Cambridge Healthtech Institute’s 5th Annual

Leveraging RWD for Clinical and Observational Research

RWD Innovation and Technologies

February 20-21, 2020


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, implementing novel data technologies, as well as close collaboration between pharmaceutical companies and organizations that possess the data. CHI’s 5th Annual Leveraging RWD for Clinical and Observational Research conference will discuss innovative approaches and technologies for RWD-based clinical and observational studies.

Arrive early and attend Part 1 (Wed - Thurs): Accessing and Generating RWD

Final Agenda

Thursday, February 20

11:30 am Registration Open

12:30 pm Bridging Luncheon Presentation to be Announced

1:00 Coffee and Dessert Break in the Exhibit HallBioTelResearch

 

2:00 Afternoon Plenary Keynotes

3:10 Booth Crawl & Refreshment Break in the Exhibit Hall. Last Chance for Exhibit Viewing

INNOVATIVE RWD-BASED STUDIES

4:10 Chairperson’s Remarks

Marc Berger, MD, Scientific Advisor, SHYFT Analytics

4:15 Hybrid Approaches for Data Collection in RWD

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

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.

4:45 Real-World Evidence Complements and Supplements Clinical Trial Designs

VanBrunt_DavidDavid Van Brunt, PhD, Senior Research Fellow and Head, HEOR Division of Evidence and Analytics AbbVie, Inc.

Regulators, notably FDA, increasingly recognize that real-world data (RWD) can improve the efficiency and effectiveness of clinical trials and can strengthen the regulator’s understanding of the benefit-risk presentation of molecules under consideration for approval. Use cases of RWD informing trial designs and being used in FDA decisions will be described, highlighting the diversity of RWD and its applications.

5:15 Improving Clinical Study Performance with Collaboration and Data during Protocol Development

DiCicco_RobRob DiCicco, Deputy Chief Health Officer, IBM Watson Health

Decisions made in clinical trial protocol design can have significant financial impacts during product development and commercialization. This session discusses the factors affecting protocol design and decision making and will present concrete ways to leverage the power of collaboration and data to overcome these challenges.

5:45 Hi-Lo: Pragmatic Trial of Higher vs. Lower Serum Phosphate Targets in Patients Undergoing Hemodialysis

MacKelfresh_AndyAndy MacKelfresh, Project Leader, Clinical Research Informatics, Duke Clinical Research Institute

The goal of this project is to determine whether less stringent control of serum phosphate versus the current standard approach will result in non-inferior rates of all-cause hospitalization among patients with ESRD undergoing hemodialysis. This presentation will describe the trial design and approach leveraging eConsent, source data from eHR, harmonization of data from multiple sources, involvement of dietitians, and reporting.

6:15 Networking Reception (Sponsorship Opportunity Available)

7:15 Close of Day

Friday, February 21

7:15 am Registration Open

7:45 Breakfast Presentation to be Announced

8:15 Session Break

RWD AS A CONTROL ARM

8:20 Chairperson’s Remarks

Chair to be Announced, Mendel Health

8:25 External Control Arms for Trial Development

David Tabano, PhD, Associate Director, Center for Observational Research & Data Sciences (CORDS), Bristol-Myers Squibb

The presentation will summarise the key methodologies used for external controls, distributing those into categories where statistical adjustment is provided to generate external control groups, and those where more simplistic selection criteria are applied to historic controls. For the purposes of this presentation, both techniques will fall under the remit of external study controls for consistency with regulatory and scientific literature surrounding this topic.

8:50 CASE STUDY: Use of Real-World Control Arm for Single Arm Trial Studies in Cancer

Jyotsna Mehta, Senior Director & Head, HEOR, Karyopharm Therapeutics

Shah_JatinJatin Shah, MD, CMO, Karyopharm Therapeutics

This case study will describe the definition of a real-world control arm, how to design a study using it alongside a single arm trial, and do’s and don’ts of this approach. It will provide examples of recently approved drugs and explain the scenarios where such approaches can be used.

PATIENT VOICE IN RWD-BASED RESEARCH

9:10 Chairperson’s Remarks

Virginia Spitzer, Executive Director, Foundation for Sarcoidosis Research

9:15 Fox Insight: Driving Discovery with the Patient Voice

Riley_LindseyLindsey Riley, MPH, Senior Associate Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson’s Research

Patient reported outcomes (PRO) data complements traditional clinical research with scale & accessibility, as well as patient-centricity. The Fox Insight (FI) study aims to gather the world’s largest dataset on the day-to-day experience of living with PD, from 125,00 participants across the globe. This comprehensive dataset holds the power to help researchers better understand patients’ unmet needs, redefine therapeutic priorities and optimize clinical trial design.

