Napkin math can change the world.

Mathematical models hold the key to equitable patient care.

An increased disease burden of autoimmune inflammatory rheumatic diseases in Korea.

OBJECTIVES: To estimate the prevalence, medical utilization, and recent changes in the economic burden of autoimmune rheumatic diseases (AIRDs) in Korea. METHODS: Using a nationwide claims database that includes all medical claims made by approximately 50 million Korean residents, the prevalences of seropositive rheumatoid arthritis (RA), ankylosing spondylitis (AS), systemic lupus erythematosus (SLE), and others between 2012 and 2016 were calculated. Changes in medical utilization and the direct medical costs of each AIRD from 2012 to 2016 were also evaluated. RESULTS: Based on the data for 2016, seropositive RA was the most common AIRD in Korea with 96,330 cases (188.5/100,000 population), followed by AS (30,006, 58.7/100,000 population), SLE (19,441, 38.0/100,000 population), Behçet's disease (BD, 14,943, 29.2/100,000 population), primary Sjögren syndrome (pSS, 12,018, 23.5/100,000 population), and systemic sclerosis (SSc, 3606, 7.1/100,000 population). In terms of medical utilization, patients with eosinophilic granulomatosis with polyangiitis visited outpatient clinics the most frequently (9.8 times/year/patient), while hospitalization was most frequent in microscopic polyangiitis patients (1.0 time/year/patient). Total medical costs for all AIRDs increased from $154,348,011 in 2012 to $262,481,974 in 2016. The annual medical cost per patient in 2016 was the highest in microscopic polyangiitis ($6223/year), followed by psoriatic arthritis ($3,362/year), and granulomatosis with polyangiitis ($2823/year). CONCLUSIONS: In Korea, the most prevalent AIRD is seropositive RA, followed by AS, SLE, BD, pSS, and SSc. The economic burden of AIRDs has risen substantially in the last 5 years due not only to an increase in their prevalence but also to an increase in medical costs per patient.

Associations of pemphigus or pemphigoid with autoimmune disorders in US adult inpatients.

BACKGROUND: The associations and predictors of the gamut of autoimmune conditions in pemphigus and pemphigoid have been examined in few large-scale controlled studies. OBJECTIVE: To examine associations of pemphigus or pemphigoid with autoimmune disorders and related outcomes in adults. METHODS: Data from the 2002-2012 National Inpatient Sample were analyzed, including an ∼20% sample of all US hospitalizations (n = 72,108,077 adults). RESULTS: In multivariable logistic regression models, pemphigus (adjusted odds ratio 1.46, 95% confidence interval 1.30-1.63) and pemphigoid (adjusted odds ratio 1.35, 95% confidence interval 1.24-1.48) were associated with ≥1 autoimmune disorder. Pemphigus was associated with 9 of 29 and pemphigoid with 13 of 32 autoimmune disorders examined in bivariable models. Among pemphigus inpatients, unspecified autoimmune disease, vitiligo, eosinophilic esophagitis, and myasthenia gravis had the strongest effect sizes. Whereas, among pemphigoid inpatients, unspecified autoimmune disease, vitiligo, and chronic urticaria had the strongest effect sizes. There were significant differences of autoimmune comorbidities by age, sex, and race/ethnicity. The estimated excess annual costs of hospital care attributed to autoimmune disorders among inpatients with pemphigus was $2,286,588 and pemphigoid $4,301,681. LIMITATION: Lack of treatment history data. CONCLUSION: Inpatients with pemphigus or pemphigoid had increased odds of multiple cutaneous, extracutaneous, and systemic autoimmune disorders, which were associated with a considerable cost burden.

Characterizing Direct-to-Consumer Stem Cell Businesses in the Southwest United States.

There are currently hundreds of businesses across the United States offering direct-to-consumer stem cell treatments that have not been through regulatory approval by the Food and Drug Administration (FDA). Here, we provide a detailed characterization of nearly 170 stem cell businesses operating in the Southwest United States. We draw specific attention to two as-yet understudied facets of these businesses. First, we identify differences in the degree to which a given business focuses their practice on stem cell treatments. Second, we compare the stated expertise of the care providers in stem cell businesses with the range of conditions they purport to treat. These findings deepen our knowledge of the growing industry around unapproved stem cell treatments, and are used here to offer suggestions for how the FDA might target its resources with respect to regulatory oversight.

