Background: Novel subcutaneous (SC) prophylactic therapies are transforming the treatment landscape of hereditary angioedema (HAE). Although questions are being raised about their cost, little attention has been paid to the cost and quality of life (QoL) impact of using on-demand-only medications. Objective: We assessed the overall economic burden of on-demand-only treatment for HAE and compared patient QoL with patients who received novel SC prophylactic therapies. Methods: US Hereditary Angioedema Association members 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. We modeled 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. QoL was assessed by using the Angioedema Quality of Life questionnaire. Results: A total of 1225 patients (31.4%) responded. Of these, 737 adults with HAE (type I or II) met the inclusion criteria and completed the survey. Per patient/year direct costs associated with modeled on-demand-only treatment totaled $363,795, with additional indirect socioeconomic costs of $52,576 per patient/year. The greatest improvement in QoL was seen in patients who used novel SC prophylactic therapies, with a 59.5% (p < 0.01) improvement in median impairment scores versus on-demand-only treatment. In addition, patients who used novel SC prophylactic therapies reported a 77% reduction in the number of attacks each year when compared with those who used on-demand-only treatment. Conclusion: Our real-world patient data showed the cost and QoL burden of HAE treatment with on-demand-only therapy. Use of novel SC prophylaxis can lead to sizeable reductions in attack frequency and statistically significant and clinically relevant improvements in QoL. These data could be useful to clinicians and patients as they consider therapy options for patients with HAE.
Background: Novel subcutaneous (SC) prophylactic therapies are transforming the treatment landscape of hereditary angioedema (HAE). Although questions are being raised about their cost, little attention has been paid to the cost and quality of life (QoL) impact of using on-demand-only medications. Objective: We assessed the overall economic burden of on-demand-only treatment for HAE and compared patient QoL with patients who received novel SC prophylactic therapies. Methods: US Hereditary Angioedema Association members 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. We modeled 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. QoL was assessed by using the Angioedema Quality of Life questionnaire. Results: A total of 1225 patients (31.4%) responded. Of these, 737 adults with HAE (type I or II) met the inclusion criteria and completed the survey. Per patient/year direct costs associated with modeled on-demand-only treatment totaled $363,795, with additional indirect socioeconomic costs of $52,576 per patient/year. The greatest improvement in QoL was seen in patients who used novel SC prophylactic therapies, with a 59.5% (p < 0.01) improvement in median impairment scores versus on-demand-only treatment. In addition, patients who used novel SC prophylactic therapies reported a 77% reduction in the number of attacks each year when compared with those who used on-demand-only treatment. Conclusion: Our real-world patient data showed the cost and QoL burden of HAE treatment with on-demand-only therapy. Use of novel SC prophylaxis can lead to sizeable reductions in attack frequency and statistically significant and clinically relevant improvements in QoL. These data could be useful to clinicians and patients as they consider therapy options for patients with HAE.
Authors: Timothy Craig; Bruce Zuraw; Hilary Longhurst; Marco Cicardi; Konrad Bork; Clive Grattan; Constance Katelaris; Gordon Sussman; Paul K Keith; William Yang; Jacques Hébert; Jana Hanzlikova; Petra Staubach-Renz; Inmaculada Martinez-Saguer; Markus Magerl; Emel Aygören-Pürsün; Henriette Farkas; Avner Reshef; Shmuel Kivity; Sergio Neri; Ioana Crisan; Teresa Caballero; Maria L Baeza; Maria Dolores Hernandez; Henry Li; William Lumry; Jonathan A Bernstein; Iftikar Hussain; John Anderson; Lawrence B Schwartz; Joshua Jacobs; Michael Manning; Donald Levy; Marc Riedl; Sandra Christiansen; Henrike Feuersenger; Ingo Pragst; Sarah Mycroft; Dipti Pawaskar; Iris Jacobs Journal: J Allergy Clin Immunol Pract Date: 2019-02-15
Authors: David A Wilson; Konrad Bork; Elizabeth P Shea; Anne M Rentz; Marc B Blaustein; William E Pullman Journal: Ann Allergy Asthma Immunol Date: 2010-04 Impact factor: 6.347
Authors: Aleena Banerji; Marc A Riedl; Jonathan A Bernstein; Marco Cicardi; Hilary J Longhurst; Bruce L Zuraw; Paula J Busse; John Anderson; Markus Magerl; Inmaculada Martinez-Saguer; Mark Davis-Lorton; Andrea Zanichelli; H Henry Li; Timothy Craig; Joshua Jacobs; Douglas T Johnston; Ralph Shapiro; William H Yang; William R Lumry; Michael E Manning; Lawrence B Schwartz; Mustafa Shennak; Daniel Soteres; Rafael H Zaragoza-Urdaz; Selina Gierer; Andrew M Smith; Raffi Tachdjian; H James Wedner; Jacques Hebert; Syed M Rehman; Petra Staubach; Jennifer Schranz; Jovanna Baptista; Wolfram Nothaft; Marcus Maurer Journal: JAMA Date: 2018-11-27 Impact factor: 56.272
Authors: K Weller; A Groffik; M Magerl; N Tohme; P Martus; K Krause; M Metz; P Staubach; M Maurer Journal: Allergy Date: 2012-08-23 Impact factor: 13.146
Authors: Marcus Maurer; Markus Magerl; Stephen Betschel; Werner Aberer; Ignacio J Ansotegui; Emel Aygören-Pürsün; Aleena Banerji; Noémi-Anna Bara; Isabelle Boccon-Gibod; Konrad Bork; Laurence Bouillet; Henrik Balle Boysen; Nicholas Brodszki; Paula J Busse; Anette Bygum; Teresa Caballero; Mauro Cancian; Anthony J Castaldo; Danny M Cohn; Dorottya Csuka; Henriette Farkas; Mark Gompels; Richard Gower; Anete S Grumach; Guillermo Guidos-Fogelbach; Michihiro Hide; Hye-Ryun Kang; Allen P Kaplan; Constance H Katelaris; Sorena Kiani-Alikhan; Wei-Te Lei; Richard F Lockey; Hilary Longhurst; William Lumry; Andrew MacGinnitie; Alejandro Malbran; Inmaculada Martinez Saguer; Juan José Matta Campos; Alexander Nast; Dinh Nguyen; Sandra A Nieto-Martinez; Ruby Pawankar; Jonathan Peter; Grzegorz Porebski; Nieves Prior; Avner Reshef; Marc Riedl; Bruce Ritchie; Farrukh Rafique Sheikh; William B Smith; Peter J Spaeth; Marcin Stobiecki; Elias Toubi; Lilian Agnes Varga; Karsten Weller; Andrea Zanichelli; Yuxiang Zhi; Bruce Zuraw; Timothy Craig Journal: World Allergy Organ J Date: 2022-04-07 Impact factor: 5.516