NEWS & EVENTS

Digital Clinical Trials Workshop: Creating a Vision for the Future

April 1 - 2, 2019
National Institutes of Health, Lister Hill Auditorium

Description

The National Heart, Lung, and Blood Institute (NHLBI), in partnership with the National Science Foundation (NSF) and the NIH Office of Behavioral and Social Sciences Research (OBSSR), hosted a workshop on April 1-2, 2019 to discuss the potential transformation of NIH-funded clinical trial research by leveraging digital technologies including data analytics. Development of diverse digital technologies and analytics has impelled a paradigm shift in how clinical trials can be conducted. This development points to opportunities to advance the clinical trial process on both scientific and cost effective grounds while moving more towards a more patient-centered trial experience. This workshop convened experts in the field of clinical trials and digital health technology to identify best practices, gaps, barriers, and future priorities for leveraging digital technologies for clinical trials.

Recap

Background

Clinical trial research is a key component of the NIH research portfolio, and using digital technology to conduct research could be transformative. Digital technology has the potential not only to reduce the cost and time for research, but it may also benefit patients by offering research opportunities that are more patient-centered with the ability to quickly address critical health-related questions.

The use of advanced analytics, such as artificial intelligence (AI), can facilitate better screening of patients, ensuring that representative groups of appropriate participants will be selected for trial interventions, and may predict response to interventions. Likewise, the use of long-distance communications technology, such as FaceTime, Skype, telehealth services, and other types of virtual visits, can assure more complete patient follow-ups — and in the end, more successful trial outcomes.

Before making any decisions about how to advance the use of this technology, however, researchers must consider a host of issues, including racial, ethnic, and socioeconomic equity, aging populations, gender differences, cultural norms, and participant preferences that might influence access and utilization, and how computational simulations could be used to reduce the sample size and length of trials.

Workshop Objectives

  1. Determine how best to leverage digital technology to transform clinical trials (e.g., decrease cost, improve enrollment and retention, adherence).
  2. Review best practices that enhance the design and conduct of clinical trials, such as the use of videos, mobile apps, text-based messaging, wearable sensors, and other platforms.
  3. Determine how to increase participant diversity and curb the "digital divide".
  4. Determine what can be done now and what are future priorities.
  5. Discuss implementation strategies using digital technology in future clinical trials, including workforce considerations and training needs.

Workshop Outcomes

Objective Strategies
1. Leverage digital technology to transform clinical trials
  1. Capitalize on existing technologies and research platforms that also serve to inform new technologies or platforms as technology advances.
  2. Use system application programming interfaces (APIs) to create a flexible information infrastructure that can connect electronic health data, from different health systems, to launch mobile apps for research enabling the use of patient-generated data.
  3. Harness direct-to-consumer technology for use in clinical trials.
  4. Leverage current knowledge on enhancing patient engagement with technology that can be utilized in clinical trials.
  5. Explore the use of AI algorithms to address data challenges (e.g., security, data access, integration with downstream systems) in implementing automation approaches to achieve cost and time savings.
  6. Use advanced analytics through AI to perform deeper learning into the data and make predictions.
2. Enhance clinical trials with technology
  1. Use standard protocol templates to increase protocol-driven automation (e.g., data-matching) for recruiting participants.
  2. Encourage or require use of standard data elements where possible.
  3. Create a database for standardized phrases/criteria to increase standardization of data for use in clinical trials through government and industry partnerships.
  4. Collect intensive longitudinal data to monitor participants for change and create models of trial impact that can shorten trial length.
  5. Reduce burden of visits by deploying technology that can be used outside of the clinic.
  6. Revisit how to monitor and report adverse events using technology to effectively monitor safety since it may be easier and more reliable to report in near real-time.
  7. Sustain digital engagement by providing research participants high treatment value: goal-related interactivity, feedback, support, accountability.
3. Increase participant diversity
  1. Use digital technology to increase access to study participants.
  2. Develop a plan to address low technology literacy.
  3. Use patient-centered design model in the trial design and technology development phase.
  4. Engage participants digitally by using a “personal touch” for recruitment and retention by tailoring messages based on personal preferences and motivating factors, which may require gathering that data on personal preferences to help develop digital trial development.
  5. Address Internet access issues.
4. Implementation strategies using digital technology; what can be done now and future priorities