Short-term costs associated with non-medical switching in autoimmune conditions.

OBJECTIVES: To estimate short-term costs associated with non-medical switch (NMS) from originator biologics to biosimilars among stable patients with autoimmune conditions in rheumatology, gastroenterology, and dermatology from a US provider's and third-party payer's perspective. METHODS: An economic model was constructed to estimate switching costs related to physician time and healthcare resource utilisation (HRU) at the initial NMS visit and over 3 months. The proportion of patients with relevant conditions treated with originators and expected NMS rate, physician time, HRU, and payer reimbursement were derived from a physician survey. Switching costs were estimated for a practice of 1,000 patients with relevant conditions by therapeutic area and for an insurance plan with 1 million individuals by therapeutic area and all areas combined. Switching cost drivers were assessed with one-way sensitivity analyses. RESULTS: Physicians expected extra 6 minutes for the NMS visit and 22 minutes over 3 months; NMS rates of 14.4%, 15.5%, and 17.7%; and 11.3%, 16.2%, and 33.2% of time not reimbursed for gastroenterology, rheumatology, and dermatology, respectively. The total switching costs for payer's were $771,460 (for n = 3,609 patients with an NMS rate of 16.6%), mostly due to follow-up visits and additional laboratory tests/procedures. In sensitivity analyses, the NMS rate was the main cost driver. Increasing the NMS rate to 25% and 50% increased payer's total switching costs to $1.19 and $2.39 million, respectively. CONCLUSIONS: Originator-to-biosimilar NMS in stable patients with autoimmune conditions could result in considerable switching costs for both providers and payers.

The Effect of Pre-Appointment Consultation Triage on Patient Selection and Revenue Generation in a University Rheumatology Practice.

OBJECTIVE: To evaluate the effectiveness of pre-appointment consult screening to identify patients with autoimmune and inflammatory rheumatic disease (AIRD) and to evaluate the revenue implications of routine outpatient care of patients with AIRD compared to that of non-AIRD patients. METHODS: Using data in the electronic medical records, we retrospectively analyzed all new outpatients who were referred for rheumatology consults during a 9-month period for a final diagnosis and revenue generation for routine outpatient care over 1 year following the consult review or initial evaluation. RESULTS: A total of 961 patients were referred to the outpatient rheumatology clinic and underwent pre-appointment triage. Overall, 673 patients were approved for evaluation of AIRD, and 288 patients were denied rheumatology consultation. Patients were seen an average of 13 days after the consult review. Among patients who were approved for consult, 597 came for evaluation, with 357 diagnosed as having an AIRD and 240 with a non-AIRD. Among patients who were denied a consult, 128 had 1-year follow-up data, with 6 patients eventually diagnosed as having an AIRD (consult triage sensitivity 98%, positive predictive value 60%). The consult triage system allowed more AIRD patients to be seen over a 1-year period. Revenue data for outpatient care was available for 318 of 357 patients with an AIRD and 192 of 240 non-AIRD patients and showed that care for patients with an AIRD generates 44 times more revenue compared to care for non-AIRD patients ($5,877 per AIRD patient versus $134 per non-AIRD patient; P < 0.001). CONCLUSION: Pre-appointment consult screening is an effective method to identify patients with an AIRD. This approach enables timely access to care for patients with the highest need for evaluation and results in significantly more revenue generation.

Association between atopic dermatitis and autoimmune disorders in US adults and children: A cross-sectional study.

BACKGROUND: Little is known about the risk and predictors of autoimmune diseases in children and adults. OBJECTIVE: To determine the prevalence, predictors, and excess costs of autoimmune disease in atopic dermatitis (AD) patients. METHODS: Cross-sectional study of the 2002-2012 National Inpatient Sample, which includes a ∼20% sample of all US hospitalizations (n = 87,053,155 adults and children). RESULTS: The prevalence of autoimmune disease was higher in adults with AD (7.9%, 95% confidence interval [95% CI] 7.3-8.5%) than without AD (5.7%, 95% CI 5.7%-5.8%) and higher in children with AD (2.0%, 95% CI 1.7%-2.3%) than without AD (1.0%, 95% CI 0.9%-1.1%). In multivariable logistic regression models controlling for sociodemographics, adult (adjusted odds ratio 1.45, 95% CI 1.32-1.58) and pediatric (adjusted odds ratio 2.08, 95% CI 1.73-2.50) AD were associated with any autoimmune disorder. In particular, AD was associated with 18 of 32 autoimmune disorders examined in adults and 13 of 24 examined in children, including disorders of the skin, endocrine, gastrointestinal, hematologic, and musculoskeletal systems. AD patients hospitalized with any autoimmune disorder had a higher cost of inpatient care, with $2.5-$50 million excess annual costs. CONCLUSIONS: Adults and children with AD had increased cutaneous and extracutaneous autoimmune disorders, which were associated with a considerable cost burden.