9:35 Patient Advocacy Collaboration: A Value RWD Resource

Spitzer_GingerVirginia Spitzer, Executive Director, Foundation for Sarcoidosis Research

This presentation would focus on an underutilized resource of RWD - the patient advocacy community. By reviewing how IRB-approved patient registries, observational studies, validated tools measuring patient experience, and other PROs collected can be utilized for industry, regulatory, and academic projects, RWD can be expanded to include the direct patient voice.

9:55 Leveraging Real World Data in Clinical Trials and Observational Research, Focusing the Patient Experience

Russell_ClaireClaire Russell, Executive Director, Patient Experience, PRA Health Sciences

Real world evidence is underutilized in interventional and observational studies. As a result, protocols are less patient-centric; patient recruitment timelines are being extended and promising drugs are not reaching patients fast enough. Due to this missed opportunity, patients are unable to access potentially life-saving treatment options.

10:25 Networking Coffee Break

AI FOR RWD APPLICATIONS

10:55 Chairperson’s Remarks

Jyotsna Mehta, Senior Director & Head, HEOR, Karyopharm Therapeutics

11:00 Machine Learning with Real World Data

John Page, MD, ScD, Medical Director and Lead of Predictive Analytics, Center for Observational Research, Amgen, Inc.

RWD provides opportunities to study drug utilization/safety of pharmaceutical products. Machine learning (ML) allows the development of automated algorithms for classification/prediction using data. ML offers opportunities to mine RWD for classification of medical images and prediction of medical events. However, RWD currently has a number of limitations, including incomplete and faulty data. The performance of ML in RWD is discussed and some solutions are offered.

11:30 Presentation to be Announced

12:00 pm Transition to Shared Sessions

AI TO ENABLE DIGITALIZATION OF CLINICAL TRIALS

12:00 Chairperson’s Remarks

Prasanna Rao, Head, AI & Data Science, Data Monitoring and Management, Clinical Sciences and Operations, Global Product Development, Pfizer Inc.

12:05 Real World Evidence Generation for Clinical Decision Making: Can AI Solve the Challenges?

Bartels_DorotheeProfessor Dr. Dorothee Bartels, Clinical and Real World Data Strategist, X The Moonshot Factory

Real World Evidence Generation (RWE) is continuously gaining importance, but trust in results, transparency and reproducibility are challenging. The same is true for prediction models based on learning algorithms. Questions are arising: who may be the best expert in big data analysis; who may have the appropriate skill sets; and can clinical decisions be based on real world data analysed by either traditional or artificial intelligence (AI) methods? Complementary approaches using synergies from the epidemiological and AI field may boost the field of RWE.

12:25 Re-Skilling for AI/ML: Leveraging Your SMEs

Katan_NechamaNechama Katan, Associate Director, Data Monitoring and Management, Clinical Sciences and Operations, Global Product Development, Pfizer

AI/ML are very powerful tools for clinical trials. However, there is a gap between those that understand what AI/ML can do for the business and the business SME (subject matter experts) who really understand the business problems. Without strong SME engagement in solutions, technical solutions are often at risk. This talk will review successful case studies for developing “lego” employees/teams who help bridge the gaps between AI/ML technologist and the SMEs. We will discuss both the how and what that makes an AI/ML project successful in clinical trials.

12:45 PANEL DISCUSSION: AI Implementation: Technology, Data, People

Rao_PrasannaModerator: Prasanna Rao, Head, AI & Data Science, Data Monitoring and Management, Clinical Sciences and Operations, Global Product Development, Pfizer Inc.


Panelists: Balazs Flink, PhD, Head, Clinical Trial Analytics, R&D Business Insights, Bristol-Myers Squibb

Faisal Khan, PhD, Executive Director, Advanced Analytics and Artificial Intelligence, AstraZeneca

Arun Patnaik, Director, Clinical Data Management, Data Operations, Novartis

Malaikannan Sankarasubbu, Vice President, AI Research, Saama Technologies

It was proven that machine learning and AI can aid clinical development in various aspects. With evolving AI technology implementation challenges become more and more noticeable. This panel discussion will brainstorm the key pain points of AI implementation:

  • What is the best technology and how to work with technology providers?
  • How to make all data machine learnable and available for AI applications
  • How to solve “the people puzzle”

1:05 Transition to Lunch

1:10 SCOPE Send Off Luncheon Presentation to be Announced

1:40 Closing Remarks

1:45 SCOPE Summit 2020 Adjourns

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