All-cause Medical Costs Research Articles

Immunotherapy in combination with chemotherapy vs. immunotherapy alone for advanced non-small cell lung cancer and programmed death ligand 1 score

IntroductionLittle is known about the effectiveness of immunotherapy alone or with chemotherapy for patients with non-small cell lung cancer (NSCLC) and programmed death ligand 1 (PD-L1) expression <50 %. We examined the outcomes of PD-L1 therapy vs. PD-L1 therapy in combination with chemotherapy as first-line treatment among NSCLC patients with PD-L1 score <50 %. MethodsWe used administrative claims and prior authorization data of a national insurer from November 2015 to July 2021. We selected patients with Stage IIIb/IV NSCLC and PD-L1 expression <50 %. Each patient was required to have ≥1 claim of a PD-L1 or PD-1 inhibitor. Treatment groups were propensity-score matched 1:1 on baseline characteristics. We measured PD-L1 therapy duration, incident immune-related adverse events (irAEs), healthcare utilization, costs, and overall survival (OS). ResultsIn the matched sample totaling 176 patients, mean duration of PD-L1 therapy was similar (4.1 [SD 3.3] months combination vs. 4.0 [SD 4.9] months monotherapy, p = 0.800). IrAEs were similar, both for FDA-recognized irAEs (48.9 % combination, 48.9 % monotherapy, p = 0.710) and other types (34.1 % combination, 39.8 % monotherapy, p = 0.473). The combination group had more all-cause inpatient stays, ER visits, and outpatient visits (all p < 0.001). Total adjusted all-cause medical cost was $112,833 (95 % CI $5,548-$251,973) higher for combination therapy. We saw no difference in OS (adjusted hazard ratio 1.09 [95 % CI 0.72–1.65]). ConclusionThis study found no difference in adverse drug effects or survival between PD-L1 monotherapy compared to combination therapy for patients with Stage IIIb/IV NSCLC and PD-L1 expression <50 %, though the combination therapy cohort had higher healthcare utilization and costs. MicroAbstractUse of immunotherapy alone or combined with chemotherapy for patients with non-small cell lung cancer and programmed death ligand 1 expression <50 % is understudied. Our observational study using claims and authorization data from a matched sample of 176 patients found no difference in survival or the rate of adverse drug effects between groups, although the chemo-immunotherapy cohort generated higher overall healthcare costs.

An Evaluation of a Care Coaching and Provider Referral Intervention for Behavioral Health Needs

To evaluate changes in health care spending and utilization associated with a telehealth-based care coach-supported and behavioral health (BH) provider referral intervention in the United States. Observational retrospective cohort study with propensity score matching of treated and control groups. Difference-in-differences (DID) analysis was used to calculate per-member per-month (PMPM) savings and changes in utilization in a treated group relative to matched controls over 36 months. The study included 1800 adults with substance use disorder (SUD), anxiety, or depression who were eligible for the intervention. Treated members (n = 900) graduated from the program. Matched control members (n = 900) were eligible but never enrolled. Primary outcomes included all-cause and disease-attributable health care cost and utilization PMPM, categorized by place of service. There were statistically significant reductions in total all-cause medical costs of $485 PMPM (P < .001) and a 66% pre-post reduction in inpatient encounters, with $488 PMPM DID savings for inpatient admissions (P < .001) among the treated cohort compared with the control cohort over 36 months. Conversely, there were statistically significant cost increases ($110 PMPM; P < .001) for all-cause office visits in the treated cohort compared with the control cohort. Similar results were seen in SUD-attributable and BH-attributablecosts. Although the results could be affected by unmeasured confounding, they suggest that care coaching interventions that offer BH provider referrals may produce long-term savings, reductions in avoidable utilization, and increases in targeted services to treat BH conditions. Rigorous evaluations are needed to confirm these findings.

Health Care Resource Utilization and Costs of CAR T Therapy in Patients with Large B-Cell Lymphoma: A Retrospective US Claims Database Analysis

Introduction: Despite its association with promising clinical outcomes, chimeric antigen receptor T-cell (CAR T) therapy may not be a feasible treatment option for some patients with relapsed or refractory large B-cell lymphoma (LBCL). Although more patients have been referred to CAR T therapy in recent years, many patients are still not treated due to anticipated costs and logistical challenges, particularly in the community setting (Gajra et al, Immunotherapy 2020). Because of long manufacturing times, patients often require bridging therapy prior to CAR T and a subset of patients die on bridging therapy without ever receiving CAR T (Chow et al, Am J Hematol 2020). Furthermore, more than one-half of patients relapse within 1 year of receiving CAR T therapy (Spiegel et al, Blood 2021). It is important to understand the clinical and economic burden associated with CAR T therapy. We assessed health care resource utilization (HCRU) and all-cause health care (medical and medication) costs from 30 days prior to through 90 days after infusion with tisagenlecleucel or axicabtagene ciloleucel CAR T therapy in patients with diffuse large B-cell lymphoma (DLBCL), the most common non-Hodgkin lymphoma (NHL) subtype, or primary mediastinal large B-cell lymphoma (PMBCL), another aggressive NHL subtype. Methods: This observational, retrospective cohort study analyzed MarketScan (IBM, Armonk, NY) administrative claims data for adult patients with DLBCL and PMBCL who received CAR T infusion therapy between October 1, 2017 and March 31, 2021, and who had continuous enrollment 6 months before and at least 90 days after the CAR T treatment date (index date). Those with mantle cell lymphoma or follicular lymphoma were excluded. Hospital length of stay (LOS) and HCRU from 30-days prior to 90-days post-infusion for inpatient and outpatient hospital visits and other facility visits were examined. All-cause medical, medication, and health care costs per patient were reported in 2021 US dollars. Results: A total of 106 patients (DLBCL, n=92; PMBCL, n=14) who received CAR T therapy (mean age, 55 years; men, 59%; commercial payer, 87%; CAR T brand information available, n=20 patients) were included in the analysis. A majority of patients (78 [74%]) had at least 5 outpatient hospital visits in the 30 days prior to CAR T therapy. Many were reported as having received bridging and/or lymphodepletion treatment prior to CAR T infusion (81 [76%]). CAR T therapy was administered in an inpatient setting for 92 patients (87%) with a mean (SD) LOS of 18.4 (11.3) days and a mean (SD) cost of $341,217 ($245,564) within 90 days. Among those receiving CAR T inpatient, 41 (45%) had intensive care unit (ICU) admissions during their stay. In the 90 days post-CAR T infusion, most patients (92 [87%]) had at least 5 outpatient hospital visits. Subsequent inpatient admissions occurred in 30 patients (28%), with a mean (SD) LOS of 12 (12.44) days and a mean (SD) cost of $83,952 ($105,961) within 90 days after CAR T infusion. From 30 days prior to 90 days after infusion, the mean (SD) total all-cause health care costs per patient, including those associated with CAR T therapy, were $511,139 ($293,631) (Figure). Conclusions: While CAR T therapy may be a treatment option as LBCL patients progress, patients face a considerable clinical and economic burden. Both the HCRU prior to infusion and after infusion included frequent outpatient hospital visits. Additionally, in this analysis, inpatient hospitalizations were the primary driver of HCRU and costs of CAR T therapy. Most patients received CAR T therapy on an inpatient basis, requiring prolonged stays. Nearly half of patients were admitted to the ICU and more than a quarter experienced secondary inpatient stays. Costs associated with the logistically challenging CAR T therapy within a month leading up to the infusion and up to 3 months post-infusion are substantial, averaging half a million dollars per patient, indicating a significant burden, given that a majority of the patients relapse within a year. These findings underscore the need for an accessible, off-the-shelf, and efficacious therapeutic option for LBCL patients. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Economic burden and antidepressant treatment patterns among patients with major depressive disorder in the United States.

