OBJECTIVES

: Preference-based patient-reported outcomes (PROs) are used to indicate patient preference of one treatment over another based on modality, symptom or treatment. There are two types of preference-based PROs: generic and condition-specific. The objective of this research is to identify commonly used preference-based PROs, evaluate their development, and assess their utility in various types of studies.

METHODS

: A literature review using PubMed was conducted to identify studies which detail the development of various preference-based PROs currently being used. These PROs were evaluated against the FDA’s FDA PRO Guidance criteria with regard to: (1) content validity, particularly patient input in concept elicitation, item generation, and cognitive debriefing; and (2) psychometric testing, particularly construct validity, internal consistency reliability, test-retest reliability, and responsiveness. Each criteria was assessed and given a rating. Finally, an attempt was made to make recommendations on which preference-based PROs should be used in different types of studies.

RESULTS

: Ten PROs were identified for inclusion in this analysis, with six having been validated in most dimensions required by the FDA PRO Guidance criteria. Although there has only been one label claim granted by the FDA based on patient preference, there has been a significant recent uptick in the number of preference based PRO studies being conducted. The approved label claim involved a modality preference of subcutaneous injection versus intravenous use of Rituxan. Most preference-based patient-reported outcomes studies currently use generic measures. There has been demonstrated benefit shown to administering condition-specific measures in order to assess dimensions relevant to individual patient populations.

CONCLUSIONS

: There are a number of commonly used PROs that can be appropriately used in preference studies that require little to no additional validation. Consideration should be given to including both generic and condition-specific measures in patient-preference studies.