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This is the first report of the health economic benefits derived from preventing infections through Immunoglobulin Replacement Therapy (IgRT) in patients with secondary immunodeficiency due to hematological malignancies. We conducted a retrospective population-based cohort study using patient medical history and pharmacy data from the Hospital Clínico San Carlos for 21 patients between 2011 and 2020. The pharmacoeconomic impact of using prophylactic IgRT was assessed by comparing characteristics of the SID patients 1 year before and after initiating IgRT measured by direct medical and tangible indirect costs. Results indicate a marked reduction in hospitalization days following IgRT initiation, decreasing from an average of 13.9 to 6.1 days per patient, with the elimination of ICU admissions. While emergency department visits decreased significantly, the number of routine consultations remained unchanged. Notably, absenteeism from work dropped substantially. The financial analysis revealed significant reductions in medication use and fewer ancillary tests, resulting in considerable cost savings. Specifically, total expenditure dropped from €405,088.18 pre-IgRT to €295,804.42 post-IgRT-including the cost of IgRT itself at €156,309.60. Overall, the annual savings amounted to €109,283.84, validating the cost-effectiveness of IgRT in managing SID in patients with hematological cancers.

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The introduction of human immunoglobulin (Ig) therapies 40 years ago reduced the risk of often life-threatening infections for individuals with one of several immune-related conditions known as primary immunodeficiencies. Since then, the use of Ig has expanded to numerous other conditions. However, even though less than 1% of covered lives under Medicare or commercial insurers require Ig, it is in the top 5 drug categories in terms of annual spending. The cost of Ig is directly related to the type of delivery method used and the site of care. Numerous studies attest to the efficacy and cost savings of shifting Ig to the home setting, as well as shifting patients from intravenous Ig (IVIG) to subcutaneous Ig (SCIG). In addition, surveys find that patients with primary immunodeficiencies prefer home delivery, with patient evaluations also finding a preference for SCIG. Payers have numerous options to ensure Ig is used appropriately for the right patient in the right setting. These include formulary management, site-of-care programs, education for providers and patients on the possibility of switching from IVIG to SCIG, preauthorization policies that restrict the use of Ig to certain specialties for specific indications, implementation of evidence-based coverage criteria, and shifting coverage from the medical to the pharmacy benefit.

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BACKGROUND: Home-based subcutaneous immunoglobulin (SCIg) administration used for immunoglobulin replacement therapy for patients with primary immunodeficiency has been demonstrated to have benefits compared with hospital-based intravenous immunoglobulin (IVIg) therapy. OBJECTIVE: To estimate the cost savings associated with treating eligible patients with primary immunodeficiency with home-based SCIg compared with hospital-based IVIg in a prospective study. METHODS: This study was a 12-month prospective observational study that collected information from patient charts, directly from the nurse for time spent with patients and materials used, and directly from the physicians for billing. Data were collected on case report forms at each follow-up. Data were entered in a web-based REDCap database and statistical comparisons were performed. RESULTS: The average hospital (including hospital personnel such as nurses) and physician costs were significantly lower in the SCIg group ($1,836 and $84, respectively) than in the IVIg group ($4,187 and $744, respectively), which supported the findings in the number of hospital and physician visits in each group. The total cost was reported from the hospital's (only hospital-related costs) and the health system's (hospital- and physician-related costs) perspectives. For the 2 perspectives, the SCIg group reported significantly lower average total costs than the IVIg group. CONCLUSION: This is the first prospective analysis of the cost savings associated with home-based SCIG therapy compared with hospital-based IVIG therapy. These findings could help justify provision of home-based therapy training to suitable patients to lower health care costs or improve the capacity of care.

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BACKGROUND: In addition to the deleterious effect on health, there is considerable economic and psychosocial morbidity associated with primary immunodeficiency diseases (PID). Also, the cost of a late diagnosis frequently results in a heavy disease burden on the patient. The objective of this study was to collect and analyze data on patients with PID in the state of Guanajuato in Mexico, to indirectly estimate the burden of the disease. METHODS: An observational, longitudinal, and comparative study was conducted. A total of 44 patients were included and grouped according to the updated classification of PID. RESULTS: The median time elapsed from the onset of symptoms to the reference and diagnosis by a tertiary hospital was of 2.17 (IQR = 6.44) years. Before diagnosis, the number of hospitalizations/year per patient was 0.86 (IQR = 2.28), the number of visit to emergency room/year per patient was 0.92 (IQR = 1.77), the number of doctor's visits/year per patient was 15 (IQR = 11.25), whereas the school/work absence days per patient were reported in 52.72 (IQR = 56.35) days per year. After diagnosis, 20 patients (45.45%) received IVIG replacement therapy, and all of them presented a significant improvement (p <0.05) in all the mentioned variables. Characteristically, even when patients with PID received IVIG, there was still an important disease burden when comparing them against healthy controls. Complications secondary to PID were detected in 19 patients (43.18%). The reported overall mortality rate was 6.82% (n = 3). CONCLUSIONS: We were able to indirectly estimate an important disease burden in patients with PID; which is considered to be preventable, at least in part, with effective interventions like health planning, research, collaboration with primary care providers, and generation of policies and practices, in order to improve the quality of life and care of families with PID.

