Per Patient Per Year Research Articles

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.

Clinical burden and healthcare resource utilization associated with managing sickle cell disease with recurrent vaso-occlusive crises in France

Objective This retrospective, real-world claims database analysis described the clinical burden and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in France. Methods The French National Health Data System database (système national des données de santé) was used to identify eligible patients from January 1, 2012, to March 1, 2019. Inclusion criteria were a SCD diagnosis, ≥2 VOCs/year for ≥2 consecutive years following the diagnosis, and ≥1 year of follow-up data to March 1, 2020. Patients with hereditary persistence of fetal hemoglobin or hematopoietic stem cell transplant in their medical records were excluded. Clinical complications, mortality, treatment use, and HCRU were evaluated during follow-up. Results Overall, 4602 patients with SCD with recurrent VOCs were eligible; their mean (standard deviation [SD]) age was 19.8 (13.5) years, and 51.8% were female. Patients experienced a mean (SD) of 3.82 (3.57) VOCs per patient per year (PPPY). Prevalent complications were anemia or leukocytosis (44.1%), infections (42.0%), and organ failure (38.2%). In total, 101 (2.2%) patients died during follow-up (mean age of death [SD]: 39.3 [17.5] years; mortality rate: 0.64 deaths per 100 person-years). Most patients received opioids (89.1%) and hydroxycarbamide (72.8%). Patients had a mean of 5.7 inpatient hospitalizations, 6.0 emergency room visits, 6.6 outpatient visits, and 13.4 outpatient prescriptions PPPY. Conclusions Patients with SCD with recurrent VOCs in France have substantial clinical complications, mortality, and HCRU despite currently available treatment options. Innovative treatments that reduce frequency of or eliminate VOCs are needed to alleviate the burden associated with SCD.

Direct healthcare expenditure on Alzheimer’s disease from healthcare providers’ perspective in Malaysia: a micro-costing approach

Alzheimer’s disease (AD) is an important geriatric disease that creates challenges in health policy planning. There is no previous attempt to quantify the actual direct healthcare cost of AD among older adults in Malaysia. This retrospective observational study with bottom-up micro-costing approach aimed to evaluate the direct healthcare expenditure on AD along with its potential predictors from healthcare providers’ perspective, conducted across six tertiary hospitals in Malaysia. AD patients aged 65 and above who received AD treatment between 1 January 2016 and 31 December 2021 were included. Direct healthcare cost (DHC) of AD was estimated by extracting one-year follow-up information from patient medical records. As a result, 333 AD patients were included in the study. The mean DHC of AD was estimated RM2641.30 (USD 572.45) per patient per year (PPPY) from the healthcare payer’s perspective. Laboratory investigations accounted for 37.2% of total DHC, followed by clinic care (31.5%) and prescription medicine (24.9%). As disease severity increases, annual DHC increases from RM2459.04 (mild), RM 2642.27 (moderate), to RM3087.61 (severe) PPPY. Patients aged 81 and above recorded significantly higher annual DHC (p = 0.003). Such real-world estimates are important in assisting the process of formulating healthcare policies in geriatric care.

Health care resource utilization and direct costs incurred over 24 months after initiating galcanezumab or standard-of-care preventive migraine treatments in the United States.

Health care resource utilization (HCRU) and direct costs incurred over 12 months following initiation of galcanezumab (GMB) or standard-of-care (SOC) preventive migraine treatments have been evaluated. However, a gap in knowledge exists in understanding longer-term HCRU and direct costs. To compare all-cause and migraine-related HCRU and direct costs in patients with migraine initiating GMB or SOC preventive migraine treatments over a 24-month follow-up. This retrospective study used Optum deidentified Market Clarity Data. The study included adults diagnosed with migraine, with at least 1 claim for GMB or SOC preventive migraine therapy (September 2018 to March 2020), with continuous enrollment for 12 months before and 24 months after (follow-up) the index date (date of first GMB or SOC claim). Propensity score (PS) matching (1:1) was used to balance cohorts. All-cause and migraine-related HCRU and direct costs for GMB vs SOC cohorts were reported as mean (SD) per patient per year (PPPY) over a 24-month follow-up and compared using a Z-test. Costs were inflated to 2022 US$. After PS matching, 2,307 patient pairs (mean age: 44.4 years; female sex: 87.3%) were identified. Compared with the SOC cohort, the GMB cohort had lower mean (SD) PPPY all-cause office visits (17.9 [17.7] vs 19.1 [18.7]; P = 0.023) and migraine-related office visits (2.6 [3.3] vs 3.0 [4.7]; P = 0.002) at follow-up. No significant differences were observed between cohorts in other all-cause and migraine-related events assessed including outpatient visits, emergency department (ED) visits, inpatient stays, and other medical visits. The mean (SD) costs PPPY were lower in the GMB cohort compared with the SOC cohort for all-cause office visits ($4,321 [7,518] vs $5,033 [7,211]; P < 0.001) at follow-up. However, the GMB cohort had higher mean (SD) PPPY all-cause total costs ($24,704 [30,705] vs $21,902 [28,213]; P = 0.001) and pharmacy costs ($9,507 [12,659] vs $5,623 [12,605]; P < 0.001) compared with the SOC cohort. Mean (SD) costs PPPY were lower in the GMB cohort for migraine-related office visits ($806 [1,690] vs $1,353 [2,805]; P < 0.001) compared with the SOC cohort. However, the GMB cohort had higher mean (SD) PPPY migraine-related total costs ($8,248 [11,486] vs $5,047 [9,749]; P < 0.001) and migraine-related pharmacy costs ($5,394 [3,986] vs $1,761 [4,133]; P < 0.001) compared with the SOC cohort. There were no significant differences between cohorts in all-cause and migraine-related costs for outpatient visits, ED visits, inpatient stays, and other medical visits. Although total costs were greater for GMB vs SOC following initiation, changes in a few categories of all-cause and migraine-related HCRU and direct costs were lower for GMB over a 24-month follow-up. Additional analysis evaluating indirect health care costs may offer insights into further cost savings incurred with preventive migraine treatment.

