OBJECTIVES: Literature attributing the factors to patient and Health Care Professional (HCP) treatment preferences in a specific rare bleeding disorder (sRBD) is lacking. This study (i) explores qualitatively with key medical experts the relevance of existing factors published in the literature attributing to sRBD (ii) identifies any other factors that pertain specifically to the rare bleeding disorder and (iii) aims to inform the development of a patient and HCP preference survey.
METHODS: A literature review preceded qualitative focus groups to understand the full scope of attributes and levels published in a sRBD. Findings of the literature review informed the design of the interview guide. Two focus groups of key medical experts from Japan, Italy, Germany, US, and UK were conducted. A content analysis was performed to identify common and unique concepts pertaining to a sRBD which further refined end selection of the attributes and levels for the patient and HCP preference surveys.
RESULTS: Focus groups resulted in the following eight attributes: Joint health improvement; number of treated bleeding events; concerns about safety of long-term treatment; risk of treatment side effects, frequency of intravenous infusions, hospital resource use, patient adherence, patient management costs considered as factors of relevance to bleeding disorders. Of these 8 factors, three were excluded (joint health improvement; patient adherence, patient management costs) as they were considered less relevant to the sRBD. The final selection of attributes and associated levels were further adjusted for appropriateness after two iterations of expert input.
CONCLUSIONS: Qualitative input from medical experts in a disease area where little is known, has advanced understanding beyond the published literature about specific factors that might impact treatment preferences for a sRBD, both from a patient and HCP perspective. This information will improve the relevancy and appropriateness of content for the design of a future HCP and preference survey.