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BACKGROUND: Patients with primary immune deficiency (PID) often require immunoglobulin G (IgG, commonly referred to as Ig) replacement therapy to prevent infections and associated comorbidities. Ig therapy can be given either through intravenous or subcutaneous routes, and both can be done in the home setting. There is limited information available on the real-world diagnosis, management, and outcomes of this patient population, given the variable disease presentation and treatment options. The Immunoglobulin Diagnosis, Evaluation, and key Learnings (IDEaL) Patient Registry is designed to capture nursing, pharmacy, and patient-reported data for patients receiving Ig at home. OBJECTIVES: To (a) present a real-world population of patients with PID who have received Ig at home and (b) examine how differences in administration, dosing, and insurance affect health and quality-of-life outcomes in these patients. METHODS: As of July 2015, 383 patients receiving Ig therapy from Coram/CVS specialty infusion services, across multiple disease states, signed consent forms and enrolled in the IDEaL Patient Registry. Patients' referral paperwork, including lab values, and standard of care nursing and pharmacy follow-up forms were collected. Patients were mailed quality-of-life surveys at the time of enrollment and every 6 months after their enrollment. RESULTS: The most common diagnosis (78%) in these PID patients was common variable immunodeficiency (CVID). For Ig-naive adult patients, the average age at the start of treatment was 59 years. For pediatric patients, average age at start of treatment was 9 years. A majority of these PID patients (80%) received subcutaneous Ig (SCIg) at home, and 20% received intravenous Ig (IVIg). The average SCIg dose was 10 grams per week, or 130 mg per kg, and the average IVIg dose was 36 grams every 4 weeks, or 472 mg per kg. In the IVIg patient population, 34% had a dose or frequency change while on treatment, while 30% of the SCIg patients had a dose or frequency change. Patient-reported health and quality-of-life scores were generally positive. Route of administration did not affect patient perception of cost (P = 0.171), but whether the patient had private or government-backed health care did affect perception of cost (P = 0.036). CONCLUSIONS: For a disease state with an extremely variable presentation, data from the IDEaL Patient Registry provides further insights into the real-world clinical and diagnostic characteristics of this population, as well as dosing and treatment outcomes of home administration of Ig therapy. The majority of patients received SCIg infusions. SCIg dosing was on the lower end of the recommended mg per kg dose range, while IVIg patients were more in the middle of the recommended dose range. Patient outcomes on treatment were correlated with baseline status, suggesting that earlier detection and treatment of primary immune deficiencies may be critical in achieving beneficial outcomes on Ig therapy. DISCLOSURES: No outside funding supported this study. Seidu was compensated by Coram Clinical Trials for acting as primary investigator and reviewing data. Study concept and design were contributed by all the authors. Kearns, Kristofek, and Kiles collected the data, and data interpretation was performed by Kearns, Seidu, and Kristofek, along with Bolgar. The manuscript was written and revised primarily by Kearns, along with Kristofek, Bolgar, and Seidu.

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Primary immune deficiency disorders (PIDs) are rare conditions for which effective treatment is available. It is critical these patients are identified at an early stage to prevent unnecessary morbidity and mortality. Treatment of these disorders is expensive and expert evaluation and ongoing management by a clinical immunologist is essential. Until recently there has been a major shortage of clinical immunologists in New Zealand. While the numbers of trained immunologists have increased in recent years, most are located in Auckland. The majority of symptomatic PID patients require life-long immunoglobulin replacement. Currently there is a shortage of subcutaneous and intravenous immunoglobulin (SCIG/IVIG) in New Zealand. A recent audit by the New Zealand Blood Service (NZBS) showed that compliance with indications for SCIG/IVIG treatment was poor in District Health Boards (DHBs) without an immunology service. The NZBS audit has shown that approximately 20% of annual prescriptions for SCIG/IVIG, costing $6M, do not comply with UK or Australian guidelines. Inappropriate use may have contributed to the present shortage of SCIG/IVIG necessitating importation of the product. This is likely to have resulted in a major unnecessary financial burden to each DHB. Here we present the case for a national service responsible for the tertiary care of PID patients and oversight for immunoglobulin use for primary and non-haematological secondary immunodeficiencies. We propose that other PIDs, including hereditary angioedema, are integrated into a national PID service. Ancillary services, including the customised genetic testing service, and research are also an essential component of an integrated national PID service and are described in this review. As we show here, a hub-and-spoke model for a national service for PIDs would result in major cost savings, as well as improved patient care. It would also allow seamless transition from paediatric to adult services.

