First-Ever Study Conducted Demonstrating Economic, Individual, and Societal Burden of Hereditary Angioedema
HONOLULU & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov 10, 2008 - Results of a ground-breaking study quantifying the economic, individual and societal burden of hereditary angioedema (HAE) bring to light the substantial direct and indirect costs of the disease on patients and society. The study reveals a compelling need for new and better therapies to ameliorate the devastating impact of this potentially life-threatening condition which is characterized by acute attacks of swelling that is often accompanied by excruciating pain. Findings from this study, which evaluated feedback collected from 457 HAE patients, were presented yesterday at the 2008 Annual Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), in Seattle, WA. The study was conducted jointly by the United States Hereditary Angioedema Association (HAEA) and Dyax Corp. (NASDAQ:DYAX).
The study surfaced HAE's pervasive and detrimental impact: On average, participants experienced 26.9 attacks per year, with the typical acute attack lasting more than 2.5 days (60 hours). Monetizable costs averaged $45,000 annually per HAE patient and were based on physician visits, missed work days, reduced productivity, hospital stays, tests and procedures, chronic therapy, and patient co-pays. Patients who suffer from severe attacks, amass an estimated $81,000 in annual per patient direct medical costs. Patients who sought hospital-based care for treatment of acute HAE attacks incurred approximately $170,000 annually in these costs alone.
"This is the first time the burden of HAE has been quantified. The formidable costs revealed in this study provide tangible evidence of HAE's significant negative impact and its harmful effect on careers as well as family and social life," stated Anthony J. Castaldo, President of the HAEA. "This study confirms that HAE imposes a significant and extensive burden on patients and society similar to what is seen in other severe, chronic diseases. We look forward to additional research which will increase awareness about HAE so that patients suffering from this disorder can get diagnosed and treated sooner."
The study also demonstrated a substantial psychological and financial burden of HAE that cannot be quantified easily in financial terms. Non-monetizable costs include missed school, missed educational and career opportunities, long-term side effects from anabolic steroids (the current standard of care for HAE treatment) and decreased overall mental and physical health. Nearly half (42.5%) of patients studied had scores indicative of clinical depression, when evaluated with a standardized instrument used to screen for depression. Notably, 69% of respondents reported they had not been able to consider certain jobs as a result of their condition, and 100% reported that HAE limited them from advancing in school.
The productivity impairment suffered by HAE patients is similar to that of patients with more common severe chronic diseases like Crohn's disease and severe asthma. The WPAI(1) assessment showed overall work impairment for HAE patients at 33.6%, falling between the range of WPAI measurements reported for Crohn's disease patients (45.9%) and severe asthma patients (28.0%).
Patients are further burdened by side effects associated with anabolic steroids, the current standard of treatment. The study found these agents impose significant burden in the form of depression, weight gain, changes in mood, sleeplessness, and agitation. Despite these substantial adverse effects, nearly 50% of study respondents reported using these agents.
"These findings emphasize the urgent need for effective treatments for this debilitating and potentially life-threatening disease," commented Henry Blair, Chairman, President and Chief Executive Officer of Dyax, which is developing a recombinant, subcutaneously-administered product, DX-88, for the treatment of HAE. "We hope this study will help to further raise awareness about this patient population and the challenges they face daily."
Findings from this study also will be shared with health insurance companies to help them make informed decisions regarding HAE coverage and reimbursement. "Now more than ever, with new treatments under development, truly understanding the impact of HAE on the individual and society will be especially important," noted Anthony J. Castaldo, adding, "These data support the need for new, effective therapies that will eliminate HAE's ominous burden and allow patients to lead normal and productive lives."
Study Methodology
The Burden of Illness study, conducted by the United BioSource Corporation, was developed in consultation with expert health economists, HAE experts, and HAE patients. The study collected responses from HAE patients via an Institutional Review Board-approved, web-based survey of HAE patients using the following parameters: attack characterization, acute attack treatment, chronic disease management, and financial, physical, and emotional burden of disease management. Disease-specific questions included: treatment patterns and providers; side-effects and burden of androgen therapy; and the quality of life impact of the disease. Three separate standardized instruments were used for comparison with normative and other disease populations: the SF-12 Health Survey, Work Productivity and Activity Impairment (WPAI) tool and the Hamilton Depression Inventory-Short Form (HDI-SF).
Data Presentation at ACAAI
The HAE Burden of Illness study results were presented on Sunday, November 9, 2008 from 12:00-1:00 pm in Exhibit Hall 4C, Washington State Convention Center, at the 2008 Annual Meeting of the American College of Allergy, Asthma & Immunology in Seattle.
