May 29, 2020: Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Experiences from the Collaboratory PCTs (Wendy Weber, ND, PhD, MPH; Jerry Jarvik, MD, MPH; Lynn DeBar, PhD, MPH; Doug Zatzick, MD; Vince Mor, PhD)

Speakers

Guest Moderator
Wendy Weber, ND, PhD, MPH
Branch Chief, Clinical Research in Complementary and Integrative Health
Division of Extramural Research
National Center for Complementary and Integrative Health, NIH

Panel
Jeffrey (Jerry) G. Jarvik, MD, MPH
LIRE Demonstration Project
Professor of Radiology, Neurological Surgery and Health Services
Adjunct Professor of Pharmacy and Orthopedics & Sports Medicine
Co-Director, Comparative Effectiveness, Cost and Outcomes Research Center
Director, UW CLEAR Center for Musculoskeletal Disorders
University of Washington School of Medicine

Lynn DeBar, PhD, MPH
PPACT Demonstration Project
Senior Scientist
Kaiser Permanente Washington Health Research Institute

Doug Zatzick, MD
TSOS Demonstration Project
Professor, Department of Psychiatry and Behavioral Sciences
University of Washington School of Medicine

Vince Mor, PhD
PROVEN Demonstration Project
Florence Pirce Grant University Professor and Professor of Health Services, Policy and Practice
Brown University School of Public Health

Topic

Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Experiences from the Collaboratory PCTs

Keywords

EHRs; PCTs; Embedded PCTs; NIH Collaboratory; Demonstration Project; PROVEN; LIRE; PPACT; TSOS; Patient-reported outcomes; PROs; Data collection

Key Points

  • Lessons and experiences from the Demonstration Projects include:
    • Pilot the intervention at the partnering health systems and work closely with site programmers who know the systems best.
    • Monitor the trial’s implementation at regular intervals to detect breakage.
    • Keep both the intervention and the outcomes as simple as possible.
    • Choose endpoints that matter to patients and providers and that are captured reliably as part of routine clinical care.
  • Enabling rapid outcome ascertainment in large pragmatic trials can be a game changer.
  • The Emergency Department Information Exchange (EDIE) in Washington state is an innovative technology platform that can help with the collection of population-level administrative data for acute care follow-up and ongoing care plans.
  • It might be better to uncouple some types of patient-reported outcome (PRO) data collection from the routine clinical care visit.

Discussion Themes

A common theme throughout all the Demonstration Project PCTs is how dynamic and ever-changing health care delivery settings are. The reality is that the trial as planned is often not the trial as piloted or conducted.

What type of education is most effective about how to use a PRO to inform care plans and decision-making?

There is nothing like a crisis (eg, the coronavirus pandemic) for people to do things that were before thought impossible. Now, the concept of a “visit” is changing, and there will likely be more uncoupling.

Read more about all 19 of the NIH Collaboratory’s Demonstration Projects.

Tags

#pctGR, @Collaboratory1

May 15, 2020: Optimized Learning While Doing: The REMAP-CAP Adaptive Platform Trial (Derek Angus, MD, MPH)

Speaker

Derek C. Angus, MD, MPH, FRCP
Distinguished Professor and Mitchell P. Fink Endowed Chair
Department of Critical Care Medicine
University of Pittsburgh and UPMC Health System

Topic

Optimized Learning While Doing: The REMAP-CAP Adaptive Platform Trial

Keywords

Adaptive study design; REMAP-CAP; Community-acquired pneumonia; Embedded research; Learning health system; Pandemic; Response-adaptive randomization; Global adaptive platform; COVID-19

Key Points

  • The Randomised, Embedded, Multifactorial, Adaptive Platform Trial for Community-Acquired Pneumonia (REMAP-CAP) aims to determine and continuously update the optimal set of treatments for community-acquired pneumonia.
  • An important aspect of adaptive trial designs is that already accrued data are used to increase the likelihood that patients within the trial are randomized to treatments that are beneficial.
  • With the onset of the COVID-19 pandemic, the REMAP study made use of an adaptive sub-platform called REMAP-COVID, which is studying multiple questions around COVID treatment simultaneously.

Discussion Themes

The COVID-19 pandemic requires us to do two things at once: learn and do. An integrated approach finds the optimal balance to treat patients as well as possible and learn as fast as possible.

Adaptive randomization is potentially more comfortable for physicians, where patients are preferentially assigned to the best therapy over time.

Read more about REMAP-CAP and Dr. Angus’ research in Optimizing the Trade-off Between Learning and Doing in a Pandemic (JAMA, March 2020).

