Obtaining useful and significant data is paramount to a successful clinical trial; however for those studies with a limited amount of quantitative scientific data, results may be highly dependent on patient-reported outcomes (PROs). The FDA definition of a PRO is “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else”.
How are PROs Used?
A PRO assessment is typically developed as a measure of patient-reported results that may be able to act as predictors and identifiers for additional complications or difficulties as well as potential benefits in the study data.
- Clinical Study Endpoints
- Researchers may define a PRO as an endpoint within a clinical study in which a statistical analysis may be planned to evaluate additional outcomes. In most studies the PRO is best captured using questionnaires in which similarities in patient reporting could be statistically analyzed and act as additional study data. It is customary that primary end points are specifically modeled to evaluate a physiological effect for the indicated treatment; however, frequently PROs are used as supportive concepts in the improvement of symptoms, outcomes, or safety analysis for secondary endpoints.
- Monitoring Adverse Events
- As stated above PROs may best be utilized in safety measures within a clinical trial. PROs can assist in data collection regarding patient satisfaction and quality of life within a clinical trial. The FDA supports direct patient reporting because some effects of the treatments are only known to the patients and may not be outlined as predicted outcomes in the clinical trial. In addition, a clinical trial that provides PRO implementation may impart improved physician and subject relations in which the research participant feels their opinions and health status are important to the Sponsor. Subjects may feel more at ease to discuss burdens in the trial or provide the research team the opportunity to clarify unrealistic treatment goals that a subject may have.
- Contribution to Clinical Care
- The National Institutes of Health (NIH) has created an initiative designed to develop, validate and standardize PROs across common medical conditions. The NIH Patient-Reported Outcomes Measurement Information System (PROMIS) was designed as a five year program that developed self-reporting measures for both adult and pediatric (ages 5-17) assessments. PROMIS has created a database in which users can access a library of common PRO tools and best practices on administering and utilizing the PROs within a study.
- The use of PROs may be difficult to utilize for those studies in which patients are unable to report for themselves.
- Blinding and Randomization: A PRO may be one way to standardize patient expectations in a blinded clinical study and truly ascertain if a placebo effect is impacting study data.
- Additional guidelines are discussed in the FDA Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (December 2009).
The FDA indicates in its Guidance document noted above that the PRO should be able to be measured in absolute terms or is utilized to note a change from a previous measure. Utilizing the NIH PROMIS database and the FDA Guidance, researchers can incorporate PRO data into their clinical studies to truly gain a critical perspective on assessing the patient’s perception on the effects of the experimental treatment.