Drivers of physician decision-making and patient perspectives across lines of therapy in multiple myeloma in the USA.

Aim: To explore treatment selection for relapsed/refractory multiple myeloma (RRMM), which remains complex due to heterogeneity of available treatments and lack of defined standard of care. Patients & methods: The Adelphi Real World MM Disease Specific Programme surveyed physicians in the USA and their patients with MM to collect real-world data on patterns and perceptions of MM treatment across lines of therapy (LOT). Results: Triplets were the most common regimens across each LOT. Physicians reported efficacy-related factors, health insurance coverage, and clinical guidelines as key determinants of treatment choice regardless of LOT. Patients identified better quality of life as the most important treatment benefit. Conclusion: The DSP RW data highlight drivers of RRMM treatment choice from physicians' and patients' perspectives and need for a more holistic approach to guidelines and clinical trials that encompasses patient perspectives.

Multiple myeloma (MM) is an incurable cancer of white blood cells. Treatments tend to become less effective when taken for long periods of time, meaning that patients often receive several different treatments over the course of their disease. Many different drugs and drug combinations are now available for patients with MM. As there is no standardized approach for the treatment of MM, it can be challenging for physicians to choose between the various complex treatment options for their patients, and the key factors that influence physicians' treatment choices remain unclear. In this study, we used information from a survey of physicians and their patients with MM in the USA to investigate which treatments were used most commonly and how physicians and patients made decisions about their treatment. We found that patients with MM were typically treated with a combination of three drugs which differed between patients. When deciding which treatment to prescribe to their patients, physicians primarily considered factors related to how well a treatment works and, in-turn, prolong a patient's life, but this was also considered by physicians to be an area for improvement. While patients also favored treatments associated with a longer life, they more commonly favored those associated with a better quality of life. These findings show that quality of life is important to patients receiving treatment for MM and should be taken into account by physicians when choosing a treatment for their patients.

Real-world patient-reported outcomes and concordance between patient and physician reporting of side effects across lines of therapy in multiple myeloma within the USA.

PURPOSE: We aimed to explore patient-reported outcomes (PROs) and patient and physician concordance of side effects perception across lines of therapy (LOT) in multiple myeloma (MM) within the United States of America (USA). METHODS: Data were drawn from the Adelphi Real World MM III Disease Specific Programme™, a point-in-time survey of hemato-oncologists/hematologists and their patients with MM conducted in the USA between August 2020 and July 2021. Physicians reported patient characteristics and side effects. Patients reported side-effect bother and health-related quality of life (HRQoL) using validated PRO tools (European Organisation for the Research and Treatment of Cancer Quality of Life Core Questionnaire/-MM Module [EORTC QLQ-C30/-MY20], EQ-5D-3L and Functional Assessment of Cancer Therapy-General Population physical item 5). Descriptive, linear regression and concordance analyses were performed. RESULTS: Records from 63 physicians and 132 patients with MM were analyzed. EORTC QLQ-C30/-MY20 and EQ-5D-3L scores were consistent across LOTs. Scores tended to be worse with higher side-effect bother; patients "very much" bothered by side effects had lower median (interquartile range) global health status scores (33.3 [25.0-50.0]) than those "not at all" bothered (79.2 [66.7-83.3]). Patient and physician concordance on side-effect reporting was poor to fair. Patients frequently reported fatigue and nausea as bothersome side effects. CONCLUSION: HRQoL of patients with MM was worse with greater side-effect bother. Discordant patient and physician reporting of side effects indicated a need for improved communication during management of MM.

Physician treatment preferences for relapsed/refractory multiple myeloma: a discrete choice experiment.

Aim: This study aimed to assess physician preferences for later lines (third to fifth) of therapy in patients with relapsed/refractory multiple myeloma (RRMM) in the USA. Materials & methods: Factors relevant to physicians' treatment preferences for RRMM were identified from a literature search and refined in a qualitative phase. Preferences were quantitatively assessed using a discrete choice experiment. Physicians (n = 227) made choices regarding treatment scenarios for RRMM. Results: Efficacy had the highest mean relative importance, with overall survival valued as most important when making treatment decisions for patients with RRMM. Reduced incidences of keratopathy and thrombocytopenia had similar relative importance in later-line treatment. Conclusion: Greater understanding of physicians' criteria for clinical decision-making may help inform wider adoption of new treatments.