BACKGROUND: The prevalence of major depressive disorder (MDD) continues to rise year over year, resulting in significant economic implications. However, when patients are treated with contemporary standard-of-care antidepressant pharmacotherapies, a suboptimal response is often attained, resulting in frequent treatment changes. OBJECTIVE: To compare health care resource utilization (HCRU) and all-cause medical and pharmacy costs between commercially insured patients with an MDD diagnosis and matched non-MDD patients and explore treatment patterns among patients with MDD initiating antidepressant pharmacotherapy. METHODS: This was a retrospective, observational analysis of IBM MarketScan US commercial claims data. Adults aged 18 years and older with continuous enrollment 12 or more months before and after the patient's first MDD diagnosis from 2017 to 2018 were included in the analysis. HCRU and all-cause medical and pharmacy costs were compared among patients with MDD and a 1:1 exact-matched cohort of non-MDD patients during the same period (Objective 1). Treatment patterns (persistence, discontinuation, switch, combination, and augmentation) were analyzed for patients with MDD starting first-line antidepressant monotherapy for up to 12 months following their antidepressant initiation index date (Objective 2). Time to first treatment change or discontinuation was calculated and treatment patterns were graphically displayed in Sankey diagrams. RESULTS: 625,272 patients with MDD were matched 1:1 to a cohort of non-MDD patients in Objective 1. Patients with MDD had statistically significantly greater all-cause medical (20.4 vs 9.4; P < 0.0001), outpatient (19.5 vs 9.0; P < 0.0001), emergency department (0.51 vs 0.23; P < 0.0001), inpatient (0.35 vs 0.11; P < 0.0001), and any mental health-related (7.7 vs 0.58; P < 0.0001) visits compared with non-MDD patients. Mean all-cause medical costs were $6,809 (P < 0.0001) higher among patients with MDD than among patients without MDD ($13,183 vs $6,374, respectively). In Objective 2, 44,485 patients with MDD who received antidepressant monotherapy as their first-line MDD treatment were examined. Among the first treatment patterns observed following initiation, 19.3% of patients persisted with their first-line therapy, 56.2% discontinued antidepressant therapy, 24.5% experienced a treatment change (switching, adding a second antidepressant, or augmenting their existing therapy). The median days until first treatment change were 65 days for those discontinuing and 47 days for those switching antidepressants. Among the 24.5% of patients with a treatment change, 50.0% experienced another change in therapy within 30 days. CONCLUSIONS: The HCRU and costs accrued for patients with MDD is significantly greater than those for non-MDD patients. A large proportion of patients with MDD experienced treatment changes shortly after initiating their first-line antidepressant therapy. The results of this study highlight the need for reevaluation of the current MDD treatment paradigm. DISCLOSURES: Drs Zhu and Namjoshi are employees of Biogen Inc. and may hold stock. Dr Ferries and Ms Suthoff are employees of Sage Therapeutics, Inc., and may hold stock and/or stock options. Dr Bera has no potential conflicts of interest to disclose. This research was funded by Sage Therapeutics and Biogen. Manuscript editorial services were provided by Boston Strategic Partners, Inc., funded by Sage Therapeutics and Biogen. This work was supported by Sage Therapeutics, Inc., and Biogen. The authors had full editorial control of the manuscript and provided final approval on all content.

Impact of medications for opioid use disorder on healthcare resource utilization and costs for patients served by a state Medicaid program: An exploratory retrospective matched-cohort analysis of South Carolina Medicaid claims data

Objectives: Although evidence-based recommendations from treatment guidelines support the use of medications for opioid use disorder (MOUD) in addition to psychosocial counseling, about 86.6% of patients diagnosed with opioid use disorder (OUD) do not receive MOUD and several barriers still restrict access to MOUD in the United States. This study assesses the impact of MOUD on healthcare resource utilization (HCRU) and costs for Medicaid beneficiaries. Methods: An exploratory retrospective matched-cohort analysis was performed among Medicaid patients not concurrently enrolled in Medicare using anonymized claims data from South Carolina Medicaid between 7/1/2016 and 12/31/2019. Patients newly diagnosed with OUD who received MOUD were matched based on age, gender, and race to patients who did not receive MOUD to evaluate HCRU and costs in the 6 months prior to and 12 months following initial OUD diagnosis. Results: A total of 397 matched pairs of MOUD and non-MOUD patients were identified for this analysis. A significantly lower percentage of patients had emergency department visits (63.2% vs 74.3%; P=0.0005) and hospitalizations (21.9% vs 37.8%; P&lt;0.0001) in the matched MOUD cohort compared to the non-MOUD cohort. All-cause total direct costs were numerically lower for the matched MOUD cohort ($15,212 vs $17,451; P=0.3388), as numerically higher all-cause pharmacy costs associated with MOUD utilization were offset by significantly lower all-cause medical costs compared to the non-MOUD cohort ($9,265 vs $14,819; P=0.0005). Conclusions: The results of this analysis suggest that MOUD utilization has a positive association with reducing HCRU and all-cause medical costs for Medicaid patients with OUD.

