BioCryst Pharmaceuticals, Inc. presents data from a cross-sectional study among patients, caregivers and physicians in the US, highlighting the unmet need in prophylaxis medication administration options through the experiences of HAE patients and caregivers, and differences in perceptions between physicians and HAE patients.
“These data are consistent across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies. New therapies with easier routes of administration may meet a significant unmet need for HAE patients seeking improved quality of life,” says study lead Cristine Radojicic, M.D., assistant professor of medicine at Duke University School of Medicine.
Overall, the burden of treatment reported across all groups surveyed suggests an unmet need still remains in HAE clinical management. These study findings collectively highlight the opportunity to strengthen the shared decision making between patients and physicians with more effective dialogue about the burden of treatment and patients’ individual needs and preferences.
The following is a brief summary of the data from the cross-sectional study conducted via three double-blinded surveys in the US with HAE patients (n=75), caregivers (n=30) and physicians (n=109), respectively:
- Almost nine in 10 patients with HAE report they have learned to tolerate difficult aspects of their treatment and 58 percent report they are tired of their injections. Even though patients are satisfied with their current prophylactic medications, 86 percent are still interested in a less burdensome route of administration.
- Over 50 percent of caregivers agree it was challenging to learn how to administer HAE treatment, specifically gaining comfort with using needles and learning how to self-administer. Seventy-one percent of caregivers agree that patients experience needle fatigue with their HAE prophylactic medications and an even greater proportion of caregivers believe a once-daily pill would provide the patient more freedom (86 percent), independence (85 percent), and reduce caregivers’ burden.
- Most physicians (94 percent) and patients (84 percent) agree there is a need for newer and more novel HAE treatments. In addition, 86 percent of caregivers believe that, while their patient is satisfied with current treatment, the patient would still be interested in one that is easier to administer.
- Over 70 percent of physicians surveyed believe that starting prophylaxis treatment was overwhelming, becoming comfortable with needles was intimidating, and learning how to self-administer was challenging for their patients. The study also shows that physicians tend to underestimate time required for preparation and administration of prophylaxis medications. Importantly, despite recognition of the burden with current treatments, there is discordance between patients and physicians regarding the person initiating conversations about medication challenges, suggesting an opportunity to improve the dialogue to help with an individualized approach to the management of HAE.