When deciding which treatment patients with relapsed or refractory multiple myeloma should receive, physicians have to weigh the benefits of each treatment against the risk of side effects. This study required physicians to complete a survey on aspects involved in their treatment decisions and identified those of highest importance. Physicians chose patient survival as the most important factor and minimization of side effects as less important considerations. Reducing patients' risk of developing corneal conditions or low platelet (a type of blood cell) count were of equal importance to doctors. Understanding physicians' treatment preferences and the reasons behind them will help identify gaps in education about new therapies as they become available.

Prevalence of rheumatoid arthritis in the United States adult population in healthcare claims databases, 2004-2014.

This study aimed to determine the prevalence of rheumatoid arthritis in the United States (US) adult insured population from 2004 to 2014. This was an observational, retrospective, cross-sectional study based on US administrative health insurance claims databases (Truven Health MarketScan® Research database and IMS PharMetrics Plus database). Trends in RA prevalence focusing on the 10-year period covering January 1, 2004-December 31, 2014 were analyzed using a validated algorithm for the identification of RA. Prevalence rates in the databases were determined and age- and gender-adjusted rates were projected to the US population in 2014. Analysis of data from the two databases indicated that the RA prevalence rate in commercially insured adult US population ranged from 0.41 to 0.54% from 2004 to 2014. The prevalence varied substantially by gender and age in each year and increased gradually across the years for most subgroups. In 2014, out of 31,316,902 adult patients with continuous enrollment in the Truven Health MarketScan® Research database, 157,634 (0.50%) patients met our criteria for RA. Similarly, out of 35,083,356 adult patients in the IMS PharMetrics Plus database, 139,300 (0.50%) patients met our criteria for RA. In 2014, the overall age-adjusted prevalence of RA ranged from 0.53 to 0.55% (0.29-0.31% for males and 0.73-0.78% for females). The prevalence of RA in the US appeared to increase during the period from 2004 to 2014, affecting a conservative estimate of 1.28-1.36 million adults in 2014.

"A Bayesian sensitivity analysis to evaluate the impact of unmeasured confounding with external data: a real world comparative effectiveness study in osteoporosis".

PURPOSE: Observational studies are frequently used to assess the effectiveness of medical interventions in routine clinical practice. However, the use of observational data for comparative effectiveness is challenged by selection bias and the potential of unmeasured confounding. This is especially problematic for analyses using a health care administrative database, in which key clinical measures are often not available. This paper provides an approach to conducting a sensitivity analyses to investigate the impact of unmeasured confounding in observational studies. METHODS: In a real world osteoporosis comparative effectiveness study, the bone mineral density (BMD) score, an important predictor of fracture risk and a factor in the selection of osteoporosis treatments, is unavailable in the data base and lack of baseline BMD could potentially lead to significant selection bias. We implemented Bayesian twin-regression models, which simultaneously model both the observed outcome and the unobserved unmeasured confounder, using information from external sources. A sensitivity analysis was also conducted to assess the robustness of our conclusions to changes in such external data. RESULTS: The use of Bayesian modeling in this study suggests that the lack of baseline BMD did have a strong impact on the analysis, reversing the direction of the estimated effect (odds ratio of fracture incidence at 24 months: 0.40 vs. 1.36, with/without adjusting for unmeasured baseline BMD). CONCLUSIONS: The Bayesian twin-regression models provide a flexible sensitivity analysis tool to quantitatively assess the impact of unmeasured confounding in observational studies. Copyright © 2016 John Wiley & Sons, Ltd.

Health care resource utilization and costs among patients with multiple myeloma with exposure to double-class or triple-class multiple myeloma treatments: A retrospective US claims database analysis.

