patient-centered outcomes research

June 23, 2025: How to Choose Patient-Reported Outcome Measures in Pragmatic Clinical Trials?

Posted on by Karen Staman

Living Textbook iconA new section of the Living Textbook of Pragmatic Clinical Trials describes considerations for choosing patient-reported outcome measures in pragmatic clinical trials.

“Where possible, investigators are encouraged to use measures with adequate support for validity that are in the public domain,” the authors wrote.

The authors provide a set of questions to guide investigators in choosing appropriate measures. For example, investigators may want to understand whether the patient-reported outcome is in electronic health records, is in the public domain, and is valid for the use case in question.

The considerations were developed by members of the NIH Pragmatic Trials Collaboratory’s Patient-Centered Outcomes Core in collaboration with the Health Care Systems Interactions Core, the Electronic Health Records Core, and colleagues at the NIH.

November 26, 2024: Hybrid Methods Improve Collection of Patient-Reported Outcome Measures in Rural and Underserved Populations

Posted on by Karen Staman

ACI

In an article published online this week, leaders from the NIH Pragmatic Trials Collaboratory share lessons learned about the collection of patient-reported outcome measures (PROMs) in medically underserved patients, including people with incomes below the federal poverty threshold, racial or ethnically minoritized groups, and rural and frontier communities.

The electronic health record and other digital modes are frequently used to collect PROM data, and engagement with these digital tools can be low among disparities-prone populations.

“Our ability to measure PROMs is hampered by patients’ limited digital proficiency, difficulty understanding PROMs as written, and access to portals and the Internet. …With low uptake among disparities prone populations, reliance on portals may inadvertently exacerbate health inequities,” the authors wrote.

Therefore, the authors recommend hybrid approaches:

  • multiple outreach modes
  • high-touch methods
  • creativity in promoting digital uptake
  • multimodal participant engagement, and
  • text messaging for the collection of PROMs

The authors draw on on experiences from 5 of the NIH Collaboratory Trials and the expertise of the Patient-Centered Outcomes Core and the Health Equity Core.

The article was published this week in Applied Clinical Informatics.

Grand Rounds August 23, 2024: Improving Quality of Life in COPD and Heart Failure: Unpacking a Successful Multicomponent Virtual Team Intervention (David B. Bekelman, MD, MPH; Lyndsay DeGroot PhD, RN, CNE)

Posted on by Sophia Ramirez

Speakers

David B. Bekelman, MD, MPH
Professor of Medicine and Psychiatry
Rocky Mountain Regional VAMC and University of Colorado Division of General Internal Medicine

Lyndsay DeGroot PhD, RN, CNE
Postdoctoral Fellow
University of Colorado
Anschutz Medical
Campus School of Medicine

Keywords

COPD; Heart Failure; Collaborative Care; Palliative Care

Key Points

  • Heart failure (HF) and Chronic Obstructive Pulmonary Disease (COPD) are common illnesses that share a number of symptoms, including persistent breathlessness and fatigue; depression; and anxiety.
  • Though HF and COPD patients could benefit from palliative care prior to the end of life, there aren’t enough palliative care specialists to provide care to the patient population.
  • ADAPT is a virtual intervention built on a collaborative care team approach. A nurse and social worker provide direct patient care and meet weekly with a collaborative care team – consisting of a primary care physician, palliative care specialist, and as-needed cardiologist and pulmonologist – to review individual cases.
  • The research team’s primary aim was to determine the effect of ADAPT on participant quality of life, measured using the FACT-G scale.
  • They concluded that a nurse and social worker palliative telecare team demonstrated early, persistent, clinically meaningful improvements in quality of life for high-risk outpatients with COPD, HF, and Interstitial Lung Disease.
  • The ADAPT virtual care model leveraged a team of nurses, social workers, and physicians across two large VA health systems to increase the reach of palliative care to common, serious non-cancer illnesses.
  • The intervention was relatively inexpensive, but implementing a multifaceted, personnel-intensive program posed its own challenges.
  • Future directions include investigating the intervention effect on utilization outcomes and end-of-life outcomes; adapting the model for patients with advanced liver or renal disease; testing other implementation and dissemination strategies; and investigating how to improve adoption.

