Patient-reported outcomes

March 17, 2021: Cultural and Linguistic Adaptations: Results of a Survey of PRISM NIH Collaboratory Trials

Posted on by Karen Staman

To better understand efforts at cultural and linguistic adaptions in clinical trials focused on pain, members of the NIH Collaboratory’s Patient-Centered Outcomes Core recently conducted a survey of the PRISM NIH Collaboratory Trials (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing), which are part of NIH’s Helping to End Addiction Long-term Initiative℠, or NIH HEAL Initiative℠.

"Having culturally valid patient-reported outcome instruments is extremely important to the conduct of embedded pragmatic trials. For the results to be valid, the intended audience needs to understand both the measure and what is being asked of them. This is critical for trials centered on pain because pain-related outcomes are best described by the person who is experiencing the symptoms."  —Emily O’Brien, PhD, Co-Chair of the Patient-Centered Outcomes Core

The results are published in the Cultural Adaptation and Linguistic Translation section of the Patient-Reported Outcomes Chapter in the Living Textbook.
For more information on the PRISM projects, study snapshots are accessible from each NIH Collaboratory Trial page and the links below:

  • BackInAction: Pragmatic Trial of Acupuncture for Chronic Low Back Pain in Older Adults
  • FM TIPS: Fibromyalgia TENS in Physical Therapy Study
  • OPTIMUM: Group-based Mindfulness for Patients with Chronic Low Back Pain in the Primary Care Setting
  • NOHARM: Nonpharmacologic Options in Postoperative Hospital-based and Rehabilitation Pain Management

Note that the 2 newest PRISM projects, GRACE and BeatPain Utah, are still in the design phase and were not included in this round of interviews.

November 5, 2020: NIH HEAL Initiative Common Data Elements Published in the Living Textbook

Posted on by Karen Staman

Logo for the NIH HEAL InitiativeThe Helping to End Addiction Long-Term Initiative℠, or NIH HEAL Initiative℠, has created common data elements (CDEs) to facilitate cross-study comparisons for pain, improve interoperability of findings for patient-reported outcomes, and compare results across trials. The core CDEs were published recently in the NIH Collaboratory’s Living Textbook. They include CDEs for adult acute pain, adult chronic pain, and pediatric acute and chronic pain, and represent patient-reported outcomes that HEAL Initiative clinical trials are required to collect.

“Having common measures to capture the patient’s perspective regarding pain will enable researchers to compare meaningful data across pain conditions, in diverse populations, and in multiple research studies, including in pragmatic clinical research. This should greatly increase our understanding of pain and how best to treat it.”

      —Emily O’Brien, PhD, co–principal investigator of the NIH Collaboratory Patient-Centered Outcomes Core

The NIH Collaboratory serves as the resource coordinating center for 6 NIH Collaboratory Trials funded through the Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing (PRISM) program, a component of the NIH HEAL Initiative:

Because the PRISM studies are pragmatic clinical trials embedded in healthcare systems, it may not be feasible for investigators to collect all the required CDEs, as the trials need to be incorporated into clinical workflow with as little burden as possible. The NIH Collaboratory’s Patient-Centered Outcomes Core Working Group will work with the NIH Collaboratory Trials to understand and document which patient-reported outcome measures are collected and the perceived burden associated with collecting them.

Learn more in the Choosing PRO Measures chapter of the Living Textbook.

June 4, 2020: New Chapter in the Living Textbook Describes the Uses and How-tos of Patient-Reported Outcomes

Posted on by Karen Staman

A new chapter in the Living Textbook describes how patient-reported outcomes (PROs) are used in different settings and how to choose and integrate PRO measures into an embedded pragmatic clinical trial.

The chapter also characterizes PRO use within the PRISM program (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing) of the NIH HEAL Initiative (Helping End Addiction Long-term Initiative). NIH Collaboratory Trials supported through the PRISM program include:

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

The main topics of the chapter are:

The authors also include Core Outcome Sets for chronic pain, low back pain, musculoskeletal pain, and cancer.

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

Posted on by Karen Staman

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

 

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

Posted on by Karen Staman

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).

March 14, 2018: Public Input on Patient-Reported Outcome Measures Due April 1

Posted on by Karen Staman

The Agency for Healthcare Research and Quality (AHRQ) is requesting information to help inform a competition in the fall to develop and implement user-friendly technical tools for the collection of patient-reported outcome (PRO) data. The AHRQ is interested in learning about experiences with physical function PRO measures that are currently in use in ambulatory care settings, including primary and secondary care, as well as the methods used to collect these data. The information will be used to select physical function measures for the competition, which aims to bolster collection and integration of PRO data in the electronic health record by developing and piloting new, user-friendly tools. Responses can be emailed to Janey.hsiao@ahrq.hhs.gov and are due April 1, 2018.