Comparison of clinical and economic burden of pulmonary arterial hypertension patients with and without connective tissue disease treated with macitentan

Abstract Introduction Patients with pulmonary arterial hypertension (PAH) and connective tissue disease (CTD) have a poorer prognosis than those diagnosed with other PAH etiologies. Purpose To compare the clinical and economic burden among PAH patients with and without CTD (PAH-CTD/PAH non-CTD) treated with macitentan. Methods This retrospective study used the Optum Clinformatics DataMart to identify PAH-CTD and PAH non-CTD patients aged ≥18 years and prescribed macitentan from 01JAN2015–30JUN2019. The index date was the date of the first macitentan claim. Patients had to have ≥12 months of continuous enrollment before index and were followed until earliest of macitentan discontinuation, health plan disenrollment, death, or study end. Patients in the PAH-CTD cohort had to have a claim for a CTD condition at any time during the 12-month baseline period. Patients with a baseline claim for any PAH medication, chronic thromboembolic pulmonary hypertension, prior atrial septostomy or lung transplant were excluded. Descriptive analysis comprised means, standard deviations, medians, and interquartile ranges for continuous variables and frequencies and percentages for categorical variables. Multivariable analyses (Cox proportional hazards and generalized linear models) adjusting for baseline characteristics were used to compare time to PAH-related and all-cause hospitalization, disease progression (death, lung transplant, atrial septostomy or addition of intravenous/subcutaneous prostanoids), and PAH- and all-cause related medical (inpatient and outpatient) costs between the two cohorts with PAH non-CTD as reference. Results The analysis included 203 PAH-CTD and 750 PAH non-CTD patients treated with macitentan. Versus PAH non-CTD, PAH-CTD patients were younger, had proportionately more females, and lower Charlson Comorbidity Index (CCI) scores (Table). The proportion of patients with a baseline hospitalization, length of stay and inpatient costs were similar across groups. There were no significant differences observed in time to PAH-related hospitalization (hazard ratio [HR]=0.99; p=0.910), all-cause hospitalization (HR=1.13; p=0.195), or disease progression (HR=1.00; p-value=0.973) for the PAH-CTD cohort (PAH non-CTD as reference). All-cause medical costs were similar between the cohorts ($3,836 per patient per month [PPPM] for PAH CTD vs $3,521 PPPM for PAH non-CTD, p=0.362). PAH-CTD patients incurred higher mean PAH-related medical costs vs PAH non-CTD ($2,806 PPPM vs $1,961 PPPM, p=0.047). Conclusion The study suggests that risk of all-cause and PAH-related hospitalization and disease progression did not differ for PAH-CTD and PAH non-CTD patients. Treatment with macitentan in the real world appears to confer similar clinical benefits in both PAH-CTD and PAH non-CTD groups. All-cause costs did not differ between the two cohorts but PAH-related costs were significantly higher in PAH-CTD patients suggesting an incremental burden associated with CTD. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): Janssen Scientific Affairs, LLC

Clinical and economic burden of chronic rhinosinusitis with nasal polyposis: A U.S. administrative claims analysis.

Background: Limited data exist on the clinical and economic burden of chronic rhinosinusitis with nasal polyposis (CRSwNP). Objective: To describe patient characteristics, health-care resource utilization (HCRU), and health-care costs among patients with CRSwNP with and without comorbid asthma (primary analysis) and with surgical management of nasal polyps (secondary analysis). Methods: This was a retrospective study of patients diagnosed with CRSwNP conducted using administrative claims data from January 1, 2013, through March 31, 2019. Study outcomes were assessed over a 2-year follow-up. Results were stratified by baseline asthma status (primary analysis) and presented separately for patients with surgically managed CRSwNP (secondary analysis). Results: The primary analysis included 10,999 patients with CRSwNP (2649 with asthma, 8350 without asthma). Patients with versus without asthma had higher medication use, HCRU, and all-cause medical costs (mean ± standard deviation $34,667 ± $42,234 versus $27,122 ± $45,573; p < 0.001) across the full follow-up period. CRSwNP-related medical costs were significantly higher for patients with versus without asthma in year 2 of follow-up. In the surgical management analysis (n = 4943), most categories of medication use and CRSwNP-related HCRU declined from baseline levels during follow-up, and CRSwNP-related pharmacy costs in year 2 were less than half of baseline levels. Conclusion: Patients diagnosed with CRSwNP and asthma had a greater burden of illness than those without asthma. Higher CRSwNP-related medical costs in year 2 of follow-up for patients with asthma may indicate worsening symptoms over time. Among patients with surgically managed CRSwNP, HCRU and costs increased in year 1 of follow-up but decreased below baseline levels in year 2, potentially reflecting improved symptom severity.

Comparative Analysis of All-Cause Health Care Resource Utilization and Costs Among Venous Thrombosis Patients Without Cancer Prescribed Apixaban or VKAs in France.