BACKGROUND: Despite recent advancements in the therapeutic landscape, multiple myeloma (MM) remains incurable. There are multiple treatment options available with a novel mechanism of action, but there is limited evidence describing the economic burden among patients with MM exposed to different drug classes and combinations and across different health care settings. OBJECTIVE: To describe all-cause and MM-related health care resource utilization (HCRU) and costs among patients with MM exposed to different drug classes and combinations (ie, double-class and triple-class-exposed) and characterize the economic burden in different health care settings among these patients with MM. METHODS: We conducted a retrospective cohort study using the IBM MarketScan databases. The study included adult patients (aged ≥18 years) diagnosed with MM between December 1, 2015, and December 31, 2019. The study sample comprised double-class-exposed (DCE) and triple-class-exposed (TCE) cohorts, categorized based on their earliest exposure to different combinations of immunomodulatory drugs, proteasome inhibitors, or targeted monoclonal antibody. Patients with at least 1 subsequent line of therapy following the categorization were included, and the start date of the first subsequent line of therapy was the index date. The primary outcomes were all-cause and MM-related HCRU and costs during the follow-up period. Costs were stratified across 8 care settings defined by place of service. The Kaplan-Meier sample average technique was used to estimate the cumulative mean outcomes, accounting for differential follow-up periods. The outcomes were reported as per patient per month (PPPM). 18 years) diagnosed with MM between December 1, 2015, and December 31, 2019. The study sample comprised double-class-exposed (DCE) and triple-class-exposed (TCE) cohorts, categorized based on their earliest exposure to different combinations of immunomodulatory drugs, proteasome inhibitors, or targeted monoclonal antibody. Patients with at least 1 subsequent line of therapy following the categorization were included, and the start date of the first subsequent line of therapy was the index date. The primary outcomes were all-cause and MM-related HCRU and costs during the follow-up period. Costs were stratified across 8 care settings defined by place of service. The Kaplan-Meier sample average technique was used to estimate the cumulative mean outcomes, accounting for differential follow-up periods. The outcomes were reported as per patient per month (PPPM). RESULTS: The study included 1,521 patients with MM, of whom 1,016 (66.8%) were DCE and 505 (33.2%) were TCE. The mean total all-cause health care costs were $20,338 PPPM, and approximately 85% of the total all-cause costs were MM-related. The mean all-cause and MM-related total costs were driven by overall drug costs primarily attributed to MM treatment and administration costs. The TCE cohort was associated with more HCRU and incurred higher costs than the DCE cohort across all categories. The hospital-based ambulatory setting had the highest all-cause and MM-related costs during the follow-up period: $7,302 (95% CI = $6,801-$7,784) PPPM and $6,695 (95% CI = $6,239-$7,136) PPPM, respectively. CONCLUSIONS: The study findings suggest that the economic burden following exposure to multiple drug classes and combinations is substantial, especially among the TCE cohort and in the ambulatory setting. These findings highlight the need for more effective treatments that can mitigate the economic burden of patients with MM. Future research on the HCRU and costs related to recently approved MM treatments with novel mechanisms is warranted. DISCLOSURES: At the time of this study, Dr Yang was a postdoctoral fellow and the fellowship was supported by GSK. Dr Boytsov is a full-time employee of GSK. Dr Carlson discloses consulting fees from Pfizer, AbbVie, and Genentech. Dr Barthold reports no disclosures.

Treatment patterns and overall survival of patients with double-class and triple-class refractory multiple myeloma: a US electronic health record database study.

Patients with relapsed/refractory multiple myeloma (RRMM) resistant to multiple drug classes remain a high unmet need population. This longitudinal retrospective cohort study assessed real-world treatment patterns and outcomes in adults with RRMM. Patients who had three or more prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (double-exposed) were further categorized as refractory to a PI and an immunomodulatory agent (double-class refractory, n = 381) or additionally to an anti-CD38 monoclonal antibody (triple-class refractory, n = 173). Treatment options are limited for patients with double-class or triple-class refractory disease. Retreatment is a part of standard of care. Bortezomib and lenalidomide had the highest retreatment rates among double-class and triple-class refractory patients. Survival outcomes remain poor among RRMM patients with median overall survival (OS) of 22.3 and 11.6 months for double-class refractory and triple-class refractory patients, respectively. This study highlights the need for novel efficacious therapies in this heavily pretreated RRMM population.

Prescription Market Share and Treatment Patterns in Atopic Dermatitis: A Retrospective Observational Study Using US Insurance Claims.

INTRODUCTION: There is limited real-world evidence on the treatments for atopic dermatitis (AD) since dupilumab was approved in 2017. The objective of this study was to assess market share of drugs commonly prescribed for the treatment of AD and describe treatment patterns in patients diagnosed with AD. METHODS: This was a retrospective, observational study in adult patients with an ICD-10 diagnosis of AD between 2017 and 2019 using insurance claims data in the US population. RESULTS: Market share cohorts consisted of 75,794 (2017) and 89,618 (2018) patients. Treatment patterns cohort had 68,588 patients with 63.56% female, mean (SD) age 43.54 (15.96) years, and mean (SD) Quan CCI 0.31 (0.85). Topicals had two-thirds market share by prescription volume (2017 = 65.56%; 2018 = 63.63%). Corticosteroids were the most prescribed topical (2017 = 71.94%; 2018 = 72.04%) and systemic (2017 = 30.59%; 2018 = 30.23%) drug class. Dupilumab had the highest medication adherence (proportion of days covered [PDC] ≥ 80%; 60.74%) and persistence (17.39%), lowest discontinuation rate (23.32%), and longest mean (SD) days on therapy 148.20 (101.77). CONCLUSION: Topicals are the primary treatment for patients with AD, even though systemic users have higher medication adherence (PDC). Systemics provide a treatment alternative to topicals.

