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).

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

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

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

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

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

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 (PRO) 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

Mobile Health (mHealth) Research Platform to Launch


In recent health information technology news, the University of California, San Francisco (UCSF), has received a 5-year National Institutes of Health award to support its launch of a cardiovascular mHealth platform. The research platform, to be named Health ePeople, will build on the successes of UCSF’s Health eHeart Study, which began in 2013. That study, with more than 30,000 participants worldwide, uses the power of mobile technologies to collect cardiovascular data and patient-reported outcomes (PROs) from study participants.

The Health ePeople platform will advance mHealth by providing researchers with easy access to a large cohort of volunteers, along with a quick, affordable means for collecting their health data through mobile and wireless technologies. Though the platform will not be ready to enroll new participants for several months, people who want to participate in the cohort can sign up through the Health eHeart Study website.

For information and short videos on mHealth technologies, visit the Living Textbook’s chapter on mHealth and PROs.


Study Shows Patient-Reported Outcomes Valid & Reliable for Adverse Events of Cancer Treatment


In a study recently published in JAMA Oncology, researchers found that patient reporting of adverse events of cancer treatment using a new scale gave valid and reliable assessments that correlated with standard measures of functioning and quality of life. The National Cancer Institute (NCI) developed a patient-reported outcome (PRO) version of its Common Terminology Criteria for Adverse Events (CTCAE), which is the standard system for reporting toxicities of cancer treatment in clinical trials. The PRO-CTCAE was then tested among more than 900 patients undergoing treatment at 9 cancer centers. As described in a commentary by Benjamin Movsas, MD, these results are encouraging for PROs to be integrated in informing treatment recommendations, symptom management, and even labeling decisions.

Read more about PROs in the Living Textbook chapter on this topic.


Patient-Reported Outcomes Workshop Report Available


Tools for ResearchIn January of 2015, the NIH HCS Collaboratory’s Patient-Reported Outcomes (PRO) Core Group convened a 2-day workshop in Baltimore devoted to identifying barriers and possible solutions to the use of NIH-supported PRO tools in comparative-effectiveness research (CER).

Findings from the meeting, which include case study presentations and reflections from multiple stakeholders representing the research, clinical, and patient communities, were distilled into a summary document available from the NIH Collaboratory Knowledge Repository at the link below:

The workshop summary is also available on the Living Textbook’s “Tools for Research” section, under “Patient-Reported Outcomes White Paper.


The National Patient-Centered Clinical Research Network (PCORnet) Adopts Definitions for Information Provided by Patients

The NaTools for Researchtional Patient-Centered Clinical Research Network (PCORnet) Patient-Reported Outcomes (PRO) Task Force has released a new white paper that delineates definitions for data contributed by patients. They hope to facilitate the incorporation of information provided by patients across the networks and to maximize data sharing and interoperability by operationalizing the terms within PCORnet.

The following three terms were adopted to describe the patient-contributed data collected across PCORnet:

PATIENT-REPORTED OUTCOME (PRO): a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.

PATIENT-CENTERED OUTCOMES: outcomes that matter to patients

PATIENT-GENERATED HEALTH DATA: health-related data (such as health history, symptoms, biometric data, treatment history, lifestyle choices, and other information) that are created, recorded, gathered, or inferred by or from patients or their designees (i.e., care partners or those who assist them) to help address a health concern. Patient-generated health data include patient-reported outcomes.

The white paper can be found under Patient-Reported Outcomes White Papers on the Tools for Research page on the living textbook, or accessed directly here (PDF).