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

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

April 6, 2020: Convening Pain Management Expertise in the Patient-Centered Outcomes Core: An Interview With Dr. Kevin Weinfurt

The Collaboratory recently interviewed Kevin Weinfurt, PhD, co-chair of the Patient-Centered Outcomes Core working group, to hear about the Core’s plans for supporting the PRISM Demonstration Projects. In this video, Dr. Weinfurt says one challenge for embedded trials of pain is in obtaining high-quality, complete patient-reported outcome data on pain intensity and functioning. Along with other national initiatives focused on pain research, such as the NIH-DoD-VA Pain Management Collaboratory, a chief task of the Core will be to look at efforts to harmonize pain outcomes across studies.

“The Core serves as a convener of expertise in pain management within the Collaboratory. We keep an eye on issues across the Demonstration Projects that might also be of interest to the broader pragmatic trial community.”

Read more about the work of the Patient-Centered Outcomes Core, and view other interviews about the HEAL Initiative and PRISM Demonstration Projects.

February 21, 2020: New Video Interviews Highlight Goals of the NIH HEAL Initiative and PRISM Demonstration Projects

Recently, the Collaboratory Coordinating Center conducted video interviews describing the critical work being done in the NIH HEAL Initiative program to address the national opioid crisis. As part of this program, the PRISM (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing) Demonstration Projects present exciting new possibilities for answering important questions along with unique challenges.

Drs. Rebecca Baker and Wendy Weber discuss the aims of the HEAL Initiative, including:

  • Leveraging expertise across all NIH institutes and centers to enhance pain management and improve prevention and treatment strategies for opioid use disorder and addiction
  • Generating evidence to help clinicians and patients make the right treatment decisions

 

Drs. Meyers, Hernandez, and Weber discuss aspects of collaborating with the four PRISM Demonstration Projects, including:

  • Leveraging broad expertise in the Collaboratory program to support embedded healthcare systems research in pain management
  • Developing new understanding across different care models such as physical therapy, surgery, acupuncture therapy, and primary care settings
  • Learning about patient-reported outcomes that will be the most meaningful to establish best practices in pain management

Learn more about the PRISM Demonstration Projects on their webpages:

  • AcuOA: Pragmatic Trial of Acupuncture for Chronic Low Back Pain in Older Adults
  • FM TIPS: Fibromyalgia TENS in Physical Therapy Study
  • NOHARM: Non-pharmacological Options in Postoperative Hospital-Based and Rehabilitation Pain Management
  • OPTIMUM: Group-based mindfulness for patients with chronic low back pain in the primary care setting

February 11, 2020: ADAPTABLE Roundtable Produces Consensus Statement on Analysis and Integration of Patient-Reported Data in Clinical Trials

A roundtable discussion organized by the NIH Collaboratory in 2017 has produced consensus findings on the analysis and integration of patient-reported health (PRH) data in clinical trials. The report is part of an effort by the ADAPTABLE Supplement project team “to address best practices for capturing PRH data in pragmatic studies and optimal analytic approaches for integrating PRH with other data sources.”

The consensus statement was published online ahead of print this month in the Journal of the American Medical Informatics Association.

The report discusses strengths and limitations of PRH data, approaches for ascertaining and classifying study end points, and methods for addressing incompleteness, data alignment, and data concordance. Roundtable participants used experiences from the ADAPTABLE trial as a case study to inform their discussions.

ADAPTABLE, the first major randomized comparative effectiveness trial conducted by the National Patient-Centered Clinical Research Network (PCORnet), seeks to determine the optimal dose of aspirin therapy for secondary prevention of atherosclerotic cardiovascular disease. The trial relies on both existing EHR data sources and PRH data.

This work was supported by a supplemental grant award to the NIH Collaboratory Coordinating Center from the National Center for Complementary and Integrative Health.

January 30, 2020: Meeting Materials from the NIH Collaboratory PRISM Kickoff Meeting

The Collaboratory has made available all the presentations from their recent PRISM Kickoff meeting held in Bethesda November 19-20, 2019.

PRISM Demonstration Project Investigators
Left to right: Kathleen Sluka, PT, PhD (FM TIPS); Andrea Cheville, MD (NOHARM); Karen Sherman, PhD, MPH (AcuOA); Jon Tilburt, MD (NOHARM); Lynn DeBar, PhD, MPH (AcuOA); Leslie Crofford, MD (FM TIPS); and Natalia Morone, MD (OPTIMUM).

The PRISM (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing) program is a component of the NIH HEAL Initiative to address the opioid crisis. Highlights of Day 1 included welcoming the 4 new PRISM UG3 Demonstration Projects—AcuOA, FM TIPS, NOHARM, and OPTIMUM—and introducing the study teams to the NIH Collaboratory Program and Coordinating Center, hearing an overview of the HEAL Initiative goals and cooperative agreement, and learning about the aims of the new studies. Day 2 included face-to-face meetings between each PRISM Demonstration Project and the Collaboratory Core working groups to discuss anticipated challenges in design, implementation, analysis, and dissemination.

View or download the meeting materials on the website.

