The Evolving Effectiveness of Biologics in Avoiding Surgery in Children With Ulcerative Colitis: At what Nutritional Cost?

BACKGROUND: Pediatric ulcerative colitis (UC) treatment has changed dramatically with the introduction of multiple biologics. The goal of this study was to determine the effectiveness of these new biologics on achieving remission, nutritional impact, and eventual need for surgery in children. METHODS: We retrospectively analyzed hospital records of UC patients (ages 1-19) seen at a pediatric gastroenterology clinic between January 2012 andAugust 2020. Patients were divided into groups: 1) medically without biologics or surgery; 2) patients treated with one biologic; and 3) patients treated with multiple biologics 4)patients that underwent colectomy. RESULTS: There were 115 UC patients with a mean follow-up of 5.9 ± 3.7 years (1 month-15.3 years). PUCAI score at diagnosis was mild in 52 patients (45%), moderate in 25 (21%), and severe in 5 (4.3%). PUCAI score for 33 patients (29%) could not be calculated. There were 48 (41.3%) in group 1 with 58% remission, 34 (29.6%) in group 2 with 71% remission, 24 (20.8%) in group 3 with 29% remission, and only 9 (7.8%) in group 4 with 100% remission. The majority (55%) of surgical patients had colectomy within the first year of diagnosis. BMI improved after surgery (P = 0.001). The change from one biologic to others did not improve nutrition over time. DISCUSSION: New biologics are changing the landscape in maintaining remission from UC. The current need for surgery is much lower than previously published studies. In medically refractive UC, nutritional status only improved after surgery. Addition of another biologic for medically refractory ulcerative colitis in order to avoid surgery must take into account the positive impact surgery has on nutrition and disease remission.

A Pharmacoeconomic Outlook of the Biological Drugs Marketed in India: A Cost Variance Analysis.

Introduction Biologic drugs are used to treat various illnesses like autoimmune diseases, cancers, hormonal irregularities, anemia, etc., and to prevent various diseases as vaccines. Though various biologic drugs are already available, they are still not within reach of the common man due to financial constraints. Through many search engines, studies evaluating the cost variation of different brands of biologics were investigated; however, only a few studies that address this problem were found. Hence, this study was planned with the objective of addressing the cost variation of various brands of biologic medicines available in the Indian market. Methods The website for the Current Index of Medical Specialties (CIMS) for India's location was used to obtain the prices of the different brands of biologic medicines in Indian National Rupee (INR) currency, which different manufacturers market with identical forms in strength and dosage. The percentage cost variation and cost ratio were calculated with the help of the minimum and maximum prices of various brands of biologic drugs. Results The prices of biologics belonging to six different classes that are available in 23 formulations were analyzed. The highest cost variability was shown by pegfilgrastim 6 mg at 1,022.92%, and the minimum-cost variation was shown by darbepoetin alfa 200 mcg at 13.07%. Conclusion Our research found a vast variance in the costs of various brands of biologic medicines in India. The government should address this cost variation problem by developing various policies, such as breaking the monopoly of manufacturers, providing tax incentives to nonprofit generic medicine manufacturers, and incorporating more biologic drugs under the protection of the Drugs Prices Control Order (DPCO).

Economics of Medical and Surgical Management of Chronic Rhinosinusitis with Nasal Polyps: A Contemporary Review.

BACKGROUND: Chronic rhinosinusitis (CRS) with nasal polyposis (CRSwNP) is a chronic inflammatory condition with significant patient morbidity and associated healthcare costs. While the economic burden of CRS overall has been previously described, the economic impact of CRSwNP has received less attention. Patients with CRSwNP have higher disease burden and healthcare resource utilization than those with CRS without nasal polyposis. Rapid evolution of medical management in recent years with the use of targeted biologics warrants further investigation into the economic burden of CRSwNP. OBJECTIVE: Provide an updated review of the literature on the economic impact of CRSwNP. METHODS: A literature review. RESULTS: Research shows that patients with CRSwNP have higher direct costs and usage of ambulatory services compared to matched non-CRS controls. Patients undergoing functional endoscopic sinus surgery (FESS) incur roughly $13,000 in costs which is particularly relevant given the rate of disease recidivism and need for revision surgery associated with CRSwNP. Disease burden additionally leads to indirect costs through loss of wages and productivity due to work absenteeism and presenteeism, with estimates of up to roughly $10,000 lost in mean annual productivity cost in refractory CRSwNP. Several studies have shown FESS to be more cost-effective in intermediate and long-term management than medical therapy with biologics, despite similar long-term outcomes with respect to quality-of-life metrics. CONCLUSION: CRSwNP is a chronic condition with high recurrence rates making it a challenge to manage over time. Current research suggests that FESS is more cost-effective than medical management, including use of newer biologics. Further investigation into both direct and indirect costs associated with medical management is warranted to perform accurate cost-effectiveness analyses and allow for the best allocation of limited healthcare resources.