IntroductionThe direct oral anticoagulant (DOAC) apixaban has shown to have non-inferior efficacy and better safety than vitamin K antagonists (VKAs) in patients with venous thromboembolism (VTE). We determined whether healthcare resource use (HCRU) and direct all-cause medical and non-medical costs in patients with VTE in France differed between VKAs and apixaban.MethodsA retrospective cohort study was conducted using French national health data from January 2013–June 2018 for anticoagulant-naïve adults hospitalized with VTE. All-cause costs and HCRU per patient per month (PPPM) were compared between apixaban and VKA cohorts created by 1:1 propensity score matching. Two-part models with bootstrapping were used to calculate marginal effects for costs and HCRU.ResultsThe matched VKA and apixaban cohorts each comprised 7503 patients. Compared to VKAs, patients prescribed apixaban had significantly lower (P < 0.0001) mean all-cause costs PPPM for outpatient visits (€438.54 vs. €455.58), overall laboratory tests (€21.26 vs. €83.73), and hospitalizations (€249.48 vs. €343.82), but significantly higher (P < 0.0001) mean all-cause costs PPPM for overall drugs (€97.06 vs. €69.56) and medical procedures (€42.12 vs. €35.50). Mean total all-cause direct medical costs (€687.93 vs. €798.70) and total all-cause direct medical and non-medical costs (€771.60 vs. €883.66) were significantly lower (P < 0.0001) for apixaban. Mean HCRU PPPM showed similar trends. Subgroup analyses showed that, among patients with recurrent VTE, patients prescribed apixaban had significantly lower (P < 0.0001) all-cause costs PPPM for total medical costs (€17.26 vs. €18.12) and total all-cause direct medical and non-medical costs (€18.37 vs. €19.20) than patients prescribed VKAs. Similarly, among patients with bleeding, patients prescribed apixaban had significantly lower (P < 0.0001) all-cause costs PPPM for total medical costs (€15.34 vs. €32.61) and total all-cause direct medical and non-medical costs (€16.23 vs. €34.63) than patients prescribed VKAs.ConclusionCompared to VKAs, apixaban may be cost-saving in the treatment of patients hospitalized for acute VTE.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02200-7.

Impact of timing of nintedanib initiation among patients newly diagnosed with idiopathic pulmonary fibrosis

Aims While nintedanib treatment has been shown to slow the progression of idiopathic pulmonary fibrosis (IPF) in patients across varying levels of lung function, the effect of treatment timing on outcomes has not been examined. We assessed hospitalization risk and medical costs among patients with IPF based on the timing of nintedanib initiation after IPF diagnosis. Materials and methods This retrospective administrative claims study included data from 04/01/2014–09/30/2019 for patients aged ≥40 years who initiated nintedanib within 1 year of IPF diagnosis. Patients were assigned to study cohorts based on the time from IPF diagnosis to nintedanib initiation. All-cause hospitalization and all-cause medical costs were modeled using marginal structural models including inverse probability weights to adjust for both baseline and time-varying characteristics. Results Of 11,195 patients diagnosed with IPF during the identification period, 449 met the study selection criteria (mean age 72.3 years, 68% male, mean follow-up time 13.3 months). Adjusted hospitalization risk and medical costs both varied significantly by the timing of nintedanib initiation (p < .001 and p = .020, respectively). Adjusted weighted hospitalization risk was higher among untreated vs. treated patients in months 2–3, months 4–6, and months 7–12 after diagnosis (hazard ratio [95% CI] 1.97 [1.09–3.56], p = .026; 2.62 [1.22–5.63], p = .014; and 5.57 [2.31–13.45], p < .001, respectively). Medical costs were 69% higher for patients initiating treatment in months 2–3 vs. month 1 (cost ratio [95% CI] 1.69 [1.20–2.38], p = .003). Limitations Disease severity could not be assessed because clinical data were unavailable; however, proxies such as oxygen use were included to adjust for between-cohort differences in disease severity. Conclusions Patients who initiate nintedanib promptly after IPF diagnosis may have reduced hospitalization risk and medical costs compared with those who start treatment later. Additional studies are warranted to improve understanding of the impact of prompt antifibrotic therapy on patient outcomes.

All-cause health care resource utilization and costs among adults with alopecia areata: A retrospective claims database study in the United States.

BACKGROUND: Alopecia areata (AA) is an autoimmune disorder characterized by hair loss. Patients may present with hair loss of the scalp, eyelashes, eyebrows, and/or body. Alopecia totalis (AT), total scalp hair loss, or alopecia universalis (AU), total body hair loss, are extensive forms. Although the impact of AA on quality of life is understood, evidence of its economic burden is limited. A better understanding of the all-cause health care costs for health plans and patients with AA is critical to comprehend disease burden. OBJECTIVE: To evaluate all-cause health care resource utilization and direct health care costs in US adults with AA with or without AT or AU, vs matched control subjects. METHODS: Patients (≥ 18 years) with AA with no less than 2 claims of AA at diagnosis (October 31, 2015, to March 3, 2018) were identified in the IBM MarketScan Commercial Claims and Encounters and Medicare Supplemental databases. Patients were enrolled no less than 12 months before and after first diagnosis (index). Patients were grouped according to AT or AU status (AT/AU group) or AA without AT/AU (non-AT/AU group) and matched 1:3 to control subjects without AA/AT/AU. Summary statistics were calculated for demographic and clinical characteristics at baseline and follow-up. RESULTS: At baseline, there were 14,972 adult patients with AA and 44,916 control subjects. Of patients with AA, 1,250 and 13,722 were in the AT/AU and non-AT/AU groups, respectively. A significantly greater proportion of patients with AA had atopic and autoimmune comorbidities vs control subjects. After index, patients with AA used significantly more corticosteroid treatments (injectable/oral/topical) than control subjects. A greater mean number of annual outpatient and dermatologist visits was observed for both AA groups vs control subjects (outpatient visits: AT/AU group: 17.8 vs 11.8; non-AT/AU group: 15.4 vs 11.2; dermatologist visits: AT/AU group: 3.4 vs 0.4; non-AT/AU group: 3.4 vs 0.4; P < 0.001 for all). Mean total all-cause medical and pharmacy costs (2018 US$) were higher in both AA groups vs control subjects (AT/AU group: $18,988 vs $11,030; non-AT/AU group: $13,686 vs $9,336; P < 0.001 for both). Patient out-of-pocket costs were higher for AA vs control subjects (AT/AU group: $2,685 vs $1,457; non-AT/AU group: $2,223 vs $1,341; P < 0.001 for both). CONCLUSIONS: Compared with control subjects, patients with AA are more likely to have atopic and autoimmune comorbidities, to use corticosteroids, and to make outpatient visits. Patients with AA have greater all-cause medical (including pharmacy) and out-of-pocket costs. The difference in total medical costs for patients with AT/AU vs control subjects is higher than the difference for patients with non-AT/AU vs control subjects. DISCLOSURES: This study was sponsored by Pfizer Inc. Pfizer Inc was involved in the study design; collection, analysis, and interpretation of data; writing of the report; and the decision to submit this report for publication. A. Mostaghimi reports consulting fees from Pfizer Inc, Concert, Lilly, AbbVie, hims, and Digital Diagnostics; reports equity from Lucid and hims; and is an associate editor at JAMA Dermatology. K. Gandhi, M. Ray, and V. Sikirica are former employees of Pfizer Inc and held stock and/or stock options with Pfizer Inc at the time of writing. N. Done, W. Gao, C. Carley, T. Wang, and E. Swallow are employees of Analysis Group, Inc, a consultancy that received payment from Pfizer Inc for participation in this analysis.