Tags

#pctGR, @Collaboratory1, @remap_cap

May 14, 2020: Healthcare Workers Invited to Join the HERO Registry

The Healthcare Worker Exposure Response & Outcomes (HERO) Registry invites both clinical and nonclinical healthcare workers to share their life experiences in order to understand the perspectives and problems faced by those on the COVID-19 pandemic frontlines. HERO Registry participants could have the opportunity to participate in future research studies to improve the understanding of COVID-19 and beyond, generating evidence to help healthcare workers stay safe and healthy.

The HERO Registry is open to all healthcare workers, including nurses, therapists, physicians, emergency responders, food service workers, environmental service workers, interpreters, transporters — anyone who works in a setting where people receive health care.

Learn more about the HERO Registry and how to join.

Don’t miss the recent COVID-19 Grand Rounds introducing the HERO Program and get the latest information and resources on COVID-19 for clinical researchers.

May 8, 2020: Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Real World Evidence: Contemporary Experience and Future Directions (Patrick Heagerty, PhD, Jacqueline Corrigan-Curay, JD, MD, Joshua C. Denny, MD, MS)

Speakers

Guest Moderator:
Patrick J. Heagerty, PhD
Professor, Department of Biostatistics, University of Washington

Panel:
Jacqueline Corrigan-Curay, JD, MD
Director of CDER’s Office of Medical Policy (OMP)
U.S. Food and Drug Administration (FDA)

Joshua C. Denny, MD, MS, FACMI
Chief Executive Officer, All of Us Research Program, NIH

Topic

Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Real World Evidence: Contemporary Experience and Future Directions

Keywords

Electronic health records; Real-world evidence; RWE; Real-world data; RWD; FDA; All of Us; Phenotypes; Regulatory; Fit-for-use data; Digital heath

Key Points

  • To create quality clinical/research records, we must design for multiuse by integrating standards-based tools in the EHR to bring together health care and research. 
  • Quality real-world evidence cannot be built without quality real-world data. With greater efficiencies in data capture, randomization with real-world data provides a pathway for reliable—and persuasive—real-world evidence.

Discussion Themes

Patient-generated health data is part of FDA’s MyStudies Application, designed to facilitate the input of real-world data directly by patients, which can be linked to electronic health data supporting traditional clinical trials, pragmatic trials, observational studies, and registries.

In assessing data quality we can ask, How does a data element travel from clinical care to a research data set?

The NIH’s All of Us program is building a diverse database that can inform thousands of studies on a variety of health conditions.

The All of Us study is tracking COVID-19 in its patients. Sites have identified their COVID-19 participants and relevant labs. Consent is obtained for future sharing of data.

Tags

#pctGR, @Collaboratory1

May 5, 2020: Dr. Robert Califf Discusses Next Steps for Reforming the Evidence Generation Ecosystem After COVID-19

In case you missed the May 1 keynote address by Robert M. Califf, MD, MACC, you can now listen to the recorded webinar and Q&A. Dr. Califf, head of strategy and policy for Verily Life Sciences and Google Health, kicked off the Collaboratory’s Grand Rounds workshop series, Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials.

His presentation outlined several opportunities to drive change and rebuild clinical research in the aftermath of COVID-19, including:

  • Evaluate what has and has not worked in the changes that have been made in response to the crisis
  • Allocate a significant part of recovery funding to transition issues in evidence generation, especially at the interface of medicine and public health
  • Do everything possible to fix the “purposefulness issue”:
    • Create methods for deciding the most important questions
    • Reward behavior that gets important questions answered quickly
  • Develop inclusive networks driven by people with the health problems of interest; increase incentives for clinicians and investigators that lead to reliable and faster evidence generation (balance financial focus with purpose); and automate mapping of EHR data beyond individual systems

“The effective use of digital information such as electronic health records, telehealth, applications, and patient-reported outcomes should free up effort to fix the human components that are holding us back.” – Dr. Robert Califf

View the full presentation for more insights from Dr. Califf.

Stay tuned for these upcoming presentations in the series:

May 1, 2020: Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Keynote-Can the COVID-19 Crisis Lead to Reformation of the Evidence Generation Ecosystem? (Robert Califf, MD, MACC)

Speaker

Robert Califf, MD, MACC
Head of Strategy and Policy
Verily Life Sciences and Google Health

Topic

Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials: Keynote-Can the COVID-19 Crisis Lead to Reformation of the Evidence Generation Ecosystem?

Keywords

Electronic health records; Digital health; Mobile health; Coronavirus; COVID-19; Ecosystem; Clinical trials; Evidence generation

Key Points

  • The HERO Registry and RECOVERY Trial are good examples of a rapid clinical research response to the urgent COVID-19 health crisis.
  • Among the essential steps to move the evidence generation system in the right direction:
    • Evaluate what has and has not worked in the changes made in response to the crisis
    • Allocate a significant part of recovery funding to transition issues in evidence generation, especially at the interface of medicine and public health
    • Increase purposefulness by creating methods for deciding the most important questions and rewarding behavior that gets those questions answered quickly

Discussion Themes

The COVID-19 pandemic has shone a spotlight on disparities in our current healthcare delivery system. How can we avoid leaving the most vulnerable of society behind?