The Utilization of Autoantibodies in Approaches to Precision Health.

Precision health (PH) applied to autoimmune disease will need paradigm shifts in the use and application of autoantibodies and other biomarkers. For example, autoantibodies combined with other multi-analyte "omic" profiles will form the basis of disease prediction allowing for earlier intervention linked to disease prevention strategies, as well as earlier, effective and personalized interventions for established disease. As medical intervention moves to disease prediction and a model of "intent to PREVENT," diagnostics will include an early symptom/risk-based, as opposed to a disease-based approach. Newer diagnostic platforms that utilize emerging megatrends such as deep learning and artificial intelligence and close the gaps in autoantibody diagnostics will benefit from paradigm shifts thereby facilitating the PH agenda.

Immunosuppressants in Brazil: underlying drivers of spending trends, 2010-2015.

BACKGROUND: Immunosuppressants are recommended for treatment of autoimmune diseases, and in transplant therapy. The high cost of these drugs has been causing an important impact on global pharmaceutical spending. OBJECTIVE: Analyzing immunosuppressant expenditure in Brazil, using data from the Federal Procurement System database (SIASG), between 2010 and 2015. METHODS: The pharmaceutical products were classified in accordance with the Anatomical, Therapeutic and Chemical (ATC) classification system recommended by World Health Organization (WHO) and aggregated by volume and by expenditure. The expenditure variation was decomposed into three broad categories: price effects, quantity effects, and drug mix effects. RESULTS: During the period, annual expenditure increased by 49%, ranging from USD 494.5 million in 2010 to USD 738.7 million in 2015, while purchased quantities increased by 294%, ranging from 49.8 million in 2010 to 196.5 million in 2015. Two factors drove expenditures: the quantity effect and the drug-mix effect. CONCLUSION: These findings may contribute to understand immunosuppressant spending trends and the factors that influence them in order to formulate effective cost containment strategies and design optimum drug policy. Rigorous evaluations are recommended to reduce the drug-mix effect, including systems to monitor price, effectiveness, safety, therapeutic value and budget impact of pharmaceutical innovations.

Healthcare resource use and cost associated with varying dosages of extended corticosteroid exposure in a US population.

AIMS: To quantify healthcare resource use (HCRU) and costs associated with varying levels of corticosteroid exposure. MATERIALS AND METHODS: Patients with a diagnosis of selected autoimmune and inflammatory diseases between 1 January 2006 and 30 September 2015 ("study period") were selected from a de-identified, privately-insured claims database. Patients were stratified into four treatment cohorts based on the dosing and duration of continuous corticosteroid use following disease diagnosis: intermittent use with <60 days of corticosteroid use and ≥60 days of corticosteroid use with low (≤7.5 mg/day), medium (>7.5-≤15 mg/day), or high (>15 mg/day) dosage. Patients were followed from the date of their highest dose episode of corticosteroid use ("treatment index date") until the earliest of the end of continuous corticosteroid use +30 days, disenrollment from health plan, or the end of the study period ("follow-up period"). HCRU and costs in the follow-up period were compared across treatment cohorts. RESULTS: Of 78,704 patients who were identified for study inclusion, 29% had extended corticosteroid use lasting ≥60 days, and 71% had intermittent use. On average, patients in the high-dose cohort incurred twice the cost of intermittent users ($68,408 vs $32,690 in annualized total all-cause healthcare costs, USD). Adverse event-related medical costs accounted for ∼40% of medical costs, and were higher than disease-related medical costs for all cohorts with extended corticosteroid exposure. Comparing the high-dose and low-dose cohorts, the smaller savings in disease-related prescriptions ($1,680) occurred along with a much larger cost in adverse event-related spend ($13,464). LIMITATIONS: The impact of corticosteroids may be under-estimated because of conservative follow-up duration, and administrative data may contain inaccuracies in coding. CONCLUSIONS: Steroid use, especially at higher doses, is associated with higher HCRU and costs.