What can be done now:

Form cross-disciplinary partnerships with industry, government and academia to learn and work through complex processes with digital trials. This partnership would work towards the following:

  1. Gather empirical research on the risks and benefits of digital trials.
  2. Establish criteria for what level of technology engagement verifies as adequate receipt or dosage of a digital intervention.
  3. Engage champions in underserved communities, including rural communities, for developing digital trials to prevent widening a digital divide by striving to meet community needs, build community trust, and include diverse research teams.
  4. Increase technological and data science knowledge among all levels involved with conducting trials – i.e., investigators, research team, clinical staff, regulatory officials - in order to successfully develop and implement digital trials.
  5. Address privacy and security concerns, training protocols, potential negative impact of using technology.

What can be done in the future:

  1. Build an evidence base of effective intervention components that can readily inform future intervention development.
  2. Aim for evidence that maximizes knowledge accumulation and transfer by looking at digital intervention individual components, proximal outcomes, and efficient study design (e.g., factorial experiments, micro-randomized trials, system ID experiments).
  3. Enable clinical research systems built around capabilities that use technologies and are focused on the needs of patients to achieve full potential of digital clinical trials.
  4. Incorporate Food and Drug Administration (FDA) guidelines for cybersecurity into digital trials.
5. Implementation strategies including workforce considerations
  1. Transform research teams to include computer scientists and engineers as critical partners.
  2. Build a workforce that can review complex and innovative designs, analytic standards, etc.
  3. Use digital security and privacy experts to tailor data safety monitoring plans.
  4. Require education on digital technology for Institutional Review Board (IRB) and Data Safety and Monitoring Board (DSMB) members along with investigators and other research staff by incorporating FDA guidelines on cybersecurity and other best practices.
  5. Invest in underrepresented communities for next generation workforce in technology, clinical trials, and research.

WORKSHOP CHAIR

  • Robert Califf, MD, MACC; Professor of Medicine, Division of Cardiology, Duke University; Verily

WORKSHOP LEADERS

  • Wendy J. Nilsen, PhD, Smart and Connected Health Program, NSF
  • Erin Iturriaga, DNP, MSN, RN, Division of Cardiovascular Sciences, NHLBI
  • Rebecca Campo, PhD, Division of Cardiovascular Sciences, NHLBI
  • Patrice Desvigne-Nickens, MD, Division of Cardiovascular Sciences, NHLBI
  • Yves Rosenberg, MD, MPH, Division of Cardiovascular Sciences, NHLBI