The current treatment landscape in adult atopic dermatitis in the United States: results from a cross-sectional real-world study.

BACKGROUND: This study describes the current treatment landscape in adult atopic dermatitis (AD), overall and by disease severity. METHODS: Adult patients with an AD diagnosis in dermatology-specific electronic medical records during 2018 were identified and linked to an administrative claims database. Disease severity was determined using Physician's Global Assessment (PGA). Written and dispensed prescriptions, within and between class cycling for AD therapies occurring in 2018 were assessed. RESULTS: In total, 4,364 patients were included. Among patients with available PGA, 43.2% had clear-to-mild, 37.3% had moderate, and 19.6% had severe disease. Most patients (71.0%) had written prescriptions for topical therapies only in 2018. Among the patients with claims for topical therapies alone, 80.7% used topical corticosteroids only. Within and between class cycling was observed in 33.7% and 12.8% of topical users, respectively. In patients with systemic therapy (40.6%), nearly 84.9% also used topical therapy, 25.8% cycled within systemic drug classes, and 24.8% cycled between systemic drug classes. Overall, cycling was more prevalent in patients with more severe disease. CONCLUSION: Cycling within and between both topical and systemic drug classes was more common in patients with more severe disease, indicating difficulty of managing these patients and highlighting a need for more treatment options.

Inadequate response and treatment patterns in adults diagnosed with atopic dermatitis and treated with topical therapy.

BACKGROUND: Treatment for atopic dermatitis (AD) is complex, particularly in patients with inadequate response to topical therapies. Currently, there is little clinical guidance for the treatment of these patients. METHODS: A real-world retrospective study utilizing electronic medical records (EMR) and administrative claims data selected patients with AD between January 01 2016 and June 30 2018. Patients had a written prescription for a topical therapy (first observed script = index date) and no prior systemic treatment. Disease severity at index, follow-up treatment response and prescriptions patterns were assessed. A subset of patients linked to claims was evaluated for treatment patterns. RESULTS: We identified 137,214 adult topical-treated AD patients with no prior systemic therapy. Among the 16,035 patients with available Physician Global Assessment (PGA) at index, 8169 (50.9%) had the moderate-to-severe disease. Among these patients, 60% had an inadequate response to topical therapy. Of 4475 patients linked to claims, 13.0% had claims for systemic therapy during follow-up, most initiated systemic steroids (95.2%), and oral immunosuppressants and biologics were initiated in 3.3% and 3.8%, respectively. CONCLUSION: In this real-world study, inadequate response to topical therapy among moderate-to-severe AD patients was high and initiation of systemic treatment was low which suggests a need for additional AD-indicated systemic treatment options in this patient population.

US health care utilization and costs in adult patients with atopic dermatitis by disease severity.

BACKGROUND: Although previous studies have reported the economic burden of atopic dermatitis (AD) in adults, updates are needed using more current data and measure of disease severity. OBJECTIVE: To describe the health care resource utilization (HCRU) and associated costs in US adults diagnosed with AD overall and by disease severity. METHODS: This real-world retrospective study identified adults aged at least 18 years who received a clinical diagnosis of AD in a dermatology electronic medical record (EMR) database between 2016 and 2018 (first record = index date), which was linked to an administrative claims database. Patients were required to have an AD diagnostic code and at least 6 months of continuous enrollment in medical and pharmacy benefits before and after the index date. Baseline severity was assessed using the Physician Global Assessment score closest to the index date. Inpatient and outpatient services, visits to specialists and its seasonality, treatment use, and associated annual direct health care costs were reported using descriptive statistics. RESULTS: Annual all-cause direct health care costs were $10,474 per patient per year and primarily driven by outpatient visits and pharmacy use. Compared with patients with clear to mild disease, more AD patients with severe disease had at least 1 dermatology (73.0% vs 58.5%) and allergy/immunology office visit (16.0% vs 5.5%) and AD-related medications (90.0% vs 64.3%). All-cause total annual costs in patients with severe disease ($23,242) were significantly higher than in patients with clear to mild disease ($8,936; P = 0.0002). Little seasonal variation in dermatology office visits was observed. CONCLUSIONS: Significant economic burden primarily driven by outpatient and pharmacy utilization was observed in AD patients, which increased with disease severity. DISCLOSURES: This work was sponsored by Eli Lilly and Company. Gorritz and Wade are employees of IQVIA, which was contracted by Eli Lilly and Company to conduct this study and develop the manuscript. Wang was employed by IQVIA at the time of this study. Malatestinic and Goldblum are employees and stockholders of Eli Lilly and Company. Boytsov was an employee of Eli Lilly at the time of this research.