Estimating the healthcare burden of Prurigo Nodularis in England: a CPRD database study

Purpose: Prurigo nodularis (PN) is a skin disease characterized by intensely itchy skin nodules and is associated with a significant healthcare resource utilization (HCRU). This study aimed to estimate the HCRU of patients in England with PN overall and moderate-to-severe PN (MSPN) in particular. Methods: This retrospective cohort study used data from the Clinical Practice Research Datalink and Hospital Episode Statistics in England. Patients with Mild PN (MiPN) were matched to patients with MSPN by age and gender for the primary analysis. Patients were enrolled in the study between 1st April 2007 and 1st March 2019. All-cause HCRU was calculated, including primary and secondary care contacts and costs (cost-year 2022). Results: Of 23,522 identified patients, 8,933 met the inclusion criteria, with a primary matched cohort of 2,479 PN patients. During follow up, the matched cohort’s primary care visits were 21.27 per patient year (PPY) for MSPN group and 11.35 PPY for MiPN group. Any outpatient visits were 10.72 PPY and 4.87 PPY in MSPN and MiPN groups, respectively. Outpatient dermatology visits were 1.96 PPY and 1.14 PPY in MSPN and MiPN groups, respectively. Conclusion: PN, especially MSPN, has a high HCRU burden in England, highlighting the need for new and improved disease management treatments.

#2598 Risk of vascular access complications in haemodialysis patients initiating dialysis with arteriovenous fistula or arteriovenous graft

Abstract Background and Aims Patients (pts) with end-stage kidney disease (ESKD) rely on successful vascular access (VA) for continual hemodialysis (HD) treatments. Possible VA complications are numerous and complex, which makes them difficult to study. We developed a comprehensive VA complication metric to evaluate VA complications holistically. The goal of this analysis was to describe the risk of having high VA complications in in-center hemodialysis (ICHD) pts whose initial VA included arteriovenous fistula (AVF) or arteriovenous graft (AVG), and who have been successfully receiving regular dialysis care for at least 2 years. Method Adult ICHD pts, with Charlson Comorbidity Index &amp;lt;8, who initiated treatment at one of the clinics of a large dialysis organization between Jan 2016 and Dec 2021, received continuous treatment for ≥2 years, remained on ICHD until censored, and whose initial VA included an AVF or an AVG were investigated. Pts could be censored by death, transplant, discharge from FKC, or study end. Initial VA was determined by use of either AVF or AVG during ICHD treatments in the 90-120 days after initiation of ICHD. Individual-level vascular access complication rates (VACR) were defined as the sum of a patient's VA complication events divided by their duration of follow-up until censor date. VA complication events include: hospitalization due to VA problems or VA infection, VA infections not requiring hospitalization, VA removal due to infection, symptoms of VA infection, symptoms of thrombosis/stenosis, documented stenosis, documented thrombosis, cannulation issues, bacteremia, sepsis. The threshold for high VACR was the highest quintile values. Logistic regression was used to determine the risk of having a high VACR by demographic and comorbid conditions. Results Analyses included 94,208 pts who met our inclusion criteria. The mean VACR for the population was 2.7 VA complications per patient per year (PPY). Pts whose VACR was in the highest quintile, ≥4.34 PPY, were categorized as having high VACR. Table 1 shows the risk of having high VACR by demographic characteristics and comorbid conditions present at ICHD initiation. In general, we observed lower or neutral risk of having high VACR in the demographic characteristics we examined. However, older patients as well as patients with higher BMI had an increase in risk of having high VACR. We observed significant increased risk of having high VACR in diabetic pts (20%), pts with cerebrovascular disease (35%), pts with COPD (10%), pts with Congestive Heart Failure (21%), patients with dementia (22%), and patients with history of myocardial infarction (26%). Conclusion We observed that being older, having increased BMI, or having several comorbid conditions are associated with increased risk of having high VACR in successful ICHD patients who initiated dialysis with an AVF or AVG. Multivariate analyses to further explore these associations are warranted.