Telemedicine can be a framework for the integration of research and clinical care. But the digital element must be integrated with the human element. The routine and effective use of digital information should free up effort to fix the human components that are holding us back.

We’ve been trying to modernize clinical trial design for decades (factorial/sequential/adaptive designs for example). While some positive movement in the past month has been made, the research enterprise remains largely conservative when it comes to design modernization. How do we make more rapid progress?

Tags

#pctGR, #COVID19, @Collaboratory1, @Califf001

April 24, 2020: The RECOVERY Trial: A UK National Platform Trial of Potential Treatments for Patients Hospitalised with COVID-19 (Martin Landray, PhD, FRCP)

Speaker

Martin Landray, PhD, FRCP
Professor of Medicine & Epidemiology
Nuffield Department of Population Health
University of Oxford, UK

Topic

The RECOVERY Trial: A UK National Platform Trial of Potential Treatments for Patients Hospitalised with COVID-19

Keywords

Coronavirus; Virus pandemic; COVID-19; SARS-CoV-2; RECOVERY Trial; Randomized trial; Epidemiology

Key Points

  • Between March 10 and April 3, 2020, the RECOVERY team developed the protocol, obtained regulatory and ethics approval, and enrolled 1,000 patients.
  • COVID-19 presents an unprecedented clinical challenge to the health system, staff, and patients. Even moderate effects from this study will be worthwhile in the generation of robust evidence.
  • RECOVERY follow up will involve linkage to national data sources for vital status and death certificates; coded hospital episode statistics (diagnoses, procedures); intensive care audit data and SARS-CoV-2 PCR laboratory results; and primary care and national outpatient prescribing data.

Discussion Themes

Informed consent for participants in the RECOVERY trial consists of a 2-page information sheet and single form written in plain language.

The urgency of this pandemic requires everyone to focus on what matters and leave orthodoxy, habits, and traditional practices behind. Our mindset has been altered by the COVID-19 disruptions and this public health crisis of extraordinary proportion.

For more information, visit the RECOVERY Trial website.

Tags
#pctGR, #COVID19, @Collaboratory1

April 17, 2020: The HERO Program: PCORnet® at Work to Create a Healthcare Worker Community for Rapid Cycle Evidence (Nakela Cook, MD, MPH; Josie Briggs, MD; Susanna Naggie, MD; Emily O’Brien, PhD; Russell Rothman, MD; Chris Forrest, MD, PhD)

Speakers

Nakela Cook, MD, MPH
Executive Director
PCORI

Josie Briggs, MD
Acting Chief Science Director
PCORI

Susanna Naggie, MD
Principal Investigator, HERO-HCQ Clinical Trial
Duke University

Emily O’Brien, PhD
Principal Investigator, HERO-Registry
Duke University

Russell Rothman, MD, MPP
Chair, HERO Steering Committee
Vanderbilt University

Chris Forrest, MD, PhD
Chair, HERO Registry
Children’s Hospital of Pennsylvania

Topic

The HERO Program: PCORnet® at Work to Create a Healthcare Worker Community for Rapid Cycle Evidence

Keywords

Coronavirus; Virus pandemic; COVID-19; PCORI; Patient-Centered Outcomes Research Institute; Frontline health workers; Hydroxychloroquine; Healthcare systems; HERO-HCQ

Key Points

  • With the onset of the coronavirus pandemic, the U.S. healthcare system faces an unprecedented stress test to adapt to meet new demands.
  • Those on the frontline of caring for patients—healthcare workers—are at risk of developing or transmitting COVID-19. Evidence is urgently needed to keep healthcare workers and their families safe and healthy, which ultimately will help protect us all.
  • The HERO (Healthcare Worker Exposure Response & Outcomes) Program has three broad focus areas to develop rapid evidence around healthcare workers’ outcomes related to COVID-19:
    • Emphasis on the adaptations in how healthcare is delivered
    • Emphasis on vulnerable populations
    • Emphasis on the well-being of the healthcare worker
  • HERO will consist of a registry study of healthcare workers and a randomized controlled trial called HERO-HCQ that will evaluate the safety and efficacy of hydroxychloroquine to prevent COVID-19 clinical infections in healthcare workers.

Discussion Themes

The goal of the HERO registry is to understand all aspects of healthcare workers’ lives—those with direct impact and those with indirect impact.

What mechanisms are you envisioning for new study proposals that would be conducted via the HERO platform?

Serologic testing will have a significant role in understanding prior infection and potential immunity.

The HERO Registry is open to any healthcare worker to join at https://heroesresearch.org/. Enrollment takes only a few minutes; participation is free and voluntary. It is not restricted by profession. All data are kept confidential.