WORKSHOP SPEAKERS/MODERATORS IN ORDER LISTED IN WORKSHOP AGENDA

  • David Goff, MD, PhD, Division Director, Division of Cardiovascular Sciences, NHLBI
  • Walter (Rance) Cleaveland, PhD, Division Director, Computing and Information Science and Engineering, NSF
  • Steven Steinhubl, MD, Director of Digital Medicine, Scripps Research Translational Institute
  • Amy Abernethy, MD, PhD, Deputy Commissioner, FDA
  • Pamela Tenaerts, MD, MBA, Executive Director, Clinical Trial Transformation Initiative, Duke University
  • Mark Pletcher, MD, MPH, Director Engagement of Participants and mHealth Data Collection, University of California San Francisco
  • Naveen Pereira, MD, FACC, FAHA, Mayo Clinic Rochester
  • Craig Lipset, MBA, Pfizer
  • John Wilbanks, BA, Chief Commons Officer, Sage Bionetworks
  • Gil Alterovitz, PhD, Harvard University
  • Scarlet Shore, Verily
  • Beth Mynatt, PhD, Institute for People and Technology, Georgia Tech
  • Robert Harrington, MD, Chair, Department of Medicine, Stanford University; President, American Heart Association
  • Mintu Turakhia, MD, MAS, Director of Center for Digital Health, Stanford University
  • Ken Mandl, MD, MPH, Harvard University/Boston Children’s
  • Adrian Hernandez, MD, Duke University
  • Kenzie Preston, PhD, National Institute on Drug Abuse
  • Bonnie Spring, PhD, Northwestern University
  • Steven Grinspoon, MD, Professor of Medicine, Harvard Medical School
  • Don S. Dizon, MD, FACP, FASCO, Director of Medical Oncology, Rhode Island Hospital; Professor of Medicine, Brown University
  • Harmony Reynolds, MD, Associate Professor of Medicine, NYU School of Medicine
  • Luis Belen, Chief Executive Officer, National Health IT Collaborative for the Underserved
  • Kandi Velarde, MPH, Veterans Administration
  • Sara Czaja, PhD, Cornell University
  • Herman Taylor, MD, Director, Cardiovascular Research Institute, Morehouse School of Medicine
  • Sheila Prindiville, MD, MPH, Director, Coordinating Center for Clinical Trials, Office of the Director, National Cancer Institute (NCI)
  • Harlan Krumholz, MD, Professor of Medicine, Yale University; Director, Yale-New Haven Hospital Center for Outcomes Research and Evaluation
  • Benjamin Marlin, PhD, College of Information and Computer Sciences, University of Massachusetts at Amherst
  • Predrag Klasjna, PhD University of Michigan
  • Bharat Rao, PhD, KPMG
  • Teresa Zayas-Caban, PhD, Office of the National Coordinator for Health Information Technology, U.S. Department of Health and Human Services (HHS)
  • Bakul Patel, PhD, Center for Devices and Radiologic Health, FDA
  • Ivor Prichard, PhD, Office of Human Research Protection, HHS
  • Anna Kravets, PhD, Merck
  • Omer Inan, PhD, School of Electrical and Computer Engineering, Georgia Tech

FEDERAL WORKING GROUP PARTICIPANTS

  • Dana Wolff-Hughes, PhD, NIH Office of Behavioral and Social Sciences Research (OBSSR)
  • Julie Bamdad, MSE, Division of Lung Disease, NHLBI
  • Katharine Cooper-Arnold, MPH, Division of Cardiovascular Sciences, NHLBI
  • Brad Newsome, PhD, Center for Translational Research and Implementation Science, NHLBI
  • Rebecca Roper, MS, MPH, Center for Translational Research and Implementation Science, NHLBI
  • Chris Sasiela, PhD, RAC, Office of Translational Alliances and Coordination, NHLBI
  • Holly Nicastro, PhD, Division of Cardiovascular Sciences, NHLBI
  • Albert Lee, PhD, Division of Cardiovascular Sciences, NHLBI
  • Nicole Redmond, MD, PhD, MPH, Division of Cardiovascular Sciences, NHLBI
  • Vandana Sachdev, MD, Division of Cardiovascular Sciences, NHLBI
  • Paula Schum, MSN, RN, Division of Cardiovascular Sciences, NHLBI
  • Susan Czajkowski, PhD, Chief of the Health Behaviors Research Branch, National Cancer Institute (NCI)
  • April Oh, PhD, Health Communication and Informatics Research Branch, NCI
  • Tiffani Lash, PhD, National Institute of Biomedical Imaging and Bioengineering (NIBIB)
  • Richard Conroy, PhD, Office of Strategic Coordination, NIH Common Fund
  • Courtney Aklin, PhD, Chief of Staff, National Institute on Minority Health and Health Disparities (NIMHD)
  • Beda Jean-Francois, PhD, Clinical and Health Services Research Division, NIMHD
  • Valerie Florance, PhD, Extramural Program Director, National Library of Medicine (NLM)
  • Nick Langhals, PhD, Division of Translational Research, National Institute of Neurologic Disorders and Stroke (NINDS)
  • Codrin Lungu, MD, Division of Clinical Research, NINDS