The direct and indirect costs of adult atopic dermatitis.

BACKGROUND: Atopic dermatitis is considered a childhood illness, and the direct and indirect health care burden of atopic dermatitis in adults is not fully understood. OBJECTIVE: To measure the direct and indirect costs of atopic dermatitis among adults in 2018. METHODS: This retrospective cohort study compared commercial and Medicare-insured adults with atopic dermatitis in 2018 with directly matched (1:3) adults without atopic dermatitis. Atopic dermatitis prevalence was reported. Health care utilization, direct health care costs, and work loss data were compared between cohorts. This analysis was repeated for adults with atopic dermatitis in 2016 and 2017. RESULTS: 31,164 adults with atopic dermatitis in 2018 were identified and directly matched (1:3) to controls. Adults with atopic dermatitis had greater utilization of outpatient services, outpatient pharmacy services, and short-term disability benefits than controls. Unadjusted annual health care costs in 2018 were $4,979 higher for adults with atopic dermatitis ($14,603) than for the matched controls ($9,624), driven by outpatient services and pharmacy. Findings were supported by analyses of adults from 2016 and 2017 and multivariable analyses. One limitation of this study was that patients with mild cases of atopic dermatitis may not seek medical treatment and may be underrepresented in the study cohort. CONCLUSIONS: The direct health care and indirect (short-term disability) health care costs of atopic dermatitis present a significant health care burden among the adult population. DISCLOSURES: This study was funded by Eli Lilly and Company. Employees of Eli Lilly were involved in the planning, execution, and interpretation of the study. Pierce is employed by Eli Lilly and Company. Boytsov and Goldblum were employed by Eli Lilly and Company Health at the time this research was conducted. Manjelievskaia and Brouillette are employed by IBM Watson Health, which received funding from Eli Lilly and Company to conduct this study. Bonafede and Onyekwere were employed at IBM Watson Health at the time this research was conducted.

Probabilistic Linkage of Randomized Controlled Trial Data to Administrative Claims: A Case Study of Patients from Baricitinib Clinical Trials.

INTRODUCTION: The aim of this work is to assess the feasibility of probabilistically linking randomized controlled trial (RCT) data to claims data in a real-world setting to inform future rheumatoid arthritis (RA) research. METHODS: This retrospective cohort study utilized IQVIA's Patient Centric Medical Claims (Dx) Database, IQVIA's Longitudinal Prescription Claims (LRx) Database, and Lilly's baricitinib RCT data from a sample of patients that consented to the linkage of their de-identified insurance claims to their de-identified RCT data. Patients were initially matched on age, gender, and three-digit ZIP code of the provider and further matched according to a point scoring system using additional clinical variables. RESULTS: A total of 245 patients from 49 US clinical trial sites were eligible for the study and 78 (31.8%) of these patients consented to participate. Of the 78 consented patients, 69 (88%) were successfully matched on age, gender, and three-digit ZIP code of the provider. Of the 69 patients successfully matched on age, gender, and three-digit ZIP code of the provider, 44 (63.8%) had at least one sufficient match using the point scoring system. Of these 44, 23 (52.3%) patients matched at a ratio of one RCT patient to one Dx/LRx patient, 11 (25.0%) at a ratio of 1:2, 7 (15.9%) at a ratio of 1:3 and three (6.8%) at a ratio of 1:4 or greater. To further improve match ratios, a variable hierarchy was applied to the 18 RCT patients with 2-3 matches. Overall, 38 of the 78 (48.7%) consented RCT patients were successfully matched 1:1 to claims database patients. CONCLUSIONS: This probabilistic linkage methodology demonstrates the feasibility, at a moderate linkage rate, of linking patients from RCTs to real-world data, which can provide a means to assess additional information not usually collected within or following a clinical trial.

A Qualitative Analysis of Provider Notes of Atopic Dermatitis-Related Visits Using Natural Language Processing Methods.