The Pharmaceutical Industry in 2022: An Analysis of FDA Drug Approvals from the Perspective of Molecules.

While 2021 ended with the world engulfed in the COVID-19 Omicron wave, 2022 has ended in almost all countries, except China, with COVID-19 being likened to the flu. In this context, the U.S. Food and Drug Administration (FDA) has authorized only 37 new drugs this year compared to an average of 52 in the last four years. Thus 2022 is the second lowest harvest after 2016 in the last six years. This ranking may be transient and will be confirmed in the coming years. In this regard, the reduction in the number of drugs accepted by the FDA this year applies only to the so-called small molecules as there has been no variation in the respective numbers of biologics or TIDES (peptides and oligonucleotides). Monoclonal antibodies (mAbs) continue to be the class with the most drugs authorized (9), while proteins/enzymes (5) and an antibody-drug conjugate complete the biologics harvest. In 2022, five TIDES and seven drugs inspired by natural products have received the green light, thus showing the same tendency as in previous years. Finally, pharmaceutical agents with nitrogen aromatic heterocycles and/or fluorine atoms continue to be predominant among small molecules this year. Furthermore, three drugs have been approved for imaging, reinforcing the trend in recent years for this class of treatments. A keyword in 2022 is bispecificity since four drugs have this property (two mAbs, one protein, and one peptide). Herein, the 37 new drugs approved by the FDA in 2022 are analyzed. On the basis of chemical structure alone, these drugs are classified as the following: biologics (antibodies, antibody-drug conjugates, proteins/enzymes), TIDES (peptide and oligonucleotides), combined drugs, natural products; nitrogen aromatic heterocycles, fluorine-containing molecules, and other small molecules.

Economic Evaluation of Selected Interleukin Inhibitors Versus Methotrexate for Moderate-to-Severe Plaque Psoriasis From the Philippine Payer Perspective.

OBJECTIVES: We conducted an economic evaluation of interleukin inhibitors (ILIs) guselkumab, ixekizumab (IXE), secukinumab (SEC), and ustekinumab to a methotrexate (MTX) comparator for biologic-naive adult Filipino patients with moderate-to-severe chronic plaque psoriasis. METHODS: A 1-year decision tree and 5-year Markov model were used to estimate incremental cost-effectiveness ratios (ICERs) in Philippine pesos (PHP) per Psoriasis Area Severity Index improvement of at least 75%. For health technology assessment purposes, we also estimated the budget impact of subsidies for SEC to a Government of the Philippines (GoP) payer. Deterministic and probabilistic sensitivity analyses were performed. Data sources included global literature and local intervention prices. RESULTS: All ILIs were more effective but also more expensive than MTX. In the base case, only IXE and SEC were cost-effective treatments at a gross domestic product-benchmarked threshold, yielding ICERs of PHP468 098.01 and PHP483 525.32 per PASI responder, respectively. GUS and UST were less likely to be cost-effective throughout a range of simulated thresholds. ICERs were most responsive to discontinuation rates and drug prices. Full subsidy of SEC for 5 years would cost the GoP PHP1.83 billion more than a similar subsidy for MTX. CONCLUSIONS: ILIs were clearly more effective than MTX, but only IXE and SEC were potentially cost-effective for a GoP payer. Any case in which SEC is fully subsidized is more expensive to the GoP than the base case. This study was limited by a lack of country-specific effectiveness data, underestimation of comparator costs, exclusion of noncutaneous and quality-of-life effects, and indirect costs.

A real-world economic analysis of biologic therapies for psoriatic arthritis in Italy: results of the CHRONOS observational longitudinal study.