 

 

January 29, 2020: Open-Source Tool From the ADAPTABLE Supplement Enables Comparisons of EHR and Patient-Reported Data

The ADAPTABLE Supplement project team released user documentation and source code for an open-source tool that enables rapid assessment of concordance between electronic health record (EHR) data and information reported directly by patients. The tool is part of a larger effort supported by the NIH Collaboratory Coordinating Center to develop and test methods for integrating patient-reported data into the EHR and to streamline data for use in pragmatic clinical trials.

ADAPTABLE, the first major randomized comparative effectiveness trial conducted by the National Patient-Centered Clinical Research Network (PCORnet), aims to identify the optimal dose of aspirin therapy for secondary prevention of atherosclerotic cardiovascular disease. The trial relies on both existing EHR data sources and direct patient report.

The ADAPTABLE Supplement project team developed a menu-driven query (MDQ) tool to enable comparison of patient-reported data with analogous EHR data. Using data for patients enrolled in ADAPTABLE at the trial’s largest US site, the team tested the MDQ tool by using it to compare patient-reported hospitalizations with hospitalizations recorded in the EHR. In this test, 46% of the encounters recorded in the EHR were an exact match with patient-reported encounters, and 85% of the EHR-recorded encounters fell within 5 days of the patient-reported encounter dates.

The study demonstrates the feasibility of using the MDQ tool to assess concordance between patient-reported data and EHR data. Because the tool is based on the PCORnet Common Data Model, it will be useful to participating sites across the network and can be used for querying this widely available data source.

The MDQ tool user documentation describes the features of the tool and provides links to the source code. A summary of the MDQ tool’s development describes how the tool performed with data from ADAPTABLE.

This work was supported by a supplemental grant award to the NIH Collaboratory Coordinating Center from the National Center for Complementary and Integrative Health.

July 2, 2019: New Living Textbook Section on Inpatient Endpoints in Pragmatic Clinical Trials

A new section in the Living Textbook describes the considerations for using “real-world” data for inpatient-based event ascertainment. There are many sources for acquiring this information, and they have different time lags in their availability and varying degrees of error and bias. In order to use inpatient endpoints in pragmatic clinical trials, these factors must be understood during the design, conduct, and analysis phases of an embedded pragmatic clinical trial.

“The pragmatic trial community needs to collectively determine which endpoints are relevant for pragmatic trials, how they can be measured and validated, and how the accuracy of these measurement methods may impact hypothesis testing sample size estimates.” —Eisenstein et al 2019

Topics in the chapter include:

  • Pragmatic trial inpatient endpoints
  • Inpatient event data sources
  • Patient-reported data
  • Secondary data sources: EHR
  • Secondary data sources: claims
  • Case studies: ICD-Pieces, TRANSFORM-HF, ADAPTABLE, and TRANSLATE ACS
  • Data source accuracy

 

January 11, 2019: FDA Releases Framework for Evaluating the Use of Real-World Evidence

To help fulfill the requirements of the 21st Century Cures Act by accelerating medical product development and fostering innovation and advances in medicine, FDA recently created a framework for evaluating the use of real-world evidence. The framework is intended to help evaluate trials that use real-world data for the creation of real-world evidence.

  • Real-world data: routinely collected information about a person’s health status in the electronic health record, claims, registries, and other sources, including patient-generated sources.
  • Real-world evidence: reliable, clinical information derived from real-world data about risks, benefits, and burdens of therapies.

This framework will apply to various pragmatic clinical trials embedded in health care systems and conducted as part of routine care (and will not apply to more traditional clinical trials conducted parallel to care).

Three main considerations are included in the framework:

  1. Will the real-world data be fit for use (do they reliably and adequately represent the concept)?
  2. Will the evidence generated by the trial provide adequate evidence to help answer regulatory questions?
  3. Will the conduct of the study meet FDA regulatory requirements?

August 28, 2018: ADAPTABLE Patient-Reported Health Data Codes Now Available

The ADAPTABLE pragmatic trial relies on patients to report key information at baseline and throughout follow-up. To capture these data, ADAPTABLE investigators developed a LOINC (Logical Observation Identifiers Names and Codes) patient-reported item set, which is now publicly available.

The development of the item set is part of the ADAPTABLE Supplement, an initiative funded by the Office of the Assistant Secretary for Planning and Evaluation to develop best practices for capturing patient-reported outcome data and optimal analytic approaches for using the data in a pragmatic clinical trial. Additional reference material can be found in the ADAPTABLE Supplement Roundtable Meeting summary, in a report describing the results of a literature review of data standards and metadata standards for variables of interest, and on GitHub. The project is expected to inform future efforts to integrate patient-reported data in the electronic health record and provide opportunities to streamline data for use in pragmatic trials. Information from the project is being added to the Living Textbook as it accumulates; learn more in the chapters on Using Electronic Health Record Data and Choosing and Specifying End Points and Outcomes.

ADAPTABLE (Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-Term Effectiveness) aims to identify the optimal dose of aspirin therapy for secondary prevention in atherosclerotic cardiovascular disease and is the first major randomized comparative effectiveness trial to be conducted by the National Patient-Centered Clinical Research Network (PCORnet).