INTRODUCTION: Real-world disease management of atopic dermatitis (AD) is hampered by a lack of consistency between providers that treat AD regarding assessment of severity, disease activity, and quality of life. Variability and inconsistency in documentation makes it difficult to understand the impact of AD. This study summarizes AD-related symptoms and concerns captured in unstructured qualitative provider notes by healthcare providers during visits with patients with AD. METHODS: Provider notes were obtained for patients with AD (n = 133,025) from a USA-based ambulatory electronic health records system. The sample included both children (n = 69,551) and adults at least 18 years of age (n = 63,474) receiving treatment from a variety of specialties including primary care, dermatology, and allergy/immunology. Key skin-related words were identified from a review of a sample of notes and natural language processing (NLP) was applied to determine the frequency of the keywords and bigram patterns. RESULTS: Provider notes largely focused on symptoms (primarily itch) and symptom relief rather than the impact of AD on work or lifestyle. Despite the known relationship between itch and skin pain, neuralgia was not widely documented. Compared to primary care providers, dermatologists' and allergist/immunologists' notes had more documentation of symptom-related issues. Personal and work/life burden issues were not widely documented regardless of specialty. CONCLUSION: The topics documented in case notes by healthcare providers about their patients with AD focus largely on symptoms and, to a lesser extent, treatment, but do not reflect the burden of AD on patients' lives. This finding highlights a potential care gap that warrants further investigation.

Impact of Plan-Level Access Restrictions on Effectiveness of Biologics Among Patients with Rheumatoid or Psoriatic Arthritis.

BACKGROUND: Novel disease-modifying antirheumatic drugs (DMARDs) can slow disease progression among patients with rheumatoid arthritis (RA) and psoriatic arthritis (PsA); however, some health plans require prior authorization (PA) or step therapy for access to treatments. OBJECTIVES: This retrospective study compared treatment effectiveness among RA and PsA patients with and without plan-level access restrictions to biologic DMARDs (bDMARDs) or targeted synthetic DMARDs (tsDMARDs). Medication adherence, a component of effectiveness, was also examined as a secondary outcome. METHODS: RA and PsA patients aged 18-64 years with one or more claims for subcutaneous bDMARDs between January 1, 2014 and December 31, 2015, with plan-level access data available, were identified within the IBM MarketScan claims database. The primary outcome was treatment effectiveness assessed during the 12 months following the first qualifying DMARD claim. Multivariate modeling examined the correlation between access restrictions and treatment effectiveness. Medication adherence during the 12-month follow-up period was also compared between patients with and without access restrictions. RESULTS: Among 3993 RA and 1713 PsA patients, 34.2 and 35.1%, respectively, had access restrictions, of whom 70.5 and 78.9%, respectively, had plans with step therapy. Compared with patients whose plans did not require step therapy, odds of treatment effectiveness were 19% lower (odds ratio [OR] 0.81, 95% CI: 0.67-0.98; p  = 0.033) for RA patients and 27% lower (OR 0.73, 95% CI: 0.55-0.98; p = 0.037) for PsA patients in plans with step therapy. Differences in effectiveness were driven by differences in medication adherence, the odds of which were 19% lower (OR 0.81, 95% CI 0.68-0.96; p = 0.014) among RA patients and 29% lower (OR 0.71, 95% CI: 0.54-0.94; p = 0.017) among PsA patients in plans with versus without step therapy. CONCLUSIONS: Compared with patients in plans without access restrictions or with PA only, RA and PsA patients in insurance plans with step therapy had lower odds of treatment effectiveness, mainly due to lower odds of adhering to treatment, during the 12 months following subcutaneous bDMARD initiation.

A Budget Impact and Cost Per Additional Responder Analysis for Baricitinib for the Treatment of Moderate-to-Severe Rheumatoid Arthritis in Patients with an Inadequate Response to Tumor Necrosis Factor Inhibitors in the USA.

BACKGROUND/OBJECTIVE: Baricitinib is a selective and reversible Janus kinase (JAK) inhibitor indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more tumor necrosis factor inhibitors (TNFis) and has been shown to improve multiple clinical and patient-reported outcomes. However, it is unclear what the budgetary impact would be for US commercial payers to add baricitinib to their formulary and how the efficacy of baricitinib compares to other disease-modifying antirheumatic drugs (DMARDs) with a similar indication. METHODS: A budget impact model (BIM) was developed for a hypothetical population of 1 million plan members that compared a world without and with baricitinib. A retrospective observational study was carried out to estimate market utilization of advanced therapies. Number needed to treat (NNT) and cost per additional responder were calculated for American College of Rheumatology (ACR) 20%/50%/70% improvement criteria (ACR20/50/70) response outcomes combining cost estimates from the BIM and efficacy values from a network meta-analysis (NMA). The model included costs related to drug acquisition and monitoring costs. RESULTS: Adding baricitinib would save a commercial payer $US169,742 for second-line therapy and $US135,471 for third-line therapy over a 2-year time horizon (all costs correspond to 2019 US dollars). Cost savings were driven by baricitinib drawing market share away from more expensive comparators. The NMA, based on nine studies, found no statistically significant differences in the median treatment difference between baricitinib and comparators except for versus a conventional synthetic DMARD (csDMARD), and thus NNT versus a csDMARD was similar. The cost per additional responder for baricitinib in patients with inadequate response to a TNFi was substantially lower than all other treatments for all three ACR response criteria at 12 weeks (ACR20: $US129,672; ACR50: $US237,732; ACR70: $US475,464), and among the lowest at 24 weeks (ACR20: $US167,811; ACR50: $US259,344; ACR70: $US570,557). CONCLUSIONS: Baricitinib, compared to other DMARDs, was a less expensive option (- $US0.01 incremental cost per member per month in second- and third-line therapy over a 2-year time horizon) with comparable efficacy in patients with inadequate response to TNFi. Adding baricitinib to formulary would likely be cost saving for US payers and expands treatment options for these patients.