Discussion Themes

The mortality rate in the population they enrolled was lower than expected. This may be because it is more difficult to enroll very sick patients from an outpatient population.

This study was designed to test the effect of the intervention on quality of life, an outcome that has a significant impact on patients. However, securing funding for work like this can be a challenge, as the program is not designed to reduce costs (unlike interventions that aim to reduce hospitalizations).

There is a need for creative solutions when it comes to funding this model; the research team is looking to collaboration with community partners and examples set by other, similar projects.

August 13, 2024: EHR Core and Patient-Centered Outcomes Core Reflect on Challenges in Pragmatic Trials

Posted on by Karen Staman

At the NIH Pragmatic Clinical Trials Collaboratory’s 2024 Annual Steering Committee Meeting, leaders of the Electronic Health Records (EHR) Core and the Patient-Centered Outcomes (PCO) Core discussed challenges and lessons learned in the NIH Collaboratory Trials.

EHR cochairs Keith Marsolo and Rachel Richesson shared challenges related to EHR usage, including EHRs switching midtrial, unanticipated changes to the EHR that impacted intervention delivery, and difficulties navigating data sharing policies.

“We continue to be challenged, but we fall back on our core principles, which is looking for good quality data and good quality research with an eye on how we can share the results in the underlying data in the future,” Richesson said.

Over the next year, the EHR Core will explore improving data access, transparency of computable phenotypes, and capturing dietary supplement information in the EHR.

PCO Core cochairs Cristy Zigler and Emily O’Brien shared goals for effective collection PCO data.

“We have advised studies about the importance of delivering patient-reported outcomes to participants in a way that is easy to complete, doesn’t overburden them, and is relevant for their population,” O’Brien said.

The Core has compiled a list of best practices for collecting PCO data and collaborated with the Health Equity Core to create a Health Equity Toolkit, designed to give investigators practical guidance for incorporating health and equity considerations in research.

The Core has also been working closely with Implementation Science Core to consider the sustainability of PCO data systems and facilitate reuse of collection methods.

“We often see separate systems set up to collect PCO data for the trial, and then those systems sunset at the end and are not in place to support an assessment over time,” O’Brien said.

Access the complete meeting materials from the 2024 Annual Steering Committee Meeting.

July 16, 2024: OS-PCORTF Report Focuses on Building Data Capacity for Patient-Centered Outcomes Research

Posted on by Damon Seils

OS-PCORTF Annual Report screenshotA recent report from the Assistant Secretary for Planning and Evaluation in the US Department of Health and Human Services outlines 36 projects funded by the Office of the Secretary Patient-Centered Outcomes Research Trust Fund (OS-PCORTF).

OS-PCORTF was created to build national data capacity and infrastructure and leverage existing clinical data and federal data for patient-centered outcomes research. OS-PCORTF projects involve collaboration across an array of federal agencies to develop products and resources (such as data linkages, data standards, and tools to improve data interoperability) that serve this core mission.

The 2023 Annual Portfolio Report focuses on the most recent advancements in data infrastructure for priority topic areas, including COVID-19, opioid use disorder, and maternal and infant health across a variety of settings and levels.

Read the full report.

October 2, 2023: Patient-Centered Outcomes Core Develops Tool Kit to Promote Health Equity in PROs

Posted on by Karen Staman

The Patient-Centered Outcomes Core has developed a new tool kit to provide resources to support the capture of patient-reported outcome (PRO) measures in diverse study populations. The tool kit is intended for research teams conducting pragmatic clinical trials, including those participating in the NIH Pragmatic Trials NIH Collaboratory Trials.