Analysis of Medicare Patients Treated with Pimavanserin versus Other Atypical Antipsychotics: A Cost-Offset Model Evaluating Skilled Nursing Facility Stays and Long-Term Care Admissions in Parkinson's Disease Psychosis.

Patients with Parkinson's disease psychosis (PDP) treated with pimavanserin (PIM) versus other atypical antipsychotics (AAPs) including quetiapine (QUE) may have health-care cost savings due to fewer skilled nursing facility-stays (SNF-stays) and long-term care admissions (LTCA). A decision analytic model was developed using the 2019 Medicare Patient Driven Payment Model (PDPM) to estimate SNF-stays and LTCA associated per-patient- per-year (PPPY) facility and rehabilitation costs among patients that initiated PIM vs QUE or vs other-AAPs (i.e, quetiapine, risperidone, olanzapine, aripiprazole). Model inputs were derived for: (i) annual SNF-stay and LTCA rates from an analysis of Medicare beneficiaries with PDP, and (ii) annual mean rehabilitation and resident care-stay costs from PDPM case-mix adjusted value-based payment rates for 5 rehabilitation components (ie, physical-therapy, occupational-therapy, nursing, speech-language pathology, non-therapy ancillary), and an additional variable-per-diem for room/board services. PPPY costs were estimated from (i) SNF-stay and (ii) LTCA rates multiplied by annual mean costs of stay in 2022 USD. Probabilistic sensitivity analysis (PSA) was performed using 1000 Monte Carlo simulations. Overall SNF-stay rates of 20.2%, 31.4%, and 31.7%, and LTCA rates of 23.2%, 33.8%, 34.6% were observed for PIM, QUE, and other-AAPs, respectively. Based on annual mean costs, PPPY SNF-stay rehabilitation and resident related costs for PIM ($41,808) vs QUE ($65,172) or vs other-AAPs ($65,664), resulted in $23,364 and $23,856 PPPY cost savings, respectively. Similarly, PPPY LTCA rehabilitation and resident related costs for PIM ($47,957) vs QUE ($70,091) or vs other-AAPs ($71,566) resulted in $22,134 and $23,609 PPPY cost-savings for PIM, respectively. PSA suggested PIM would provide cost-savings vs QUE or other-AAPs in >99% of iterations. In this analysis, PIM demonstrated nearly 36% and 32% lower PPPY SNF-stays and LTCA costs, respectively, vs QUE or other-AAPs. Research examining additional cost-offsets (i.e., fewer falls/fractures) associated with SNF-stay or LTCA may be needed.

Association of Osteoporosis-related Healthcare Costs with the Use of Tenofovir Disoproxil Fumarate and Tenofovir Alafenamide in Chronic Hepatitis B Patients: a Population-based National Cohort Study in Korea

Long-term administration of tenofovir disoproxil fumarate (TDF) for chronic hepatitis B (CHB) may lead to bone mineral density loss. Tenofovir alafenamide (TAF) developed to address these concerns. This study aimed to investigate whether there is a significant difference in osteoporosis-related healthcare costs between CHB patients treated with TDF and TAF. This study is a retrospective cohort study using claims data from the Health Insurance Review and Assessment Service (HIRA) covering the entire population in Korea. The cohort included CHB patients treated with TDF or TAF from November 2017 to April 2022. We applied inverse probability of treatment weighting (IPTW) to balance baseline characteristics observed for one-year preceding prescription date. Osteoporosis-related costs per patient per year (PPPY) included all healthcare costs with an osteoporosis diagnosis code including outpatient and hospitalization costs. 7,172 and 3,837 patients were administered TDF and TAF respectively. After IPTW, TDF group had higher outpatient costs ($11.2) compared to TAF group ($6.1), but the difference was not statistically significant (p=0.1001). The total hospitalization cost was $24.6 in TDF group and $9.8 in TAF group, not statistically significant (p=0.1633). This largescale population-based study found no significant difference in osteoporosis-related healthcare costs between CHB patients treated with TDF and TAF.

Patient characteristics, treatment patterns, healthcare resource utilization, and costs among patients diagnosed with neurofibromatosis type 1 with and without plexiform neurofibromas in Japan

Objectives: The objectives of this study were to retrospectively investigate the patient characteristics, treatment patterns, healthcare resource utilization (HCRU), and healthcare costs related to management of neurofibromatosis type 1 (NF1) in Japan. Methods: Cohorts of NF1 patients with or without plexiform neurofibromas (PN) were identified from the Medical Data Vision database in 2008-2019. Baseline characteristics, NF1 medications, HCRU, and associated costs were assessed using descriptive statistics. All-cause HCRU and costs following the first confirmed NF1 diagnosis date were analyzed per patient per year (PPPY) in Japanese Yen (JPY) and United States Dollar (USD). Results: A total of 4,394 NF1 patients without PN and 370 NF1 patients with PN were identified. The mean age was 35.0 and 36.9 years, respectively. The proportion of patients with PN treated with medications was higher than that in patients without PN (except for antirheumatic/immunologic agents). Analgesics/non-steroidal anti-inflammatory drugs were the most frequently prescribed NF1 medications (44.3% and 56.0% in patients without and with PN, respectively), followed by inpatient prescriptions of opioids/opioid-like agents (17.8% and 27.6%, respectively). Inpatient admissions accounted for the highest costs in both cohorts with the average cost PPPY being JPY 2,133,277 (USD 19,861) for patients without PN and JPY 1,052,868 (USD 9,802) for patients with PN. Conclusions: NF1 is treated primarily with supportive care with analgesics/non-steroidal anti-inflammatory drugs being the most frequently prescribed NF1 medications in Japan. Findings underscored the unmet need and substantial economic burden among patients with NF1 and highlighted the need for new treatment options for patients with this disease.

