patient-centered outcomes research

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

March 15, 2023: In This Friday’s PCT Grand Rounds, PROs for Adverse Event Monitoring in Oncology

Posted on by Damon Seils

Headshot of Dr. Ethan BaschIn this Friday’s PCT Grand Rounds, Ethan Basch of the University of North Carolina at Chapel Hill will present “Patient-Reported Outcomes for Symptom and Adverse Event Monitoring in Oncology.” The Grand Rounds session will be held on Friday, March 17, 2023, at 1:00 pm eastern.

Basch is the Richard M. Goldberg Distinguished Professor of Medical Oncology in the UNC School of Medicine and a professor of health policy and management in the UNC Gillings School of Global Public Health. He led the National Cancer Institute’s initiative to develop a patient-reported adverse event monitoring system for use in clinical research (the “PRO-CTCAE”), and he is the study chair for multiple trials employing patient-reported endpoints.

Join the online meeting.

August 30, 2022: FDA Announces Webinar on Patient-Focused Drug Development Draft Guidance

Posted on by Damon Seils

FDA logoThe US Food and Drug Administration (FDA) will host a webinar on September 9 for industry, patient groups, and other interested stakeholders to discuss and answer questions about the draft guidance, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.

The draft guidance, known as “Guidance 3,” is the third of 4 methodological guidance documents for patient-focused drug development that describe how patients, caregivers, researchers, medical product developers, and others can collect and submit patient experience data and other relevant information to be used for medical product development and regulatory decision making. Guidance 3 discusses approaches to selecting, modifying, developing, and validating clinical outcome assessments to measure outcomes of importance to patients in clinical trials.

Register for the webinar at https://www.eventbrite.com/e/public-webinar-patient-focused-drug-development-pfdd-draft-guidance-3-tickets-397246183027.

July 7, 2022: Patient-Centered Outcomes Core Values Cross-Core Collaboration and Real-World Knowledge

Posted on by terren.green@duke.edu

Headshot of Dr. Emily O'Brien
Dr. Emily O'Brien

In connection with the NIH Pragmatic Trials Collaboratory’s annual Steering Committee meeting and 10th anniversary celebration, we interviewed Dr. Christy Zigler and Dr. Emily O’Brien, asking them to reflect on the role of the Patient-Centered Outcomes Core in helping the NIH Collaboratory Trial teams plan and implement their trials, and to discuss their focus for the Core’s future contributions to pragmatic clinical trials.

Based on your experience working with the NIH Collaboratory Trials, what are some of the common challenges of patient-centered outcomes?

Based on our experience, the challenges that NIH Collaboratory Trials face with patient-centered outcomes can occur across several project periods. In the planning phase, we see a number of basic questions about patient-reported outcomes (PROs), including “What outcomes should be measured?” and “What measures should be used to capture these outcomes?”

Once appropriate measures have been identified, there are often challenges related to implementation of these measures and resources. For example, project teams are usually interested in understanding the availability of existing PROs within the electronic health records that study sites could leverage, as well as which measures have appropriate translations or cross-cultural validation versions that are already established.

We also have come across some interesting Core-crossing challenges, including ethical considerations for collecting PROs (Who is responsible for monitoring patient responses?) and data collection methods (Should PROs be integrated within the EHR systems or in a separate repository?). Finally, sites are sensitive to patient and system burden of PRO collection, how to reduce the likelihood of missing data, and how patient-centered outcomes fit into the overall statistical analysis plan.

Dr. Christy Zigler

What strategies have NIH Collaboratory Trials used to overcome these barriers?

So far, the NIH Collaboratory Trials have been able to share resources and test out some direct strategies to overcome these challenges. For example, some projects have created outcome-specific data silos and also tested different administration methods. The Cores have also been able to provide insight into best practices for making these decisions, such as when to use particular formats for PRO administration (eg, in clinic, via telephone, via text, via electronic capture).

How are the NIH Collaboratory Trials’ experiences with patient-centered outcomes helping the field of pragmatic research?

We’ve really be able to pull together the lessons learned around patient-centered outcomes in pragmatic research to help inform the field. In particular, even for NIH Collaboratory Trials that have not conferred directly with our Core, we have been able to interview them about their work. For example, we highlighted critical advice from the Guiding Good Choices for Health (GGC4H) NIH Collaboratory Trial on the Living Textbook. Their real-world experience with determining and utilizing patient-centered outcomes provide a road map for other investigators.

What do you think the Core can contribute over the next decade?

Our wish list for future contributions includes:

  • More cross-core collaboration—specifically, conferring with the Biostatistics and Study Design Core on quantitative measurement questions around topics like meaningful change and longitudinal responsiveness within the context of pragmatic trials.
  • More focus on lessons learned from NIH Collaboratory Trial teams who have worked through their enrollment periods and have successful response rates. This real-world knowledge is very valuable to the field.
  • More focus on how to create equity within patient-centered outcomes in pragmatic trials.