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Primary immunodeficiencies (PI) are defects of the immune system that cause severe, sometimes life-threatening, infections if not diagnosed and treated appropriately. Many patients with PI are undiagnosed, under-diagnosed, or misdiagnosed. To raise awareness and assure earliest diagnosis, appropriate treatment, and proper care management, the Jeffrey Modell Foundation (JMF) implemented a physician education and public awareness program beginning in 2003. Data are requested annually from physician experts within the Jeffrey Modell Centers Network (JMCN), consisting of 602 expert physicians, at 253 academic institutions, in 206 cities, and 84 countries spanning six continents. Center Directors reported on patients' specific PI defects and treatment modalities including immunoglobulins, transplantation, and gene therapy as well as data on gender and age. Center Directors also provided physician-reported patient outcomes as well as pre- and post-diagnosis differences. Costs were assigned to these factors. In collaboration with the Network, JMF advocated, funded, and implemented population-based newborn screening for severe combined immunodeficiency and T cell lymphopenia, covering 96.2 % of all newborns in the US. Finally, 21 JMF Centers participated in a polio surveillance study of patients with PI who either received or have been exposed to the oral polio vaccine. These initiatives have led to an overall better understanding of the immune system and will continue to improve quality of life for those with PI.

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BACKGROUND: A subset of patients with chronic rhinosinusitis (CRS) has refractory disease. The risk factors for refractory CRS include atopy, a disrupted mucociliary transport system, medical conditions affecting the sinonasal tract mucosa, and immunodeficiency. METHODS: We review four primary immunodeficiencies reported in individuals with CRS: common variable immune deficiency (CVID), selective IgA deficiency, IgG subclass deficiency, and specific antibody deficiency. We also review treatment options for individuals with both CRS and a concomitant immune defect. RESULTS: There is a high prevalence of CRS in individuals with CVID and selective IgA deficiency. While many reports describe IgG subclass deficiency in individuals with CRS, the clinical relevance of this is unclear. Specific antibody deficiency may play a more significant role in the pathogenesis of refractory CRS. CONCLUSION: Screening for a primary immunodeficiency should be part of the diagnostic workup of refractory CRS, as its identification may allow for more effective long-term therapeutic options.

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OBJECTIVE: The objective of this study is to evaluate the economic benefits of immunoglobulin replacement therapy achieved subcutaneously (subcutaneous immunoglobulin, SCIG) by the rapid push method compared to intravenous infusion therapy (intravenous immunoglobulin, IVIG) in primary immune deficiency (PID) patients from the healthcare system perspective in the context of the adult SCIG home infusion program based at St Paul's Hospital, Vancouver, Canada. MATERIALS AND METHODS: SCIG and IVIG options were compared in cost-minimisation and budget impact models (BIMs) over 3 years. Sensitivity analyses were performed for both models to evaluate the impact of varying modality of IVIG treatments and proportion of patients switching from IVIG to SCIG. RESULTS: The cost-minimisation model estimated that SCIG treatment reduced cost to the healthcare system per patient of $5736 over 3 years, principally because of less use of hospital personnel. This figure varied between $5035 and $8739 depending on modality of IVIG therapy. Assuming 50% of patients receiving IVIG switched to SCIG, the BIM estimated cost savings for the first 3 years at $1·308 million or 37% of the personnel and supply budget. These figures varied between $1·148 million and $2·454 million (36 and 42%) with varying modalities of IVIG therapy. If 75% of patients switched to SCIG, the reduced costs reached $1·962 million or 56% of total budget. CONCLUSION: This study demonstrated that from the health system perspective, rapid push home-based SCIG was less costly than hospital-based IVIG for immunoglobulin replacement therapy in adult PID patients in the Canadian context.

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Since 2005, when changes in Medicare reimbursement for IgG replacement therapy went into effect, physicians and patients with primary immunodeficiency disease (PIDD) have encountered a number of challenges to administering and receiving appropriate immunoglobulin therapy. A 2006 membership survey conducted by the American Academy of Allergy, Asthma & Immunology found that 95 % of responders thought that the health of their patients was at risk due to Medicare changes; many patient surveys also found a significant number adversely affected by these changes. Decisions critical for optimal care being made by third-party payors are often in conflict with guidelines on recommended standard of care. Many payors, for example, are dictating where infusions can occur despite evidence clearly demonstrating that choice of the site of care needs to be determined by the particular patient's circumstance and experience. Another critical issue is the lack of product availability due to the determination by payors of which IgG products appear on formularies. Patients, physicians, and payors all bring their own perspective to these issues, and finding a solution to these challenges requires balancing the needs of all three groups.