BACKGROUND: Psoriatic arthritis (PsA) is a chronic, immune-mediated, spondyloarthropathy characterised by musculoskeletal signs and symptoms with associated joint pain and tenderness. The average worldwide PsA prevalence is 133/100,000, while in the Italian population is 90-420/100,000. Traditionally, nonsteroidal anti-inflammatory drugs, glucocorticoid, and disease-modifying antirheumatic drugs have been used in the treatment of PsA. However, for those patients who are not adequately controlled with conventional therapies, the new biologics compounds represent a valid option. Biologic therapies have been shown to be more effective but also more expensive than conventional systemic treatments. Based on the CHRONOS study, the economic analyses presented in this paper aim to assess the annualised direct costs and the cost-per-responder of biologics in a real-world context assuming the Italian National Health System perspective. METHODS: The economic assessments were carried out on the overall cohort of patients, and on the tumour necrosis factor alpha inhibitors (TNFi) and the secukinumab subgroup, the most prescribed biologic therapies within the CHRONOS study. RESULTS: The annual economic impact of PsA in the overall group was €12,622, €11,725 in the secukinumab subgroup, and €12,791 in the TNFi subgroup. Biologics absorbed the main expenditure costs in the treatment of PsA accounting for about the 93% of total costs. At 6 months, secukinumab performed better in all the considered outcomes: cost-per-responder according to EULAR DAS28 and ACR50 response criteria were €12,661- €28,975, respectively, while they were €13,356 - €33,368 in the overall cohort and €13,138 - €35,166 in the TNFi subgroup. At 12 months secukinumab remained the subgroup with the lowest cost-per-responder ratio in EULAR DAS28 and ACR50 response criteria, while TNFi subgroup was the lowest one considered the ACR20. CONCLUSION: Despite some potential methodological limitations, our cost-per-response analysis provides physicians and payers additional insights which can complement the traditional risk-benefit profile assessment and drive treatment decisions.

Economic Burden of Comorbidities in Patients with Psoriasis in the USA.

INTRODUCTION: This study assessed the comorbidity burden, healthcare resource utilization (HCRU), and costs associated with patients with moderate to severe psoriasis (PsO) compared with a matched cohort of the general population without PsO in the USA. METHODS: Comorbidity-related HCRU (incidence rate ratios [IRRs]) and direct medical cost burden (per patient per month [PPPM] 2020 USD) in patients with moderate to severe PsO in the USA, previously apremilast- and biologic-naive, but currently treated, versus the general population were assessed through a retrospective cohort study using IBM (now Merative) MarketScan Commercial and Medicare Claims data (1 January 2006 to 31 December 2019). Comorbidities included cardiovascular, mental health, pulmonary, diabetes, hyperlipidemia, hypertension, peripheral vascular, liver, obesity, and other autoimmune disorders. RESULTS: There are increased all-cause HCRU and costs in patients with PsO compared with the general population. These differences (PsO-general population) in HCRU and costs (IRR visits; PPPM) are associated with specific comorbidities, including mental health (1.08; $372), chronic pulmonary disease (1.07; $135), diabetes (1.10; $159), hyperlipidemia (1.13; $203), hypertension (1.13; $305), liver disease (1.21; $360), and obesity (1.12; $145, all P < 0.01). CONCLUSIONS: Patients with PsO experience a higher economic burden of comorbidities than the general population despite using currently available systemic treatments for PsO.

Psoriasis is a disease that causes itchy and painful sores on the skin. People with psoriasis can develop several other diseases, known as comorbidities. These comorbidities include cardiovascular disease, depression, diabetes, hypertension, and obesity, and they pose a large economic burden to individuals, households, and society. Existing estimates of this burden are outdated because new treatments have become available for psoriasis. The aim of this study was to assess the economic burden of comorbidities in people with psoriasis compared with the economic burden in the general population in the USA. Healthcare claims reported in the IBM (now Merative) MarketScan Commercial and Medicare Claims database were used. This study assessed the total number of health-related visits to a doctor's office, hospital, or emergency department and the total costs of these visits in both groups. This study found that people with psoriasis had more health visits and costs because of comorbidities than the general population, despite using advanced treatments for their psoriasis.

Insurance delays in the approval of biologic medications for patients with psoriasis and psoriatic arthritis.