Updated cost-effectiveness of MDMA-assisted therapy for the treatment of posttraumatic stress disorder in the United States: Findings from a phase 3 trial.

Severe posttraumatic stress disorder (PTSD) is a prevalent and debilitating condition in the United States. and globally. Using pooled efficacy data from six phase 2 trials, therapy using 3,4-methylenedioxymethamphetamine (MDMA) appeared cost-saving from a payer's perspective. This study updates the cost-effectiveness analysis of this novel therapy using data from a new phase 3 trial, including the incremental cost-effectiveness of the more intensive phase 3 regimen compared with the shorter phase 2 regimen. We adapted a previously-published Markov model to portray the costs and health benefits of providing MDMA-assisted therapy (MDMA-AT) to patients with chronic, severe, or extreme PTSD in a recent phase 3 trial, compared with standard care. Inputs were based on trial results and published literature. The trial treated 90 patients with a clinician administered PTSD scale (CAPS-5) total severity score of 35 or greater at baseline, and duration of PTSD symptoms of 6 months or longer. The primary outcome was assessed 8 weeks after the final experimental session. Patients received three 90-minute preparatory psychotherapy sessions, three 8-hour active MDMA or placebo sessions, and nine 90-minute integrative psychotherapy sessions. Our model calculates the per-patient cost of MDMA-AT, net all-cause medical costs, mortality, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). We reported results from the U.S. health care payer's perspective for multiple analytic time horizons, (base-case is 30 years), and conducted extensive sensitivity analyses. Costs and QALYs were discounted by 3% annually. Costs were adjusted to 2020 U.S. dollars according to the medical component of the U.S. Bureau of Labor Statistics' Consumer Price Index (CPI). MDMA-AT as conducted in the phase 3 trial costs $11,537 per patient. Compared to standard of care for 1,000 patients, MDMA-AT generates discounted net health care savings of $132.9 million over 30 years, accruing 4,856 QALYs, and averting 61.4 premature deaths. MDMA-AT breaks even on cost at 3.8 years while delivering 887 QALYs. A third MDMA session generates additional medical savings and health benefits compared with a two-session regimen. Hypothetically assuming no savings in health care costs, MDMA-AT has an ICER of $2,384 per QALY gained. MDMA-AT provided to patients with severe or extreme chronic PTSD is cost-saving from a payer's perspective, while delivering substantial clinical benefit.

Immune checkpoint inhibitors: immune-related adverse events, healthcare utilization, and costs among commercial and Medicare Advantage patients

BackgroundImmune checkpoint inhibitors (ICI) are increasingly used across multiple cancer types and stages and little is known about real-world outcomes. This study sought to determine healthcare utilization, costs, immune-related adverse events (irAEs), and all-cause mortality of single-agent versus combination ICI in the USA.Materials and methodsThis is a retrospective study conducted with 2016–2018 data from the HealthCore Integrated Research Database, consisting of commercial and Medicare-insured adult patients with a cancer diagnosis using ICI in the USA. Outcomes were healthcare utilization, costs, and irAEs (FDA-recognized and others) up to 1-year post-index between patients using ICI monotherapy (mono, PD-1/PD-L1 inhibitor) and combination therapy (combo, PD-1/PD-L1 with CTLA-4 inhibitors).ResultsIn total, 9084 patients received monotherapy and 904 patients received combo therapy. Mean age 65 years for mono and 58 years for combo. Overall, the combo arm had higher rates of FDA-recognized irAEs (67.4% vs. 45.9%), especially endocrinopathies (27.7% vs 14.7%) and dermatitis (25.9% vs. 12.4%). All-cause mortality over 1-year follow-up was similar, 30.7% in mono vs 30.8% in combo arms. The combo group had higher rates of all-cause inpatient hospitalizations (55.4% mono vs 65.6% combo) and emergency department (ED) visits (33.7% mono vs 41.4% combo). IrAE-related hospitalizations were higher in combo (55.2% vs 42.1%). IrAE-related ED visits were 15.7% mono vs 22.7% combo. This increased toxicity and health care utilization was reflected in significant differences in healthcare costs. Stark differences were seen in all-cause medical costs as well as costs related to inpatient and ED utilization and costs attributed to irAEs.ConclusionsHigher rates of irAEs, healthcare utilization, and costs occur with combination immunotherapy. As further indications are approved for combination ICI, our study highlights the real-world tradeoffs involved with combination therapy regarding burdens of toxicity and increased healthcare utilization.

Healthcare Costs and Resource Utilization Associated with the Use of Empagliflozin Versus Other Antihyperglycemic Agents Among Patients with Type2 Diabetes Mellitus and Cardiovascular Disease: A Real-World Retrospective Cohort Analysis.

