Advances in Patient Reported Outcomes: The NIH PROMIS Measures
Patient reported outcomes (PROs) are questionnaire measures of patients’ symptoms, functioning, and health-related quality of life. They are designed to provide important clinical information that generally cannot be captured with objective medical testing. Recent legislative developments in the United States support greater efforts to include patients’ reports of health experience to evaluate treatment outcomes, engage in shared decision-making, and prioritize the focus of treatment. PROs have garnered increased attention by the Food and Drug Administration (FDA) for evaluating drugs and medical devices. Recent calls for comparative effectiveness research favor inclusion of PROs. In 2004, the National Institutes of Health launched a research initiative to improve the clinical research enterprise by developing state-of-the-art PROs. The NIH Patient Reported Outcomes Measurement System (PROMIS) and Assessment Center are the products of that initiative. Adult, pediatric, and parent-proxy item banks have been developed using contemporary psychometric methods, yielding rapid, accurate measurement. PROMIS currently provides tools for assessing physical, mental, and social health using short-form and computer adaptive testing methods. The PROMIS tools are being adopted for use in clinical trials and translational research. They are also being introduced in clinical medicine to assess a broad range of disease outcomes. There is potential for use of PROs to improve quality of care and disease outcomes, to provide patient-centered assessment for comparative effectiveness research, and to enable a common metric for tracking outcomes across providers and medical systems.
Tools and systems to capture PROs
Data Collection Tools/Healthcare Settings
The NIH PROMIS initiative was designed to “reengineer the clinical research enterprise” by creating generic health measures with improved reliability, validity, and precision based on use of item response theory IRT) methods. The program funded over a dozen research sites across the country to engage in fundamental psychometric item bank and scale development. One important outcome was item calibrations that allows for computer adaptive testing (CAT) delivery of scales. Researchers/clinicians can access large item banks for physical, mental, and social health domains for administration of short forms and CATs.
Generalizability to other settings and best practices
This paper outlines the thinking behind the PROMIS initiative and the psychometric advances applied to the initiative. It also addresses contemporary health care policy mandates relating to PROs as well as opportunities for novel use of PROs beyond outcomes in clinical trials. The goal of this paper is to acquaint the reader with PROMIS and PRO measurement issues and to demonstrate how recent developments have the potential to modernize measurement in clinical trials, facilitate comparative effectiveness research (CER), and improve clinical care.
Acknowledgement of Funders
PROMIS® was funded with cooperative agreements from the National Institutes of Health (NIH) Common Fund Initiative.