TRAPPED - an insight into two sisters' struggle to access treatment for a rare genetic disease.

Medical student training is largely focused on acquiring knowledge of diseases and their management, which may leave one with a naïve perception of what is achievable in practice, particularly in the field of rare diseases. Tumour Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS) is a rare autoinflammatory disorder with a prevalence of one in a million. Its features include recurrent disabling episodes of high-grade fever associated with rash and arthralgia. Its rarity, combined with its somewhat vague and heterogenous clinical presentation, means that patients often suffer with TRAPS for years before they are diagnosed. Although it has a licensed treatment, Interleukin-1 blocker Anakinra, this is not currently funded by the NHS. This report provides an insight into the experiences of two sisters recently diagnosed with TRAPS, and the barriers they face preventing them from accessing the treatment they need, without which they are likely to suffer life-threatening organ failure. I have argued that the commissioning policy model for rare diseases needs reconsideration to improve access to Anakinra on a national level, and have highlighted the significant impact that clinicians can have on an individual level by being advocates for their patients.

Productivity Losses and Costs in the Less-Common Systemic Autoimmune Rheumatic Diseases.

PURPOSE OF REVIEW: We synthesised the literature on productivity losses and costs in the less-common systemic autoimmune rheumatic diseases: Sjogren's syndrome (SjS), systemic sclerosis (SSc), poly/dermatomyositis (PM/DM), and systemic vasculitides (SV). RECENT FINDINGS: Of 29 studies located, 12 were published 2012 onwards (SSc = 6, SjS = 2, PM/DM = 2, SV = 2). In these, 25% of PM/DM, and 21-26% of SV, were work disabled, 22% of SSc stopped work within 3 years of diagnosis, and annual costs of absenteeism in SSc averaged $12,024 2017 USD. Very few studies reported on costs, presenteeism (working at reduced levels), or unpaid productivity loss. Across multiple systemic autoimmune rheumatic diseases (SARDs), major drivers of lost productivity were generalised items like pain, depression, and fatigue, rather than disease-specific factors. Evidence suggests that work disability is common in SSc and strikes quickly. However, in SSc and other SARDs, more comprehensive estimates are needed, which include absenteeism and presenteeism from paid and unpaid work, costs, and drivers of productivity loss.

Clinical and Economic Evaluation of Repository Corticotropin Injection: A Narrative Literature Review of Treatment Efficacy and Healthcare Resource Utilization for Seven Key Indications.

INTRODUCTION: Repository corticotropin injection (RCI; H.P. Acthar® Gel; Mallinckrodt Pharmaceuticals Inc., Hampton, NJ) is a highly purified, prolonged-release porcine preparation of adrenocorticotropic hormone (ACTH) analogue that is FDA-approved for treatment of 19 autoimmune and inflammatory disorders. The diverse physiological actions of RCI at the melanocortin receptors (MCRs) affect processes involved in inflammation, pigmentation, steroidogenesis, and immunomodulation. Although RCI has been approved to treat inflammatory and autoimmune diseases for more than 60 years, recent progress in understanding both MCRs and the effects of RCI in modulating immune responses has led to increased interest in RCI as a therapeutic choice. The objective of this narrative literature review is to summarize key clinical and economic data on RCI treatment of seven disorders: infantile spasms (IS), multiple sclerosis (MS) relapses, proteinuria in nephrotic syndrome, rheumatoid arthritis (RA), dermatomyositis/polymyositis (DM/PM), systemic lupus erythematosus (SLE), and symptomatic sarcoidosis based on published literature and product information. An extended report is available as the Academy of Managed Care Pharmacy (AMCP) Formulary dossier for H.P. Acthar® Gel. METHODS: Key studies of clinical efficacy and healthcare utilization and cost from 1956 to 2016 are summarized. RESULTS: The evidence supports the efficacy of RCI across the seven indications. RCI is effective as a first-line therapy for IS. For the other six conditions, RCI may improve clinical outcomes during exacerbations or when the condition is resistant to conventional treatments. Use of RCI is associated with reduced use of biologics, corticosteroids, and disease-modifying antirheumatic drugs. Initiation of RCI therapy in patients with IS, MS, RA, SLE, or DM/PM has been associated with lower post-therapy healthcare utilization and medical costs, including decreases in hospitalizations, hospital length of stay, outpatient visits, and emergency department visits. CONCLUSION: The evidence suggests that RCI may improve inflammatory and autoimmune disease control and patient quality of life, particularly in complex patients, and yield healthcare cost savings that demonstrate the medicine's value. FUNDING: Mallinckrodt Pharmaceuticals Inc.

