: Blockchain in Clinical Research

Najm_RamziModerator: Ramzi Najm, Senior Associate, Waife & Associates

The most significant costs to clinical trials are in time and resources to insure the com-pleteness, accuracy and integrity of patient data. Blockchain technology has the potential to transform and simplify the exchange of data among business partners in clinical re-search. Can blockchain solutions be applied to reduce the time to bring new biopharmaceu-tical products to market while reducing the cost of achieving that objective? The presenta-tions and discussion will address this opportunity and the path to its implementation.

  • What is the realistic path for the adoption of innovations such as block chain for sponsors, sites and CROs?
  • Do service providers (CROs) play a leading or trailing role in the facilitating for the industry and why?
  • Unlike EDC, block chain technology requires sites to take an active role rather than waiting for sponsors/CROs to deliver the capabilities, how does that impact adoption?
  • Thoughts on global adoption
  • Thoughts on business process implications and feasibility for transition

1:20 Transition to Lunch

1:25 LUNCHEON PRESENTATION (Sponsorship Opportunity Available)

1:55 Closing Remarks

2:00 SCOPE Summit 2019 Adjourns

Arrive early and attend Part 1 (Tues-Wed): Late Stage Research Strategy and Operations


“SCOPE Featured Author”
Emmanuel Fombu, MD,
Director, Digital Health Solutions

Signature Sponsors

Premier Sponsors







Mendel Health

PRA Health Sciences 

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