Poster 248
Health-Related Quality of Life Burden Associated with Androgen Therapies for Hereditary Angioedema
M.B. Blaustein(1), M.K. Vernon(2), A.M. Rentz(2), D. Wilson(3), A.J. Castaldo(4), 1. Cambridge, MA; 2. Bethesda, MD; 3. Boston, MA; 4. New Bedford, MA.
Poster 249
Humanistic Burden of Hereditary Angioedema
A.J. Castaldo(1), M.K. Vernon(2), W.R. Lumry(3), H.H. Li(4), A.M. Rentz(2), M.B. Blaustein(5), D. Wilson(6), 1. New Bedford, MA; 2. Bethesda, MD; 3. Dallas, TX; 4. Wheaton, MD; 5. Cambridge, MA; 6. Boston, MA.
Poster 250
Economic Costs Associated with Acute and Chronic Management of Hereditary Angioedema
D. Wilson(1), A.M. Rentz(2), B.L. Zuraw(3), K. Bork(4), M.K. Vernon(2), A.J. Castaldo(5), M.B. Blaustein(6), 1. Boston, MA; 2. Bethesda, MD; 3. San Diego, CA; 4. Mainz, Germany; 5. New Bedford, MA; 6. Cambridge, MA.
(1) Work Productivity and Activity Impairment (WPAI)
About the Hereditary Angioedema Association
The United States Hereditary Angioedema Association (HAEA) is a not-for profit organization that represents approximately 2,300 HAE families. The HAEA provides the HAE patient community with a wide variety of services that include education, advocacy, case management, counseling, and referrals for diagnosis. Additional information on HAE and the services provided by the HAEA can be obtained at www.haea.org.
About Dyax
Dyax is focused on advancing novel biotherapeutics for unmet medical needs, with an emphasis on oncology and inflammatory indications. Dyax utilizes its proprietary drug discovery technology to identify antibody, small protein and peptide compounds for clinical development. Dyax's lead product candidate is DX-88 (ecallantide), a recombinant small protein that is currently in clinical trials for its therapeutic potential in two separate indications. On September 23, 2008, Dyax submitted a Biologic License Application with the U.S. Food and Drug Administration for approval of DX-88 for the treatment of hereditary angioedema (HAE). DX-88 has orphan drug designation in the U.S. and E.U., as well as Fast Track designation in the U.S., for the treatment of acute attacks of HAE. Additionally, DX-88 is being evaluated for the prevention of blood loss during on-pump cardiothoracic surgery (CTS) through its partner Cubist Pharmaceuticals. Dyax licensed to Cubist Pharmaceuticals the intravenous formulation of DX-88 for surgical indications in North America and Europe. DX-88 and other compounds in Dyax's pipeline were identified using its patented phage display technology, which rapidly selects compounds that bind with high affinity and specificity to therapeutic targets. Dyax leverages this technology broadly with over 70 revenue generating licenses and collaborations for therapeutic discovery, as well as in non-core areas such as affinity separations, diagnostic imaging, and research reagents.
Dyax is headquartered in Cambridge, Massachusetts. For online information about Dyax Corp., please visit www.dyax.com.
Dyax Disclaimer
This press release contains forward-looking statements, including statements regarding clinical trials, the potential administration, dosing, safety and therapeutic benefit of DX-88 for HAE, and plans for development of DX-88 for CTS. Statements that are not historical facts are based on Dyax's current expectations, beliefs, assumptions, estimates, forecasts and projections about the industry and markets in which Dyax competes. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors which may affect clinical trials, the potential administration, dosing and therapeutic benefit of DX-88 for HAE and plans for development of DX-88 for CTS, include the risks that: DX-88 may not show sufficient therapeutic effect or an acceptable safety profile in clinical trials or could take a significantly longer time to gain regulatory approval than Dyax expects or may never gain approval; others may develop technologies or products superior to DX-88 or that are on the market before DX-88; DX-88 may not gain market acceptance; Dyax is dependent on the expertise, effort, priorities and contractual obligations of third parties in the clinical trials, manufacture, marketing, sales and distribution of DX-88; and other risk factors described or referred to Item 1A, "Risk Factors" in Dyax's most recent Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. Dyax cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this release, and Dyax undertakes no obligations to update or revise these statements, except as may be required by law.
Dyax and the Dyax logo are registered trademarks of Dyax Corp.
Contact
HAEA
Anthony J. Castaldo, 202-297-3409
President, US HAE Association
ajaycas@yahoo.com
or
Dyax Corp.
Ivana Magovcevic-Liebisch, 617-250-5759
Executive Vice President of Administration
and General Counsel
imagovcevic@dyax.com
or
Nicole Jones, 617-250-5744
Director, Investor Relations
njones@dyax.com
Posted: November 2008