Biologics are the most effective treatment for moderate-to-severe psoriasis. Insurance approval and need for prior authorization continue to be a barrier to care for many patients with psoriasis and psoriatic arthritis. We sought to determine whether race/ethnicity, insurance type, and provider specialty affect biologic approval times. Records from the University of Miami Health System were reviewed, and 101 patients were included. Need for a prior authorization was significantly associated with long waits (p = 2.4 × 10-5). We did not identify a significant difference in wait times between non-Hispanic Whites and non-Whites. The average wait time for biologic approval for Whites was 29.7 days and for non-Whites was 27.2 days. Biologics were approved the same day for 23.7% of HMO carriers, 11.5% of PPO carriers, 63% of Medicare carriers, and 40% of Medicaid carriers (p < 0.001). There was no difference in the biologic type prescribed based on insurance type. Medicaid (p < 0.05) and the need for prior authorization (p = 2.4 × 10-5) significantly predicted approval wait time in our multilinear regression model. Patients with Medicare had the shortest wait time with a mean of 7.3 days. Medicaid patients waited a mean of 11.3 days. Private insurance patients waited the longest, regardless of whether they had a PPO (37 days) or HMO (41.3 days).

Equity in the usage of biologics for psoriasis in the working poor.

Biologic therapy often produces excellent outcomes for psoriasis; however, their high cost may create a barrier to appropriate usage, especially in the working poor population. This study defines working poor as income below 150% of the federal poverty level and holding or seeking work at least half a year. Our study aims to identify gaps in access to biologic therapy for psoriasis based on working poor status. This retrospective cross-sectional study was conducted utilizing data from the Medical Expenditure Panel Survey (MEPS) from 2007 to 2018. Patients were stratified into working poor (57,091), non-working poor (43,421), and non-poor (693,841) groups for analysis. In univariate analysis, WP (4.0%, ph p = 0.003) and NWP (2.8%, ph p = 0.006) were less likely to use biologics than NP (15.8%) (X2 p < 0.001). A binary logistic regression showed that WP vs. NP status (OR 0.27, p = 0.05), female vs. male sex (OR 0.55, p = 0.05), Black vs. White race (OR 0.14, p = 0.02), and Medicare vs. private insurance (OR 0.09, p = 0.03) had lower odds of using biologics. After correcting for age, sex, race, and insurance, WP confers an independent risk factor to lower biologic prescriptions. The high cost of biologics in the setting of financial barriers for some patients should be considered by physicians prescribing biologic therapy for psoriasis.

Extension of mepolizumab injection intervals as potential of saving costs in well controlled patients with severe eosinophilic asthma.

Background: Present guidelines recommend a life-long therapy with mepolizumab in patients suffering from severe eosinophilic asthma as several studies proved the disadvantages of treatment cessation. This study evaluated the possibility of extending the dosage intervals of mepolizumab in those patients with severe eosinophilic asthma after being well controlled. Methods: Eighteen patients diagnosed with severe eosinophilic asthma were started on treatment with mepolizumab in regular 4-week intervals. Symptom control was measured using the asthma control test (ACT) and pulmonary function test every 3 months. The amount of oral corticosteroids needed to maintain symptom control was monitored at every visit. After achieving good symptom control, defined as well controlled ACT ≥20, injection intervals were prolonged from 4 up to 6 to 8 weeks. The evaluation of this data was approved by the ethics committee. Results: ACT and pulmonary function values significantly improved after initiating therapy with mepolizumab on a regular 4-weekly injection interval. After extending the dosage intervals, both ACT and pulmonary function remained on a stable level without significant changes during the follow-up visits for 1 year. Median dosage of prednisolone declined significantly in the studied group under mepolizumab therapy and stayed on a low level during the follow-up visits with only a single patient using prednisolone after 1 year. Conclusion: In patients with fully or well controlled eosinophilic asthma treated with mepolizumab extending the dosage intervals between the injections up to 8 weeks bears the potential to save costs for the health care system.

Estimating the impact of biosimilar entry on prices and expenditures in rheumatoid arthritis: a case study of targeted immune modulators.