IntroductionEmpagliflozin has demonstrated lower rates of cardiovascular outcomes vs. standard of care among patients with type 2 diabetes mellitus (T2DM) and cardiovascular disease (CVD). However, the impact of empagliflozin compared to other branded antihyperglycemic agents (AHAs) on total cost of care has yet to be quantified.Methods and ResultsThis retrospective cohort study evaluated the impact of empagliflozin (n = 441) on costs and healthcare resource utilization (HCRU) vs. other branded AHAs (n = 13,122) among patients with T2DM and CVD, using the IQVIA PharMetrics® Plus Claims Database (1 August 2013–31 December 2017). Date of the first prescription (index date) for empagliflozin or other branded AHAs was used to classify patients into study cohorts. All-cause costs and HCRU were computed on a per patient per month (PPPM) basis and compared across study cohorts using outcome-appropriate statistical models. Overall, the empagliflozin cohort was younger and had a lower comorbidity burden. After covariate adjustment, the total all-cause costs (mean difference − $412 PPPM; 95% CI − $593, − $214) were significantly lower for the empagliflozin cohort. These cost differences were mainly driven by lower all-cause medical costs (mean difference − $400 PPPM; 95% CI − $577, − $196). For HCRU, the mean adjusted all-cause visits in the physician office and other outpatient settings were lower with empagliflozin vs. other branded AHAs (p < 0.001).ConclusionsThis study demonstrated that the all-cause healthcare costs and HCRU were significantly lower for patients with T2DM and CVD who initiated empagliflozin vs. other branded AHAs. Along with the positive clinical evidence base of empagliflozin, these results can guide healthcare decision makers during therapy selection.

Transitioning from Once-Monthly to Once-Every-3-Months Paliperidone Palmitate Among Veterans with Schizophrenia

PurposeCompared to once-monthly paliperidone palmitate (PP1M), once-every-3-months paliperidone palmitate (PP3M) reportedly increases treatment adherence. The objective of this study was to compare treatment patterns, utilization, and costs among Veterans Health Administration (VHA) patients with schizophrenia who transitioned to PP3M versus those remaining on PP1M.Patients and MethodsAdult VHA patients with ≥2 health care encounters (inpatient or outpatient) that included a schizophrenia diagnosis who initiated PP1M between January 1, 2015, and March 31, 2018 (identification period) were included in this exploratory retrospective cohort study. Propensity scores were used to match cases (PP1M users who transitioned to PP3M during the identification period) with controls (any patient initiating PP1M during the identification period). Data were assessed until death, health plan disenrollment, or study end. Outcomes were compared using chi-square and t-tests.ResultsA total of 257 eligible PP3M and 2973 eligible PP1M patients were identified among adult VHA patients; mean ages were 53.1 and 53.7 years, respectively. After propensity score matching, the PP3M and PP1M cohorts each held 111 patients. Comorbidities of patients treated with PP3M versus PP1M, respectively, included anxiety (12.5% vs 20%; standardized difference [STD] = 20.6), tobacco use (28.4% vs 43.2%; STD = 31.2), depressive disorder (26.5% vs 36.2%; STD = 21.1), and substance abuse (37.4% vs 44.2%; STD = 13.9). For the PP3M cohort, adherence (proportion of days covered ≥80%) to any antipsychotic agent was higher (78.4% vs 57.7%, P = 0.0009), and all-cause inpatient lengths of stay (LOS) were shorter (3.0 vs 8.3 days, P = 0.0354). Increased all-cause pharmacy costs with PP3M were offset by reduced all-cause medical costs, resulting in overall health care cost-neutrality.ConclusionRelative to those remaining on PP1M, VHA patients with schizophrenia who transitioned to PP3M experienced improved antipsychotic medication adherence and significantly shorter all-cause inpatient LOS; costs remained neutral.

Prevalence and Financial Burden of Digestive Diseases in a Commercially Insured Population

Digestive diseases represent a diverse group of clinical conditions that impact the population. Their heterogeneity in classification, presentation, acuity, chronicity, and need for drug therapy presents a challenge when comparing and contrasting the burden associated with these conditions. Prior studies use an outdated classification system and aggregate costs at the population level or focus on specific diseases, limiting the ability to characterize the overall landscape. Our aim was to provide the most up-to-date assessment of cost, utilization, and prevalence associated with digestive diseases. We examined digestive disease claims and payment data for a commercially insured adult population between 2016 and 2018 to provide a comprehensive summary of costs, utilization, and prevalence across 38 conditions. Outcome variables included point prevalence and relative prevalence, annualized all-cause medical and drug costs, digestive disease-specific average medical cost, digestive disease-specific cost per fill, and utilization by clinical setting and by clinical condition. A total of 7,297,435 individuals with a digestive disease diagnosis were included in the study. The point prevalence of having a digestive disease in the total population was 24%. Annualized total costs by clinical category ranged from $10,038 (eosinophilic esophagitis) to $107,007 (hepatitis C), with medical costs accounting for most of the expenditures in a majority of conditions. Annualized total costs for common conditions included $39,653 for alcoholic liver disease, $42,554 for acute pancreatitis, $62,735 for Crohn's disease, $13,948 for functional gastrointestinal disorders, $53,214 for nonalcoholic cirrhosis, and $36,441 for ulcerative colitis. Average cost of inpatient stays ranged from $12,218 (noninfectious gastroenteritis/colitis) to $78,259 (nonalcoholic steatohepatitis). Outpatient visits ranged from $784 (gastrointestinal infection) to $4629 (gallbladder and biliary tract disease). Average drug cost per fill ranged from $83 (gastroesophageal reflux disease) to $1458 (hepatitis C). A total of 27,429,046 clinical encounters occurred across all conditions during the study period, with 90% taking place as outpatient visits. Abdominal pain was the single largest contributor to outpatient visits and emergency department to home encounters. Inpatient stays were considerably more heterogeneous, with no condition accounting for more than 12% (gallbladder and biliary tract disease) of the total. The results demonstrate digestive diseases are common, heterogeneous in cost and utilization, and collectively exact a significant financial burden on the U.S. adult population.

