RESUMO
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.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/prevenção & controle , Anti-Inflamatórios não Esteroides/administração & dosagem , Quimioprevenção , Proteína Inibidora do Complemento C1/administração & dosagem , Custos de Medicamentos/estatística & dados numéricos , Qualidade de Vida , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Angioedemas Hereditários/economia , Anti-Inflamatórios não Esteroides/economia , Anti-Inflamatórios não Esteroides/uso terapêutico , Anticorpos Monoclonais Humanizados/economia , Anticorpos Monoclonais Humanizados/uso terapêutico , Bradicinina/análogos & derivados , Bradicinina/economia , Bradicinina/uso terapêutico , Antagonistas de Receptor B2 da Bradicinina/economia , Antagonistas de Receptor B2 da Bradicinina/uso terapêutico , Quimioprevenção/economia , Quimioprevenção/métodos , Estudos de Coortes , Proteína Inibidora do Complemento C1/economia , Proteína Inibidora do Complemento C1/uso terapêutico , Progressão da Doença , Esquema de Medicação , Feminino , Inquéritos Epidemiológicos , Humanos , Injeções Subcutâneas , Masculino , Pessoa de Meia-Idade , Peptídeos/economia , Peptídeos/uso terapêutico , Proteínas Recombinantes/economia , Proteínas Recombinantes/uso terapêutico , Autorrelato , Resultado do Tratamento , Estados Unidos , Adulto JovemRESUMO
BACKGROUND AND OBJECTIVES: Patients with the rare disease hereditary angioedema (HAE) suffer from recurrent acute attacks of edema. There is no curative therapy, but the frequency of attacks and quality of life of severely affected patients can be improved by prophylactic therapy. The monoclonal antibody lanadelumab has been approved for routine prophylaxis in patients with HAE since November 2018. PATIENTS AND METHODS: In this prospective assessment, a long-term therapy with lanadelumab was initiated in 12 adult patients with HAE. We analyzed their course of disease 6 months after the start of long-term prophylactic therapy using a validated quality-of-life questionnaire and evaluated the frequency and severity of attacks as well as side effects. Furthermore, the therapy with lanadelumab was compared with the previous medication. RESULTS: To date, our study is the first prospective quality of life analysis in HAE patients under treatment with lanadelumab in real life conditions. Mean attack frequencies were reduced from 6.4 to 0.3 attacks per month and patient in our cohort (P<0.0001). No severe attacks occurred under lanadelumab prophylaxis. In all patients, quality of life increased significantly. CONCLUSIONS: Lanadelumab is an effective but expensive long-term prophylaxis for HAE patients. A favorable side-effect profile has been shown. J Drugs Dermatol. 2020;19(10):978-983. doi:10.36849/JDD.2020.5269.
Assuntos
Angioedemas Hereditários/prevenção & controle , Anticorpos Monoclonais Humanizados/administração & dosagem , Qualidade de Vida , Prevenção Secundária/métodos , Exacerbação dos Sintomas , Adolescente , Adulto , Idoso , Angioedemas Hereditários/economia , Angioedemas Hereditários/genética , Angioedemas Hereditários/psicologia , Anticorpos Monoclonais Humanizados/economia , Proteína Inibidora do Complemento C1/genética , Análise Custo-Benefício , Custos de Medicamentos/estatística & dados numéricos , Feminino , Humanos , Calicreínas/antagonistas & inibidores , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Doenças Raras , Recidiva , Adulto JovemRESUMO
Assuntos
Angioedemas Hereditários/epidemiologia , Ansiedade/epidemiologia , Depressão/epidemiologia , Absenteísmo , Angioedemas Hereditários/economia , Animais , Efeitos Psicossociais da Doença , Progressão da Doença , Humanos , Prevalência , Qualidade de VidaRESUMO
BACKGROUND: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease. Low levels of functional C1-INH can lead to recurrent attacks of severe swelling occurring in areas such as the limbs, face, gastrointestinal tract, and throat. These attacks are both painful and disabling and, if not treated promptly and effectively, can result in hospitalization or death. Agents targeting the specific physiologic pathway of HAE attacks can offer improved outcomes with limited side effects compared with nonspecific therapies. However, these treatments display varying efficacy in HAE patients, including the need to redose or seek additional care if the treatment does not resolve symptoms effectively. OBJECTIVE: To analyze the expected cost and utility per HAE attack when treated on-demand with HAE therapies indicated for the treatment of acute attacks. METHODS: A decision-tree model was developed using TreeAge Pro software. Four on-demand HAE treatments were included: ecallantide, icatibant, plasma-derived (pd)C1-INH, and recombinant human (rh)C1-INH. The model uses probabilities for redosing, self-administration versus health care provider administration, and risk of hospitalization. Costs within the model consisted of the HAE treatments and associated health care system expenses. Nonattack