OBJECTIVES: The treatment landscape in hemophilia is characterized by multiple clotting factor products that makes it difficult for patients, caregivers, and clinicians to select the optimum treatment regimen. In this context it is valuable to understand the benefit-risk preferences of these stakeholders for various treatment attributes. Such studies are commonly conducted using discrete choice experiments (DCEs). This systematic review of the literature was conducted to identify DCE studies and characterize common treatment-related attributes evaluated in hemophilia.

METHODS: A systematic literature review was conducted using PubMed, ProQuest, and ISPOR scientific presentations database to identify published studies and abstracts reporting DCEs in hemophilia. Search terms included: hemophilia, discrete choice experiments, conjoint analysis, and best worst scaling. Only English language studies were included.

RESULTS: Fourteen published studies were identified. Sample sizes ranged between 30 and 222, and 4-12 attributes were typically evaluated. The attributes were stratified into convenience, treatment efficacy, safety, cost and other medication-related factors. 13 (93%) studies included frequency of administration/administration time as a convenience attribute. The most common efficacy-related attribute assessed was bleeding risk (N =12 [86%]). Risk of viral infection (N = 6 [43%]) and risk of inhibitor development (N = 6 [43%]) were the most common safety-related attributes evaluated. Cost was assessed as an attribute in 9 (64%) studies. Infusion diluent volume (N = 7 [58%]) and dosage form (N = 5 [36%]) were the most frequently assessed other medication-related attributes.

CONCLUSIONS: Existing DCE studies suggest that convenience, efficacy, and safety were the most key treatment attributes. With the introduction of newer treatment options, results from previous DCEs may not hold. Therefore, future studies should assess the benefit-risk preferences of key stakeholders for these newer hemophilia therapies.