Rates Of Healthcare Resource Utilization Research Articles

Healthcare resource utilization and costs associated with first versus subsequent use of cariprazine for bipolar I disorder

Aims To evaluate the healthcare resource utilization (HRU) and costs of patients who initiated cariprazine as their first versus subsequent atypical antipsychotic (AA) following a bipolar I disorder (BP-I) diagnosis. Methods Adults with a BP-I diagnosis (first claim = index), commercial, Medicare Supplemental, or Medicaid insurance, and ≥1 outpatient cariprazine dispensing were identified from Merative MarketScan database. Cohorts included patients who initiated cariprazine as either their first or subsequent AA after initial BP-I diagnosis. Characteristics were balanced between cohorts using inverse probability of treatment weighting (IPTW). Outcomes evaluated post-index included all-cause and mental health (MH)–related HRU (hospitalizations, emergency department [ED] visits, outpatient visits), total healthcare costs (medical + pharmacy), and treatment patterns. HRU and healthcare costs were reported per patient-year (PPY) and compared between cohorts using rate ratios and 95% CIs estimated using nonparametric bootstrap procedures. Treatment patterns were analyzed descriptively, with standardized differences ≥10% considered important. Results After IPTW, cohorts included 1,409 patients who initiated cariprazine first and 1,621 patients who initiated cariprazine subsequently; the average (standard deviation, SD) observation period was 678 (373) and 758 (389) days for first and subsequent initiators, respectively. Patients who initiated cariprazine first had 23% fewer all-cause hospitalizations and 28% fewer MH-related hospitalizations PPY (each comparison, p < 0.001). Rates of all-cause and MH-related outpatient visits were significantly lower in patients who initiated cariprazine first versus subsequently (each comparison, p < 0.001), while rates of ED visits were similar. Relative to subsequent initiators, first initiators incurred $2,587 and $2,130 lower all-cause and MH-related total healthcare costs PPY, respectively (each comparison, p < 0.05). Before starting cariprazine, first initiators used fewer BP-I–related medications on average than subsequent initiators (2.6 vs 3.9; standardized difference = 23.9%). Limitations Potential coding inaccuracies and residual confounding. Conclusions In this real-world database analysis, patients with BP-I who initiated cariprazine as their first AA had lower rates of HRU and incurred lower costs than patients who initiated cariprazine as a subsequent AA.

US Clinical Practice Experience with Eculizumab in Myasthenia Gravis: Acute Clinical Events and Healthcare Resource Utilization.

The terminal complement inhibitor eculizumab is approved in the USA for the treatment of patients with acetylcholine receptor antibody-positive generalized myasthenia gravis (MG). The ELEVATE study aimed to examine clinical-practice outcome data on eculizumab effectiveness in US adults with MG (generalized or ocular). This paper reports the findings on MG exacerbations and crises and associated healthcare resource utilization, and the use of rescue therapy. A retrospective chart review was conducted of US adults with MG who initiated eculizumab. Outcomes assessed for up to 2 years before and after eculizumab initiation included percentages and rates per patient per year (PPPY) of exacerbations and crises (the latter defined as intubation/impending intubation), healthcare resource utilization, and rescue therapy administration. A total of 119 patients diagnosed with MG were enrolled in the study; 92 patients had ≥3 months of data both before and during eculizumab therapy and were included in the analyses. The mean rate of MG exacerbations decreased from 0.385 PPPY before eculizumab initiation to 0.152 PPPY during eculizumab treatment (p = 0.0034); the mean rate of MG crises decreased from 0.411 to 0.056 PPPY (p = 0.0018). Rates of healthcare resource utilization and rescue therapy use also decreased significantly during eculizumab treatment. This retrospective chart review analysis provides evidence for a beneficial impact of eculizumab treatment on the incidence of MG exacerbations and crises and associated healthcare resource utilization in clinical practice, and on rescue therapy use. These data further support the therapeutic benefits of eculizumab in patients with MG.

Healthcare resource use and costs associated with the misdiagnosis of migraine.

To compare healthcare resource utilization and healthcare costs in patients with migraine with or without a history of misdiagnosis. Despite the high prevalence of migraine, migraine is commonly misdiagnosed. The healthcare resource use and cost burden of a misdiagnosis is unknown. This retrospective cohort study identified adults with an incident migraine diagnosis from the Merative™ Marketscan® Commercial and Medicare Supplemental Databases between June 2018 and 2019. Patients with a diagnosis of commonly considered misdiagnoses (headache, sinusitis, or cervical pain) before their migraine diagnosis were classified as the "misdiagnosed cohort." Patients in the misdiagnosed cohort were potentially misdiagnosed, then eventually received a correct diagnosis. Patients without a history of commonly considered misdiagnoses prior to their migraine diagnosis were classified as the "correctly diagnosed cohort." Healthcare resource utilization and healthcare costs were assessed in the period before migraine diagnosis and compared between the cohorts. Outcomes were reported as per patient per month and compared with incidence rate ratios. A total of 29,147 patients comprised the correctly diagnosed cohort and 3841 patients comprised the misdiagnosed cohort and met the inclusion criteria. Patients in the misdiagnosed cohort had statistically significantly higher rates of inpatient admissions (0.02 vs. 0.01, incidence rate ratio [IRR] 1.61, 95% confidence interval [CI] 1.47-1.74), emergency department visits (0.10 vs. 0.05; IRR 1.89, 95% CI 1.79-1.99), neurologist visits (0.12 vs. 0.02; IRR 5.95, 95% CI 5.40-6.57), non-neurologist outpatient visits (2.64 vs. 1.58; IRR 1.67, 95% CI 1.62-1.72) and prescription fills (2.82 vs. 1.84; IRR 1.53, 95% CI 1.48-1.58) compared to correctly diagnosed patients. Misdiagnosed patients had statistically significantly higher rates of healthcare cost accrual for inpatient admissions ($1362 vs. $518; IRR 2.62, 95% CI 2.50-2.75), emergency department visits ($222 vs. $98; IRR 2.27, 95% CI 2.18-2.36), neurologist visits ($42 vs. $9; IRR 4.39, 95% CI 4.00-4.79), non-neurologist outpatient visits ($1327 vs. $641; IRR 2.07, 95% CI 1.91-2.24), and prescription fills ($305 vs. $215; IRR 1.41, 95% CI 1.18-1.70) compared to correctly diagnosed patients. Patients with migraine who have a history of misdiagnoses have higher rates of healthcare resource utilization and cost accrual versus those without such history.