baseline utility and attack utility were implemented for effectiveness calculations; time to attack resolution was considered as well. Effectiveness and overall costs per attack were calculated and used to estimate cost per quality-adjusted life-year (QALY). Variability and ranges in cost-effectiveness were determined using probabilistic sensitivity analyses. Finally, a budget impact model for a health plan with 1 million covered lives was also developed. RESULTS: The base case model outputs show costs and calculated effectiveness per attack at $12,905 and 0.806 for rhC1-INH, $14,806 and 0.765 for icatibant, $14,668 and 0.769 for pdC1-INH, and $21,068 and 0.792 for ecallantide, respectively. Cost per QALY was calculated using 26.9 attacks per person-year, leading to results of $420,941 for rhC1-INH, $488,349 for icatibant, $483,892 for pdC1-INH, and $689,773 for ecallantide. Sensitivity analyses demonstrate that redose rates (from 3% for rhC1-INH to 44% for icatibant) are a primary driver of variability in cost-effectiveness. Annual health plan costs from the budget impact model are calculated as $6.94 million for rhC1-INH, $7.97 million for icatibant, $7.90 million for pdC1-INH, and $11.33 million for ecallantide. CONCLUSIONS: Accounting for patient well-being and additional cost components of HAE attacks generates a better estimation of cost-effectiveness than drug cost alone. Results from this model indicate that rhC1-INH is the dominant treatment option with lower expected costs and higher calculated effectiveness than comparators. Further analyses reinforce the idea that low redose rates contribute to improved cost-effectiveness. DISCLOSURES: Funding support was contributed by Pharming Healthcare. Relan and Adams are employed by Pharming Healthcare. Tyson and Magar are employed by AHRM, which received fees to perform the analysis and develop the manuscript. Bernstein reports grants, personal fees, and nonfinancial support from Shire, CSL Behring, and Pharming Healthcare; grants and personal fees from Biocryst; and nonfinancial support from HAEA, unrelated to this study.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Bradicinina/análogos & derivados , Proteína Inibidora do Complemento C1/administração & dosagem , Peptídeos/administração & dosagem , Angioedemas Hereditários/economia , Anti-Inflamatórios não Esteroides/administração & dosagem , Anti-Inflamatórios não Esteroides/economia , Bradicinina/administração & dosagem , Bradicinina/economia , Proteína Inibidora do Complemento C1/economia , Análise Custo-Benefício , Árvores de Decisões , Humanos , Modelos Econômicos , Peptídeos/economia , Anos de Vida Ajustados por Qualidade de Vida , Proteínas Recombinantes , Resultado do TratamentoRESUMO
DISCLOSURES: Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, AHIP Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Cambia Health Solutions, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, Johnson & Johnson, Merck, National Pharmaceutical Council, Prime Therapeutics, Sanofi, Spark Therapeutics, Health Care Service Corporation, Editas, Alnylam, Regeneron, Mallinkrodt, Biogen, HealthPartners, and Novartis. Agboola, Dreitlein, and Pearson are ICER employees. Lin reports personal fees from ICER, during the conduct of this study, and grants from the National Institutes of Health and the California Department of Insurance, outside the submitted work. Carlson and Lubinga report grants from ICER, during the conduct of this study.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Anticorpos Monoclonais Humanizados/uso terapêutico , Proteína Inibidora do Complemento C1/administração & dosagem , Angioedemas Hereditários/economia , Angioedemas Hereditários/fisiopatologia , Anticorpos Monoclonais Humanizados/economia , Proteína Inibidora do Complemento C1/economia , Proteína Inibidora do Complemento C1/genética , Humanos , Resultado do TratamentoRESUMO
OBJECTIVES: To explore treatment behaviours in a cohort of Italian patients with hereditary angioedema due to complement C1-inhibitor deficiency (C1-INH-HAE), and to estimate how effects and costs of treating attacks in routine practice differed across available on-demand treatments. DESIGN: Cost analyses and survival analyses using attack-level data collected prospectively for 1 year. SETTING: National reference centre for C1-INH-HAE. PARTICIPANTS: 167 patients with proved diagnosis of C1-INH-HAE, who reported data on angioedema attacks, including severity, localisation and duration, treatment received, and use of other healthcare services. INTERVENTIONS: Attacks were treated with either icatibant, plasma-derived C1-INH (pdC1-INH) or just supportive care. MAIN OUTCOME MEASURES: Treatment efficacy in reducing attack duration and the direct costs of acute attacks. RESULTS: Overall, 133 of 167 patients (79.6%) reported 1508 attacks during the study period, with mean incidence of 11 attacks per patient per year. Only 78.9% of attacks were treated in contrast to current guidelines. Both icatibant and pdC1-INH