February 28, 2018: New Meeting Summary Examines How to Integrate Patient‐Reported Health Data for Pragmatic Research

Posted on by Karen Staman

A recently released summary from the ADAPTABLE Roundtable Meeting explores ways to better understand the sets of circumstances and considerations that could guide when and how to gather and integrate patient-reported health data with other data sources in pragmatic trials.

For outcomes that represent subjective experiences, such as pain, symptoms, and physical functioning, the patient is the unique and privileged source of information. Other patient-reported health data may not have a clear source of truth, such as co-morbidities and hospitalizations. In such cases, patient-reported health data may supplement, contradict, or agree with EHR and claims data. For example, medication data reported by patients might be a more accurate reflection of what patients are actually taking than medication data in the EHR, especially for over-the-counter medications.

Patient-reported health data come from various sources and can be feasibly collected in the conduct of a pragmatic clinical trial, but the optimal approaches for capturing and analyzing these data are unclear. Questions include how to integrate this information with other data collected as part of a study, including data from the EHR.

To better understand patient-reported health data and how to use them in pragmatic trials, 18 experts from 8 institutions convened at the roundtable meeting, coming from a wide variety of backgrounds including biostatistics, epidemiology, oncology, nursing, psychiatry, health policy, and regulation. Representatives from the NIH Collaboratory included Drs. Lesley Curtis and Rachel Richesson from the EHR Core and Dr. Kevin Weinfurt from the Patient-Reported Outcomes Core.

In addition to the meeting summary, two white papers are forthcoming. For more information about using patient-reported data in pragmatic trials, see the Living Textbook Chapter on Endpoints and Outcomes.

This effort was funded by Office of the Assistant Secretary for Planning and Evaluation at the U.S. Department of Health and Human Services through a supplement provided to the NIH Collaboratory Coordinating Center.

January 22, 2018: AHRQ Announces Competition to Develop Patient-Reported Outcomes Tools

Posted on by Karen Staman

Patient-reported outcome (PRO) data are critical for informing patient-centered care and patient-centered outcomes research, although they are not commonly collected in routine care, and are often not available for use in clinical care or research. To counter this problem, the Agency for Healthcare Research and Quality (AHRQ) announced an upcoming competition to develop tools to collect and integrate PRO data in electronic health records and other health IT products. During this multi-phase Challenge Competition in the fall of 2018, developers will be asked to create user-friendly application programming interfaces (APIs) and other tools that can be used to collect physical function data in ambulatory care settings based on specifications provided by AHRQ. The tools will enable PRO data to be shared more easily and regularly with clinicians and researchers.

December 12, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Patient-Reported Outcomes Core

Posted on by Karen Staman

We recently asked Dr. Kevin Weinfurt, Chair of the Patient-Reported Outcomes (PRO) Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. He says the biggest impact of the Core has been working with national initiatives to improve inclusion of PROs in the electronic health record (EHR). Further, Core members have contributed to new knowledge through white papers and chapters in the Living Textbook. In the coming years, he’s hoping the Core will be able to identify the value proposition of PROs.

“Because there are costs associated with collecting PROs, we need to determine when PROS are essential, supporting, or not at all informative for the clinical questions. This gets at the value proposition: When are they of value and to whom?” —Dr. Kevin Weinfurt.

Read more from Dr. Weinfurt in the full interview (pdf).

New White Paper from Collaboratory PRO Core on the Impact of Patient-Reported Outcomes on Clinical Practice

Posted on by Karen Staman

Patient-reported outcome (PRO) measures are often used in pragmatic clinical trials to assess endpoints that are meaningful to stakeholders. These measures may also support patient care, although there is mixed evidence about effects of PROs on (1) improved patient-provider communication, clinical decision-making, and patient satisfaction; (2) enhanced patient outcomes; and (3) helped ensure better quality of care from a healthcare systems perspective. In a new white paper from the Collaboratory Patient-Reported Outcomes Core, the available evidence in the literature is examined to determine when PROs have the potential to provide added value to patient care.

The full text of the white paper can be found here: Impact of Patient-Reported Outcomes on Clinical Practice_V1.0