PROMIS® tools as endpoints in clinical trials: what should you know? A review of PROMIS® capabilities and the current regulatory space

Abstract

<p>NIH funded PROMIS measures are increasingly at the forefront of discussions in clinical trial endpoint measurement. In the US alone, 242 clinical trials have included PROMIS measures 2016. Regulatory agencies also recently appear to have more interest in the applications and interpretations of PROMIS tools. This paper provides an overview of the PROMIS tools, how and when they can be used, how they are scored, what modalities exist and what considerations one should make before choosing to use a PROMIS instrument. PROMIS spans across a wide range of areas ranging from mental, physical and social health status, from short form to profile instruments, and to electronic IRT scoring based methods. Psychometric properties measurement testing in a few therapeutic areas has also been evident and continues to develop. The regulatory agency and governmental bodies continue to focus their efforts on having a more profound understanding of the application and use of this patient reported toolbox. PROMIS measures are promising for use within the context of clinical trials, but stakeholders should prudently consider their use, thinking about both the pros and cons. It is likely that for endpoint measurement, PROMIS tools may be used on a case by case basis, but that a consideration of additional disease specific instruments may be recommended.</p>

Reporting and utilization of Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures in orthopedic research and practice: a systematic review

“…While the field of orthopedics is focused on improved functioning and reduced pain, we would encourage a more holistic view of the patient by incorporating more psychological constructs that may affect patient prognosis. This review provides evidence that the prevalence and support for use of PROMIS measures is growing in orthopedics and that PROMIS is being recognized as a PRO measure of choice for clinical trials [ 109 ].…”

Comparing Patient Reported Outcomes among Anti-TNF Experienced Patients with Ulcerative Colitis Initiating Vedolizumab versus Tofacitinib

“…Minimal important differences have been reported to be in the range of 2–6. 25 Secondary outcomes measured at the same time point included PRO2, 26 a patient-reported measure of stool frequency and bleeding, the PROMIS domain of Social Satisfaction, continued use of the index medication (persistence), and need for colectomy.…”

The validity, reliability and minimal clinically important difference of the patient specific functional scale in snake envenomation

“…[34] We correlated these functional outcomes with the PSFS score, and considered a positive moderate association as an indicator of good external validity. All functional assessments have shown good psychometric properties in prior literature [33][34][35][36][37][38][39].…”