Advancing the Science of Outcomes Measurement: the Patient-Reported Outcomes Measurement Information System (PROMIS)

David Cella, PhD
Professor and Chair, Department of Medical Social Sciences
Northwestern University Feinberg School of Medicine
Chicago, Illinois
d-cella@northwestern.edu

What's the problem?

Traditional endpoints in clinical research include tumor response or survival. However, in a growing trend over the past two decades, investigators have moved beyond these traditional endpoints to evaluate the effectiveness of an intervention by its effect on a patient's health-related quality of life (HRQOL). HRQOL includes physical, mental, and social aspects of a person's health. It is universally accepted that patients themselves are the best source for reporting their HRQOL.

HRQOL endpoints in a trial may be an exploratory variable, a secondary endpoint with survival as the primary, or a primary endpoint when the intervention goal is to reduce a symptom such as pain, fatigue, or depression. To capture patient-reported HRQOL, investigators need access to valid questionnaires that precisely measure a patient's HRQOL level and when their HRQOL changes over the course of cancer care.

Precise measurement of HRQOL has historically required many questions to capture unobservable components of HRQOL (depression, for example), but completing long questionnaires creates a burden for patients. Also, because multiple HRQOL questionnaires exist in the cancer research field, investigators have no way of comparing results from one clinical study to another if different measures are used. We need a set of standardized HRQOL measures that are easy to answer for patients and that capture the full extent of the burden of cancer disease and treatment on patients in a way that provides meaningful information on the effectiveness of an intervention.

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How is this research addressing the problem?

In 2004, the NIH, through the Roadmap Initiatives to reengineer the clinical research enterprise, launched the development of the Patient-Reported Outcomes Measurement Information System (PROMIS). Now, PROMIS is a publicly available web-based resource that can be used to measure key HRQOL domains across a range of chronic diseases, including cancer. PROMIS researchers are building and validating common, accessible item banks that will help investigators and clinicians accurately assess and interpret patient-reported outcomes data from clinical trials and apply these findings in clinical practice.

The first phase of PROMIS (2004-2009) involved a network of six primary research sites across the US and a Statistical Center, led by Dr. David Cella. Dr. Cella's group led all activities related to coordinating research across the six PROMIS sites, creating the standards for the PROMIS study design and analyses, conducting the statistical and psychometrical analyses, and developing the PROMIS Assessment System software to administer the PROMIS measures and facilitate clinical research.

During the second PROMIS phase (2009-2013), Dr. Cella and his research group continue to serve as the PROMIS Statistical Center, providing both psychometric and qualitative research methods to support the network. The network has now expanded to 12 primary research sites. The group also will develop and carry out processes to ensure data quality and will provide leadership in developing PROMIS-approved translations of new and existing HRQOL domains. Another area of support to network members will be help with developing and carrying out research protocols; providing consultation on research design, sample size, sampling plans, and power estimates and providing psychometric and statistical support. All of this support is essential to fostering high-quality data collection across the network and ensuring data analysis integrity.

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Significance of the study & results

Measuring patient-reported HRQOL is particularly important in research studies where changes in clinical measurements or imaging results may not translate into recognizable benefits to patients. Assessing patient-reported outcomes also is important in clinical trials where two treatments may have similar effects in controlling or curing disease but different effects on symptoms, function, or other HRQOL issues. Having a validated, dynamic PROMIS system to measure patient-reported outcomes efficiently in study participants with a wide range of chronic diseases and demographic characteristics will greatly enhance the clinical research enterprise and facilitate comparisons between research studies.

Dr. Cella and his group have been pioneers in the development and evaluation of HRQOL instruments and item banks, especially in cancer. They have extensive expertise in designing, implementing, and coordinating clinical and survey research studies and in developing and applying psychometric, statistical, and qualitative methods for analyzing self-report data. Dr. Cella's group also has expertise in developing, evaluating, and translating culturally appropriate patient-reported outcomes instruments.

Dr. Cella himself is an internationally recognized leader in the field of HRQOL research and instrument development and also works closely with the Eastern Cooperative Oncology Group (ECOG) and the Gynecologic Oncology (GOG). Primarily funded by NCI, ECOG and GOG are large networks of clinical cancer researchers that conduct multi-center cancer clinical trials. Thus, Dr. Cella brings unique skills in HRQOL and cancer research to bear on the problem of understanding and measuring the complex and multifaceted physical and psychosocial burden of cancer.

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Recent related publications of interest

Hays R, Bjorner J, Revicki D, Spritzer K, Cella, D. Development of physical and mental health summary scores from the patient reported outcomes measurement information system (PROMIS) global items. Quality of Life Research 2009;18(7):873-880. [View Abstract]

Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, Ader D, Fries J, Bruce B, Rose M. The Patient-Reported Outcomes Measurement Information System (PROMIS): Progress of an NIH Roadmap Cooperative Group During its First Two Years. Medical Care 2007;45(5):S3_S11. [View Abstract]

Garcia S, Cella D, Clauser S, Flynn K, Lad T, Lai J-S, Reeve B, Wilder Smith A, Stone A, Weinfurt K. Standardizing patient-reported outcomes assessment in cancer clinical trials: a patient-reported outcomes measurement information system initiative. Journal of Clinical Oncology 2007;25(32):5106-5112. [View Abstract]

Reeve B, Hays R, Bjorner J, Cook K, Crane P, Teresi J, Thissen D, Revicki D, Weiss D, Hambleton R, Liu H, Gershon R, Reise S, Cella, D. Psychometric evaluation and calibration of health-related quality of life items banks: Plans for the Patient-Reported Outcome Measurement Information System (PROMIS). Medical Care 2007;45(5):S22-S31. [View Abstract]

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