significantly reduced attack duration compared with no treatment (median times from onset 7, 10 and 47 hours, respectively), but remission rates with icatibant were 31% faster compared with pdC1-INH (HR 1.31, 95% CI 1.14 to 1.51). However, observed treatment behaviours suggest patterns of suboptimal dosing for pdC1-INH. The average cost per attack was 1183 (SD 789) resulting in 1.58 million healthcare costs during the observation period (11 912 per patient per year). Icatibant was 54% more expensive than pdC1-INH, whereas age, sex and prophylactic treatment were not associated to higher or lower costs. CONCLUSIONS: Both icatibant and pdC1-INH significantly reduced attack duration compared with no treatment, however, icatibant was more effective but also more expensive. Treatment behaviours and suboptimal dosing of pdC1-INH may account for the differences, but further research is needed to define their role.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Bradicinina/análogos & derivados , Proteína Inibidora do Complemento C1/uso terapêutico , Fatores Imunológicos/uso terapêutico , Adulto , Angioedemas Hereditários/economia , Bradicinina/economia , Bradicinina/uso terapêutico , Proteína Inibidora do Complemento C1/economia , Proteína Inibidora do Complemento C1/genética , Proteína Inibidora do Complemento C1/isolamento & purificação , Análise Custo-Benefício , Feminino , Humanos , Fatores Imunológicos/economia , Fatores Imunológicos/isolamento & purificação , Itália , Masculino , Pessoa de Meia-Idade , Plasma , Estudos Prospectivos , Resultado do TratamentoRESUMO
Although several new therapies to both treat acute hereditary angioedema (HAE) attacks and provide prophylaxis for patients with recurrent episodes have been introduced in the past several years, HAE remains a disease with a substantial physical, psychosocial, and economic burden to both affected patients and society in general. Not all treatments work for all patients, and research is ongoing to improve HAE diagnosis and selection of therapy for individual patients to optimize outcomes. But the disease has a significant economic impact with high direct and indirect costs, and high charges related to the new therapies developed for patients to reduce symptoms and attack recurrence. Overall, effective management of HAE is often complicated by clinical and economic barriers to optimal patient outcomes that must be overcome to provide the best care possible and prevent future attacks and complications associated with this rare disease.
Assuntos
Angioedemas Hereditários/economia , Efeitos Psicossociais da Doença , Custos de Cuidados de Saúde , Programas de Assistência Gerenciada/economia , Melhoria de Qualidade/economia , Doença Aguda , Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/prevenção & controle , Custos de Medicamentos , Humanos , Medidas de Resultados Relatados pelo Paciente , Resultado do TratamentoRESUMO
BACKGROUND: Despite the availability of guidelines for the specific treatment of hereditary angioedema (HAE) attacks, HAE morbidity and mortality rates remain substantial. HAE attacks are a major medical issue requiring specific treatment as well as a considerable socio-economic burden. We report a protocol designed to test whether a dedicated call centre is more effective than usual practice in the management of patients experiencing an HAE attack. METHODS/DESIGN: This prospective, cluster-randomised, single-blind, parallel-group, multicentre trial evaluates the morbidity and consequent socio-economic costs of the management of patients experiencing an HAE attack by a dedicated call centre as compared to usual practice. The trial aims to recruit 200 patients. Patients in the intervention arm are provided with an SOS-HAE card with the call centre's freephone number that they can access in the case of an attack. The centre's mission is to provide recommended expert advice on early home treatment. The centre can route the call to a local emergency medical service with competency in HAE management or even arrange for the drugs needed for the specific treatment of an HAE attack to be sent to the emergency department of the local hospital. The primary outcome measure is the number of hospital admissions for an HAE attack. Each patient will be followed up every 2 months for 2 years. The study has been approved by the ethics committee (Comité de Protection des Personnes d'Ile de France 10; registration number: 2012-A00044-39; date of approval: 19 January 2012). DISCUSSION: The SOS-HAE protocol has been designed to address the handling of attacks experienced by patients with HAE in the home. The proposed trial will determine whether the setting up of a dedicated call centre is more effective than usual practice in terms of reducing morbidity as given by the numbers of hospital admissions. The results are also anticipated to have important implications in terms of socio-economic costs for both healthcare services and patients. TRIAL REGISTRATION: ClinicalTrials.gov NCT01679912 .