OBJECTIVE: To model changes in prices, utilization, and expenditures of targeted immune modulators (TIMs) for rheumatoid arthritis, accounting for biosimilar entry. METHODS: Using IQVIA National Sales Perspective data between 2013 and 2019, we examined sales and expenditures of biologics and non-biological complex molecules, 20 quarters before and after patent exclusivity milestones. We estimated the impact of a molecule's exclusivity milestones and biosimilar entry on prices, using a regression discontinuity design (RDD). We then combined the RDD estimate with historical trends to assess the impact of adalimumab's exclusivity milestones on future TIM expenditures. RESULTS: Changes in average molecule prices were associated largely with biosimilar uptake. For molecules with relatively high biosimilar uptake (>60%), prices fell considerably (-21.2% to -59.3%) one year after exclusivity milestones, whereas molecules with lower biosimilar uptake (<10%) experienced smaller price decreases (-2.4% to -8.4%). Average price reduction at the molecule level after biosimilar entry was not significant (-18.6%; p = .657). When applying the RDD results after adalimumab's exclusivity milestones, its projected share of total TIM market expenditures decreased from 48.0% in 2019 to 26.0% in 2025, whereas expenditures on Janus kinase inhibitors increased from 4.0% to 34.0%. CONCLUSIONS: Biologics facing biosimilar competition may experience price decreases, potentially offering substantial savings to payers, patients, and society, although the magnitude of these estimates depends on biosimilar uptake. Formulary placement, along with manufacturer-payer dynamics, may also play a role in determining the impact on price and market uptake of biosimilars.

Social, political, and economic determinants of access to biologics: A scoping review of structural determinants in the clinical disparities literature.

BACKGROUND: The number of biologics among new medication approvals is increasing. Social, political, and economic factors influence access to these expensive medications. Disparities in access to new medications can exacerbate health disparities. The notion of "structural determinants" provides a theoretical framework for broadly evaluating the integration of upstream social, political, and economic determinants in the clinical study of access. OBJECTIVE: To review the literature on access to FDA approved biologic medications with particular focus on the integration of social, political, and economic determinants into study design and interpretation. METHODS: We used PRISMA guidelines to review studies on racial and socioeconomic disparities in biologic access through August 2020. We assessed whether the design or interpretation of studies considered key economic determinants of access: the biologics supply chain, trade agreements, patents, drug research and development, insurance reimbursement, and non-insurance drug policies. RESULTS: 100 studies met our inclusion criteria. Sixty-six studies considered insurance reimbursement, but trade law, patents, and other key economic determinants were rarely considered. The literature focuses on a small number of older biologics. CONCLUSIONS: A small number of studies model the integration of structural determinants into clinical research on access to biologics, but overall this literature has many limitations and lacks integration of structural determinants. Increased interdisciplinary collaboration, availability of manufacturer data, and use of disease registries can help create structurally grounded understandings of the relationship between the political economy of expensive medications and clinical disparities.

Assessment of Real-World Escalation to Biologics in US Patients With Asthma.

BACKGROUND: Previous studies indicate that suboptimal medication adherence may contribute to uncontrolled asthma. Global Initiative for Asthma (GINA) guidelines recommend treatment escalation to biologics for patients with uncontrolled asthma despite adherence to high-dose maintenance medication and who have eosinophilic/allergic biomarkers or require maintenance oral corticosteroids. OBJECTIVE: This study aimed to describe the clinical status of patients with asthma escalated to biologic therapy. METHODS: This retrospective claims database analysis enrolled US patients with asthma who were escalated to biologics between January 2016 and June 2020. Exacerbations, control status, GINA step, and maintenance medication adherence during the 12 months before biologic therapy initiation were analyzed. Asthma control was assessed using both the European Respiratory Society/American Thoracic Society (ERS/ATS) and Stempel criteria. Adherence was defined as the proportion of days covered (PDC) by maintenance medication claims. RESULTS: Of 1786 patients escalated to biologics, 506 were included for analysis. During the 12 months before escalation, 346 patients had confirmed exacerbations. Uncontrolled asthma status was estimated in 55% and 70% of patients (ERS/ATS and Stempel criteria, respectively). GINA step was inferred for 395 patients: 154 were at step 2, 11 at step 3, 104 at step 4, and 126 at step 5. Of 403 patients with maintenance medication claims, 63% had suboptimal maintenance medication adherence (PDC <80%). CONCLUSION: In this study, most patients initiating biologic therapy had mild-to-moderate asthma or suboptimal maintenance medication adherence, possibly indicating inappropriate escalation. Incorporating objective medication adherence monitoring into existing guidelines may reduce inappropriate escalation to biologics.

