OBJECTIVES:

Emerging, systematic approaches for capturing patient input, such as preference elicitation, can provide valuable information for the benefit-risk assessment of medical products. This study aims to identify and develop methods to capture, rank and summarize preference scores across different studies in hemophilia patients.

METHODS:

Patient preference data were compiled from studies identified through literature review and publicly available FDA Patient-Focused Drug Development meeting documents on bleeding disorders. Text mining was performed using R to identify major themes across studies. Standardized relative attribute importance (RAI) was estimated and aggregated across relevant studies.

RESULTS :

Twelve preference studies that employed direct qualitative (n=4), and quantitative methods (n=8) met the inclusion criteria. Text mining of qualitative and quantitative studies revealed similar themes (e.g. the importance of inhibitor and viral risk, dosing frequency, and efficacy attributes) as the standardized attribute ranking. We found that 7 out of the 8 quantitative studies employed discrete choice experiments (DCE)/conjoint analysis (CA), which examined a range of 5 to 12 attributes per study. For DCE/CA studies published prior to 2014 (n=4) safety attributes (inhibitor and viral risk) were among the most highly ranked attributes, accounting for ~46% of the total utility measured. DCE/CA studies published after 2014 (n=3) focused on frequency of infusion (convenience) and bleeding risk (efficacy), accounting for ~67% of the total utility. Interestingly, two studies using different preference elicitation approaches, one DCE and one a monadic conjoint approach, both ranked infusion frequency as the most important attribute. These results follow a consistent trend with current scientific innovation and regulatory approval toward extension of half-life and personalized dosing of clotting factors.

CONCLUSIONS :

Although there are few published patient preference studies for hemophilia, the results of this study can be viewed in the larger context of enhancing scientific methods of incorporating patient input in medical product development.