Applications in Research
HealthMeasures can be used in clinical research settings, including clinical trials, observational studies, and comparative effectiveness research.
HealthMeasures in Drug Development
- HealthMeasures can be used as primary or secondary endpoints in clinical and observational studies on the effectiveness of treatment.
- HealthMeasures, particularly PROMIS, can measure symptomatic adverse events, physical function, and disease-related symptoms (e.g., dyspnea) as recommended for cancer clinical trials. Learn more>>
- Researchers and sponsors should review the evidence to support the context of use.
- For publications and webinars on assessment of patient-reported outcomes (PROs) in children and adolescents, content validity in PRO instruments and good practices for outcomes research visit the website of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
FDA Drug Development Tool Qualification
The U.S. Food and Drug Administration (FDA) recognizes the importance of research tools that aid the development and evaluation of medical treatments. These tools include performance measures of function and patient-reported outcome (PRO) instruments that may serve as clinical outcome assessments (COAs) in clinical trials. To expedite the use of a specific PRO measure as an outcome in clinical trials, a person or group may submit the PRO for qualification by a center of the FDA, e.g., the Center for Drug Evaluation and Research (CDER). Qualification is a multi-step process that requires substantial qualitative and quantitative evidence to support the reliability, validity, and interpretability of the PRO for its intended use. Once a PRO has been qualified, FDA reviewers can be confident that it is valid for its specific context of use, e.g., assessing the effect of treatment relative to placebo on fatigue in children with Crohn’s disease aged 8-17 years old. Hence, the PRO may be employed in drug development trials within the context of use without the need for further review of its validity.
(1) Submission of a Letter of Intent that describes the PRO and preliminary supporting evidence
(2) Submission of a Qualification Plan that provides detailed information about qualitative research conducted with the intended patient population and plans for further research
(3) Submission of a Full Qualification Package with data and results supporting the PRO as well as instructions for administration, scoring, handling missing data, and interpreting scores.
At each stage, FDA reviewers provide feedback and may request revisions and additional information. Learn more>>
Qualification of HealthMeasures
Qualification of several PROMIS measures is in progress (e.g., PROMIS Physical Function in sarcopenia and oncology, PROMIS fatigue in Systemic Exertion Intolerance Disease, Rheumatoid Arthritis, and Crohn’s Disease). For the full list of submissions and contacts, visit the FDA’s Clinical Outcome Assessment webpage. Learn more>>
Evidence Supporting the Validity of HealthMeasures
HealthMeasures have substantial qualitative and quantitative evidence for their validity in a range of populations and for different purposes. Read more about validity for
Patient-Centered Outcomes Research Institute (PCORI) acknowledges the importance of the PROMIS initiative with extensive funding opportunities
From PCORI’s first funding announcement through the end of 2019, PCORI has awarded nearly $34 million to studies using PROMIS measures. In addition to a PROMIS-specific funding announcement in 2014 totaling $5 million in support, PCORI continues to recognize the importance of PROMIS by soliciting input on the future involvement of PROMIS’ methodology and measures within its research portfolio.
Results of the studies featuring PROMIS measures are available at www.pcori.org.