Agenda

9:00 - 9:10 AM
-
Welcome and Logistics

9:10 - 9:25 AM
-
Opening Remarks from the Division Director of NHLBI and NSF Division Director for Computer and Information Science and Engineerin
Investment in Clinical Trials: A Path Forward

David Goff, MD, PhD; Director, Division of Cardiovascular Sciences, National Heart, Lung, Blood Institute

Walter (Rance) Cleaveland, PhD; Director, Division of Computing and Communication Foundations, National Science Foundation

9:25 - 9:50 AM
-
Workshop Objectives, Charge, and Presentation
Digital Clinical Trials and the 21st Century Cures Act

Robert M. Califf, MD, MACC; Duke University

9:50 – 10:30 AM
-
KEYNOTE SESSION

Digital Clinical Trials: It’s Not (all) About The Tech
Steven Steinhubl, MD; Scripps Research Translational Institute - Director of Digital Medicine

Digital Clinical Trials: It’s Not (all) About The Tech (Part Deux)
Amy Abernethy, MD, PhD: Deputy Commissioner, Food and Drug Administration

10:30 – 10:45 AM
-
MORNING BREAK

10:45 AM – 12:10 PM
-
SESSION 1
CURRENT STATUS OF DIGITAL TRIALS AND THE PATH FORWARD

Session Moderator: Pamela Tenaerts, MD - Executive Director, Clinical Trial Transformation Initiative (CTTI)

Clinical Trials Transformation Initiative: Decentralized Clinical Trials and Mobile Technologies
Pamela Tenaerts, MD, MBA; Duke University

Direct Engagement of Participants and mHealth Data Collection using The Eureka Research Platform
Mark Pletcher, MD, MPH; University California San Francisco

Trials, Tribulations, Victories - Transitioning a Clinical Trial to a Registry Using Digital technology
Naveen Pereira, MD; Mayo Rochester

What Makes a Digital Trial in Medicine Development?
Craig Lipset, MBA; Pfizer

PANEL DISCUSSION

12:10 – 12:45 PM
-
LUNCH BREAK

12:45 – 1:45 PM
-
SESSION 2
RECRUITMENT AND RETENTION IN DIGITAL TRIALS

Session Moderator: John Wilbanks, Chief Commons Officer at Sage Bionetworks

Ethics Frameworks for Digital Clinical Trials
John Wilbanks, B.A.; Sage Bionetworks

Recruitment with social media, digital registries, EHR and other digital strategies
Gil Alterovitz, PhD; Harvard University

A Patient-Centric Approach to Clinical Research
Scarlet Shore; Verily

Techniques to Improve Patient Recruitment, Engagement and Retention in Digital Trials
Beth Mynatt, PhD; Institute for People and Technology (IPaT), Georgia Tech

PANEL DISCUSSION

1:45 – 3:15 PM
-
SESSION 3
DELIVERING INTERVENTIONS IN DIGITAL SPACE

Session Moderator: Robert Harrington, MD; Professor of Medicine and Chairman of the Department of Medicine at Stanford University Stanford University, President, American Heart Association

Measuring Interventions, Outcomes and Biomarkers Digitally
Mintu Turakhia, MD, MAS; Director of Center for Digital Health Stanford University

A SMART Approach to Clinical Trial Data Generation and Workflow: A Standards-based Interface to Health Systems and the Patient
Ken Mandl, MD, MPH; Harvard University/Boston Children’s (Pediatric Perspective)

Bridging the Digital Divide in Health Systems for Clinical Trials
Adrian Hernandez, MD; Duke University (Adult Perspective)

Navigating Safety Monitoring in Digital Clinical Trials
Kenzie Preston, PhD; National Institute on Drug Abuse/National Institutes of Health