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Decisions by third-party payors that are restricting delivery of appropriate IgG treatment for primary immunodeficiency disease (PIDD) are summoning action from patients, physicians, and their organizations to ensure that high quality treatment remains accessible. Some of the strongest advocacy to date is from patient organizations, such as the Immune Deficiency Foundation (IDF), which strive to educate stakeholders on key issues that determine patient access to appropriate IgG treatment. These issues include the ability to choose the appropriate site of care based on a patient's experience and circumstance and greater awareness of product choice. Advocacy by physicians on these issues at the local level is needed, as are national efforts by organizations such as the American Academy of Allergy, Asthma & Immunology and their regional societies.

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Treatment decisions made in clinical practice, based on current guidelines, often conflict with decisions by third-party payors that restrict the ability of patients with primary immunodeficiency disease (PI) to adhere to appropriate treatment. This is seen by many physicians as potentially placing the health of patients at risk. Key treatment decisions challenged by third-party payors and discussed here include dosing, product safety, and routes of administration. Data on safety issues emphasize that IgG products are not generic drugs and each of the products currently licensed by the Food and Drug Administration (FDA) must be regarded as an individual therapy, given the products' different manufacturing processes and stabilizing ingredients. The issue of switching patients to a different product needs careful consideration as evidence shows that infusion-related adverse events in many patients are frequently related to this activity. Decisions regarding the route of therapy should also be individualized to the patient, weighing such factors as side effects, adherence with therapy, and lifestyle.

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Kiovig is a ready-to-use 10% liquid immunoglobulin preparation that is medically indicated for the treatment of primary immunodeficiency. This study aims to conduct an economic evaluation which compares the intravenous immunoglobulin (IVIg) preparations Kiovig, Multigam, and Sandoglobulin from the Belgian societal perspective. As three prospective studies have observed no difference in outcomes, a cost-minimization analysis is considered appropriate to evaluate differences in treatment costs that can arise from IVIgs. A decision-analytic model simulated treatment costs attributed to one infusion. Resource use data were derived from a Dutch costing study. Cost items included immunoglobulin costs, pharmacy administration and nursing costs, mini-forfait for hospital infusion, costs of adverse events, and lost productivity with 2009 as base year. Cost data were identified from published sources and Belgian hospital administrators. A probabilistic sensitivity analysis explored the impact of parameter uncertainty on cost results. Costs per infusion cycle in adult primary immunodeficiency patients were €1,046 (95% confidence interval: €1,006-1,093) with Kiovig; €1,102 (€1,064-1,147) with Multigam; and €1,147 (€1,108-1,193) with Sandoglobulin. The average cost savings per infusion with Kiovig as compared to Multigam and Sandoglobulin amounted to €56 and €101 per infusion. In conclusion, treatment costs with Kiovig were shown to be lower as compared to other IVIgs in Belgium. Reduced costs per infusion were attributed to lower costs associated with treating adverse events and the opportunity cost of nursing time and time off work for working adults.

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Experts from six Latin American countries met to discuss critical issues and needs in the diagnosis and management of primary immunodeficiency diseases (PIDD). The diagnosis of PIDD is generally made following referral to an immunology centre located in a major city, but many paediatricians and general practitioners are not sufficiently trained to suspect PIDD in the first place. Access to laboratory testing is generally limited, and only some screening tests are typically covered by government health programmes. Specialised diagnostic tests are generally not reimbursed. Access to treatment varies by country reflecting differences in healthcare systems and reimbursement policies. An online PIDD Registry Programme for Latin America has been available since 2009, which will provide information about PIDD epidemiology in the region. Additional collaboration across countries appears feasible in at least two areas: a laboratory network to facilitate the diagnosis of PIDD, and educational programmes to improve PIDD awareness. In total, these collaborations should make it possible to advance the diagnosis and management of PIDD in Latin America.

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Primary immunodeficiency disorders are associated with increased patient susceptibility to recurrent infections. Since the 1950s, intramuscular, intravenous and subcutaneous immunoglobulin products have been used to replace functionally deficient or absent immunoglobulins, reduce the incidence of infections and prevent organ damage caused by infections. This article aims to review the use of immunoglobulin therapy in primary immunodeficiency by focusing on costs, effectiveness, cost-effectiveness, supply and off-label use. To date, the economic burden of primary immunodeficiency is unknown. Past studies have supported minimal differences in effectiveness between intravenous and subcutaneous immunoglobulins. Subcutaneous therapy may be considered for patients who prefer treatment at home. The small number of economic evaluations and their methodological limitations precludes the recommendation of a specific product for use in primary immunodeficiency on pharmacoeconomic grounds. Demand for immunoglobulins has increased over time, leading to periodic shortages and emphasizing the importance of its appropriate use.

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