Correction to: A Budget Impact and Cost Per Additional Responder Analysis for Baricitinib for the Treatment of Moderate-to-Severe Rheumatoid Arthritis in Patients with an Inadequate Response to Tumor Necrosis Factor Inhibitors in the USA.

Due to a single error in the annual cost of sarilumab the following needs to be corrected in the article.

Do Patients With Moderate or High Disease Activity Escalate Rheumatoid Arthritis Therapy According to Treat-to-Target Principles? Results From the Rheumatology Informatics System for Effectiveness Registry of the American College of Rheumatology.

OBJECTIVE: Despite strong recommendations for routine measurement of rheumatoid arthritis (RA) disease activity and associated treatment changes to attain remission/low disease activity, the measurement tools that clinicians use to evaluate RA patients' disease activity and frequency of treatment change have not been well characterized. Therefore, we evaluated different measurement tools that physicians used to assess RA disease activity and associated RA treatment changes. METHODS: Using data from the Rheumatology Informatics System for Effectiveness (RISE) registry from January 2016 through June 2017, and using the following criteria: age ≥18 years, diagnosis of RA (International Classification of Diseases, Ninth and Tenth Revision, codes), ≥2 RISE visits, and ≥1 RA disease activity measure scored in 2016, we classified eligible patients' drug use at the index visit as monotherapy or combination therapy with conventional synthetic (cs) and biologic disease-modifying antirheumatic drugs (bDMARDs). Outcomes include change in treatment over 12 months. Mixed models identified factors associated with treatment change. RESULTS: Among 50,996 eligible patients, 27,274 had longitudinal data. The most commonly used measures were RAPID3 (78.9%) and the Clinical Disease Activity Index (CDAI) (34.2%). The frequency of treatment change during follow-up was relatively low (35.6-54.6%), even for patients with moderate/high disease activity according to RAPID3 or CDAI scores. Older patients (age ≥75 years; adjusted odds ratio [ORadj ] 0.63 [95% confidence interval (95% CI) 0.50-0.78]) and those already receiving combination therapy with csDMARDs (ORadj 0.45 [95% CI 0.33-0.61]) or combination therapy with bDMARDs (ORadj 0.30 [95% CI 0.24-0.38]) were less likely to change RA treatment even after multivariable adjustment. CONCLUSION: Using the American College of Rheumatology's national RISE registry, one- to two-thirds of RA patients failed to change their treatment, even when experiencing moderate/high disease activity. Multimodal interventions directed at both patients and providers are needed to encourage shared decision-making, goal-directed care, and to overcome barriers to treatment escalation.

Health Care Effect of Disease-Modifying Antirheumatic Drug Use on Patients with Rheumatoid Arthritis.