Tags
#HeroRegistry, #pctGR, @Collaboratory1, @PCORI, @PCORnetwork, @HeroesResearch

April 10, 2020: Hydroxychloroquine for the Early Treatment of COVID-19 in Hospitalized Adults: A Multicenter Randomized Clinical Trial (Sean Collins, MD, MSc)

Speaker

Sean Collins, MD, MSc
Professor and Executive Vice Chair
Department of Emergency Medicine
Director, Center for Emergency Care Research and Innovation
Vanderbilt University Medical Center

Topic

Hydroxychloroquine for the Early Treatment of COVID-19 in Hospitalized Adults: A Multicenter Randomized Clinical Trial

Keywords

Coronavirus; Virus pandemic; COVID-19; Randomized controlled trial; Acute respiratory distress syndrome (ARDS); Hydroxychloroquine; FDA; Emergency Use Authorization; ORCHID study

Key Points

  • Hydroxychloroquine is a biologically plausible agent for early treatment of acute respiratory distress syndrome in patients with COVID-19, but its effects remain to be evaluated in a high-quality, multicenter, blinded, placebo-controlled trial.
  • In an Emergency Use Authorization, the FDA has encouraged the conduct and participation in randomized controlled clinical trials that may produce evidence concerning the effectiveness of hydroxychloroquine in treating patients with COVID-19.
  • Trial results of the effects of this agent will be informative, whether showing benefit or harm.

Discussion Themes

The study team for this trial determined that one-to-one randomization would yield the best data quickly.

Efficacy and safety of hydroxychloroquine must be closely monitored in a health setting.

This is not the only study of chloroquine going on around the world; is there any collaboration with other studies?

Because of the urgency of the pandemic, people are collaborating on a level never seen before. We have a common goal and must maintain momentum through accelerating clinical trials with large teams of parallel studies.

Read more about this COVID-19 study at NCT04332991.

Tags
#pctGR, @Collaboratory1

April 3, 2020: Innovative Support for Patients with SARS-COV-2 Infections Registry (INSPIRE): Participant-Centered, Rapidly-Deployed, Digitally-Enabled Research (Harlan Krumholz, MD; Bala Hota, MD, MPH; Graham Nichol, MD, MPH)

Speakers

Harlan M. Krumholz, MD
Harold H. Hines Jr. Professor of Medicine, Yale University
Director, Yale New Haven Hospital Center for Outcomes Research and Evaluation
Co-Founder Hugo Health

Bala Hota, MD, MPH
Professor of Internal Medicine, Rush University
Chief Analytics Officer, Rush University Medical Center

Graham Nichol, MD, MPH
Medic One Foundation Endowed Chair for Pre-hospital Emergency Care
Professor of Emergency Medicine
University of Washington

Other Panelists:

Jacqueline Rollin, Administrative Fellow
Rush University Medical Center

Wade Schulz, MD, PhD
Assistant Professor of Laboratory Medicine
Director, CORE Center for Computational Health

Matthew J. Thompson, MB, ChB, DPhil
Helen D. Cohen Endowed Professorship in Family Medicine
Professor of Global Health and Medicine, University of Washington

Deb R. Chromik, Participant Experience
Hugo Health

Dave Hutton, Product Lead
Hugo Health

Topic

Innovative Support for Patients with SARS-COV-2 Infections Registry (INSPIRE): Participant-Centered, Rapidly-Deployed, Digitally-Enabled Research

Keywords

Coronavirus; Virus pandemic; INSPIRE Registry; COVID-19 directed research program; SARS-COV-2; Longitudinal data; Hugo Health digital research platform

Key Points

  • In the face of the coronavirus pandemic, there is an urgent need for rapid knowledge generation and actionable insights. Evidence needed includes:
    • The number of cases, including milder ones
    • Risk factors and timing of transmission
    • Severity and attack rate
    • Risk factors for infection and severe outcomes, including death
    • Infectiousness timing and intensity
  • Patients must be considered part of the team; involved, engaged, and respected, with agency over their data.
  • To better understand the experience of people with COVID-19, Rush University Medical Center and Hugo health are piloting the COVID INSPIRE registry. INSPIRE is a rapidly-deployed, digitally-enabled, participant-centered platform to collect longitudinal data and facilitate observational and experimental studies.

Discussion Themes

Even with social distancing, the coronavirus is in a rapid escalation phase; this rapid pace has our attention.

People are interested in participating in research now more than ever. The call to action is to build a human-connected system that treats patients compassionately and supports patients in real time.

Are there existing systems that could be built on or adapted for COVID-19? Are there potential for linkages to other systems?

Tags
#pctGR, @Collaboratory1, @HMKYale, @BalaHota, @GrahamNichol