Assuntos
Angioedemas Hereditários/terapia , Call Centers , Prestação Integrada de Cuidados de Saúde , Acessibilidade aos Serviços de Saúde , Angioedemas Hereditários/diagnóstico , Angioedemas Hereditários/economia , Call Centers/economia , Efeitos Psicossociais da Doença , Prestação Integrada de Cuidados de Saúde/economia , Serviços Médicos de Emergência , França , Custos de Cuidados de Saúde , Gastos em Saúde , Acessibilidade aos Serviços de Saúde/economia , Serviços de Assistência Domiciliar , Humanos , Admissão do Paciente , Estudos Prospectivos , Qualidade de Vida , Projetos de Pesquisa , Método Simples-Cego , Inquéritos e Questionários , Fatores de Tempo , Resultado do TratamentoRESUMO
BACKGROUND: Hereditary angioedema (HAE) due to C1 inhibitor deficiency is a rare but serious and potentially life-threatening disease marked by spontaneous, recurrent attacks of swelling. The study objective was to characterize direct and indirect resource utilization associated with HAE from the patient perspective in Europe. METHODS: The study was conducted in Spain, Germany, and Denmark to assess the real-world experience of HAE via a cross-sectional survey of HAE patients, including direct and indirect resource utilization during and between attacks for patients and their caregivers over the past 6 months. A regression model examined predictors of medical resource utilization. RESULTS: Overall, 164 patients had an attack in the past 6 months and were included in the analysis. The most significant predictor of medical resource utilization was the severity of the last attack (OR 2.6; p < 0.001). Among patients who sought medical care during the last attack (23%), more than half utilized the emergency department. The last attack prevented patients from their normal activities an average of 4-12 hours. Patient and caregiver absenteeism increased with attack severity and frequency. Among patients who were working or in school (n = 120), 72 provided work/school absenteeism data, resulting in an estimated 20 days missing from work/school on average per year; 51% (n = 84) indicated that HAE has hindered their career/educational advancement. CONCLUSION: HAE poses a considerable burden on patients and their families in terms of direct medical costs and indirect costs related to lost productivity. This burden is substantial at the time of attacks and in between attacks.
Assuntos
Angioedemas Hereditários/economia , Efeitos Psicossociais da Doença , Fatores Socioeconômicos , Europa (Continente) , HumanosRESUMO
Hereditary angioedema (HAE) and acquired angioedema (AAE) are rare life-threatening conditions caused by deficiency of C1 inhibitor (C1INH). Both are characterized by recurrent unpredictable episodes of mucosal swelling involving three main areas: the skin, gastrointestinal tract and larynx. Swelling in the gastrointestinal tract results in abdominal pain and vomiting, while swelling in the larynx may be fatal. There are limited UK data on these patients to help improve practice and understand more clearly the burden of disease. An audit tool was designed, informed by the published UK consensus document and clinical practice, and sent to clinicians involved in the care of HAE patients through a number of national organizations. Data sets on 376 patients were received from 14 centres in England, Scotland and Wales. There were 55 deaths from HAE in 33 families, emphasizing the potentially lethal nature of this disease. These data also show that there is a significant diagnostic delay of on average 10 years for type I HAE, 18 years for type II HAE and 5 years for AAE. For HAE the average annual frequency of swellings per patient affecting the periphery was eight, abdomen 5 and airway 0·5, with wide individual variation. The impact on quality of life was rated as moderate or severe by 37% of adult patients. The audit has helped to define the burden of disease in the UK and has aided planning new treatments for UK patients.
