November 22, 2022: ADAPTABLE Points to Need for Better Integration of Patient-Reported Health Data Into Pragmatic Trials

Headshot of Dr. Emily O'BrienA concordance analysis from ADAPTABLE, a large pragmatic, comparative effectiveness trial, found low to moderate agreement between patient-reported health data and data derived from the electronic health record (EHR). The findings highlight the need for better integration of patient-reported health data into pragmatic research studies.

Results of the study were published in JAMA Cardiology.

Patient-reported health data can assist investigators in capturing clinical events in pragmatic clinical trials. However, little information is available about the fitness for use of patient-reported data in large-scale health research.

In this study, analysts evaluated the concordance of a set of variables that were both reported by patients through an online portal and available in the EHR as part of the trial. The validity of participant self-report varied by the method of data collection, the participant population, and the type of information collected. For example, patient report of clinical events had low sensitivity compared with EHR data for myocardial infarction, stroke, and major bleeding events. Coronary revascularization was the most concordant event by data source, but agreement was only moderate.

“As the use of direct-to-participant research designs grows, it’s important to understand how participant-reported data lines up with other data types,” said Emily O’Brien, an associate professor in population health sciences at Duke University and the lead author of the study.

“Our findings suggest that some caution is warranted in this space—especially if relying on participant report alone for capture of cardiovascular events—and we need more research on how to best design questions and set up participant portals to maximize validity of this type of data,” O’Brien said.

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 patient-reported health data captured through an online portal.

This is the first study to assess validity of participant-reported health data collected through an online portal. The study’s results are consistent with prior evidence suggesting that the validity of participant-reported events varies by data collection modality, participant subgroup, and the type of information collected.

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

Read the full report in JAMA Cardiology.

October 8, 2021: Lessons Learned and Patient Partnership in ADAPTABLE (Schuyler Jones, MD; Madelaine Faulkner Modrow, MPH)

Speakers

Schuyler Jones, MD
Associate Professor of Medicine
Duke University School of Medicine

Madelaine Faulkner Modrow, MPH
Program Director
Department of Epidemiology and Biostatistics
University of California, San Francisco

Topic

Lessons Learned and Patient Partnership in ADAPTABLE

Keywords

Patient engagement; Pragmatic clinical trial; Aspirin; ADAPTABLE; Study design; Stakeholder engagement; Recruitment

Key Points

  • ADAPTABLE is a pragmatic clinical study of 15,000 patients to examine a simple, everyday decision, whether to take 81mg or 325mg of aspirin daily, to identify if that decision could prevent heart attacks.
  • ADAPTABLE used both pragmatic and personalized approaches to participant recruitment.
  • Pragmatic recruitment involves broad-based email and social media outreach to a large pool of potential subjects. This method of recruitment was lower cost, less time intensive, and faster paced than personalized recruitment.
  • Personalized recruitment involves traditional in-clinic or phone call personal outreach to potential participants. This method of recruitment was higher cost, time intensive, and slower paced than personalized recruitment.
  • ADAPTABLE learned a critical lesson in patient engagement: in-clinic enrollment was much higher at 81% of those approached versus e-communication enrollment at 35% of those contacted.
  • Key principles of patient engagement include trust and partnership, respect and listening, empowering patients to find solutions, and value and design the trial for the patient experience.

Discussion Themes

We are moving from a traditional model of research to a relational model with participants coming together with researchers and forming a kind of social contract where a participant’s voice is valued in the research process.

We have lessons left to learn in order to fully utilize technology for patient engagement including how to use broad outreach methods to increase enrollment of diverse populations.

Patients continue to look to their clinician or doctor for advice on whether to participate in a clinical trial. Trusted contacts will continue to play an important role in the recruitment process.

 

Read more about the ADAPTABLE trial.

Tags

#pctGR, @Collaboratory1

October 6, 2021: Patient Partnership in ADAPTABLE Featured in This Friday’s PCT Grand Rounds

Headshots of Madelaine Faulkner Modrow and Dr. Schuyler Jones
Madelaine Faulkner Modrow (left) and Dr. Schuyler Jones

In this Friday’s PCT Grand Rounds, Madelaine Faulkner Modrow of the University of California, San Francisco, and Dr. Schuyler Jones of Duke University will present “Lessons Learned and Patient Partnership in ADAPTABLE.”

The Grand Rounds session will be held on Friday, October 8, at 1:00 pm eastern. Join the online meeting.

For webinar recordings and slides from previous Grand Rounds sessions, see the Grand Rounds hub.