P1188 Epidemiology and direct healthcare costs of patients with Inflammatory Bowel Disease (IBD) in an Asian cohort in the first year following diagnosis

Abstract Background Inflammatory Bowel Disease (IBD) is a chronic condition requiring multi-disciplinary care with significant investigation and treatment burden. Indeed, the global treatment market has exceeded US$ 20 billion and is expected to exceed 27 billion in 2030. We aim to study the epidemiology and direct healthcare costs of newly diagnosed IBD patients in their first year of diagnosis in Singapore General Hospital (SGH) and identify factors associated with high direct healthcare costs. Methods The study included patients identified from a prospectively maintained IBD registry of SGH. We enrolled patients newly diagnosed with IBD from 2015 to 2022. Patients who received prior treatment or investigation at other institutions were excluded. Data on clinical characteristics and direct costs (investigations, medications, clinic visits, hospitalisations and surgeries) were collected. Results A total of 199 patients were enrolled across 2015 - 2022. 96 patients had Ulcerative Colitis (UC) and 103 had Crohn’s disease (CD) (Table 1). There were 26 (20-28) new cases of IBD diagnosed at SGH yearly. Direct healthcare costs of patients in their first year of diagnosis remained steady with an overall 7.1% change in total expenditure from 2015 through 2022. A drop in expenditure happened in 2020 and 2021 due to COVID outbreak and country lockdown. (Fig 1A) The mean cost per patient-year (PPY) was US$12995 for CD US$104345 for UC. 10 (8-11) patients required admission within the first year of diagnosis, with a mean hospitalisation cost PPY of $269717 (± 73771). Overall, inpatient costs accounted for 39.3 % (IQR 34.8 - 44) of yearly expenditure (Fig 1B). A median of 16.7% (14.6-22.1) of patients were started on biologics within the first year of diagnosis. Patients with CD had significantly greater mean expenditure than those with UC on imaging ($1036 vs $328, p = 0.001) and biologics ($1535 vs $603, p = 0.033). Patients above the 75th percentile of mean cost PPY were defined as having high healthcare cost. This was $12265 and $15957 for UC and CD respectively. Factors associated with high costs were hospitalisation (p &amp;lt; 0.001), biologics (p = 0.001), surgery (p = 0.01) for both UC and CD. UC extent, CD behaviour, history of smoking and age&amp;gt; 40 were not predictive of high costs. Conclusion Patients with CD have greater expenditure compared to patients with UC in the first year following diagnosis. Biologics use, surgery and hospitalisation are significant drivers of costs. The proportion of newly diagnosed patients requiring admission and their corresponding costs have remained consistent during our period of study. These findings encourage early identification of at-risk patients with institution of treatment to minimise hospitalisation and surgical burden.

Incremental Risk of Adverse Events with Oral Janus Kinase Inhibitor Use in Atopic Dermatitis and Other Indications: A Systematic Review and Meta-Analysis

Introduction: The number of treatment opportunities for patients with moderate-severe atopic dermatitis (AD) is increasing, however with varying treatment safety profiles. The safety profile of oral JAKis remains a concern because of their ubiquitous mechanism of action inhibiting many cytokines. Safety warnings for these products have combined data across indications, making it difficult to assess risk for specific populations.&#x0D; Objective: We aimed to estimate the incremental risk of pre-specified adverse events (AEs) per patient-year (PY) of oral JAKi compared with standard of care (SoC), incl. topical corticosteroids, biologic agents, and other treatments, among those with AD or with other diagnoses.&#x0D; Methods: A systematic literature review was conducted on 17 preselected AEs based on randomized controlled trials of oral JAKis.&#x0D; Meta-analyses (MA) estimated the incidence rate difference (IRD) for each AE between oral JAKis and SoC, both in AD and non-AD populations. Number needed to harm (NNH) was calculated as the inverse of the IRD for each AE. &#x0D; Results: We identified 82 studies, including 19 in AD population, for use in the MA.&#x0D; In the AD population, the estimated NNH in PY for acne was 5, indicating 1 additional acne event for every 5 patients treated with oral JAKis vs SoC per year. Moreover, the NNH was 33 for herpes zoster, 62 for anemia, 94 for serious adverse events (SAEs), and 452 for malignancies. In the non-AD population, the NNH was 52 for HZ, 56 for hyperlipidemia, 58 for SAEs, 113 for anemia, 153 for acne, and 254 for malignancies.&#x0D; Conclusions: The MA identified an increased risk with oral JAKis compared with SoC for multiple AEs, spanning from less to more severe AE, including malignancies in both AD and non-AD populations. As safety risks are observed in both the AD and non-AD populations, the use of oral JAKis should be carefully considered.