Assuntos
Angioedemas Hereditários , Efeitos Psicossociais da Doença , Auditoria Médica , Qualidade de Vida , Adulto , Angioedemas Hereditários/diagnóstico , Angioedemas Hereditários/economia , Angioedemas Hereditários/mortalidade , Angioedemas Hereditários/terapia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Fatores de Tempo , Reino Unido/epidemiologiaRESUMO
OBJECTIVE: Hereditary angioedema (HAE) is a rare genetic disease characterized by long-term recurrent attacks of subcutaneous or submucosal edema in different parts of the body. A comprehensive review of the literature on burden of illness for patients with HAE is presented. DATA SOURCES: A Boolean search was performed using MEDLINE and EMBASE databases and the Internet. STUDY SELECTION: Articles discussing aspects of the burden of illness in HAE were selected. Topics focused on the course of the disease, nature of attacks, treatment, quality of life, and costs. RESULTS: Hereditary angioedema is associated with a significant and multifaceted disease burden. Diagnosis is often delayed for years, with patients receiving ineffective treatment and unnecessary medical procedures before diagnosis. HAE attacks are painful, unpredictable, and debilitating and often require emergency medical attention. Attacks can affect a patient's daily activities, including work or schooling. Depression and anxiety are prevalent in patients with HAE. Recent advances in treatment provide patients with effective and well-tolerated prophylactic and on-demand therapeutic options. However, end points specific to HAE that better measure the impact of treatment on disease burden are lacking. Furthermore, there is a notable paucity of literature directed toward physicians who are instrumental in diagnosing and treating patients with HAE (eg, emergency department). CONCLUSION: More publications are broadening the understanding of HAE. However, important gaps remain. Effective management of HAE requires a more comprehensive understanding of the disease burden so that disease management can be individualized to meet specific patient needs.
Assuntos
Angioedemas Hereditários , Proteínas Inativadoras do Complemento 1/genética , Efeitos Psicossociais da Doença , Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/economia , Angioedemas Hereditários/patologia , Proteínas Inativadoras do Complemento 1/deficiência , Proteínas Inativadoras do Complemento 1/metabolismo , Proteína Inibidora do Complemento C1 , Fator XII/genética , Humanos , Qualidade de VidaRESUMO
Hereditary angioedema (HAE) is a genetic syndrome caused by a functional deficit in complement C1 inhibitor that results in recurrent episodes of nonpruritic swelling of the hands, feet, arms, legs, trunk, face, genitalia, bowels, and larynx beginning in childhood or adolescence and continuing throughout the patient's lifetime. Delays in the diagnosis of HAE average several years owing to the rarity of the disorder and the mimicking of clinical symptoms of those of a variety of other diseases. The lives of people with HAE have been transformed in recent years by the approval of several effective new therapies for the treatment and prevention of angioedema attacks. Nonetheless, evidence from surveys shows that many patients still report suboptimal disease control accompanied by significant morbidity, psychological stress, and diminished productivity and quality of life. Further, new HAE agents are costly-the average wholesale price of a single dose of ecallantide (Kalbitor) is $9540-which will further strain an already stressed healthcare system. This article reviews the economic burdens, both direct and indirect, of HAE, and how changes in treatment practices, such as the wider adoption of home-based therapy, may improve patient outcomes and reduce costs.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Programas de Assistência Gerenciada , Adolescente , Adulto , Idoso , Angioedemas Hereditários/diagnóstico , Angioedemas Hereditários/economia , Criança , Pré-Escolar , Efeitos Psicossociais da Doença , Diagnóstico Tardio , Erros de Diagnóstico , Feminino , Pesquisas sobre Atenção à Saúde , Gastos em Saúde , Serviços de Assistência Domiciliar , Humanos , Lactente , Masculino , Pessoa de Meia-Idade , Qualidade de Vida , Adulto JovemRESUMO
Angioedema (AE) is characterized by swelling of the mucosa or submucosa and/or the subcutaneous tissue of the skin. Hereditary AE (HAE) is an autosomal dominant genetic disorder caused by dysfunctional C1 esterase inhibitor, which affects an estimated 1:10,000 to 1:50,000 patients. A survey study that was conducted between 2007 and 2008 to assess the burden of illness of HAE patients revealed they had significant decreases in physical and mental health, impaired work productivity, and a significantly greater amount of depression compared with patients afflicted with other chronic diseases. The cost of treating HAE is quite significant ranging from $14,000 a year for mild disease to $96,000 for severe disease. Given the changing paradigm for evaluation and treatment with the advent of increased recognition and availability of novel prophylactic and on-demand therapeutic agents, updated survey studies of this nature to assess burden of illness, quality of life, and associated treatment costs should be conducted to provide a better understanding of the optimal management of HAE patients in the United States.