[Practice of Human Resource Development Related to Biopharmaceutical Manufacturing and Quality Control and the International Contribution as Centers of Excellence of Asia-Pacific Economic Cooperation-Life Sciences Innovation Forum-Regulatory Harmonization Steering Committee].

In recent years, the development of biologics such as therapeutic antibodies has increased. Around 1980, Japan was the world's leading country in terms of research and development on biologics. However, after that, the development of biologics in Japan has become stagnant. Especially, research and development of therapeutic antibodies, which are critical medical products, is compared to that being conducted in Europe and the United States. The products distributed in Japan are developed and manufactured overseas. Furthermore, related emerging technologies are developed overseas. Therefore, to improve this situation, Biologics Center for Research and Training (BCRET) was established in August 2017 in Kobe city to develop human resources for biologics with the cooperation of Japan Pharmaceutical Manufacturers Association, Japan Agency for Medical Research and Development, Kobe University, and related ministries and agencies. BCRET will develop human resources by providing useful information and experience related to the development and manufacture of biologics in the form of classroom lectures and practical training for process and analysis developers and pharmaceutical affairs related to manufacturing, quality control, and approval applications in the biologics field of pharmaceutical companies, along with reviewers and inspectors of Pharmaceuticals and Medical Devices Agency and inspectors from countries belonging to Asia-Pacific Economic Cooperation (APEC). This is the first attempt in the world to develop human resources in a field of chemistry, manufacturing and control and Good Manufacturing Practice of biologics for inspectors in countries belonging to APEC, and it is expected to contribute internationally for human resources for biologics.

Clinical Characteristics and Disease Burden of Severe Asthma According to Oral Corticosteroid Dependence: Real-World Assessment From the Korean Severe Asthma Registry (KoSAR).

PURPOSE: Oral corticosteroids (OCS) are commonly used in patients with severe asthma, but they are associated with several adverse events. We estimated the prevalence of patients with OCS-dependent asthma in a large nationwide registry for severe asthma and delineated their clinical characteristics. METHODS: This cross-sectional study analyzed enrollment data of the patients recruited in the Korean Severe Asthma Registry (KoSAR) from 2010 to 2019. The clinical characteristics of patients were compared according to OCS dependency, which was defined as maintenance OCS treatment lasting at least 6 months during the 12 months prior to enrollment. RESULTS: Among the 562 patients with severe asthma, 121 (21.5%) patients were defined as having OCS-dependent asthma. Compared with the OCS-independent group, the OCS-dependent group was older at symptom onset and had a higher prevalence of anxiety, worse lung function, and used more medication than the control group. Despite the higher doses of daily ICS and 6-month cumulative OCS, the OCS-dependent group reported greater consumption of relievers and a higher prevalence of unscheduled emergency room visits and repeated OCS bursts. Although anti-interleukin-5 was more commonly prescribed for patients with OCS-dependent asthma, only a limited proportion of patients with severe asthma received biologics. CONCLUSIONS: One-fifth of patients with severe asthma had OCS-dependency, which was associated with a greater disease burden compared to those with OCS-independent asthma. Active intervention including initiation of biologics and regular assessment of OCS-induced morbidities is warranted to reduce the use of OCS and its potential adverse effects.

Real-world single-centre experience of rheumatoid arthritis patients requiring four or more higher cost drugs: response and duration of treatment.

The cost-effectiveness of higher cost drugs (HCDs) after several failures is disputed by some purchasers of services for people with rheumatoid arthritis (RA). We were interested to explore our service experience of using HCDs beyond the third choice to document response rates and duration of treatments. METHOD: Records from our multi-disciplinary team meeting (MDT) that is used to decide on the use of HCDs were used to identify all RA patients who had been exposed to four or more HCDs. Notes were scrutinised for sequence of treatments, duration and response to treatments and reasons for stopping at each choice point. RESULTS: From a total of 2648 RA patients in our service, 49 (< 2%) had been exposed to four or more HCDs. Response rates based on descriptive assessments for fourth to sixth choices were between 50 and 55% as well as some partial responders. There were responders and failures to all drugs at every choice point. Patients who had responded to one drug were more likely to respond to the next. Patients often responded to drugs for approximately 2 years. Only four patients had stopped looking for the next HCD. CONCLUSION: Patients often respond to late choice HCDs. There are responders and failures at each time point and they are difficult to predict. There is no justification for restricting the number of HCDs that can be tried for RA. Key Points • Less than 2% of our RA patients required 4 or more higher cost drugs. • Fourth to sixth choice drugs still worked in 50 to 55% of patients. • There is no justification for CCGs restricting the number of drugs that can be tried.