This tool kit contains 3 sections:

  • Brief background about how PROs can provide critical information about the patient experience, but they must be culturally and linguistically appropriate to be valid.
  • A checklist focused on health equity considerations and PROs, which was adapted from the Center for Nursing Research at Duke University School of Nursing (DUSON) INFusE (Implementing a New Focus on Equity in Research) Checklist in order to focus on PRO measures and health equity.
  • An additional resources document that provides links to virtual libraries containing a range of existing translated and/or culturally adapted PRO measures, links to relevant peer-reviewed literature focused on PRO collection in pragmatic trials, and other tool kits to support equity in the collection of patient-centered outcomes and the conduct of pragmatic research.

For more information, see the Patient-Reported Outcomes chapter of the Living Textbook.

September 27, 2023: This Friday’s PCT Grand Rounds to Highlight PROTEUS Consortium

Posted on by Damon Seils

Headshots of Claire Snyder, Norah Crossnohere, and Anne SchusterIn this Friday’s PCT Grand Rounds, Claire Snyder of Johns Hopkins University and Norah Crossnohere and Anne Schuster, both of Ohio State University, will present “Navigating the Use of Patient-Reported Outcomes in Research and Practice: The PROTEUS Consortium.”

The Grand Rounds session will be held on Friday, September 29, 2023, at 1:00 pm eastern.

The PROTEUS Consortium provides tools and resources to assist researchers in the the use of patient-reported outcomes in clinical trials and clinical practice.

Snyder is a professor of medicine and the director of the Program for Building Lifestyle, Outcomes, and Care Services Research in Cancer (BLOCS) at Johns Hopkins University. Crossnohere is an assistant professor of medicine and Schuster is a research scientist at Ohio State University.

Join the online meeting.

April 24, 2023: Webinar Will Highlight Meaningful Engagement of Patients and Communities in NIH HEAL Initiative Studies

Posted on by Damon Seils

HEAL Headliners: Engaging Patients and Communities in HEAL Research

An upcoming webinar in the NIH HEAL Initiative’s HEAL Headliners series will highlight the experiences of 2 projects in meaningfully engaging patients and community partners throughout the research process to ensure that the findings have the greatest public health impact. The webinar will be held on Friday, May 5, 2023, at 2:00 pm eastern.

Rosemarie Martin of Brown University and Linda Hurley of CODAC Behavioral Heathcare will report on a study of a systems-change approach for increasing use of medications for opioid use disorder across 7 probation and parole sites to improve linkage of justice-involved individuals to evidence-based treatment. In addition, Kirsten Johansen of Hennepin Healthcare will describe the role of patient and community engagement in the HOPE clinical trial of tailored patient-centered interventions to manage pain and reduce opioid use among patients receiving hemodialysis.

HEAL Headliners: Engaging Patients and Communities in HEAL Research
Venue: Live online via Zoom
Date and Time: May 5, 2023; 2:00 pm ET
Register free online

March 22, 2023: A Decade of PCORnet Research in This Week’s PCT Grand Rounds

Posted on by Damon Seils

Headshots of Erin Holve, Russell Rothman, Schuyler Jones, and Neha PagidipatiIn this Friday’s PCT Grand Rounds, Erin Holve, Russell Rothman, Schuyler Jones, and Neha Pagidipati will present “From Observational Studies to Pragmatic Clinical Trials: (Almost) A Decade of Research in PCORnet®.” The Grand Rounds session will be held on Friday, March 24, 2023, at 1:00 pm eastern.

Holve is the chief research infrastructure officer for PCORI. Rothman is the principal investigator of the PCORI-funded Mid-South Clinical Data Research Network and a member of the PCORnet Executive Committee. Jones is an associate professor of medicine and population health sciences at Duke University. Pagidipati is an associate professor of medicine at Duke University.

Join the online meeting.