Grand Rounds October 8: Lessons Learned and Patient Partnership in ADAPTABLE

Speakers:
Madelaine Faulkner Modrow, MPH
Program Director
Department of Epidemiology and Biostatistics
University of California, San Francisco

Schuyler Jones, MD
Associate Professor of Medicine
Duke University School of Medicine

Topic: Lessons Learned and Patient Partnership in ADAPTABLE

Date: Friday, October 8, 2021, 1:00-2:00 p.m. ET

Meeting Info: To check whether you have the appropriate players installed for UCF (Universal Communications Format) rich media files, go to https://dukemed.webex.com/dukemed/systemdiagnosis.php.

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May 19, 2021: ADAPTABLE Results: High- and Low-Dose Aspirin Have Similar Effectiveness and Safety

Recently published results of the ADAPTABLE study demonstrate no difference in rates of death, hospitalization for heart attack or stroke, or bleeding in those who were assigned to high- versus low-dose aspirin (325 mg versus 81 mg). Trial participants frequently switched their dose, which may have biased the results toward the null: 41.6% of patients who were assigned to take the 325 mg dose switched to 81 mg daily dose. The study, funded by PCORI,  is the first randomized controlled trial conducted using PCORnet®, the National Patient-Centered Clinical Research Network, which involves patient representatives during all phases of the trial. It enrolled 15,076 patients with cardiovascular disease at 40 health centers across the United States.

“As interest grows for real-world evidence, the trial provides a demonstration that randomized clinical trials can leverage electronic health record data, direct-to-patient methods, and patient-reported outcomes to address important, patient-centered questions.” —Jones, et al. 2021, New England Journal of Medicine

An accompanying editorial states that this trial represents a step forward for pragmatic clinical trials, demonstrating proof-of-concept for PCORnet. The study and used electronic health record data to identify patients; low-touch recruitment strategies; and a patient portal for consent, collection of patient-reported outcomes, and follow-up.

“ADAPTABLE is a major achievement […] because it has shown a method of conducting trials efficiently and at low cost in the United States, and the method can now be adapted and used more widely. This should allow many more clinical questions to be answered, with obvious benefits to health care consumers.” —Baigent 2021, New England Journal of Medicine.

Read the plain language summary of results and learn more about the ADAPTABLE study.

January 19, 2021: Using Claims and CMS Files: New Enhancements in the Living Textbook

Many of the NIH Collaboratory NIH Collaboratory Trials use data from claims and research-identifiable files from the Centers for Medicare and Medicaid Services (CMS) to support their research. We have enhanced the Living Textbook and added new sections to better present the wealth of information on how to acquire, use, and link CMS data for research.

July 27, 2020: ADAPTABLE Roundtable Produces Consensus Statement on Capturing Patient-Reported Health Data in Clinical Trials

A new report from an NIH Collaboratory roundtable discussion held in 2017 discusses considerations for the capture 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 statement was published online ahead of print this month in Healthcare.

The new consensus statement is the second report from the ADAPTABLE roundtable. A statement published in February offered guidance for the analysis and integration of PRH data in clinical trials.

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.

April 22, 2020: New Article Details the Design of ADAPTABLE, PCORnet’s First Pragmatic Trial

A new article in JAMA Cardiology discusses the design of ADAPTABLE, a pragmatic clinical trial (PCT) which strives to answer a three-decade-old question with great potential significance for public health. ADAPTABLE is the first PCT to use many of the data-driven and patient-centric capabilities of the National Patient-Centered Clinical Research Network (PCORnet).

Researchers in ADAPTABLE pursued the unanswered question of whether a low dose or high dose of aspirin is optimal for secondary prevention of atherosclerotic cardiovascular disease. Complexities of running a randomized clinical trial and the expenses associated with it have previously prevented researchers from answering this question.

The capabilities of PCORnet have aided in this research. Though not all randomized clinical trials can be designed as PCTs, ADAPTABLE demonstrates the possibility of incorporating pragmatic elements into future studies with the goal of producing real-world evidence.

A key objective of involving PCORnet in ADAPTABLE was the ability for large-scale recruitment (15,000 participants) using electronic health records and electronic informed consent. The PCORnet component permitted complete electronic participation, from randomization to data collection. Patients reported their own data during scheduled electronic follow-ups, which replaced conventional follow-up visits.

NIH Collaboratory Coordinating Center co–principal investigator Dr. Adrian Hernandez also serves as co–principal investigator of ADAPTABLE and contributed to the article.

See the accompanying editorial describing lessons learned from the design features of ADAPTABLE.

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 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.