Strategies for Maintaining Participant Engagement with Digital Trials
Bonnie Spring, PhD; Northwestern University

PANEL DISCUSSION

3:15 – 3:35 PM
-
CLINICAL TRIALIST PANEL DISCUSSION

  1. Steven Grinspoon, MD; Professor of Medicine, Harvard Medical School
  2. Don S. Dizon, MD, FACP, FASCO; Director of Medical Oncology, Rhode Island Hospital, Professor of Medicine, Brown University
  3. Harmony Reynolds, MD, Saul J. Farber Associate Professor of Medicine at NYU School of Medicine

3:35 – 3:45 PM
-
AFTERNOON BREAK

3:45 – 5:00 PM
-
SESSION 4
ARE WE WIDENING OR BRIDGING A DIGITAL DIVIDE WITH DIGITAL CLINICAL TRIALS?

Session Moderator: Luis Belen; Chief Executive Officer at National Health IT Collaborative for the Underserved

Title: pending
Kandi Verlarde, MPH; VA Cooperative Studies Program

Older Adults and Digital Research: What Have We Learned and Where Do We Need to Go
Sara Czaja, PhD; Cornell University

Access, Utilization Patterns, and Preferences Among Subgroups That Can Lead to Differential Benefits or Disparities
Herman Taylor, MD; Director of Cardiovascular Research Institute, Morehouse School of Medicine

PANEL DISCUSSION

5:00 PM
-
AJOURN DAY ONE

9:00 - 9:15 AM
-
Opening Remarks from the Director of NCI’s Coordinating Center for Clinical Trials (CCCT}
Leveraging Digital Technology for Cancer Clinical Trials

Sheila Prindiville, MD, MPH; Director, Coordinating Center for Clinical Trials, Office of the Director, National Cancer Institute

9:15 – 10:15 AM
-
SESSION 5
DIGITAL TRIAL ANALYTICS

Session Moderator: Dr. Harlan Krumholz, MD; Professor of Medicine, Yale University, Director of the Yale-New Haven Hospital Center for Outcomes Research and Evaluation

Advanced Analytics Including Artificial Intelligence 
Benjamin Marlin, PhD; College of Information and Computer Sciences at UMass Amherst

Learning How and When to Optimally Intervene: Trials and Analytics for the Design of Precision Behavioral Interventions
Predrag Klasjna, PhD; University of Michigan

DIGITAL TRIAL ANALYTICS: Analytics at Scale with Real World Data
Bharat Rao, PhD; KPMG

PANEL DISCUSSION

10:15 – 11:15 AM
-
SESSION 6
ENVIRONMENTAL AND REGULATORY ASPECTS OF DIGITAL CLINICAL TRIALS

Session Moderator: Teresa Zayas-Caban, PhD; Office of the National Coordinator

FDA Mobile Digital Health Guidance
Bakul Patel, PhD; Food and Drug Administration, Center for Devices and Radiologic Health

Digital Clinical Trials and Human Research Subject Protections: Some Thoughts
Ivor Pritchard, PhD; Office of Human Research Protection, Health and Human Services

PANEL DISCUSSION

11:15 AM – 12:30 PM
-
SESSION 7
Workshop Summary: Critical Implications, Remaining Questions & Digital Trial Needs

Session Moderator: Omer Inan, PhD; Georgia Tech, School of Electrical and Computer Engineering

Clinical Trialist Panel Discussion:

Steven Grinspoon, MD; Professor of Medicine, Harvard Medical School

Don S. Dizon, MD, FACP, FASCO; Director of Medical Oncology, Rhode Island Hospital, Professor of Medicine, Brown University

Harmony Reynolds, MD, Saul J. Farber Associate Professor of Medicine at NYU School of Medicine

  1. Critical Implications for Clinical Trials
  2. Digital Trial Needs
  3. Remaining Questions

12:30 – 1:00 PM
-
WRAP UP AND CONCLUSION