Health Care Utilization and Costs Associated With Empagliflozin in Older Adults With Type 2 Diabetes.

We compared health care resource utilization (HCRU) and costs for inpatient and outpatient services and dispensed medications in older adults with type 2 diabetes initiating empagliflozin versus dipeptidyl peptidase 4 inhibitors (DPP-4is). The study population included U.S. Medicare fee-for-service beneficiaries with diabetes (age ≥65 years) initiating empagliflozin or DPP-4is (August 2014 to September 2018). We estimated rate ratios (RRs) for HCRU outcomes using zero-inflated negative binomial regression and per-member per-year (PMPY) cost differences using generalized linear model with gamma distributions, overall and stratified by baseline cardiovascular disease (CVD), after adjusting for 143 baseline covariates using 1:1 propensity score matching. We identified 23,335 matched pairs (mean age 72 years, 51% with baseline CVD). HCRU rates were lower in empagliflozin versus DPP-4i initiators (number of inpatient days, RR 0.89 [95% CI 0.82, 0.97]; number of emergency department [ED] visits, 0.86 [0.82, 0.91]; number of hospitalizations, 0.86 [0.79, 0.93]; number of office visits, 0.96 [0.95, 0.98]). Inpatient cost (-$713 PMPY [95% CI -847, -579), outpatient cost (-$198 PMPY[-272, -124]), and total cost of care (-$1,109 PMPY [-1,478, -739]) were lower for empagliflozin versus DPP-4is, although diabetes medication cost was higher in empagliflozin initiators ($454 PMPY [95% CI 284, 567]). In the CVD subgroup, total cost was lower for empagliflozin initiators (-$2,005 PMPY [-2,451, -1,337]), while the difference was attenuated in the non-CVD subgroup (-$296 PMPY[-740, 148]). Among older adults with diabetes, empagliflozin was associated with a lower number of inpatient days, hospitalizations, ED visits, and office visits and with lower costs of care compared with DPP-4is, especially in those with history of CVD.

Real-world safety and healthcare resource utilization (HCRU) of lurbinectedin (lurbi) in patients (pts) with small cell lung cancer (SCLC): Jazz EMERGE 402 updated analysis.

e23266 Background: Lurbi monotherapy received approval in multiple countries, including accelerated US FDA approval and conditional Health Canada approval for adults with metastatic (US) or stage III/metastatic (Canada) SCLC with disease progression on or after platinum-based chemotherapy, based on a phase 2, open-label, single-arm trial (Trigo et al, Lancet Oncol 2020). Jazz EMERGE 402 (NCT04894591) is a phase 4 observational study assessing the effectiveness, safety profile, and HCRU of lurbi in pts with extensive-stage SCLC treated in real-world clinical settings. Here, we report updated safety data and HCRU. Methods: This study is enrolling pts with extensive-stage SCLC treated with lurbi according to the local approved label in the US and Canada. Results: Enrollment is ongoing with a target of 300 pts. Between 28JUN2021 and 27DEC2022, 105 pts were enrolled and followed for ≥6 months after the first dose of lurbi (data cutoff: 27JUN2023). The median (range) age was 67 (44-87) years; 72 (69%) pts had prior immunotherapy; 22 (21%) had an Eastern Cooperative Oncology Group performance status of ≥2; and 25 (24%) had central nervous system involvement. All pts received ≥1 cycle of lurbi; 56 (53%) as second-line (2L) and 37 (35%) as third-line (3L) therapy. The median (range) number of lurbi cycles was 4 (1-31) and the median (range) duration of exposure was 97 (21-694) days. Dose modifications occurred in 27 (26%) pts, with 17 (16%) requiring dose reductions. Granulocyte colony-stimulating factor was administered in 43 (41%) pts (34 [79%] as primary and 7 [16%] as secondary prophylaxis; 2 [5%] were unknown). At the time of data extraction, lurbi treatment was ongoing in 11 (10%) pts and 23 (22%) pts received subsequent anticancer therapy after lurbi. Treatment-related adverse events (TRAEs) were reported in 40 (38%) pts and treatment-related serious adverse events (SAEs) in 13 (12%) pts. The most common TRAEs were anemia (n = 13, 12%), neutropenia (n = 10, 10%), nausea (n = 8, 8%), and fatigue (n = 7, 7%). Thirty-six pts were hospitalized for a median (interquartile range [IQR]) duration of 10 (4, 18) days (Table). No pts were hospitalized on the first day of treatment; 4 pts were hospitalized during the first 21-day cycle. The most common reasons for hospitalization were pneumonia (n = 10, 28%) and sepsis (n = 6, 17%; one was an SAE). Conclusions: In this phase 4 study, lurbi was well tolerated, with low rates of HCRU and a safety profile generally consistent with that reported in the phase 2 trial. Clinical trial information: NCT04894591 . [Table: see text]

#2533 Real-world challenges associated with the use of systemic glucocorticoids in a US IgAN cohort