Economic Burden in US Patients with Relapsed or Refractory Classical Hodgkin Lymphoma Treated with Brentuximab Vedotin or Chemotherapy after Failure of Autologous Hematopoietic Cell Transplantation

Introduction: Classical Hodgkin lymphoma (cHL) is a lymph node cancer of germinal center B-cell origin. Although most patients diagnosed with cHL are cured with multiagent chemotherapy (ChT), those with advanced-stage disease are more likely to harbor refractory disease or relapse. Brentuximab vedotin (BV) was approved by the FDA in 2011 for patients who have relapsed following autologous hematopoietic cell transplantation (auto-HCT), and for patients ineligible for auto-HCT who have received 2 prior lines of therapy. Real-world data are needed to understand the direct costs of medical care among patients with relapsed or refractory cHL treated with BV or ChT after auto-HCT failure.Methods: US patients with a primary diagnosis of cHL were identified from the Truven MarketScan® databases (Commercial, Medicare, and Medicaid) from August 1, 2011 to June 30, 2016 and followed until last visit. The BV cohort comprised all patients with observed BV use, while the non-BV cohort comprised patients who received ChT after auto-HCT but never received BV. Index therapy was defined as BV-based therapy for the BV cohort and first ChT after auto-HCT failure for the non-BV cohort, and the date of first claim for index therapy was defined as the index date. The BV cohort were further grouped into subcohorts based on any observed receipt of auto-HCT (BV alone vs BV + auto-HCT). Demographics were summarized and descriptively compared between cohorts. The total all-cause costs (inpatient, outpatient, and pharmacy claims) were assessed every 6 months in a 2-year period and also calculated using a standard cost per-patient-per-month (PPPM) metric over the entire follow-up period. All costs were inflated to 2016 US dollars.Results: 795 HL patients met the study eligibility criteria (mean age: 43.0 years; 58% male); 575 (72%) were in the BV cohort and 220 (28%) in the non-BV cohort. Median follow-up time for the BV and non-BV cohorts was 10.9 and 10.6 months, respectively. Patients in these 2 cohorts were similar in terms of sex and prevalence of multiple cancers (Table), although compared to those in the non-BV cohort, patients on BV were younger on average (mean age: 42.9 vs 45.9 years, p=0.0284). Mean PPPM all-cause medical costs were $51,245 for non-BV and $30,387 for BV. The use of BV was associated with higher mean PPPM all-cause pharmacy costs than non-BV ($17,934 vs $7645), but mean PPPM inpatient and outpatient costs were higher for non-BV than for BV ($35,165 vs $8021 and $8436 vs $4431, respectively).Within the BV cohort, 357 (62%) received BV alone and 218 (38%) received BV + auto-HCT. Similarly, there was no significant difference in sex and prevalence of multiple cancers between the 2 groups, although those treated with BV + auto-HCT were younger (mean age: 36.0 vs 47.1 years, p<0.0001) and were more likely to be insured with commercial insurance (72% vs 60%, p<0.0001) than those on BV alone. Mean PPPM total all-cause costs were $28,313 for patients treated with BV alone and $33,782 for patients treated with BV + auto-HCT. While mean PPPM pharmacy costs were similar between BV alone and BV + auto-HCT ($18,412 vs $17,152), mean PPPM inpatient and outpatient costs were both higher for BV + auto-HCT than for BV alone ($5992 vs $11,346 and $3909 vs $5285, respectively).Mean total direct medical costs were calculated at 6, 12, 18, and 24 months from index date for those with available follow-up over the corresponding time period. Costs were consistently higher for non-BV and BV + auto-HCT relative to BV alone. At 24 months, mean total all-cause costs reached $550,000 for non-BV, $497,000 for BV + auto-HCT, and $379,000 for BV alone. Inpatient, outpatient, and pharmacy represent approximately 69%, 16%, and 15% of total all-cause costs for non-BV; 34%, 16%, and 51% for BV + auto-HCT; and 21%, 14%, and 65% for BV alone. These patterns were relatively consistent across all 4 timepoints considered (Table).Conclusions: While drug acquisition costs result in higher pharmacy costs for patients receiving newer agents, lower medical costs (including costs of inpatient and outpatient care for the management of cHL, cHL-related events, and other clinical conditions) may be associated with the use of newer efficacious therapies with potentially lower toxicity; thus, the use of newer agents may lead to overall cost savings.Study support: Bristol-Myers Squibb. [Display omitted] DisclosuresChen:Bristol-Myers Squibb: Employment. Johnston:Bristol Myers Squibb: Consultancy. Szabo:Bristol Myers Squibb: Consultancy. Connors:Bayer Healthcare: Research Funding; Cephalon: Research Funding; Takeda: Research Funding; Seattle Genetics: Research Funding; NanoString Technologies, Amgen, Bayer, BMS, Cephalon, Roche, Genentech, Janssen, Lilly, Merck, Seattle Genetics, Takeda,: Research Funding; Amgen: Research Funding; Bristol-Myers Squibb: Research Funding; NanoString Technologies: Research Funding; F Hoffmann-La Roche: Research Funding; Genentech: Research Funding; Lilly: Research Funding; Merck: Research Funding; Janssen: Research Funding. Yasenchak:Seattle Genetics: Consultancy; Bristol-Myers Squibb: Consultancy.

PRO18 A Systematic Literature Review of the Economic Burden and Cost Drivers in Pulmonary Arterial Hypertension

The aim of this study was to identify cost drivers in Pulmonary Arterial Hypertension (PAH) in the US through a systematic literature review of economic and humanistic burden. A systematic literature review was conducted adhering to methodology published by the Cochrane Collaboration and the National Institute for Health and Clinical Excellence (NICE) in the UK. Population, Intervention & Comparators, Outcomes and Study Design (PICOS) criteria were used to identify resource utilization and economic burden studies in PAH published from credible databases (EMBASE, MEDLINE, MEDLINE In-Process) over the last 10 years. A total of 170 publications were extracted with most publications from the US. Total all-cause costs per patient per month (PPPM) in the US (all costs reported in 2019 $USD) were found to range from $2,494 to $11,037, driven primarily by medical costs. Medical costs accounted for a larger portion of total costs than pharmacy costs within both all-cause ($2,113 to $7,625 PPPM) and PAH-related ($293 to $5,086 PPPM) health care costs; among all-cause medical cost components, inpatient admission costs ($1,115 to $2,146 PPPM) contributed the highest costs, followed by outpatient costs ($954 to $1,944 PPPM). Although total costs increased after initiation of PAH therapies, increased pharmacy costs were partially offset by reduced medical costs ($1,506 to $57,409 decreased annual medical costs from baseline). Management of PAH carries a considerable economic burden with the major cost drivers consisting of primarily medical costs. All-cause costs vary widely in current literature, which may be due to heterogeneity in study population and cost components.

