Re-engineering the Clinical Research Enterprise

Clinical Outcomes Assessment

Overview

As part of the NIH Roadmap for Medical Research, the PROMIS (Patient-Reported Outcomes Measurement Information System) initiative is developing new ways to measure patient-reported outcomes (PROs), such as pain, fatigue, physical functioning, emotional distress, and social role participation that have a major impact on quality-of-life across a variety of chronic diseases. Clinical measures of health outcomes, such as x-rays and lab tests, may have minimal relevance to the day-to-day functioning of patients with chronic diseases. Often, the best way patients can judge the effectiveness of treatments is by changes in symptoms. The goal of PROMIS is to improve the reporting and quantification of changes in PROs.

This initiative applies to a wide range of disorders including cancer, congestive heart failure, depression, arthritis, and multiple sclerosis, as well as chronic pain conditions. PROMIS is creating new paradigms for how clinical research information is collected, used, and reported. The PROMIS initiative addresses a need in the clinical research community for a rigorously tested PRO measurement tool that utilizes recent advances in information technology, psychometrics, and qualitative, cognitive, and health survey research.

The PROMIS program is developing, testing, and implementing a reporting system to collect and quantify clinically important outcomes such as pain, fatigue, physical functioning, emotional distress, and social role participation. During the first phase of the initiative (2004 to present), PROMIS formed a network of researchers that developed questions or “items” to analyze these five outcomes or “domains.” PROMIS is creating a psychometrically-robust computer adaptive testing (CAT) system, based on item response theory (IRT), to administer these items. In addition, it is developing a web-based system to give clinical researchers access to the item banks and the CAT system. Whether administered through an 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 PROs. Long-term trials are planned to address issues of validity and sensitivity to change in clinical populations. The increased efficiency, flexibility, and sensitivity of PROMIS holds potential to become a widely-accepted, standardized PRO measurement tool that will allow greater comparability of studies, with reduced burden on patients.

As the PROMIS initiative moves to a second phase of Roadmap support, it will continue to advance the field of patient self-reporting in clinical research and practice, by:

• developing new items and domains,
• translating current and future items and domains into other languages such as Spanish and Chinese to facilitate international studies;
• conducting validation studies in large-scale clinical trials in a variety of clinical populations;
• making PROMIS tools accessible to a wider range of clinical researchers and patient-care communities, and optimizing its usability for rapid adoption;
• providing on-going education and outreach to familiarize users with new developments in PROMIS;
• improving PROMIS tools to allow for better outcomes in clinical trials, and, potentially, better individual and clinical decisions;
• engaging stakeholders at all levels, by continuing interactions with other health-related federal agencies, forging new relationships with patients and patient organizations, and establishing public-private partnerships to sustain PROMIS once Roadmap funding ends.

For more information on the Re-engineering the Clinical Research Enterprise Dynamic Assessment of Patient-Reported Chronic Disease Outcomes initiative, contact Wm. Phil Tonkins, Dr. PH., National Institute of Arthritis and Musculoskeletal and Skin Diseases, tonkinsw2@mail.nih.gov, 301-594-5032.

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