Special Section on Patient-Reported Outcomes and Informatics: Collection of Patient-Reported Outcome Measures in Rural and Underserved Populations.

The NIH Pragmatic Trials Collaboratory supports the design and conduct of 31 embedded pragmatic clinical trials, and many of these trials use patient-reported outcome measures (PROMs) to provide valuable information about their patients' health and wellness. Often these trials enroll medically underserved patients, including people with incomes below the federal poverty threshold, racial or ethnically minoritized groups, or rural or frontier communities. In this series of trial case reports, we provide lessons learned about collecting PROMs in these populations.

Pain Interference in Juvenile Idiopathic Arthritis.

Objective: Despite treatment advances, pain remains a serious problem for many children with juvenile idiopathic arthritis (JIA). To better understand pain in children with JIA and identify potentially modifiable factors, this study evaluated Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference (PI) and its relationships with other pain measures and demographic, clinical, psychosocial, and functional variables.

Methods: This cross-sectional, observational, multicenter study used descriptive statistics and a mix of bivariate and multivariable analyses to describe PI and characterize relationships with other measures and variables.

Endpoints and outcomes for localized scleroderma/morphea: a scoping literature review.

Background: Current treatment for localized scleroderma (LS) has been shown to halt disease activity, but little is still known about patient experiences with these treatments, nor is there consensus about optimal measurement strategies for future clinical trials.

Objective: Conduct a scoping review of the literature for the types of outcomes and measures (i.e.

Understanding reliability of the observer-reported communication ability measure within Angelman syndrome through the lens of generalizability theory.

Aims: Caregivers rate improved communication ability as one of the most desired outcomes for successful interventions for individuals with Angelman syndrome (AS). When measuring communication ability in clinical trials, the reliability of such measures is critical for detecting significant changes over time. This study examined the reliability of the Observed-Reported Communication Ability (ORCA) measure completed by caregivers of individuals with AS.

Collecting patient-reported outcome measures in the electronic health record: Lessons from the NIH pragmatic trials Collaboratory.

The NIH Pragmatic Trials Collaboratory supports the design and conduct of 27 embedded pragmatic clinical trials, and many of the studies collect patient reported outcome measures as primary or secondary outcomes. Study teams have encountered challenges in the collection of these measures, including challenges related to competing health care system priorities, clinician's buy-in for adoption of patient-reported outcome measures, low adoption and reach of technology in low resource settings, and lack of consensus and standardization of patient-reported outcome measure selection and administration in the electronic health record. In this article, we share case examples and lessons learned, and suggest that, when using patient-reported outcome measures for embedded pragmatic clinical trials, investigators must make important decisions about whether to use data collected from the participating health system's electronic health record, integrate externally collected patient-reported outcome data into the electronic health record, or collect these data in separate systems for their studies.

Patient experience of spontaneous intracranial hypotension (SIH): qualitative interviews for concept elicitation.

Background & Objectives: Spontaneous intracranial hypotension (SIH) is an underdiagnosed and debilitating condition caused by a spinal cerebrospinal fluid (CSF) leak. Although SIH can lead to substantial morbidity and disability, little data exists about patients' perspectives. Without hearing directly from patients, our understanding of the full experience of having SIH is limited, as is our ability to identify and use appropriate patient-reported outcome measures (PROMs) within clinical care and research.

Validation of the Observer-Reported Communication Ability (ORCA) measure for individuals with Rett syndrome.

Purpose: The study goal was to validate the Observer-Reported Communication Ability (ORCA) measure for use with females with Rett Syndrome (RTT).

Methods: Qualitative interviews, including concept elicitation and cognitive interviewing methods, were conducted with 19 caregivers of individuals with RTT ages 2 and older. A quantitative study was then conducted in 279 caregivers to evaluate construct validity and reliability.

Equity and bias in electronic health records data.

Embedded pragmatic clinical trials (ePCTs) are conducted during routine clinical care and have the potential to increase knowledge about the effectiveness of interventions under real world conditions. However, many pragmatic trials rely on data from the electronic health record (EHR) data, which are subject to bias from incomplete data, poor data quality, lack of representation from people who are medically underserved, and implicit bias in EHR design. This commentary examines how the use of EHR data might exacerbate bias and potentially increase health inequities.

Exploring Communication Ability in Individuals With Angelman Syndrome: Findings From Qualitative Interviews With Caregivers.

Communication deficits have a substantial impact on quality of life for individuals with Angelman syndrome (AS) and their families, but limited qualitative work exists to support the necessary content of measures aiming to assess communication for these individuals. Following best practices for concept elicitation studies, we conducted individual qualitative interviews with caregivers and clinicians to elicit meaningful aspects of communication for individuals with AS. Caregivers were able to discuss their child's specific communication behaviors within a large number of expressive, receptive, and pragmatic functions via numerous symbolic and non-symbolic modalities.

Validation of the Observer-Reported Communication Ability (ORCA) Measure for Individuals With Angelman Syndrome.

There is a critical need for high-quality clinical outcome assessments to capture the important aspects of communication ability of individuals with Angelman syndrome (AS). To center the perspective of caregivers, our team developed the novel Observer-Reported Communication Ability (ORCA) measure using best practice guidelines, with the goal of developing a measure that could be administered to caregivers directly without the need for a certified administrator for use in clinical trials. To refine the draft measure, we conducted two rounds of cognitive interviews with 24 caregivers and a quantitative study including 249 caregivers.

Current Utilization of Qualitative Methodologies in Dermatology: A Scoping Review.