Grand Rounds March 17, 2023: Remote Symptom Monitoring with Electronic Patient-Reported Outcomes (ePROs) in Oncology (Ethan Basch, MD, MSc)

Posted on by Elizabeth Mccamic

Speaker

Ethan Basch, MD, MSc
Richard M. Goldberg Distinguished Professor and Chief of Oncology
Physician-in-Chief, North Carolina Cancer Hospital
Director, Cancer Outcomes Research Program
University of North Carolina

 

Keywords

Patient-reported outcomes, Oncology, Clinical Trials

 

Key Points

  • Symptoms are common in cancer care. They interfere with physical function and daily activities, lead to avoidable ER/hospital visits and readmissions, and preclude treatment. Symptom management is a cornerstone of quality care, but do we adequately detect and manage symptoms?
  • In the standard approach to symptom monitoring, the clinical team may not be aware of a patient’s symptoms, so when the symptoms become worse the care team has to be reactive. In a model for systematic symptom monitoring, a patient might report symptoms electronically in real-time via e-reports or e-alerts so the care team can respond proactively and have data to review at the next visit.
  • Clinicians think they know how patients are doing already and do not need self-reported data; however in a study that compared patient-reported and clinician-reported symptoms, there was a substantial gap between the symptoms that were reported by patients and symptoms that were not reported by clinicians. There is an opportunity to do better through navigation, portals, and patient-reported outcomes to bridge the gap.
  • We conducted a large single-center “STAR” Study to find out if the gap between patient and clinician reported symptoms makes an impact on clinical outcomes. There were 766 patients enrolled in the study with a median follow up of 7 years. Quality of life (QOL) was assessed at 6 months. Compared to standard care, 31% more patients in the self-reporting arm experienced QOL benefits; 7% fewer patients in self-reporting arm visited the ER with durable effects throughout the study; and median survival was 5.2 months longer among patients in the self-reporting arm. There was a 5-year absolute survival benefit of 8%.
  • Proactive monitoring prompts clinicians to act early before symptoms worsen and cause downstream complications. Symptom control enables patients to stay more functional, which is known to be associated with better survival. Symptom monitoring enables control of chemotherapy side effects, enabling more intensive and longer duration of cancer treatment.
  • The PRO-TECT Cancer Symptom Study was a cluster randomized trial at 52 U.S. community oncology practices across 25 states. At each practice up to 50 patients with metastatic cancer, receiving systemic therapy, not on a therapeutic trial, were randomized to an intervention arm with digital monitoring or a control arm with usual care. The intervention arm had to complete weekly PRO survey items from NCI PRO-CTCAE (pain, nausea, vomiting, constipation, diarrhea, dyspnea, insomnia, depression, oral intake).
  • In this study adherence with week ePROs was 91.5%. When patients didn’t report, they received a phone call to make sure they were OK. In the PRO arm there was a statistically significant improved physical function score, the study found significant benefits for the PRO arm for symptoms, and significant benefits on health-related quality of life.
  • Patient self-reporting improves symptom monitoring and outcomes in routine cancer care and clinical research by expanding our understanding of the patient experience and engaging patients. From a health services research perspective, it demonstrates how hard it is to change a simple process, even if it makes a lot of intuitive sense.

Learn more

Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

Discussion Themes

– How have clinician views changed in terms of patient-reported symptoms verses clinician understanding of symptoms? The culture has fundamentally changed. The research in this area has grown over time. 10 years ago or even 5 years ago many clinicians had not heard about patient-reported outcomes. Now almost any physician has heard of patient-reported outcomes. Technology has advanced during this time, and there are all kinds of ways for patients to communicate with providers. Regulatory authorities have become interested in PROs and it is thought of as a fundamental part of drug development. I have seen a remarkable change in views on patient-reported outcomes.

– How do we take all of these ePRO options and choose something that is right for a trial? It depends on the goals of the study. When you have more than one choice for the symptom domain or you run the risk of duplication how do you choose? FDA and other regulatory authorities want to think about symptoms separately from adverse events. You will want a dedicated tool for adverse events.

Tags

#pctGR, @Collaboratory1