Abstract Background and Aims Immunoglobulin A nephropathy (IgAN) is the most common primary glomerular disease and up to 50% of patients progress to kidney failure within 10-20 years. Until early 2022, management of IgAN was primarily focused on supportive care. As outlined in the Kidney Disease: Improving Global Outcomes (KDIGO) guidelines, patients at high risk of progressive chronic kidney disease despite maximal supportive care can be considered for a 6-month course of systemic glucocorticoids (SGC), although important risks of toxicity and contraindications must be considered. There is currently limited real-world evidence describing the impact of the use of SGC on treatment‑emergent toxicity and healthcare resource utilization (HCRU) in patients with IgAN. We report the incidence of adverse events (AEs) commonly associated with SGC, as well as HCRU rates and costs, in a real-world cohort of IgAN patients, comparing new initiators of SGC with patients who have never initiated SGC. Method This non-interventional, retrospective cohort study was conducted in the TriNetX Dataworks - United States of America Network, a database of de-identified, patient-level electronic medical records (EMR) of approximately 100 million patients. The cohort was selected from a total of 19, 687 patients with IgAN, identified using proxy ICD‑10 codes N02.8 (recurrent and persistent hematuria with other morphologic changes) or N04.1 (nephrotic syndrome) between January 1, 2011, and May 4, 2022. Eligible patients were required to have: an incident diagnosis of IgAN with the same diagnosis within 30-180 days; at least 12 months of EMR history available prior to and after initial IgAN diagnosis; not experienced kidney failure prior to or on the initial date of IgAN diagnosis; and not received SGC prior to the date of diagnosis. Data assessed included demographic and clinical characteristics, IgAN-directed medications, AEs, and HCRU. To promote balance between cohorts, new initiators of SGC (dexamethasone, prednisone, prednisolone, methylprednisolone) were matched with patients who did not receive SGC using a propensity score (PS) approach based on their characteristics at diagnosis. To avoid immortal-time bias, the index date was set as the time of SGC initiation in patients in the SGC cohort; for the non-SGC cohort, a pseudo-index date was assigned using the same lag from diagnosis to index date as their 1:1 PS-matched patient in the SGC cohort. Differences in the cumulative incidence of individual AEs were analyzed using the chi-square test or Fisher's exact test, and differences in HCRU rates and costs were analyzed using the Kruskal-Wallis test. Results After implementing the PS approach, the final analysis included 802 patients (401 PS-matched pairs), with a mean age of 41.2 years and 55% being male. Median duration of follow-up was 3.5 and 3.1 years for the SGC and non-SGC cohorts, respectively. Of AEs that occurred in at least ten of the 802 patients, patients who received SGC experienced a significantly increased incidence of all AEs except for fractures, new onset of diabetes, and reported onset of glaucoma (Table 1). Patients who received SGC had a 12-fold increased incidence of severe infections requiring hospitalization compared with the non-SGC cohort. Annualized mean HCRU rates and costs were significantly greater across all HCRU types for patients who received SGC vs those who did not receive SGC, including an 8-fold increase in inpatient visits, a 4-fold increase in emergency department admissions, and twice as many ambulatory visits (Fig. 1). Results were consistent when the cohort was restricted to patients with IgAN identified using only the ICD-10 code N02.8 (recurrent and persistent hematuria with other morphologic changes). Conclusion The findings of this real-world analysis demonstrate significant side effects and costs for IgAN patients treated with SGC compared with patients not treated with SGC, underscoring the recommendation in the 2021 KDIGO guidelines to carefully consider treatment-emergent toxicity before treating IgAN patients with SGC.

#1241 Healthcare resource utilisation in adults with chronic kidney disease by KDIGO categorisation in England: 2010-2019

Abstract Background and Aims Chronic kidney disease (CKD) is associated with high clinical burden for patients and substantial economic costs for healthcare systems. Detailed estimates of healthcare resource utilization (HCRU) for patients with CKD in the UK are limited, particularly by stage of CKD. This study assessed outpatient and non-elective inpatient HCRU by stage of CKD in England. Method This non-interventional study utilised data from primary care electronic health records from Clinical Practice Research Datalink (CPRD) AURUM and Hospital Episode Statistics (HES) admitted patient care (APC). Patients aged ≥18 years registered in CPRD with eligible linkage to HES between January 1, 2010 and December 31, 2019 with 2+ eGFR measurement during the period were identified. CKD was defined by eGFR &amp;lt;60 ml/min/1.73 m2 and/or presence of a uACR measurement ≥3 mg/mmol, both confirmed by a second measurement 90-365 days later. Start of follow-up (index) was set to the earliest date during the study period that CKD was confirmed. Follow-up for HCRU ended at earliest of death, administrative censoring, or 31st December 2019. Patients with end-stage kidney disease (eGFR &amp;lt;9 ml/min/1.73 m2) at index were excluded. Number of HCRU events for each HCRU category was estimated as rates per 1,000 person years (PY) for each calendar year based on cumulative number of events. Event rates were calculated as number of outcomes/total time at risk. Confidence intervals (95%) were calculated assuming a Poisson distribution. Age- and sex-adjusted rates were calculated to compare rates across CKD stages. Results Of 6.1 million adult patients meeting study inclusion criteria, 743,945 adults (12.2%) with CKD were identified. Mean age (SD) was 76.1 (11.5) years, 55.2% were female, and 89.8% White, contributing a total of 3,055,600 years of follow-up. Most (60.9%) patients had stage 3a CKD (eGFR 45-&amp;lt;59 ml/min/1.73 m2). Among patients with uACR measurements (44.4%), 48.0% were A1 (&amp;lt;3 mg/mmol), 44.4% A2 (3-30 mg/mmol), and 7.6% A3 (&amp;gt;30 mg/mmol). Overall, per 1000 PY, there were 24,763 primary care visits, 475 non-elective all-cause hospitalisations, 4,950 attended (722 unattended) outpatient visits, including 308 (43 unattended) outpatient nephrology visits and 368.1 attended (61.2 unattended) cardiology visits. For all HCRU categories, greater KDIGO risk category at index was associated with higher HCRU. For example, after age and sex adjustment, patients with CKD stage 3b had a mean of 0.53/PY non-elective all-cause hospitalisations compared to 0.77/PY for patients with CKD stage 4. Additionally, a mean of 5.18 outpatient visits per PY was observed for patients with CKD stage 3b, of which 0.91 were nephrology outpatient visits compared to 8.39 outpatient visits per PY for stage 4, of which 2.65 were nephrology outpatient visits. Generally, both lower eGFR and higher uACR were individually associated with higher HCRU. Conclusion This study demonstrates high HCRU for patients with CKD, with increasing rates of HCRU with lower kidney function and greater kidney damage. Patients with more advanced CKD contribute at a disproportionally higher rate of outpatient healthcare encounters and non-elective hospitalisations. Efforts to prevent the development and progression of CKD and improving clinical outcomes will reduce frequency and cost of HCRU.