Assuntos
Angioedemas Hereditários/economia , Angioedemas Hereditários/epidemiologia , Efeitos Psicossociais da Doença , Angioedemas Hereditários/genética , Proteína Inibidora do Complemento C1/genética , Progressão da Doença , Feminino , Custos de Cuidados de Saúde , Humanos , Trabalho de Parto , Gravidez , Complicações Cardiovasculares na Gravidez/fisiopatologia , Qualidade de Vida , Estados UnidosAssuntos
Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/economia , Indústria Farmacêutica/economia , Doença Aguda , Angioedemas Hereditários/fisiopatologia , Proteínas Inativadoras do Complemento 1/uso terapêutico , Proteína Inibidora do Complemento C1 , Custos de Medicamentos , Humanos , Doenças Raras/tratamento farmacológico , Doenças Raras/economia , Fatores de TempoRESUMO
Hereditary angioedema (HAE) is a potentially life-threatening autosomal dominant disease characterized by recurrent episodes of oedema, commonly occurring in the skin, abdomen, and upper respiratory tract. After many years during which limited treatment options were available, a range of newer therapies with proven efficacy have been approved in Europe by the European Commission for the treatment of HAE attacks. However, due to differing legislation and financial restrictions, these treatment options are not available in all countries. Home therapy and self-administration of treatment are recommended in order to minimize the burden of disease upon the patient, with the ideal treatment option being effective, well-tolerated, and easy to prepare and administer. Recently, the Hereditary Angioedema International Working Group (HAWK) consensus recommended early, on-demand treatment for HAE. This article reviews the current treatment options available, and considers the need for treatment guidelines to recommend the appropriate therapy.
Assuntos
Obstrução das Vias Respiratórias/tratamento farmacológico , Angioedemas Hereditários/tratamento farmacológico , Anti-Inflamatórios não Esteroides/uso terapêutico , Proteína Inibidora do Complemento C1/uso terapêutico , Seguro de Serviços Farmacêuticos/economia , Obstrução das Vias Respiratórias/economia , Obstrução das Vias Respiratórias/etiologia , Angioedemas Hereditários/complicações , Angioedemas Hereditários/economia , Anti-Inflamatórios não Esteroides/administração & dosagem , Anti-Inflamatórios não Esteroides/economia , Bradicinina/administração & dosagem , Bradicinina/análogos & derivados , Bradicinina/economia , Bradicinina/uso terapêutico , Proteína Inibidora do Complemento C1/administração & dosagem , Proteína Inibidora do Complemento C1/economia , Custos e Análise de Custo , Europa (Continente) , Conhecimentos, Atitudes e Prática em Saúde , Hospitalização/economia , Humanos , Cobertura do Seguro/economia , Seguro de Serviços Farmacêuticos/normas , Legislação de Medicamentos , Peptídeos/administração & dosagem , Peptídeos/economia , Peptídeos/uso terapêutico , Guias de Prática Clínica como Assunto , Padrões de Prática Médica/tendências , Autoadministração/economia , Autoadministração/normasRESUMO
PURPOSE: Because little is known about the burden of illness associated with hereditary angioedema (HAE), this article reviews the challenges in identifying and managing this rare disease and its humanistic and economic burdens. DESIGN: We identified studies examining the burdens associated with HAE. Conducting larger studies using claims analyses for HAE is particularly challenging, owing to the rarity of the disease in health plans and to diagnostic/coding challenges. Because the data have been limited, larger surveys of patients have been conducted. They are explored here. METHODOLOGY: We searched for studies addressing attack characterization, acute treatment, chronic disease management, adverse events, psychosocial burden, effect on work, and patient costs. PRINCIPAL FINDINGS: HAE may result in physical and/or psychological disability because of the lack of effective treatments and the unpredictability of symptom severity. The reported average annual total costs per patient are approximately $42,000, ranging from approximately $14,000 for mild cases to approximately $96,000 for severe disease. It is not known how much of this is paid by the patients, although it can be assumed that it is substantial for some. CONCLUSION: The appropriate use of disease-specific treatments for HAE may improve patients' quality of life and reduce HAE-associated morbidity and mortality while also reducing costs associated with hospitalizations and other interventions. Future cost-effectiveness studies are needed to examine these issues. Disease-specific agents are expected to significantly change the HAE treatment paradigm in the United States and dramatically improve the efficacy of medical care for these patients.