 

June 27, 2022: Special Edition of Contemporary Clinical Trials Offers New Perspectives on Pragmatic and Virtual Clinical Trials

Posted on by Elizabeth Mccamic

Contemporary Clinical TrialslsA special series of articles addressing pragmatic and virtual trials appears this week in the journal Contemporary Clinical Trials and offers practical approaches to the many challenges clinical trials face.

The 14 articles bring together leaders, researchers, biostatisticians, and bioethicists—including members of the NIH Pragmatic Trials Collaboratory—who are rethinking key aspects of the development, conduct, and oversight of clinical trials.

The articles include recommendations and real-world experience on how clinical trials can be done better while maintaining high-quality results using digital technology, direct-to-participant methods, and electronic health records.

The special issue can be found on the Contemporary Clinical Trials website.

Adrian F. Hernandez, MD, MHS, a co-principal investigator for the NIH Pragmatic Trials Collaboratory Coordinating Center, and Tammy Reece, MS, PMP, CCRA, the program’s project director, served as the issue co-editors. Dr. Hernandez is also vice dean and executive director of the Duke Clinical Research Institute and professor of medicine at Duke University. Ms. Reece is a Senior Project Leader in the Government Trials and Networks Group at the Duke Clinical Research Institute.

Following an introduction written by Hernandez, the remaining 13 articles focus on topics including simplification by design, digital technologies and clinical trials, iterative approaches to the use of electronic health records data for large pragmatic studies, ethics and regulatory considerations, and lifestyle and behavioral modification trials.

This body of work offers pragmatic approaches to overcoming the many reasons clinical trials are underperforming, such as high financial costs, long duration, administrative complexity, low recruitment and retention of participants, and difficulty translating knowledge to actual medical care.

“The articles in this issue recognize advances in evidence generation while sharing the challenges and gaps that still need to be addressed,” Hernandez said. “Ultimately, the clinical research ecosystem must improve, not only to keep pace with the advances in science, but to improve public health in meaningful ways.”

“The NIH Pragmatic Trials Collaboratory has been working to re-engineer clinical trials for a decade, yet there is still progress to be made,” Reece said. “Sharing knowledge and innovations across disciplines is critical to continue the momentum of transformation toward a learning health system.”

May 23, 2022: Solving Problems Collaboratively in Partnership With the Patient-Centered Outcomes Research Trust Fund

Posted on by Elizabeth Mccamic

Over the last 10 years, the Patient-Centered Outcomes Research Trust Fund (OS-PCORTF) has brought together agencies to work on joint problems and made data investments to enable research that generates knowledge about patient-centered outcomes.

Nancy DeLew, MA, MPA, Associate Deputy Assistant Secretary for Health Policy in the Office of the Assistant Secretary for Planning and Evaluation in the Department of Health and Human Services, and Wendy Weber, ND, PhD, MPH, NIH Project Officer for the NIH Pragmatic Trials Collaboratory, discussed the fund in an interview during the NIH Pragmatic Trials Collaboratory’s annual Steering Committee meeting in April.

The OS-PCORTF has three purposes: to fund research, to fund dissemination and training, and to fund data infrastructure.

Trials, such as the ADAPTABLE study, have used funds from the OS-PCORTF for additional research. In the ADAPTABLE study, for instance, investigators wanted to ask whether the information that is in the electronic health record matches what the patient would report on a variety of patient-reported data. The NIH Pragmatic Trials Collaboratory also organized a roundtable discussion with researchers from the ADAPTABLE study to produce consensus findings on the analysis and integration of patient-reported health data in clinical trials.

“That’s been a really great example of working together,” Weber said. “As the programs evolved and especially in the new strategic planning efforts, there were a lot of questions about how the Collaboratory investigators use all of the various data. They were interviewed and part of the workshops and other elements to help frame the new strategic priorities for the Trust Fund.”

The OS-PCORTF has made prioritizing patient-centered research more possible.

“I think the Trust Fund has helped identify patient-centered, person-centered work as a high priority, which might not have been in the forefront of people’s minds before the Trust Fund was enacted in the Congress,” DeLew said. “By the 10-year reauthorization, we know we have additional funds to do more work. We’re trying to help agencies create evidence that we can then use in decision making in the Department, and we think that will help drive health outcomes over the next decade.”

As the OS-PCORTF moves into its next 10 years of funding, there are a several goals the Trust Fund wants to achieve, DeLew said.

The future focus will be on projects that address national health priorities and projects that will link data over time and help build a longitudinal track record for patients and their experience. Another goal is to take advantage of innovation that is happening in the data field, such as machine learning and artificial intelligence. The Trust Fund also aims to improve health equity and ensure its research is inclusive of populations who might not have been in clinical trials in the past.

“We also want to branch out and move into climate change. That’s an area we haven’t addressed at all in the first decade of the Trust Fund and that’s a high priority for us going forward as well,” DeLew said.

View the full interview.

See the complete materials from the 2022 Steering Committee meeting.