Impact of combined pulmonary fibrosis and emphysema on lung cancer risk and mortality in rheumatoid arthritis: A multicenter retrospective cohort study.

Combined pulmonary fibrosis and emphysema (CPFE) is a syndrome characterized by the coexistence of emphysema and fibrotic interstitial lung disease (ILD). The aim of this study was to examine the effect of CPFE on lung cancer risk and lung cancer-related mortality in patients with rheumatoid arthritis (RA). We conducted a multicenter retrospective cohort study of patients newly diagnosed with lung cancer at five community hospitals between June 2006 and December 2021. Patients were followed until lung cancer-related death, other-cause death, loss to follow-up, or the end of the study. We used the cumulative incidence function with Gray's test and Fine-Gray regression analysis for survival analysis. A total of 563 patients with biopsy-proven lung cancer were included (82 RA patients and 481 non-RA patients). The prevalence of CPFE was higher in RA patients than in non-RA patients (40.2% vs.10.0%) at lung cancer diagnosis. During follow-up, the crude incidence rate of lung cancer-related death was 0.29 and 0.10 per patient-year (PY) in RA and non-RA patients, and 0.32 and 0.07 per PY in patients with CPFE and patients without ILD or emphysema, respectively. The estimated death probability at 5 years differed between RA and non-RA patients (66% vs. 32%, p<0.001) and between patients with CPFE and patients without ILD or emphysema (71% vs. 24%, p<0.001). In addition to clinical cancer stage and no surgery within 1 month, RA and CPFE were identified as independent predictive factors for increased lung cancer-related mortality (RA: adjusted hazard ratio [HR], 2.49; 95% confidence interval [CI], 1.65-4.76; CPFE: adjusted HR 2.01; 95% CI 1.24-3.23). RA patients with lung cancer had a higher prevalence of CPFE and increased cancer-related mortality compared with non-RA patients. Close monitoring and optimal treatment strategies tailored to RA patients with CPFE are important to improve the poor prognosis of lung cancer.

Healthcare Resource Utilisation of Severe Uncontrolled T2low and Non-T2low Asthma in Finland During 2018-2021.

Patients with asthma and low levels of type 2 inflammatory biomarkers (T2 low) have limited effective treatment options. Such biomarkers include eg blood eosinophils (b-eos) and fractional exhaled nitric oxide (FeNO). The healthcare resource utilisation (HCRU) of severe uncontrolled T2 low asthma remains unexplored. Thus, this study aimed to estimate the HCRU of T2 low and non-T2 low severe uncontrolled asthma patients using real-world data in Finland. Adult patients with an asthma diagnosis during baseline (2012-2017) at the pulmonary department of Turku University Hospital were included and followed during 2018-2021, or until death. Total HCRU costs and respiratory-related HCRU costs were evaluated. The main drivers for the HCRU and costs were assessed with gamma and negative binomial regression models. Of the severe uncontrolled asthma patients with T2 status available, 40% (N=66) were identified with T2 low and 60% (N=103) with non-T2 low asthma. The average cumulative cost per patient was similar in patients with T2 low compared with non-T2 low, with all-cause costs cumulating in four years of follow-up to 37,524€ (95% CI: 27,160, 47,888) in T2 low compared to 34,712€ (25,484, 43,940) in non-T2 low. The corresponding average cumulative respiratory-related costs were 5178€ (3150, 7205) in T2 low compared to 5209€ (4104, 6313) in non-T2 low. Regression modelling identified no differences between the T2-status groups when assessing all-cause healthcare costs per patient-year (PPY). On the other hand, the regression modelling predicted more inpatient days PPY for severe uncontrolled patients with T2 low status compared to the patients with non-T2 low status. Patients with uncontrolled severe T2 low asthma use equal healthcare resources as corresponding non-T2 low patients. This study brought new insights into the HCRU of severe uncontrolled asthma patients per T2 status, which has not previously been investigated.

Treatment patterns and economic burden of bacterial vaginosis among commercially insured women in the USA.