Healthcare Resource Utilization Associated with Intermittent Oral Corticosteroid Prescribing Patterns in Asthma.

Oral corticosteroid (OCS) use for asthma is associated with considerable healthcare resource utilization (HCRU) and costs. However, no study has investigated this in relation to patterns of intermittent OCS prescription. This historical UK cohort study used primary care medical records, linked to Hospital Episode Statistics, from 2008 to 2019, of patients (≥4 years old) with asthma prescribed intermittent OCS. Patients were categorized by OCS prescribing pattern (one-off [single], less frequent [≥90-day gap] and frequent [<90-day gap]) and matched 1:1 (by sex, age and index date) with people never prescribed OCS with/without asthma. HCRU (reported as episodes, except for length of hospital stay [days] and any prescription [records]) and associated costs were compared between intermittent OCS and non-OCS cohorts, and among intermittent OCS prescribing patterns. Of 149,191 eligible patients, 50.3% had one-off, 27.4% less frequent, and 22.3% frequent intermittent OCS prescribing patterns. Annualized non-respiratory HCRU rates were greater in the intermittent OCS versus non-OCS cohorts for GP visits (5.93 vs 4.70 episodes, p < 0.0001), hospital admissions (0.24 vs 0.16 episodes, p < 0.0001), and length of stay (1.87 vs 1.58 days, p < 0.0001). In the intermittent OCS cohort, rates were highest in the frequent prescribing group for GP visits (7.49 episodes; p < 0.0001 vs one-off), length of stay (2.15 days; p < 0.0001) and any prescription including OCS (25.22 prescriptions; p < 0.0001). Mean per-patient non-respiratory related and all-cause HCRU-related costs were higher with intermittent OCS than no OCS (£3902 vs £2722 and £8623 vs £4929, respectively), as were mean annualized costs (£565 vs £313 and £1526 vs £634, respectively). A dose-response relationship existed; HCRU-related costs were highest in the frequent prescribing cohort (p < 0.0001). Intermittent OCS use and more frequent intermittent OCS prescription patterns were associated with increased HCRU and associated costs. Improved asthma management is needed to reduce reliance on intermittent OCS in primary care.

Healthcare Resource Utilization and Economic Burden of Cytokine Release Syndrome and Neurotoxicity in Patients with Relapsed and Refractory Multiple Myeloma (RRMM) Receiving Idecabtagene Vicleucel in Earlier-Line Settings in the KarMMa-3 Clinical Trial

Introduction: Adverse events (AE), such as cytokine release syndrome (CRS) and neurological toxicity (NT), have been associated with chimeric antigen receptor (CAR) T cell therapies. Idecabtagene vicleucel (ide-cel, bb2121), a B-cell maturation antigen-directed CAR T cell therapy, demonstrated deep and durable responses in triple-class exposed (TCE) patients (pts) with RRMM in the pivotal, single-arm, open-label phase 2 KarMMa (3 prior lines of therapy [LOTs]; NCT03361748) (Munshi NC, et al. NEJM 2021;384:705-716) and superior progression-free survival with ide-cel vs standard regimens in the phase 3 KarMMa-3 trial (2-4 prior LOTS; NCT03651128) (Rodríguez-Otero P, et al. NEJM 2023;388:1002-1014). Healthcare resource utilization (HCRU) and costs associated with CRS/NT management were estimated previously for pts receiving ide-cel in the KarMMa trial (McGarvey N, et al. Value Health 2023;26:S89) and suggested low rates of severe (grade ≥ 3) CRS/NT following ide-cel infusion and higher HCRU and costs with increasing AE severity. To further assess resource and economic burden associated with care of treatment-emergent AEs, this analysis evaluated HCRU and estimated cost of CRS/NT management among pts treated with ide-cel in earlier-line settings in the KarMMa-3 study. Methods: In this retrospective study, case report forms from the KarMMa-3 clinical trial database among pts experiencing a CRS and/or NT event that initiated within 90 days post ide-cel infusion were assessed. Pt-level HCRU data, including hospitalizations and length of hospital stay (LOS) (standard inpatient [IP], intensive care unit [ICU] days), diagnostics (eg, lab work, imaging), procedures (eg, dialysis, intubation), and medications (eg, oncology supportive care, prophylactics, other AE management) were identified from CRS/NT onset through resolution. AE management costs were estimated using a micro-costing methodology in which unit costs sourced from public databases or literature (eg, United States Centers for Medicare and Medicaid Services, Healthcare Cost and Utilization Project, IBM ® Micromedex ® RED BOOK ®) were adjusted to 2022 US dollars (USD) using the Consumer Price Index and applied to each HCRU identified. Results: Of 225 pts treated with ide-cel in the KarMMa-3 trial, 203 (90.2%) experienced any grade CRS and/or NT event. Pts experiencing a CRS/NT event had a mean (standard deviation [SD]) age of 61.9 (8.9) years and most were male (123/203 [60.6%]), White (138/203 [68.0%]), and non-Hispanic or Latino (138/203 [68.0%]). The majority (187/203 [92.1%]) of pts experienced less severe CRS/NT events (grade ≤ 2), which resolved quicker and relatively few pts (16/203 [7.9%]) had a more severe (grade ≥ 3) event, which took longer to resolve. Among pts who received ide-cel, the estimated median cost of AE management ranged from USD 17,124 (CRS only grade 1) to USD 181,552 (nonconcurrent CRS or NT grade ≥ 3) (Figure 1). Additional median costs for a pt with a grade ≥ 3 CRS/NT event was USD 31,045 higher than a grade ≤ 2 CRS/NT event. Across AE categories, the main contributors to CRS/NT management were facility costs, namely standard IP hospitalizations and ICU stays. Almost all pts (202/203 [99.5%]) had a standard IP hospitalization; 3.9% (8/203) had an ICU stay (Table 1). The trial protocol required a stay of ≥ 15 days post infusion unless admitted to the ICU; some hospitalization days recorded during the AE overlapped with the protocol-required stay. ICU admissions were more common among pts with CRS/NT grade ≥ 3 (7/16 [43.8%]) than those with grade ≤ 2 (1/187 [0.5%]). Median (range) total LOS was 10 days longer among pts with grade ≥ 3 (14.5 [2-84] days) than those with grade ≤ 2 (4 [1-34] days) CRS/NT. Pts with grade ≥ 3 CRS/NT had higher rates of HCRU than those with grade ≤ 2 for several interventions, such as medications (eg, tocilizumab + corticosteroids: 13/16 [81.3%] vs 63/187 [33.7%]), diagnostics (eg, imaging: 14/16 [87.5%] vs 48/187 [25.7%]), and procedures (eg, dialysis:3/16 [18.8%] vs 0/187 [0%]). Conclusions: Most pts with RRMM treated with ide-cel in the KarMMa-3 trial experienced a CRS and/or NT event, however, few were of high severity (grade ≥ 3). HCRU and costs for CRS/NT increased with AE severity and management. CAR T cell therapies associated with lower-severity AEs and planned interventions to prevent higher-grade AEs may provide an opportunity to better manage HCRU and costs.

