On behalf of the U.S. Hereditary Angioedema Association (HAEA) as well as the patient and medical professional stakeholder community, thank you for the opportunity to provide comment during the associated materials open input period for your upcoming review of hereditary angioedema (HAE) prophylaxis therapies.

The HAEA was founded by a passionate group of patients and caregivers motivated to (1) alleviate the unmitigated pain, suffering, disability and extraordinarily high mortality rate associated with HAE, and (2) take full advantage of the Orphan Drug Act (the Act) to encourage clinical research into alternatives to the only medicine available to our patients--toxic and dangerous anabolic steroids. We are proud of the progress made in garnering FDA approval for modern HAE medicines, but much more needs to be done to upgrade patient care. Accordingly, we continue working to spark further advancements in drug discovery under the aegis of the Act. 

HAE is a serious, life threatening, and debilitating ultra-rare disease (incidence estimated at 1-50,000) that has a devastating impact on patients and their families. In addition to being painful and potentially fatal, HAE causes crippling ongoing anxiety because patients never know when their next attack may occur.

There is significant variability in patient response to therapies, and achieving a meaningful clinical result often requires trial and error by an expert physician. Therefore, patient quality of life can only be attained with full access to all FDA licensed HAE therapies that our patient and physician community worked so hard to get approved.

For many, access to treatment (particularly prophylactic therapy) is the difference between death, disability, pain, fear, and a productive and fulfilling life. As of yet, none of the prophylactic therapies are approved for pediatric patients; some of which have such frequent and incapacitating HAE attacks that preventive therapy would be the only satisfactory treatment.

Our Medical Advisory Board and the broader specialist community recognize that HAE is an ultra-rare disease that is often misdiagnosed, and when not appropriately treated leads to frequent high cost emergency room visits and/or hospitalizations, not to mention a severely diminished quality of life. As a result, they have put forth significant time and effort into developing peer reviewed expert treatment recommendations and other disease-related information.

Since the enactment of guaranteed issue requirements, the HAE patient community has been working tirelessly to fend off discriminatory coverage and limited access practices by non-regulatory entities. These efforts often seek to steer patients into health status or need-based government-funded healthcare programs that they would not otherwise qualify for when their illness is properly managed. Unfortunately, actions that jeopardize patient health such as limiting coverage options and restricting access to medication have become common tactics. In this regard, we do not understand why ICER would expend resources reviewing cost-effectiveness for therapies used by such a small subset of rare disease patients. We also find surprising that ICER is including two products that have yet to receive FDA approval, as well as another new therapy that has yet to undergo utilization/post-market study.

The HAEA hopes that ICER will approach its review with an appreciation of the expert clinician’s perspective on properly managing the disease as outlined in the Medical Advisory Board HAE management recommendations. HAE patients want to live an independent life and not be forced to rely on tax payer-funded healthcare programs. Moreover, we expect that your review will protect the important patient-physician relationship and give proper deference to expert clinical judgment, continued access to all approved HAE medicines, and the personalized treatment necessary in this ultra-rare disease. Finally, we urge you to assign significant value to the extraordinary quality of life improvements and overall prevention of human suffering brought by modern HAE therapies.

Sincerely,

The Hereditary Angioedema Association (HAEA)