Currently Viewing:
Supplements New Horizons in the Diagnosis and Treatment of Hereditary Angioedema: Overcoming Barriers to Management and Improving Patient Outcomes
Severity of Hereditary Angioedema, Prevalence, and Diagnostic Considerations
Jonathan A. Bernstein, MD
Current and Emerging Therapies to Prevent Hereditary Angioedema Attacks
William R. Lumry, MD
Currently Reading
Economic Burden Limiting Proper Healthcare Delivery, Management, and Improvement of Patient Outcomes
William J. Cardarelli, PharmD

Economic Burden Limiting Proper Healthcare Delivery, Management, and Improvement of Patient Outcomes

William J. Cardarelli, PharmD
Although prompt and effective diagnosis and treatment of HAE are paramount to successful patient outcomes, the economics and costs of HAE and its therapy must also be addressed and managed by healthcare systems treating patients with this disorder. Costs of effective therapies must be balanced against the substantial costs of the disease, both in terms of direct medical costs and the impact of HAE on patient functioning and productivity. It is critical to consider PROs in all steps of HAE management and use validated questionnaires to assess patient preferences and status. This truly individualizes HAE management for every patient and potentially provides meaningful and interpretable measures of patient function and an assessment of the effectiveness of treatments and overall management.

Author affiliation: Director of Pharmacy Revenue & Supply, Atrius Health, Watertown, MA.
Funding source: This activity is supported by educational grants from CSL Behring and Shire.
Author disclosure: Dr Cardarelli has no relevant financial relationships with commercial interests to disclose.
Authorship information: Acquisition of data; analysis and interpretation of data; concept and design; critical revision of the manuscript for important intellectual content; drafting of the manuscript.
Address correspondence to:
Dr Cardarelli gratefully acknowledges Elizabeth Paczolt, MD, for her contributions to the development of this article.
  1. Banerji A, Busse P, Christiansen SC, et al. Current state of hereditary angioedema management: a patient survey. Allergy Asthma Proc. 2015;36(3):213-217. doi: 10.2500/aap.2015.36.3824.
  2. Cardarelli W. Managed care implications of hereditary angioedema. Am J Manag Care. 2013;19(suppl 7):S119-S124.
  3. Wilson DA, Bork K, Shea EP, Rentz AM, Blaustein MB, Pullman WE. Economic costs associated with acute attacks and long-term management of hereditary angioedema. Ann Allergy Asthma Immunol. 2010;104(4)314-320. doi: 10.1016/j.anai.2010.01.024
  4. Lumry WH. Hereditary angioedema: the economics of treatment of an orphan disease. Front Med (Lausanne). 2018;5:22. doi: 10.3389/fmed.2018.00022.
  5. Zilberberg MD, Nathanson BH, Jacobsen T, Tillotson G. Descriptive epidemiology of hereditary angioedema emergency department visits in the United States, 2006-2007. Allergy Asthma Proc. 2011;32(5):390-394. doi: 10.2500/aap.2011.32.3478.
  6. Zilberberg MD, Nathanson BH, Jacobsen T, Tillotson G. Descriptive epidemiology of hereditary angioedema hospitalizations in the United States, 2004-2007. Allergy Asthma Proc. 2011;32(3)248-254. doi: 10.2500/aap.2011.32.3452.
  7. Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedema: impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc. 2010;31:407-414. doi: 10.2500/aap.2010.31.3394.
  8. Lumry WR. Pharmacoeconomics of orphan disease treatment with a focus on hereditary angioedema. Immunol Allergy Clin North Am. 2017;37(3):617-62 doi: 10.1016/j.iac.2017.03.004.
  9. Orphan Drug Act. January 4, 1983. FDA website. Accessed May 25, 2018.