Understanding the long-term impact of incident osteoporotic fractures on healthcare utilization and costs in Korean postmenopausal women.

To evaluate healthcare resource utilization (HCRU) and costs of care over 5 years after the incident osteoporotic fractures (OF) in postmenopausal women. We used data from the National Health Insurance Service databases 2011-2018. Women aged ≥ 50 years with incident OF (OF group) were matched to women without OF (non-OF group). HCRU (inpatient, outpatient, and emergency room [ER] visits) and costs of care (inpatient, outpatient, and ER visits) during the 5-year follow-up period were derived after propensity score matching (PSM). Additionally, we identified women with subsequent fractures within the first 2 years after the incident OF. After PSM, 47,238 OF and 134,813 non-OF women were identified. HCRU rates and costs of care were highest in the first year after OF and decreased substantially, but remained higher in the OF group during the entire follow-up period. The increase in cumulative HCRU rates over 5 years was highest in inpatient admissions with ER visits (138% higher in OF vs non-OF). The cumulative total costs over 5 years were 73% higher in the OF group than in the non-OF group, which was mostly driven by inpatient costs. Trends were similar for women with subsequent fractures, but they generally showed higher HCRU and costs than those in the total OF group. OF imposes a substantial and sustained economic burden on women, resulting in an approximately twofold increase in the cumulative cost over 5 years compared to women without fracture, which highlights the need for early diagnostics and treatment of osteoporosis.

Treatment Patterns and Healthcare Resource Use in Medicare Beneficiaries with Parkinson's Disease.

Studies on real-world treatment patterns and long-term economic burden of Parkinson's disease (PD) have been limited. To assess treatment patterns, healthcare resource utilization (HRU), and costs associated with PD symptoms and treatment-related adverse events (AEs) among Medicare beneficiaries in the United States. A 100% Medicare Fee-For-Service data (2006-2020) of patients with PD were analyzed. PD treatment patterns were described for the subset of patients who had no previously observed PD treatments or diagnoses (ie, the incident cohort). HRU and healthcare costs associated with PD symptoms were assessed for all patients with PD (ie, the overall cohort) and that associated with treatment-related AEs were assessed for the subset of patients who received PD treatments after PD diagnosis (ie, the active treatment cohort), using longitudinal models with repeated measures. Overall, 318,582 patients were included (mean age at PD diagnosis: 77.4 years; 53.3% female). Among patients in the incident cohort (N=214,829), 51.1% initiated levodopa monotherapy and 5.9% initiated dopamine agonists (DAs) monotherapy as first-line treatment. The proportion of incident patients treated with DAs and other PD therapies generally increased from post-diagnosis years 1 to 10. The median time from diagnosis to PD treatment initiation was 2.0 months; the median time to treatment discontinuation was the longest with levodopa (18.7 months), followed by DAs (9.5 months). In the overall cohort, PD symptoms, especially motor symptoms and severe motor symptoms, were associated with significantly higher rates of HRU and costs. In the active treatment cohort (N=234,298), treatment-related AEs were associated with significantly higher rates of HRU and medical costs. While levodopa is still the mainstay of PD management, considerable heterogeneity exists in real-world treatment patterns. Overall, PD symptoms and AEs were associated with significantly higher HRU and healthcare costs, suggesting unmet medical needs for PD treatments with better tolerability profiles.