Filling the gaps in SARDs research: collection and linkage of administrative health data and self-reported survey data for a general population-based cohort of individuals with and without diagnoses of systemic autoimmune rheumatic disease (SARDs) from British Columbia, Canada.

PURPOSE: Systemic autoimmune rheumatic diseases (SARDs) are a group of debilitating autoimmune diseases, including systemic lupus erythematosus and related disorders. Assessing the healthcare and economic burden of SARDs has been challenging: while administrative databases can be used to determine healthcare utilisation and costs with minimal selection and recall bias, other health, sociodemographic and economic data have typically been sourced from highly selected, clinic-based cohorts. To address these gaps, we are collecting self-reported survey data from a general population-based cohort of individuals with and without SARDs and linking it to their longitudinal administrative health data. PARTICIPANTS: Using administrative data from the province of British Columbia (BC), Canada, we established a population-based cohort of all BC adults receiving care for SARDs during 1996-2010 (n=20 729) and non-SARD individuals randomly selected from the general population. BC Ministry of Health granted us contact information for 12 000 SARD and non-SARD individuals, who were recruited to complete the surveys by mail or online. FINDINGS TO DATE: Four hundred individuals were initially invited to participate, with 135 (34%) consenting and 127 (94%) submitting the first survey (72% completed online). Sixty-three (49.6%) reported ≥1 SARD diagnosis. The non-SARDs group (n=64) was 92% female with mean age 57.0±11.6 years. The SARDs group (n=63) was 94% female with mean age 56.5±13.1 years. Forty-eight per cent of those with SARDs were current-or-former smokers (mean 10.6±16.2 pack-years), and 33% were overweight or obese (mean body mass index of 24.4±5.3). FUTURE PLANS: Health and productivity data collected from the surveys will be linked to participants' administrative health data from the years 1990-2013, allowing us to determine the healthcare and lost productivity costs of SARDs, and assess the impact of patient-reported variables on utilisation, costs, disability and clinical outcomes. Findings will be disseminated through scientific conferences and peer-reviewed journals.

The Corrona US registry of rheumatic and autoimmune diseases.

The Corrona US national registry collects data concerning patient status from both the rheumatologist and patient at routine clinical encounters. Corrona has functioning disease registries in rheumatoid arthritis, psoriatic arthritis, spondyloarthropathies, psoriasis and inflammatory bowel disease. Corrona merges data concerning long-term effectiveness and safety, as well as comparative and cost effectiveness of agents to treat these autoimmune diseases.

Indirect costs of absenteeism due to rheumatoid arthritis, psoriasis, multiple sclerosis, insulin-dependent diabetes mellitus, and ulcerative colitis in 2012: a study based on real-life data from the Social Insurance Institution in Poland.

INTRODUCTION: The aim of this study is to assess the indirect costs of six major autoimmune diseases including seropositive rheumatoid arthritis, other types of rheumatoid arthritis, psoriasis, multiple sclerosis, Type 1 diabetes, and ulcerative colitis. METHODS: Relevant data for 2012 on sick leave and short- and long-term work disabilities were obtained from the Social Insurance Institution in Poland. Indirect costs were estimated using the human capital approach based on gross domestic product per capita, gross value added per worker, and gross income per worker in Poland in 2012 and expressed in euro. RESULTS: We recorded data on the total number of 45,500 patients. The total indirect costs were EUR 146,862,569; 353,683,508; and 108,154,271, calculated using gross domestic product, gross value added, and gross income, respectively. CONCLUSIONS: Considering only data on absenteeism collected by the Social Insurance Institution in Poland, we can conclude that the selected autoimmune diseases are associated with great indirect costs.

The Disease Burden of the Most Common Autoimmune Diseases.

By some measures, the financial and health toll from inflammatory bowel disease, rheumatoid arthritis, and psoriasis is comparable to stroke and lower back pain. Collectively, they affect about 10 million patients in the United States. They are chronic conditions characterized by immune-mediated inflammation of unknown etiology and for which there is no cure.