BACKGROUND: Disease-modifying antirheumatic drugs (DMARDs) are recommended as the standard of care for patients with rheumatoid arthritis (RA) due to their ability to reduce pain and disability; however, DMARD use is low in some subgroups of the RA population. OBJECTIVE: To identify characteristics associated with DMARD use in the overall cohort of patients with RA and newly diagnosed RA patients. METHODS: This retrospective observational study used claims from a large national health plan. Use of DMARDs was measured according to the Healthcare Effectiveness Data and Information Set (HEDIS) as the proportion of patients with RA receiving DMARDs. Following HEDIS measure technical specifications, we identified patients aged 18-89 years with continuous enrollment during 2014 (measurement year) with ≥ 2 claims for RA outpatient visits and/or discharges on different dates between January and November 2014. Additionally, we identified a subset of patients newly diagnosed with RA in 2014 based on absence of any claims for RA or DMARDs in 2013. Descriptive analyses and bivariate associations were used to compare demographic and clinical characteristics of patients with RA with or without DMARD use in 2014. Health care resource utilization (HCRU) and costs were compared in 2014 for patients enrolled in Medicare Advantage Prescription Drug (MAPD) plans during both 2014 and 2015. Regression models were used to evaluate patient and provider characteristics associated with DMARD use in 2014 and the effect on HCRU and costs. RESULTS: Among the 33,880 patients identified with RA in 2014, most patients received a DMARD (75.2%); 29.4% of patients newly diagnosed with RA had been treated with DMARDs in 2014. Patients with DMARD use, on average, were younger (aged 67 years ± 10.7 vs. 69 years ± 10.7) and healthier (Deyo-Charlson Comorbidity Index [DCCI] 2.4 ± 1.9 vs. 2.6 ± 2.1) and included a greater proportion of women (75.9% vs. 71.0%) than those with no DMARD use (P < 0.0001). Use of DMARDs (P < 0.0001) was associated with 14.5% fewer hospitalizations and 18.0% fewer emergency department visits. Although total costs increased by 15.0% with use of DMARDs, when the cost of DMARDs was excluded, the total cost decreased by 13.7% (P < 0.0001). Female gender (32.2%), higher claims-based index for RA severity score (47.0%), higher RxRisk-V score (26.7%), visit to a rheumatologist (34.3%), and use of glucocorticoids (17.7%) increased the odds of DMARD use (P < 0.0001). Use of certain classes of medication, such as nonsteroidal anti-inflammatory drugs (12.3%), opioids (19.5%), antidepressants (20.0%), muscle relaxants (12.5%), and anticonvulsants (15.5%), were associated with lower use of DMARDs (P < 0.0001). CONCLUSIONS: We found significant differences in demographic and clinical characteristics between patients with and without DMARD use, which can potentially inform treatment decisions regarding DMARD use as deemed necessary by the provider. Future research should investigate the reasons for lack of treatment. DISCLOSURES: This study was supported by funding from Eli Lilly to Humana as a collaborative research project involving employees of both companies. Boytsov, Saverno, Zhang, and Gaich are employees of Eli Lilly. Nair, Bhattacharya, Abbott, and Dixon are employees of Humana, which received funding from Eli Lilly to complete this research.

The Effectiveness of a Private Orthopaedic Practice-Based Osteoporosis Management Service to Reduce the Risk of Subsequent Fractures.

BACKGROUND: Osteoporosis is prevalent in the United States, with an increasing need for management. In this study, we evaluated the effectiveness of a private orthopaedic practice-based osteoporosis management service (OP MS) in reducing subsequent fracture risk and improving other aspects of osteoporosis management of patients who had sustained fractures. METHODS: This was a retrospective cohort study using the 100% Medicare data set for Michigan residents with any vertebral; hip, pelvic or femoral; or other nonvertebral fracture during the period of April 1, 2010 to September 30, 2014. Patients who received OP MS care with a follow-up visit within 90 days of the first fracture, and those who did not seek OP MS care but had a physician visit within 90 days of the first fracture, were considered as exposed and unexposed, respectively (first follow-up visit = index date). Eligible patients with continuous enrollment in Medicare Parts A and B for the 90-day pre-index period were followed until the earliest of death, health-plan disenrollment, or study end (December 31, 2014) to evaluate rates of subsequent fracture, osteoporosis medication prescriptions filled, and bone mineral density (BMD) assessments. Health-care costs were evaluated among patients with 12 months of post-index continuous enrollment. Propensity-score matching was used to balance differences in baseline characteristics. Each exposed patient was matched to an unexposed patient within ± 0.01 units of the propensity score. After propensity-score matching, Cox regression examined the hazard ratio (HR) of clinical and economic outcomes in the exposed and unexposed cohorts. RESULTS: Two well-matched cohorts of 1,304 patients each were produced. The exposed cohort had a longer median time to subsequent fracture (998 compared with 743 days; log-rank p = 0.001), a lower risk of subsequent fracture (HR = 0.8; 95% confidence interval [CI] = 0.7 to 0.9), and a higher likelihood of having osteoporosis medication prescriptions filled (HR = 1.7; 95% CI = 1.4 to 2.0) and BMD assessments (HR = 4.3; 95% CI = 3.7 to 5.0). The total 12-month costs ($25,306 compared with $22,896 [USD]; p = 0.082) did not differ significantly between the cohorts. CONCLUSIONS: A private orthopaedic practice-based OP MS effectively reduced subsequent fracture risk, likely through coordinated and ongoing comprehensive patient care, without a significant overall higher cost. LEVEL OF EVIDENCE: Therapeutic Level III. See Instructions for Authors for a complete description of levels of evidence.