Health care resource utilization in 3L + patients with chronic phase chronic myeloid leukemia receiving asciminib or bosutinib

ObjectivesTo assess and compare health care resource utilization (HCRU) rates of asciminib and bosutinib at the Week 24, Week 48, and Week 96 cutoffs among 3 L + patients with chronic myeloid leukemia in chronic phase (CML-CP) in the randomized ASCEMBL trial.MethodsPatients in the ASCEMBL trial (Clinicaltrials.gov: NCT03106779) were randomized to receive asciminib 40 mg twice daily (n = 157) or bosutinib 500 mg once daily (n = 76). At each scheduled visit, investigators conducted HCRU assessment on hospitalization, emergency room visit, general practitioner visit, specialist visit and urgent care visit; duration and type of hospitalization for the hospitalized patients; and reasons for HCRU. The number of patients with HCRU, rate of HCRU per patient-year, and length of hospital stay by ward type were compared at Week 24, Week 48, and Week 96 analyses.ResultsLower proportions of patients receiving asciminib versus bosutinib used any resources including hospitalizations, emergency room visits, general practitioner visits, specialist visits, and urgent care visits (23.6% versus 36.8%, 26.1% versus 39.5%, and 28.6% versus 42.6% at Week 24, Week 48, and Week 96 analyses, respectively). After normalizing for treatment exposure, rates of HCRU for any resource per patient-year were significantly lower for asciminib versus bosutinib: 0.25 (95% CI: 0.18–0.34) versus 0.80 (95% CI: 0.55–1.16) at the Week 24 analysis, 0.20 (95% CI: 0.15–0.27) versus 0.47 (95% CI: 0.32–0.66) at the Week 48 analysis, and 0.17 (95% CI: 0.12–0.22) versus 0.40 (95% CI: 0.27–0.55) at the Week 96 analysis. Among the hospitalized patients, mean length of hospital stay was lower for asciminib than bosutinib for most wards at all three timepoints.ConclusionsIn the ASCEMBL trial, asciminib-treated patients with CML-CP in 3 L + maintained lower resource utilization compared to bosutinib over the long-term.

Economic and Clinical Burden of Herpes Zoster Among Patients With Inflammatory Bowel Disease in the United States.

Patients with ulcerative colitis (UC) or Crohn's disease (CD) are at increased risk of herpes zoster (HZ); however, relevant cost and healthcare resource utilization (HCRU) data are limited. We estimated HCRU (hospitalization, emergency department [ED], and outpatient visits) and costs in patients with UC or CD, with and without HZ, using administrative claims data (October 2015-February 2020). HCRU and costs (2020 US dollars) were compared at 1 month, 1 quarter, and 1 year after the index date, using propensity score adjustment and generalized linear models. In total, 20 948 patients were included: UC+/HZ+ (n = 431), UC+/HZ- (n = 10 285), CD+/HZ+ (n = 435), and CD+/HZ- (n = 9797). Patients with HZ had higher all-cause HCRU rates and all-cause total healthcare costs relative to those without HZ. In the first month, adjusted incidence rate ratios (aIRRs) for hospitalizations and ED visits for patients with UC and HZ compared with UC alone were 2.87 (95% confidence interval [CI], 1.93-4.27) and 2.66 (95% CI,1.74-4.05), respectively; for those with CD and HZ, aIRRs were 3.34 (95% CI, 2.38-4.70) and 3.31 (95% CI, 2.32-4.71), respectively, compared with CD alone (all P < .001). Adjusted cost differences in UC and CD cohorts with HZ over the first month were $2189 and $3774, respectively, chiefly driven by higher inpatient costs. The incremental impact on HCRU and costs in cohorts with HZ predominantly occurred during the first quarter following diagnosis. HZ is associated with increased HCRU and costs in patients with UC and CD, especially shortly after diagnosis.

Healthcare Resource Utilization Among Patients With Activated PI3Kδ Syndrome in the United States: A Real-world Assessment by Patient Age

ImportanceDespite the known morbidity associated with activated PI3Kδ syndrome (APDS), little is understood about the healthcare resource utilization (HRU) attributable to this condition. ObjectiveTo describe HRU and treatment patterns of patients with APDS by age group (≤11 and ≥12 yrs). MethodsPatients with APDS were identified via genetic testing and tokenized to the Symphony Health Integrated Dataverse database comprising medical/pharmacy claims (10/1/2015–3/31/2021). HRU rates (hospitalizations, emergency room [ER], outpatient, and other visits) per person-year, main reason for HRU visit, and treatment patterns were assessed from the start of patients’ clinical activity to the earliest of end of clinical activity or data availability (observation period). ResultsOf 31 patients with APDS identified, 14 were ≤11 yrs and 17 were ≥12 yrs. The mean±SD observation period was 3.9 ±2.1 and 5.2 ± 1.8 yrs, respectively; mean age was 3.9 ± 2.7 and 29.4 ± 13.1 yrs, respectively. Common clinical manifestations in ≤11 yrs included lymphoproliferation (71.4%), fever (64.3%), allergy (57.1%), otitis media (50.0%), diarrhea (21.4%), and lymphoma (7.1%). Common clinical manifestations in ≥12 yrs included sinusitis (58.8%), bronchitis (58.8%), allergy (47.1%), diarrhea (23.5%), and lymphoma (17.6%). Outpatient visit rates were high overall; ER visits and hospitalization rates were low in both groups, whereas other visit rates were high in ≥12 yrs (Figure 1). Immunoglobulin replacement therapy (IRT)-related HRU accounted for 14.4% and 17.9% of outpatient visits in ≤11 yrs and ≥12 yrs, respectively, and 36.6% and 34.3% of other visits in ≤11 yrs and ≥12 yrs, respectively. Reasons for outpatient visits included enlarged lymph nodes and otitis media in ≤11 yrs and long-term drug therapy, common variable immunodeficiency, and acute sinusitis in ≥12 yrs. IRT was used by 42.9% and 70.6% in ≤11 yrs and ≥12 yrs, respectively. Antibiotic, corticosteroid, and mTor inhibitors were used by 92.9% and 88.2%, 78.6% and 76.5%, and 21.4% and 11.8% in ≤11 yrs and ≥12 yrs, respectively; no rituximab use was observed. [Display omitted] ConclusionsAPDS presents with various complex clinical manifestations requiring intervention. Among patients analyzed, outpatient and other visits were the most commonly used healthcare resources and were often associated with respiratory disease and immunodeficiency management. To our knowledge, this is the first study to assess HRU among patients with APDS using administrative claims data.