Adalimumab in Pediatric Inflammatory Bowel Disease.

The introduction of biological agents with strong anti-inflammatory action, such as antitumor necrosis factor (TNF) agents, has changed inflammatory bowel disease (IBD) treatment strategy and goals, and has contributed significantly to improve the long-term prognosis of patients. Moreover, several biological agents are being used or researched in pediatric populations. However, only two biological agents, infliximab (IFX) and adalimumab (ADL), are currently approved for children and adolescents. In pediatric IBD, there are limitations and burdens associated with facilitating mucosal healing (MH) when utilizing these two biological agents. ADL is effective in both naïve patients and those with previous experience with biologics. Beyond clinical remission, this drug is also effective for MH and histological remission. The use of therapeutic drug monitoring to further enhance the effectiveness of ADL treatment can be expected to reduce treatment failure of ADL and pave the way for appropriate treatment in the treat-to-target era. This review paper focuses on ADL, examine studies conducted in children, and determine the role this agent plays against pediatric inflammatory bowel disease.

Factors Related to Biologic Adherence and Outcomes Among Moderate-to-Severe Asthma Patients.

BACKGROUND: Adherence barriers to asthma biologics may not be uniform across administration settings for patients with moderate-to-severe asthma. OBJECTIVE: To examine differences in asthma biologic adherence and associated factors, as well as association with a 1-year all-cause emergency department (ED) visit, across administration settings. METHODS: A retrospective study of biologic naïve moderate-to-severe asthma patients with initial biologic therapy between January 1, 2016, and April 30, 2020, in the Optum Clinformatics Data Mart was performed. Three administration settings were identified: Clinic-only (outpatient office/infusion center), Home (self-administration), and Hybrid setting (mixture of clinic and self-administration). Asthma biologic adherence was the proportion of observed over expected biologic dose administrations received within 6 months from initial therapy. Factors associated with adherence were identified by administration setting, using Poisson regression analyses. A relationship between a 1-year all-cause ED visit and adherence was assessed for each administration setting using Cox regression analyses. RESULTS: The study cohort was 3932 patients. Biologics adherence was 0.75 [0.5, 1] in Clinic setting, the most common administration setting, and 0.83 [0.5, 1] in both Home and Hybrid settings. Specialist access was consistently associated with better biologic adherence, whereas Black race, Hispanic ethnicity, lower education, Medicare only insurance, and higher patient out-of-pocket cost were associated with worse biologic adherence in some settings. In the Hybrid setting, hazard for a 1-year all-cause ED visit decreased with biologic adherence. CONCLUSIONS: Asthma biologic adherence varied by administration setting. Efforts to improve asthma biologic adherence should consider promoting self-administration when beneficial, improving prior specialist access, and targeting patients with higher risk of suboptimal adherence particularly Black and Hispanic patients.

The Role of Access and Cost-Effectiveness in Managing Asthma: A Systematic Review.

BACKGROUND: Inconsistent and unequal access to medical care is an issue that predates the COVID19 pandemic, which only worsened the problem. Limited access to care from asthma specialists and other specialists treating comorbid diseases may adversely affect asthma. OBJECTIVE: The purpose of this review is to identify health disparities associated with access to care for asthma, and cost-effectiveness of therapies and interventions addressing this health disparity. METHODS: A narrative systematic review was undertaken using MeSH searches of English language articles published in CINAHL, Scopus, or PubMed. RESULTS: A total of 725 articles were identified. Barriers recognized from the literature included access to diagnostic spirometry, access to specialists, medication formulary restrictions, and issues leading to medical nonadherence. Telemedicine, school-based health care interventions, digital applications, and non-office-based digital spirometry could be used to address these gaps in access to asthma care while potentially being cost-effective. CONCLUSION: With the widespread adoption of telemedicine because of the pandemic, and adoption of other mobile services, we now have potential tools that can increase access to asthma care, which can help address this health care inequity. Evidence is limited, but favorable, that some of these tools may be cost-effective.