Research Tools for Patient-Reported Outcomes

Powerful new tools can be used to advance nursing science.

Today, more people are living with chronic illnesses and the adverse symptoms that result from them. New advances in genomics and other fields have allowed scientists to better understand the symptoms of chronic illness, such as pain, fatigue, and disordered sleep. NINR supports research to develop improved, personalized strategies to treat and prevent the adverse symptoms of illness across diverse populations and settings. With support from the NIH, new tools are being developed to assess symptoms and health-related quality of life.

One example is the Patient-Reported Outcomes Measurement Information System (PROMIS®). Begun with funding from the NIH Roadmap for Biomedical Research, followed by additional support from the NIH Common Fund, PROMIS® is developing new ways to measure patient-reported outcomes (PROs) across “domains,” which are categories of specific feelings, functions, or perceptions, such as pain, fatigue, physical functioning, emotional distress, and social role participation. Existing research indicates that each of these areas has a major impact on quality of life across a variety of chronic illnesses. The PROMIS initiative addresses a need in the clinical research community for rigorously tested tools that utilize recent advances in information technology, psychometrics, and qualitative [1-3], cognitive, and health survey research to improve the reporting and quantification of changes in PROs associated with disease progression or response to treatment.

PROMIS® has developed a psychometrically-robust computer adaptive testing (CAT) system, based on item response theory (IRT), to administer questions, or “items,” which adds quantitative rigor to qualitative assessments. Whether delivered through the iterative CAT system, that allows research flexibility, or by paper version short forms, PROMIS® has already demonstrated improved efficiency and sensitivity in comparison with existing PRO instruments and reduced burden on patients.[4] Because it is domain-specific (e.g., pain, fatigue, physical functioning) rather than disease-specific, PROMIS® could facilitate comparisons across studies, the general population, and with other patient populations.

The NIH Toolbox and NeuroQOL are additional tools that focus on neurological function and behavior. Like PROMIS®, they use state-of-the-art psychometric approaches, as well as CAT, and are adaptable to advances in measurement and technology.

  • The NIH Toolbox is an integrated set of measures for cognitive, emotional, motor and sensory function; its development was supported, in part, by the NIH Blueprint for Neuroscience Research and the NIH Office of Behavioral and Social Science Research. By using multiple constructs of each domain, the NIH Toolbox’s can be used to monitor neurological and behavioral function over time, by assessing  domain constructs, such as executive function, psychological well-being, and pain. The measurement of these domain constructs facilitates the study of functional changes across the lifespan (ages 3-85), including evaluating intervention and treatment effectiveness and, similar to PROMIS®, could allow cross-study comparisons. The NIH Toolbox is expected to provide a more complete picture of neurological and behavioral health in clinical trials and observational studies, and across diverse populations and clinical settings.
  • NeuroQOL, which was developed with support from the National Institute of Neurological Disorders and Stroke, offers short forms and item banks to obtain information from patients with neurological diseases, such as epilepsy, Parkinson’s disease, and multiple sclerosis. These tools are designed to gather self-reported health-related quality of life data (e.g., anxiety, activities of daily living, applied cognition, sleep disturbance) within and across multiple neurological conditions.

The NIH Toolbox and PROMIS® tools have been validated for use in diverse cultures, ethnic and geographic groups, and ages, and they meet the need for a standard set of measures that can be used as a “common currency” across diverse study designs and settings. These factors facilitate the assembly of larger datasets, as well as allow more rigorous comparison of studies and bolster conclusions from related research.

The NIH Toolbox, NeuroQOL, and PROMIS® tools are available in English and Spanish, and some of the PROMIS® short forms have been translated into several other languages, such as Mandarin Chinese, Hebrew, and Portuguese. However, some researchers have reported that not all of the Spanish translation of PROMIS® is suitable for their study populations.[5-7] Although some of these new tools include items from past or current PRO instruments, PROMIS®, the NIH Toolbox, and NeuroQOL may not be compatible with existing tools, which could hamper comparisons with previous research.

The web-based Assessment CenterSM is a no-cost[8] platform that provides access to PROMIS®, the NIH Toolbox, and NeuroQOL, including their item banks, CAT system, short forms, and other tools under development. Assessment CenterSM also facilitates training in the use of the tools, customization of items or instruments, real-time scoring of CATs and short forms, data export and storage, and collaboration with other researchers. Assessment CenterSM experts are readily available for guidance and support in navigating the website.

NINR encourages investigators to explore these powerful new tools and the opportunities to incorporate them into study designs to further symptom science, self-management, wellness, and end-of-life/palliative care research.

Patricia A. Grady, PhD, RN, FAAN
Director
National Institute of Nursing Research
National Institutes of Health

[1] Bevans KB, et al., J Pediatr Psychol. 2013 Mar;38(2):173-91

[2] Flynn KE, et al., Psychooncology. 2010 Oct;19(10):1086-93

[3] Salsmana JM, et al. Neurology. 2013 Mar 12;80(11 Suppl 3):S76-86

[4] Fries JF, et al., J Rheumatol. 2009 Sep;36(9):2061-6

[5] Paz SH, et al., Qual Life Res. 2013 Sep: 22(7):1819-30

[6] Bevans M, Ross A, Cella D. Nurs Outlook. 2014 Jun 12.

[7] Badger T, et al., Nurs Outlook. 2014 Jul 10.

[8] Fees may apply for use of the NIH Toolbox in projects not funded by NIH.