Epidemiology and patient journey of Rett syndrome in the United States: a real-world evidence study

BackgroundRett syndrome (RTT) is a neurodevelopmental disorder that almost exclusively affects females and is associated with high clinical burden. However, literature characterizing the real-world journey of patients with RTT is limited. This study provided an overview of the epidemiology, patient characteristics, clinical manifestations, healthcare resource utilization (HRU), costs, and treatment patterns of patients with RTT in the US.MethodsIQVIA™ Medical Claims Data and Longitudinal Prescription Data (11/01/2016–10/31/2019) were used to identify female patients with RTT, with the first observed diagnosis defined as the index date. Annual incidence and prevalence of RTT were assessed over the entire study period; clinical manifestations, all-cause and RTT-related HRU and costs, and treatment patterns were evaluated during the observation period—from the index date to end of clinical activity or end of data availability, whichever occurred first. Results were further stratified into pediatric (< 18 years) and adult (≥ 18 years) subgroups.ResultsIn 2019, prevalence and incidence of RTT was 0.32 and 0.23 per 10,000 enrollees, respectively. Among 5,940 female patients (pediatric: 3,078; adult: 2,862) with mean observation period of 2.04 years, the most prevalent clinical manifestations were neurological disorders (72.8%), gastrointestinal/nutritional disorders (41.9%), and orthopedic disorders (34.6%). The incidence rate of all-cause HRU was 44.43 visits per-patient-per-year and RTT-related HRU comprised 47% of all-cause HRU. Mean all-cause healthcare costs were $40,326 per-patient-per-year, with medical costs driven by home/hospice care visits, therapeutic services, outpatient visits, and inpatient visits. RTT-related healthcare costs comprised 45% of all-cause healthcare costs. The most prevalent supportive therapy and pharmacologic agent were feeding assistance (37.9%) and antiepileptic drugs (54.8%), respectively. Trends were similar by subgroup; although, rates of HRU were generally higher among pediatric patients relative to adult patients (all-cause: 52.43 and 35.86, respectively), which translated into higher mean healthcare costs (all-cause: $45,718 and $34,548, respectively).ConclusionsPatients with RTT have substantial disease burden, including prevalent clinical manifestations, high rates of HRU and annual healthcare costs, and reliance on pharmacologic and supportive therapies. These findings underscore the unmet need for effective therapies to target the multifactorial manifestations of RTT.

Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants

Background: The effect of gestational age (GA) on comorbidity prevalence, healthcare resource utilization (HCRU), and all-cause costs is significant for extremely premature (EP) infants in the United States. Objectives: To characterize real-world patient characteristics, prevalence of comorbidities, rates of HCRU, and direct healthcare charges and societal costs among premature infants in US Medicaid programs, with respect to GA and the presence of respiratory comorbidities. Methods: Using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes, diagnosis and medical claims data from 6 state Medicaid databases (1997-2018) of infants born at less than 37 weeks of GA (wGA) were collected retrospectively. Data from the index date (birth) up to 2 years corrected age or death, stratified by GA (EP, ≤28 wGA; very premature [VP], &gt;28 to &lt;32 wGA; and moderate to late premature [M-LP], ≥32 to &lt;37 wGA), were compared using unadjusted and adjusted generalized linear models. Results: Among 25 573 premature infants (46.1% female; 4462 [17.4%] EP; 2904 [11.4%] VP; 18 207 [71.2%] M-LP), comorbidity prevalence, HCRU, and all-cause costs increased with decreasing GA and were highest for EP. Total healthcare charges, excluding index hospitalization and all-cause societal costs (US dollars), were 2 to 3 times higher for EP than for M-LP (EP $74 436 vs M-LP $27 541 and EP $28 504 vs M-LP $15 892, respectively). Conclusions: Complications of preterm birth, including prevalence of comorbidities, HCRU, and costs, increased with decreasing GA and were highest among EP infants during the first 2 years in this US analysis.

Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants.

Background: The effect of gestational age (GA) on comorbidity prevalence, healthcare resource utilization (HCRU), and all-cause costs is significant for extremely premature (EP) infants in the United States. Objectives: To characterize real-world patient characteristics, prevalence of comorbidities, rates of HCRU, and direct healthcare charges and societal costs among premature infants in US Medicaid programs, with respect to GA and the presence of respiratory comorbidities. Methods: Using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes, diagnosis and medical claims data from 6 state Medicaid databases (1997-2018) of infants born at less than 37 weeks of GA (wGA) were collected retrospectively. Data from the index date (birth) up to 2 years corrected age or death, stratified by GA (EP, ≤28 wGA; very premature [VP], >28 to <32 wGA; and moderate to late premature [M-LP], ≥32 to <37 wGA), were compared using unadjusted and adjusted generalized linear models. Results: Among 25 573 premature infants (46.1% female; 4462 [17.4%] EP; 2904 [11.4%] VP; 18 207 [71.2%] M-LP), comorbidity prevalence, HCRU, and all-cause costs increased with decreasing GA and were highest for EP. Total healthcare charges, excluding index hospitalization and all-cause societal costs (US dollars), were 2 to 3 times higher for EP than for M-LP (EP $74 436 vs M-LP $27 541 and EP $28 504 vs M-LP $15 892, respectively). Conclusions: Complications of preterm birth, including prevalence of comorbidities, HCRU, and costs, increased with decreasing GA and were highest among EP infants during the first 2 years in this US analysis.

EE427 Annual Cost Savings From the Use of Empagliflozin Over DPP4I in Select Asia-Pacific Countries – Estimates Based on the Emprise East Asia Study

The recently published findings of the EMPRISE East Asia study that looked at patients with type 2 diabetes mellitus (T2DM) in Japan, South Korea, and Taiwan demonstrated that empagliflozin treatment was associated with lower inpatient care needs and other healthcare resource utilization (HRU) than dipeptidyl peptidase-4 inhibitors (DPP4i) in routine clinical practice. The aim of this analysis was to estimate the annual costs savings arising from the reduction in the rates of HRU arising from the use of empagliflozin over DPP4i in select Asia-Pacific countries (Australia, Indonesia, Malaysia, Philippines, Thailand and Vietnam) based on the EMPRISE East Asia study findings.