The focus of this review was to determine how qualitative methods are used in dermatology research and whether published manuscripts meet current standards for qualitative research. A scoping review of manuscripts published in English between January 1, 2016 and September 22, 2021 was conducted. A coding document was developed to collect information on authors, methodology, participants, research theme, and the presence of quality criteria as outlined by the Standards for Reporting Qualitative Research.

Assessing Patient-Reported Outcomes in Pediatric Rheumatic Diseases: Considerations and Future Directions.

For children with pediatric rheumatic diseases (PRDs), the inclusion of patient-reported outcomes (PROs) is critical to inform decision making in health care delivery and research settings. PROs are direct reports from a child on their health status, without interpretation by anyone else. PROs improve understanding of the patient experience, allow clinicians to provide patient-centered care, and add value to clinical trials.

Enhancing the use of EHR systems for pragmatic embedded research: lessons from the NIH Health Care Systems Research Collaboratory.

Objective: We identified challenges and solutions to using electronic health record (EHR) systems for the design and conduct of pragmatic research.

Materials And Methods: Since 2012, the Health Care Systems Research Collaboratory has served as the resource coordinating center for 21 pragmatic clinical trial demonstration projects. The EHR Core working group invited these demonstration projects to complete a written semistructured survey and used an inductive approach to review responses and identify EHR-related challenges and suggested EHR enhancements.

Malaria Diagnosis by U.S. Providers on Short-term Medical Mission Trips to Uganda.

Malaria is one of the top 10 leading causes of death in Uganda. Short-term medical missions (STMMs) to address unmet medical needs in lower-resource settings are increasingly common. Th is study evaluates correlations between patient and clinician variables and accurate malaria diagnosis by providers on STMMs to Uganda.

Exploratory investigation of the outcomes of wheelchair provision through two service models in Indonesia.

The World Health Organization (WHO) estimates that only 17-37% of the approximately 77 million people who need a wheelchair have access to one. Many organizations are trying to address this need through varying service delivery approaches. For instance, some adhere to WHO's recommended 8-steps service approach while others provide wheelchairs with little to no service.

Use of healthcare resources in patients with low back pain and comorbid depression or anxiety.

Background Context: Psychological comorbidities are important prognostic factors for low back pain (LBP). To develop improved treatment paradigms, it is first necessary to characterize and determine current patterns of treatment in this population.

Purpose: Identify how comorbid depression or anxiety in patients with LBP is related to use of healthcare resources.

Assessing the Field of Pediatric Rehabilitation Medicine's Interest in Expanding Fellowship Training to Pediatricians.

Background: Training opportunities to pursue a career in pediatric rehabilitation medicine (PRM) have evolved over the last 40 years, with the most recent change being the development and accreditation of PRM fellowships and subspecialty certification in PRM. Currently the American Board of Physical Medicine & Rehabilitation (ABPMR) requires all candidates for PRM subspecialty certification to have completed a physical medicine and rehabilitation (PM&R) residency. The small number of certified PRM physicians has prompted debate within the field about permitting pediatricians to enter PRM fellowships without having to complete a PM&R residency.

Association Between Public Knowledge About COVID-19, Trust in Information Sources, and Adherence to Social Distancing: Cross-Sectional Survey.

Background: The success of behavioral interventions and policies designed to reduce the impact of the COVID-19 pandemic depends on how well individuals are informed about both the consequences of infection and the steps that should be taken to reduce the impact of the disease.

Objective: The aim of this study was to investigate associations between public knowledge about COVID-19, adherence to social distancing, and public trust in government information sources (eg, the US Centers for Disease Control and Prevention), private sources (eg, FOX and CNN), and social networks (eg, Facebook and Twitter) to inform future policies related to critical information distribution.

Methods: We conducted a cross-sectional survey (N=1243) between April 10 and 14, 2020.

The importance of development standards for anchoring vignettes: an illustrative example from pediatric localized scleroderma quality of life.

Purpose: Anchoring vignettes (AVs) are a promising measurement technique to reduce bias in patient-reported outcome (PRO) measures by helping researchers understand differences in how individuals and groups interpret response options. However, little attention has been paid to ensure quality development of AVs, and their performance has not been well assessed in pediatric populations. In this study, we explore the application of a rigorous development process for AVs based upon current standards for PROs, as well as feasibility of AVs when administered to children and adolescents.

Epigenetic Effects on Pediatric Traumatic Brain Injury Recovery (EETR): An Observational, Prospective, Longitudinal Concurrent Cohort Study Protocol.

Unexplained heterogeneity in outcomes following pediatric traumatic brain injury (TBI) is one of the most critical barriers to the development of effective prognostic tools and therapeutics. The addition of personal biological factors to our prediction models may account for a significant portion of unexplained variance and advance the field toward precision rehabilitation medicine. The overarching goal of the Epigenetic Effects on Pediatric Traumatic Brain Injury Recovery (EETR) study is to investigate an epigenetic biomarker involved in both childhood adversity and postinjury neuroplasticity to better understand heterogeneity in neurobehavioral outcomes following pediatric TBI.

Factors Associated With Ambulation in Myelomeningocele: A Longitudinal Study From the National Spina Bifida Patient Registry.

Objective: Evidence is limited regarding clinical factors associated with ambulation status over the lifespan of individuals with myelomeningocele. We used longitudinal data from the National Spina Bifida Patient Registry to model population-level variation in ambulation over time and hypothesized that effects of clinical factors associated with ambulation would vary by age and motor level.

Design: A population-averaged generalized estimating equation was used to estimate the probability of independent ambulation.