Healthcare utilization and costs associated with first-line treatment with obinutuzumab- and rituximab-based regimens for follicular lymphoma.

e19536 Background: Current real-world data on healthcare utilization (HCU) and costs of common first-line (1L) follicular lymphoma (FL) regimens, including obinutuzumab (G), remain limited. The aim of this study was to examine real-world HCU and costs for the most common National Comprehensive Cancer Network (NCCN)-recommended 1L FL treatments. Methods: This was a retrospective cohort study using administrative claims data from the IQVIA PharMetrics Plus and IBM MarketScan Commercial and Medicare Supplemental databases. We identified patients (pts) ≥18 years, who had ≥1 inpatient claim or ≥2 outpatient claims, with a diagnosis of FL from February 1, 2015 to March 31, 2020, and had received 1L FL treatment (per NCCN guidelines) between February 1, 2016 and September 30, 2019. The first claim for FL treatment was the index date. All pts had ≥12 months of pre- and ≥6 months of post-index continuous enrollment in medical and pharmacy benefits. Pts with other primary cancers, FL treatment, or stem cell transplant during the pre-index period, and clinical trial participation or end-stage renal disease during the study period were excluded. All-cause HCU and costs (2020 USD) per pt during the 6-month post-index period were reported for the five most common 1L FL regimens (only complete NCCN regimens were considered). Results: Overall, 1991 pts met the eligibility criteria, and 53% were male. The mean (standard deviation [SD]) age and Charlson Comorbidity Index at index were 58 (10.4) years, and 1.7 (1.0), respectively. The most common 1L regimens were rituximab-bendamustine (R-benda; n=818 [41.1%]), R-monotherapy (R-mono; n=592 [29.7%]), R-CHOP (n=461 [23.2%]), G-benda (n=86 [4.3%]), and R-CVP (n=34 [1.7%]). The proportion of pts who had ≥1 hospitalization or an emergency room visit was highest with R-CHOP (26.7% and 33.6%, respectively) and lowest with R-mono (11.3% and 18.1%, respectively). Mean (SD) all-cause total healthcare costs were highest with R-benda and G-benda, followed by R-CHOP, R-CVP, and R-mono (Table). Mean (SD) all-cause medical and FL treatment costs (drug and administration) were highest for R-benda ($174,407 [$110,520]; $135,520 [$96,492]) and lowest for R-mono ($87,368 [$83,910]; $54,271 [$40,433]). Conclusions: This real-world study provides an update on HCU and costs among pts initiating current NCCN-recommended 1L treatment for FL. Unadjusted 6-month total healthcare costs were highest with R-benda, followed by G-benda, while the lowest costs were seen with R-CVP and R-mono. Future studies with adjustment for pt characteristics are needed to compare HCU and costs between FL regimens.[Table: see text]

Disease-free survival as a predictor of overall survival in localized renal cell carcinoma (RCC) following first nephrectomy.

4581 Background: Intermediate endpoints (e.g., disease free survival [DFS]) have gained traction lately as potential surrogates for OS in oncology as they require shorter follow up to show clinical benefit. Given the high post-nephrectomy survival in patients (pts) with localized RCC, evidence on if DFS can be used as a predictor of OS in the disease is warranted. We assessed the association between DFS and OS in pts with newly diagnosed, completely resected, intermediate-high (pT2N0 high grade, pT3N0) or high-risk (pT4N0, pTanyN1) RCC post-nephrectomy. Methods: This retrospective observational study used the SEER-Medicare database (2007–2016). DFS was defined as time from initial nephrectomy date to first recurrence (diagnosis of metastatic disease, additional surgery, starting systemic treatment for advanced RCC) or death, whichever occurred first. OS from time of recurrence in pts with recurrence were compared with OS from comparable time point in pts without, using Kaplan-Meier analyses and adjusted Cox models. OS was also compared between pts with and without recurrence by landmark time points at 1, 2, 3, 4 and 5 years (yrs) post-nephrectomy; hazard ratios (HRs) between the two cohorts were estimated using adjusted Cox models. Correlation between DFS and OS was assessed using the Kendall’s τ rank correlation. Monthly healthcare costs were compared between the two cohorts using generalized linear model. Results: 643 post-nephrectomy RCC pts (269 with recurrence vs 374 without) met the inclusion criteria (Median follow-up: 23 months). The mean age was 75.5 yrs, 61% male, and 86% white. The median post-nephrectomy OS and DFS was 8.61 and 4.44 yrs, respectively. Pts with and without recurrence had comparable baseline characteristics. Pts with recurrence had significantly shorter OS than those without [median: 2.53 yrs vs not reached; adjusted HR (95% confidence interval [CI]): 6.00 (4.24–8.48)]. Pts with recurrence by each landmark time point had significantly shorter OS than those without [1 yr post-nephrectomy median OS: 2.35 vs 9.66 yrs, and the OS 1, 3, and 5 after the 1 yr landmark was 69.9 vs 96.5%, 41.8 vs 83.8%, and 37.0 vs 70.1%, respectively; all Ps (log-rank test) &lt; 0.001]. Cox models indicated that pts with recurrence by each landmark time point had 2.6–3.5 times increased risk of death compared with those without. Kendall’s τ rank correlation model demonstrated a statistically significant correlation between DFS and OS (Kendall’s τ = 0.70; 95% CI: 0.65–0.74; P &lt; 0.001). Pts with recurrence had $4,924 and $1,387 higher adjusted all-cause medical costs and pharmacy costs per month (P &lt; 0.001). Conclusions: Post-nephrectomy recurrence is associated with significantly shorter OS among pts with intermediate-high or high-risk RCC, resulting in a strong positive association between DFS and OS in the population. Higher healthcare cost was also seen among pts with recurrence.

Making a business plan for starting a transitional pain service within the US healthcare system

Chronic pain imposes a tremendous economic burden of up to US$635 billion per year in terms of direct costs (such as the costs of treatment) and indirect costs (such as...