Aim: Bacterial vaginosis (BV) is a common vaginal dysbiosis associated with adverse clinical sequelae, most notably, increased risk of sexually transmitted infections (STIs). The aims of this study were to estimate the frequency of BV recurrence, treatment patterns, other gynecological (GYN) conditions, and the associated healthcare resource utilization (HCRU) and costs among commercially insured patients in the USA. Patients & methods: Female patients aged 12-49years with an incident vaginitis diagnosis and ≥1 pharmacy claim for a BV medication (fungal treatment only excluded) were selected from the Merative™ MarketScan commercial database (2017-2020). During a minimum 12-month follow-up, additional treatment courses, treatment patterns, frequency of other GYN conditions, and HCRU and costs were assessed. Generalized linear models were used to identify baseline predictors of total all-cause healthcare costs and number of treatment courses. Results: The study population included 140,826 patients (mean age: 31.5years) with an incident vaginitis diagnosis and ≥1 BV medication claim. During the follow-up, 64.2% had 1 treatment course, 22.0% had 2, 8.1% had 3, and 5.8% had ≥4; 35.8% had a BV recurrence (≥2 BV medication claims). The most commonly prescribed BV medication was oral metronidazole (73.6%). Approximately 12% (n=16,619) of patients had a new diagnosis of another GYN condition in the follow-up; 8.2% had a new STI, which were more common among patients with ≥4 treatment courses (12.9%). During follow-up, total all-cause healthcare costs averaged $8987 per patient per year (PPPY) of which $470 was BV-related. BV-related healthcare costs increased from $403 PPPY among those with 1 treatment course to $806 PPPY among those with ≥4 with nearly half the costs attributed to outpatient office visits. Conclusion: BV recurrence among this population represented a substantial clinical and healthcare economic burden warranting improvements in women's healthcare.

Budget impact of oral nirmatrelvir/ritonavir in adults at high risk for progression to severe COVID-19 in the United States.

Nirmatrelvir/ritonavir (NMV/r) is indicated for the treatment of mild-to-moderate COVID-19 in adults who are at high risk for progression to severe COVID-19. NMV/r has also been authorized for emergency use by the US Food and Drug Administration for the treatment of mild-to-moderate COVID-19 in pediatric patients (aged 226512 years and weighing at least 40 kg) who are at high risk for progression to severe COVID-19. Understanding the budget impact of introducing NMV/r for the treatment of adults with COVID-19 is of key interest to US payers. To estimate the annual budget impact of introducing NMV/r in a US commercial health plan setting in the current Omicron COVID-19 era. A budget impact model was developed to assess the impact of NMV/r on health care costs in a hypothetical 1-million-member commercial health insurance plan over a 1-year period in the US population; clinical and cost inputs were derived from published literature with a focus on studies in the recent COVID-19 era that included vaccinated population and predominance of the Omicron variant. In the base-case analysis, it was assumed the only effect of NMV/r was a reduction in incidence (not severity) of hospitalization or death; its potential effect on post-COVID conditions was assessed in a scenario analysis. Outcomes included the number of hospitalizations, total cost, per patient per year (PPPY) costs, and per member per month (PMPM) costs. Sensitivity and scenario analyses were conducted to assess uncertainty around key model inputs. An estimated 29,999 adults were eligible and sought treatment with oral antiviral for COVID-19 over 1 year. The availability of NMV/r was estimated to reduce the number of hospitalizations by 647 with a total budget impact of $2,733,745, $91 PPPY, and $0.23 PMPM. NMV/r was cost saving when including post-COVID conditions with a -$1,510,780 total budget impact, a PPPY cost of -$50, and a PMPM cost of -$0.13. Sensitivity analyses indicated results were most sensitive to the risk of hospitalization under supportive care, risk of hospitalization with NMV/r treatment and cost of NMV/r. Treatment with NMV/r in the current COVID-19 era is estimated to result in substantial cost offsets because of reductions in hospitalization and modest budget impact to potential overall cost savings.

Incremental health care resource utilization and costs associated among patients with Parkinson's disease psychosis and incident dementia: An analysis of medicare beneficiaries.

Real-world evidence examining the incremental health care resource use (HCRU) and cost burden of incident dementia among patients with Parkinson's disease psychosis (PDP) are needed within the United States (US). To compare HCRU and cost burden between PDP patients with incident dementia (PDP+D) versus without incident dementia (PDP). A retrospective analysis of inpatient (Part A), outpatient (Part B), and prescription drug (Part D) claims from the 100% Medicare sample was conducted to compare PDP+D patients versus PDP patients between 01/01/14-12/31/18. Patients with a diagnosis of dementia, psychosis, secondary parkinsonism, or other psychotic disorders, during 12-month pre-index were excluded. Patients in both groups were matched using 1:1 propensity score matching (PSM) methodology using 31 variables (age, sex, race, region and 27 Elixhauser comorbidity characteristics). Differences in 12-month post-index HCRU rates and mean per patient per year (PPPY) costs for all-cause inpatient (IP) hospitalizations, and by type of IP stay (i.e., short-term [ST-stay], skilled nursing facility [SNF-stay] and long-term [LT-stay]) were analyzed via logistic and gamma log-link regression models. Of the 12,484 patients who met our study criteria, 1855 PSM-matched cohorts were identified. Mean age, gender, and comorbidities were similar in PSM groups. Approximately, 50.3% with PDP+D reported ≥1 all-cause IP hospitalizations versus 36.0% with PDP (p<0.05) during 12-month follow-up. Specifically, all-cause ST-stay, SNF-stay, and LT-stay among PDP+D versus PDP patients were: 45.2% versus 35.7%, 28.3% versus 15.7%, and 8.5% versus 6.0% (p<0.05), respectively. Psychiatric-related ST-stay, SNF-stay, and LT-stay among PDP+D versus PDP patients were: 12.3% versus 9.0%, 7.5% versus 3.4%, and 2.4% versus 1.2% (p<0.05), respectively. Mean PPPY all-cause IP hospitalization costs for PDP+D patients versus PDP patients was $17,891 (±29,882) versus $11,599 (±$25,247) (p<0.05). Patients with PDP+D experience significantly higher all-cause and psychiatric-related IP hospitalizations, including ST-stays, LT stays, and SNF stays. They also had 54% greater mean PPPY IP hospitalization costs versus PDP patients.

