Prophylaxis therapies can lead to significant improvements in quality of life

According to HAEi President & CEO Anthony J. Castaldo, “A study of HAE medicines published by a group of economists who receive funding from the insurance industry concluded that new preventive HAE medicines are not cost-effective. The US HAE Association and HAEi joined forces in questioning the methods and conclusions of this analysis and responded by initiating a comprehensive study to assess both on-demand treatment and use of the new preventative medicines in the real-world. Although the focus is on the new preventative medicines, we wanted to make sure to also look at cost and quality of life impact of using on-demand–only medications.”

Almost 750 people with HAE participated in the study and the results point to a high cost and quality of life burden of HAE treatment with on-demand only therapy. The real-world data also reveals that the new preventive therapies can deliver (1) remarkable decreases in attack frequency and (2) statistically significant and clinically relevant improvements in patient quality of life. After an extensive peer review, the study ‘Assessing the cost and quality-of-life impact of on-demand-only medications for adults with hereditary angioedema’ is now published and available in print and free online at the ingenta website.

Co-authors Anthony J. Castaldo, Deborah Corcoran, Henrik Balle Boysen from HAEi, Christian Jervelund from Copenhagen Economics, along with Bruce L. Zuraw and Sandra C. Christiansen from the Department of Medicine at the University of California San Diego, noted that, “Novel subcutaneous prophylactic therapies are transforming the treatment landscape of HAE. Although questions are being raised about their cost, little attention has been paid to the cost and quality of life impact of using on-demand-only medications. Therefore, we decided to assess the overall economic burden of on-demand-only treatment for HAE and compare patient quality of life with patients who received novel subcutaneous prophylactic therapies.”

For the study, members of the US HAEA were invited to complete an anonymous online survey to profile attack frequency, treatment use, and the presence of comorbidities as well as economic and socioeconomic variables. The authors modelled on-demand treatment costs by using net pricing of medications in 2018, indirect patient and caregiver costs, and attack-related direct billed costs for emergency department admissions, physician office visits, and/or hospitalizations. Quality of life was assessed by using the Angioedema Quality of Life questionnaire.

The study shows that per patient/year, direct costs associated with modeled on-demand-only treatment totalled 363,795 USD, with additional indirect socioeconomic costs of 52,576 USD per patient/year. The most significant improvement in quality of life was seen in patients who used novel subcutaneous prophylactic therapies, as there was a 59.5 percent improvement in median impairment scores versus on-demand-only treatment. In addition, patients who used novel subcutaneous prophylactic therapies reported a 77 percent reduction in the number of attacks each year compared with those who used on-demand-only treatment.

The authors concluded that the use of novel subcutaneous prophylaxis can lead to sizeable reductions in attack frequency and statistically significant and clinically relevant improvements in quality of life. In addition, the study noted that these data could be useful to clinicians and patients as they consider therapy options.