Real-World Effectiveness and Economic Impact Associated with Chimeric Antigen Receptor T-Cell Therapy Among Older Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma in US

Introduction: Chimeric antigen receptor (CAR) T-cell therapy has changed the treatment landscape of relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). However, there are still limited real-world evidence of survival outcomes from CAR T-cell therapy in older patients with DLBCL. The aim of the study was to examine the outcomes and economic impact among older patients with DLBCL receiving CAR T-cell therapy in a real-world setting in the United States. Methods: Using administrative medical and pharmacy claims from the 100% Medicare Fee-for-Service database, older patients (≥65) with at least one inpatient or two outpatient claims with a diagnosis of DLBCL between 4/1/2016 and 12/1/2020, followed by at least one claim for the administration of CAR T-cell therapy between 1/1/2018 and 12/31/2020 were selected. Patients with evidence of clinical trial participation were excluded. Bridging therapies were reported based on the presence of therapy in the 4-week period preceding the infusion. Outcomes included progression-free survival (PFS) and overall survival (OS). The initiation of any subsequent treatment was used as a proxy for progression. Additionally, healthcare resource utilization (HrU) and total costs within the 90-day period following the infusion were examined among patients with ≥90 days of enrollment post-infusion. The proportion of patients receiving inpatient care, emergency room (ER) services, and outpatient services post CAR T-cell therapy, were reported by location of CAR T-cell therapy administration (inpatient vs. outpatient). Descriptive analyses included means, medians, and standard deviations (SD) for continuous variables, and frequencies and proportions for categorical variables. PFS and OS were analyzed using Kaplan-Meier curves, calculated from infusion date, and tested for significance using the Log-Rank test. Select analyses were further stratified by age category (65-69; 70-74; 75+ years). Results: A total of 551 patients with R/R DLBCL receiving CAR T-cell therapy were included, with mean age of 72.2 and 173 patients (31.4%) aged ≥ 75. A total of 262 patients (47.5%) received bridging therapy (Table 1). The oldest patients (≥75) had shorter median PFS (160 days) and OS (403 days), compared to patients aged 70-74 who had median PFS (379 days) and OS (603 days) and patients aged 65-69 who had a median PFS (194 days) and OS (518 days) (Figure 1). The 12-month failure rate based on PFS was highest in patients aged ≥ 75 at 66%, compared to patients aged 70-74 at 48% and patients aged 65-69 at 57% (p = 0.001). The majority of patients received CAR T-cell infusion in an inpatient setting (n=456, 82.8%), with an average length of stay 21.4 days. Within 90 days following infusion, 28.7% of patients who received CAR T-cell therapy in an inpatient setting presented a re-hospitalization, 30.3% had an ER visit, and all patients had ≥1 outpatient visit, with a mean of 17.4 outpatient visits. Among patients who received CAR T-cell therapy in an outpatient setting, 41.8% were hospitalized later, 31.6% had an ER visit, and all patients had ≥1 outpatient visit, with a mean of 19.0 outpatient visits. Similar HrU rates were observed across age groups, except hospitalization rate, which was lowest among patients with age ≥ 75. Total median cost within 90 days from the infusion date (inclusive of CAR T-cell infusion) was $352,572 and was similar across all age groups (Table 1). Conclusion: This study is one of the largest real-world studies to date of patients with R/R DLBCL receiving CAR T-cell therapy leveraging Medicare claims data. Older patients in this study appeared to have shorter PFS and OS compared to clinical trials, with the worst outcomes observed in patients aged 75+. Similar costs within the 90-day period post CAR T-cell therapy were observed across age groups, and intensive use of HrU was noted. The results provide important reference to consider real-world healthcare utilization and cost effective treatment approaches in older patients with R/R DLBCL. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Reductions in acute medication use and healthcare resource utilization in patients with chronic migraine: a secondary analysis of a phase 3, randomized, double-blind, placebo-controlled study of galcanezumab with open-label extension (REGAIN)

Aims To analyze secondary objectives of the REGAIN study related to acute headache medication use and healthcare resource utilization (HCRU) in patients with chronic migraine treated with galcanezumab, a monoclonal antibody to calcitonin gene-related peptide. Methods Adults with chronic migraine (N = 1,113) were randomized (2:1:1) and treated with double-blind monthly injections of placebo, galcanezumab-120 mg, or galcanenzumab-240 mg for 3 months, followed by a 9-month open-label extension with 120 or 240 mg/month galcanezumab. Headache and medication information was collected by daily eDiary. HCRU was reported for the 6 months before randomization, monthly thereafter, and converted to rate per 100-patient-years. Results At baseline, 63–64% of patients met criteria for acute headache medication overuse. At Month 3, incidence of headache medication overuse in the galcanezumab groups (33% and 33%) was significantly lower than in the placebo group (46%, both p < .001) and was 16% and 23% in the previous-galcanezumab groups at Month 12. From a baseline of 14.5 to 15.5, reduction in mean number of monthly migraine headache days with acute headache medication use was also significantly greater in the galcanezumab groups at Month 3 (−4.2 and −4.9) than in placebo (−2.6, both p < .001), with reductions of −6.8 and −7.6 in the previous-galcanezumab groups at Month 12. Migraine-specific HCRU rates decreased for all groups, with no significant between-group differences at Month 3. At Month 12, in the two previous-galcanezumab groups, emergency room visits decreased by 58% and 75%, hospital admissions by 100%, and healthcare professional visits by 54% and 67%. Limitations Only 3 months of double-blind, placebo-controlled data, a longer HCRU recall period for baseline than postbaseline, and patients receiving care in the clinical trial itself, may limit generalizability. Conclusions Treatment with galcanezumab resulted in significant reductions in headache medication overuse and migraine headache days requiring acute medication use, with notable reductions in migraine-specific HCRU.