  10. Divino V, DeKoven M, Kleinrock M, Wade RL, Kaura S. Orphan drug expenditures in the United States: a historical and prospective analysis, 2007-18. Health Aff (Millwood). 2016;35(9):1588-1594. doi: 1377/hlthaff.2016.0030.
  11. IMS Health. Market prognosis 2014–2018, USA. London: IMS Health; 2014.
  12. Tilles SA, Borish L, Cohen JP. Management of hereditary angioedema in 2012: scientific and pharmacoeconomic perspectives. Ann Allergy Asthma Immunol. 2013;110(2):70-74. doi: 10.1016/j.anai.2011.014.
  13. Study: Drug costs for hereditary angioedema tripled in 2 years. April 15, 2015. Formulary Journal website.
    angioedema-tripled-2-years. Accessed May 25, 2018.
  14. Prime Therapeutics finds drug costs drive nearly entire cost of care for hereditary angioedema [press release]. St Paul, Minnesota: April 19, 2018.  Prime Therapeutics website. Accessed May 25, 2018.
  15. Vande Walle SE, Starner CI, Gleason PP.  Hereditary angioedema: a comprehensive integrated medical and pharmacy claims analysis of utilization and costs among 15 million commercially insured members. Prime Therapeutics website. Accessed May 25, 2018.
  16. Rodriguez-Monguio R, Spargo T, Seoane-Vazquez E. Ethical imperative of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? Orphanet J Rare Dis. 2017;12:1. doi: 10.1186/s13023-016-0551-7.
  17. Basch E. New frontiers in patient-reported outcomes: adverse event reporting, comparative effectiveness, and quality assessment. Annu Rev Med. 2014;65:307-3 doi: 10.1146/annurev-med-010713-141500.
  18. Basch E, Bennett AV. Patient-reported outcomes in clinical trials of rare diseases. J Gen Intern Med. 2014;29(suppl 3):S801-S803. doi: 10.1007/s11606-014-2892-z.
  19. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. December 2009. FDA website. Accessed May 25, 2018.
  20. Rüther A, Elstein D, Wong-Rieger D, Guyatt G. Aspects of patient reported outcomes in rare disease: a discussion paper. Int J Technol Assess Health Care. 2016;32(3):126-130. doi: 10.1017/S0266462316000271.
  21. Wiebe S, Guyatt GH, Weaver B, Matijevic S, Sidwell C. Comparative responsiveness of generic and specific quality-of-life instruments. J Clin Epidemiol. 2003;56(1):52-60.
  22. Morel T, Cano SJ. Measuring what matters to rare disease patients – reflections on the work
    by the IRDiRC taskforce on patient-centered outcome measures. Orphanet J Rare Dis. 2018;12:171. doi: 10.1186/s13023-017-0718-x.
  23. Weller K, Groffik A, Magerl M, et al. Development, validation, and initial results of the angioedema activity score. Allergy. 2013;68(9):1185-1192. doi: 10.1111/all.12209.
  24. Caballero T, Ayala A, Caminoa M, et al. Development of a clinical activity score for hereditary angioedema with C1-inhibitor deficiency. J Angioedema. 2015:28-29.
  25. Bygum A, Busse P, Caballero T, Maurer M. Disease severity, activity, impact, and control and how to assess them in patients with hereditary angioedema. Front Med (Lausanne). 2017;4:212. doi: 10.3389/fmed.2017.00212.
  26. Weller K, Groffik A, Magerl M, et al. Development and construct validation of the angioedema quality of life questionnaire. Allergy. 2012;67(10):1289-1298.
  27. Prior N, Remor E, Perez-Fernandez E, et al. Psychometric field study of hereditary angioedema
    quality of life questionnaire for adults: HAE-QoL. J Allergy Clin Immunol Pract. 2016;4(3):464-473. doi: 10.1016/j.jaip.2015.12.010.
  28. Squeglia V, Barbarino A, Bova M, et al. High attack frequency in patients with angioedema due to C1-inhibitor deficiency is a major determinant in switching to home therapy: a real-life observational study. Orphanet J Rare Dis. 2016;11:133. doi: 10.1186/s13023-016-0518-8.
Copyright AJMC 2006-2020 Clinical Care Targeted Communications Group, LLC. All Rights Reserved.
Welcome the the new and improved, the premier managed market network. Tell us about yourself so that we can serve you better.
Sign Up