Director Benefit Risk Sanofi R&D epidemiology & BR Bridgewater, United States
Background: Patients with hemophilia, including children, often face difficulties in managing their disease due to the need for frequent and often invasive treatments. Various studies have been done to evaluate different attributes among varying populations, but there remains a lack of understanding around what aspects of therapeutic interventions are valued most by patient groups.
Objectives: To better understand the perspectives of patients while considering the relative values of attributes of available treatment options and how patients weigh and trade- off among multiple benefits and risks.
Methods: A literature review was performed on hemophilia patient preference studies focusing on treatments for both hemophilia A and B patients. Of the 70 studies that were reviewed, 41 were included in the final evaluation: 7 qualitative, 30 quantitative and 4 studies with mixed methods. The studies were primarily conducted in Western countries and looked almost exclusively at male patients (due to the substantially higher prevalence in male patients) but included predominantly female caregivers to evaluate children’s preferences.
Results: Among the most frequently reported preferences, efficacy in controlling bleeding and a less difficult and time-consuming treatment process are the two first benefits considered. Less frequent, less invasive treatment regimens were preferred. Prophylactic subcutaneous treatments with low frequency of dosing most often preferred to other routes and frequency of administration. Cost was found to be of low or no significance in almost all studies. Risks were often significant in patients’ decisions, especially those risks that impacted quality of life. Some differences arose between children and adult patients particularly regarding safety and the ability to dose. One study looking at switching to extended half-life products found caregivers (representing children’s preferences) were more skeptical than adult patients and only 47.1% of caregivers would switch to a new product compared to 63.8% of adults. No differences between hemophilia A and B were heavily evaluated or found. Additional differences were found in multiple studies correlating with patients’ severity of disease, but due to the differing amount of knowledge and experience those with more severe disease often have, this mediation may explain some of the differences.
Conclusions: Overall, patients want treatment that allowed for normalcy in their lives: the ability to travel, participate in physical activity and not disturb life with frequent injections. Further research needs to be done to continue evaluating patient desires in treatment options to make sure future medications are being tailored towards preferences.