Assuntos
Angioedemas Hereditários/fisiopatologia , Efeitos Psicossociais da Doença , Angioedemas Hereditários/diagnóstico , Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/economia , Angioedemas Hereditários/psicologia , Gerenciamento Clínico , Inquéritos Epidemiológicos , HumanosRESUMO
Hereditary angioedema (HAE) is a rare inherited disorder of complement factor C1 inhibitor. There are â¼6000 HAE cases in the United States, nearly one-half of whom suffer a monthly exacerbation. Little is known about hospital use patterns by patients with HAE attacks in the United States. This study was designed to examine burden, epidemiology, and outcomes of hospitalizations among HAE patients. We evaluated epidemiology, resource use, and discharge destinations of HAE (ICD-9-CM code 277.6) hospitalizations within the NIS, part of Agency's for Healthcare Research and Quality Healthcare Costs and Utilization Project in 2004 through 2007. There were 10,125 hospitalizations with HAE, of which 3216 (31.8%) had HAE as the principal diagnosis (HAE-PD). Two-thirds of all HAE hospitalizations were among women, and 60% were white. Hypertension was the most common comorbidity (26.9%, all HAE, and 28.0%, HAE-PD). Mortality was 1.4% in HAE and 0.3% in the HAE-PD group. Mean hospital length of stay (3.7, 95% CI 3.0-4.4 days vs. 5.0, 95% CI 4.6-5.4 days) and costs ($4,760, 95% CI $3,612-$5,907 vs. $8,383, 95% CI $7,432-$9,334) were lower in HAE-PD than in the HAE cohort. Although >80% in each group were discharged home routinely, 15.9% of HAE and 4.9% of HAE-PD required either home health care or a transfer to another short-term hospital or a skilled nursing facility. HAE hospitalization volume is substantial. Because diagnostic uncertainty is likely, HAE and its related resource use may be underestimated. HAE prevention strategies need to be examined in the context of these outcomes.
Assuntos
Angioedemas Hereditários/epidemiologia , Hospitalização/estatística & dados numéricos , Hipertensão/epidemiologia , População Branca , Adolescente , Adulto , Angioedemas Hereditários/economia , Angioedemas Hereditários/mortalidade , Comorbidade , Feminino , Custos de Cuidados de Saúde/estatística & dados numéricos , Visitadores Domiciliares , Humanos , Tempo de Internação/estatística & dados numéricos , Masculino , Pessoa de Meia-Idade , Transferência de Pacientes , Análise de Sobrevida , Resultado do Tratamento , Estados UnidosRESUMO
BACKGROUND: Hereditary angioedema (HAE) is a potentially life-threatening condition that affects approximately 1 in 50,000 persons. There are no known surveys of US physicians' knowledge and experience regarding the epidemiology, diagnosis, and management of HAE. OBJECTIVE: This survey of physicians treating patients with HAE assessed physician characteristics, diagnostic and treatment practices, factors that influence physician and patient treatment choices, and physician awareness of new therapies. METHODS: From October 2009 to February 2010, physicians (N = 172) voluntarily completed an online survey developed by physician-investigators. Data were analyzed with descriptive statistics. RESULTS: Most physicians (73.8%) managed 5 patients or fewer in diverse practice settings. Laboratory testing was considered most important in establishing a diagnosis. Fresh frozen plasma and C1 esterase inhibitors were the most frequently administered treatments for acute events; nearly 50% of respondents prescribed C1 esterase inhibitor for acute attacks. More than 80% of respondents prescribed androgens for long-term prophylaxis. Approximately half of respondents were aware of, and likely to use, new therapies for HAE. Other than efficacy, adverse effects were the most important factor that influenced physicians' treatment recommendations, whereas physicians perceived that patients were most influenced by adverse effects and cost. CONCLUSIONS: Wide variability exists in the treatment of patients with HAE. Many patients experience acute attacks that require emergency care or hospitalization. Androgens and fresh frozen plasma are frequently used despite recent availability of effective condition-specific agents, and many physicians are only somewhat aware of medications newly approved by the US Food and Drug Administration. Because the survey was completed shortly after approval of additional HAE therapies by the US Food and Drug Administration, these data will be useful for tracking changes in HAE treatment over time.