The economic value of insulin glargine 300 U/mL (Gla-300) in people ≥18 years of age with type 2 diabetes mellitus: a value-based economic model from a U.S. payer perspective

AimsThis study aimed to evaluate the value and affordability of insulin glargine 300 U/mL (Gla-300) in a budget impact model from a United States (U.S.) payer perspective by leveraging recent real-world evidence (RWE) studies and incorporating the recent insulin price caps where applicable.Materials and methodsAn economic model for a hypothetical one million U.S. health-plan population was developed to assess the budgetary impact of therapeutic interchanges in either direction between the two long- and longer-acting basal insulins (BIs) for patients with type 2 diabetes over a three-year model horizon. The utilization of long-acting BIs, longer-acting BIs, biosimilar BIs, and insulin degludec (IDeg-100) were informed by IQVIA data and internal forecasting at Sanofi. The DELIVER-2 and DELIVER-naïve studies provided healthcare resource utilization (HCRU) parameters. In the model base case, 24% of patients switched from long-acting BIs to insulin glargine biosimilars, IDeg-100, and other longer-acting BIs (Gla-300) by projected year 3.ResultsThe base case total costs were $10,145 per patient per year (PPPY) in year 3 for the cumulative population. When all patients switched to Gla-300, the total costs in year 3 were $8,799, reflecting a net savings of -$660 PPPY compared to the budget increase of $686 PPPY in the base case. However, the longer-acting to long-acting BIs reversal scenario demonstrated a budgetary decrease of $676 PPPY over the model horizon. The reduction in incremental PPPY cost of $93 was observed using net drug costs rather than wholesale acquisition costs (WAC).LimitationsThe market shares for years 1–3 were based on expectations supported by the clinicians’ expert opinions and were not obtained from real-world data.ConclusionsThe economic value of increased utilization of Gla-300 was driven by the reduction in HCRU, costs and market shares assumptions. Budgetary reductions were achieved by switching patients from long-acting BIs to Gla-300.

Dravet syndrome: A systematic literature review of the illness burden.

We performed a systematic literature review and narrative synthesis according to a pre-registered protocol (Prospero: CRD42022376561) to identify the evidence associated with the burden of illness in Dravet syndrome (DS), a developmental and epileptic encephalopathy characterized by drug-resistant epilepsy with neurocognitive and neurobehavioral impairment. We searched MEDLINE, Embase, and APA PsychInfo, Cochrane's database of systematic reviews, and Epistemonikos from inception to June 2022. Non-interventional studies reporting on epidemiology (incidence, prevalence, and mortality), patient and caregiver health-related quality of life (HRQoL), direct and indirect costs and healthcare resource utilization were eligible. Two reviewers independently carried out the screening. Pre-specified data were extracted and a narrative synthesis was conducted. Overall, 49 studies met the inclusion criteria. The incidence varied from 1:15 400-1:40 900, and the prevalence varied from 1.5 per 100 000 to 6.5 per 100 000. Mortality was reported in 3.7%-20.8% of DS patients, most commonly due to sudden unexpected death in epilepsy and status epilepticus. Patient HRQoL, assessed by caregivers, was lower than in non-DS epilepsy patients; mean scores (0 [worst] to 100/1 [best]) were 62.1 for the Kiddy KINDL/Kid-KINDL, 46.5-54.7 for the PedsQL and 0.42 for the EQ-5D-5L. Caregivers, especially mothers, were severely affected, with impacts on their time, energy, sleep, career, and finances, while siblings were also affected. Symptoms of depression were reported in 47%-70% of caregivers. Mean total direct costs were high across all studies, ranging from $11 048 to $77 914 per patient per year (PPPY), with inpatient admissions being a key cost driver across most studies. Mean costs related to lost productivity were only reported in three publications, ranging from approximately $19 000 to $20 000 PPPY ($17 596 for mothers vs $1564 for fathers). High seizure burden was associated with higher resource utilization, costs and poorer HRQoL. The burden of DS on patients, caregivers, the healthcare system, and society is profound, reflecting the severe nature of the syndrome. Future studies will be able to assess the impact that newly approved therapies have on reducing the burden of DS.

SAT233 Clinical And Economic Burden Of Postsurgical Chronic Hypoparathyroidism: A US Medicare Retrospective Analysis

Abstract Disclosure: W. Noori: Employee; Self; Ascendis Pharma, Inc. S. Inguva: Employee; Self; OPEN Health. Other; Self; Research Funding (institution): Ascendis. C.T. Sibley: Employee; Self; Ascendis Pharma, Inc. V. Chirikov: Employee; Self; OPEN Health. Stock Owner; Self; OPEN Health. Other; Self; Research Funding (institution): Ascendis. A.R. Smith: Employee; Self; Ascendis Pharma, Inc. Background: Hypoparathyroidism is a rare endocrine disease characterized by insufficient parathyroid hormone (PTH) production and is associated with substantial burden of illness. Individuals with hypoparathyroidism often report physical, emotional, and cognitive symptoms indicating impaired health-related quality of life (HRQoL). Objective: To describe demographics, clinical characteristics, healthcare resource utilization (HCRU), and costs among individuals with postsurgical chronic hypoparathyroidism compared to individuals without hypoparathyroidism in Medicare Fee-For-Service (FFS). Methods: Adults with newly diagnosed postsurgical chronic hypoparathyroidism were identified from the Medicare 100% Limited Data Set between July 1, 2017, to March 31, 2020. All had a confirmed diagnosis within 6-12 months after index diagnosis. Individuals were required to be continuously enrolled for ≥6 months pre- and ≥12 months post-index for baseline and follow-up assessments. Those with a prior hypoparathyroidism diagnosis in the baseline period were excluded. A random sample of enrollees without hypoparathyroidism was synthetically assigned an index date of diagnosis to ensure similar baseline and follow-up periods as individuals with postsurgical chronic hypoparathyroidism. Baseline demographics and comorbidities were compared descriptively. All-cause HCRU and costs during baseline and follow-up were evaluated. All costs were inflated to 2021 US dollars. Results: Individuals with postsurgical chronic hypoparathyroidism (N=1,166) were older than those without hypoparathyroidism (N=11,258) (median age of 69 vs 64 years) and more were female (76% vs 57%); they also had higher Charlson Comorbidity Index scores at baseline (3.24 vs 0.73) and a higher prevalence of moderate or severe renal disease (28.8% vs 5.6%), nephrolithiasis (8.3% vs 1.0%), urinary tract infections (9.9% vs 2.2%), hospitalizations for infections (11.9% vs 2.5%), congestive heart failure (13.1% vs 3.0%), among others. Over a median follow-up of 30 months, mean [SD] all-cause medical costs per patient per year (PPPY) were significantly higher among individuals with postsurgical chronic hypoparathyroidism ($199,297 [$443,294] vs $61,897 [$210,825]). This difference was largely attributable to higher all-cause medical utilization among individuals with postsurgical chronic hypoparathyroidism (22.0 vs 11.6 inpatient days PPPY; 15.3 vs 5.8 outpatient visits PPPY). Individuals with postsurgical chronic hypoparathyroidism also had higher mortality compared to those without hypoparathyroidism (HR=2.75 (95% CI: 2.15-3.51). Conclusions: The clinical and economic burden of individuals with postsurgical chronic hypoparathyroidism in Medicare FFS is substantial, highlighting the need for innovative treatment options to replace the missing PTH hormone. Presentation: Saturday, June 17, 2023

DO GENOMIC ALTERATIONS INflUENCE THE DEVELOPMENT OF BRAIN METASTASES IN ADVANCED AND METASTATIC NON-SMALL CELL LUNG CANCER? A SYSTEMATIC REVIEW AND META-ANALYSIS

Abstract AIMS Establish prevalence at diagnosis, and incidence of BM in patients with advanced and metastatic non-small cell lung cancer (NSCLC), stratified by genomic alterations. METHOD A systematic review and meta-analysis was conducted in accordance with PRISMA guidelines (PROSPERO ID CRD42022315915). Three databases were searched (Search date 30th April 2022). Prevalence at diagnosis, and per-patient year incidence of brain metastases were obtained, including patients with EGFR, ALK, KRAS, ROS1, RET, and other genomic alterations. Pooled incidence rates were calculated using random effects models. RESULTS Of 960 articles identified, 65 were included (24,784 patients). The combined BM prevalence at diagnosis was 28.6% (45 studies, 95% Confidence Interval [CI] 26.1-31.0). Median follow-up was 24 months (Interquartile range [IQR] 16-36 months). The combined incidence of BM development was 10.8% per year (40 studies, 95% CI 9.2- 12.6). The per-year incidence of new BM was 15.6%/year in the EGFR +ve group (16 studies, 95% CI 11.4-21.2), 17.0% in the ALK+ve group (7 studies, 95% CI 11.3-25.8), 9.8% in the KRAS +ve group (4 studies, 95% CI 5.5-17.5), 12.5% in the ROS1 +ve group (3 studies, 95% CI 5.7-27.6), and 11.8% in the RET +ve group (2 studies, 95% CI 8.0-17.5). Combined overall prevalence was 55.0% (21 studies, IQR 44.2-67.8). CONCLUSIONS This systematic review indicates a high rate of brain metastases in NSCLC and is the first and only study to describe incidence, stratified by genomic alterations. This will have a major clinical impact by stratifying brain imaging at staging and follow-up by patient subgroup, particularly in ALK and EGFR mutations.