Journal of managed care & specialty pharmacy最新文献

Background: Bruton's tyrosine kinase inhibitors (BTKis) and the BCL-2 inhibitor venetoclax in combination with obinutuzumab (VEN-O) are both recommended as frontline therapy in chronic lymphocytic leukemia (CLL). However, VEN-O is a 12-month fixed-duration therapy generating durable remissions whereas BTKis are continuous treat-to-progression treatments.

Objective: To examine costs before and after the fixed-duration treatment period for VEN-O relative to that observed for BTKis in a national sample of older US adults with CLL in the frontline setting.

Methods: This retrospective analysis used Medicare Parts A, B, and D claims from 2016 to 2021. Fee-for-service Medicare beneficiaries aged 66 years or older initiating frontline CLL treatment with VEN-O or a BTKi treatment between June 1, 2019, and June 30, 2020 (index date = first prescription fill date), were included in the sample. Mean cost measures were captured for both groups over 2 fixed time periods calculated from the index date: Month 0 to 12 (proxy for VEN-O on-treatment period) and Month 13 to 18 (proxy for VEN-O off-treatment period). A difference-in-difference approach was used. Multivariate generalized linear models estimated changes in adjusted mean monthly costs during Month 0 to 12 vs Month 13 to 18, for the VEN-O group relative to the BTKi group.

Results: The final sample contained 193 beneficiaries treated with VEN-O and 1,577 beneficiaries treated with BTKis. Risk-adjusted all-cause monthly total costs were similar for VEN-O patients ($13,887) and BTKi patients ($14,492) between Month 0 and 12. Moreover, during Month 13 to 18, the mean monthly all-cause total costs declined by 67% for VEN-O ($13,887 to $4,462) but only by 10% for BTKi ($14,492 to $13,051). Hence, the relative reduction in costs across the 2 periods was significantly larger for VEN-O (-$9,425) vs BTKi (-$1,441) patients (ie, difference in difference = -$7,984; P < 0.001). Similar patterns were observed for CLL-related costs, with the substantially larger reductions in CLL-related total monthly costs (-$9,880 VEN-O vs -$1,753 BTKi; P < 0.001) for the VEN-O group primarily driven by the larger reduction in CLL-related monthly prescription costs (-$9,437 VEN-O vs -$2,020 BTKi; P < 0.001).

Conclusions: This real-world study of older adults with CLL found a large reduction in monthly Medicare costs in the 6 months after completion of the fixed-duration treatment period of VEN-O, largely driven by the reduction in CLL-related prescription drug costs. A similar decline in costs was not observed among those treated with BTKis. Our study highlights the substantial economic benefits of fixed-duration VEN-O relative to treat-to-progression therapies like BTKis in the first-line CLL setting.

The AMCP Poster Abstract Program provides a forum for authors to share their research with the managed care pharmacy community. Authors submit their abstracts to AMCP, and each abstract is reviewed by a team of peer reviewers and editors. All accepted abstracts are presented as posters at AMCP's Annual and Nexus meetings. These abstracts are also available through the AMCP meeting app. This JMCP supplement publishes all abstracts that were peer reviewed and accepted for presentation at Nexus 2024. Abstracts submitted in the Student and Encore categories did not undergo peer review; therefore, these abstracts are not included in the supplement.

Background: At present, 4 prescription therapies have been approved by the US Food and Drug Administration for the treatment of chronic idiopathic constipation (CIC) in adults.

Objectives: To compare persistence with and adherence to prucalopride vs 3 other prescription medications for CIC in a US population.

Methods: This retrospective, observational cohort study used data from the IBM MarketScan Commercial Claims and Encounters and Medicare Supplemental Databases (January 2015-June 2020). Inclusion criteria were patients (aged ≥18 years) with at least 1 prescription fill for prucalopride, lubiprostone, linaclotide, or plecanatide on or after April 2, 2019 (commercial availability of prucalopride), and at least 1 constipation-related diagnosis code. Persistence was assessed by time to discontinuation, and adherence was assessed by the proportion of days covered (PDC) and the proportion of patients who achieved PDC of at least 80%. Adjusted hazard ratios (HRs) for discontinuation and odds ratios for adherence were calculated.

Results: A total of 14,700 patients (mean age = 48.3 years; female = 81.9%) were included (prucalopride, n = 675; lubiprostone, n = 1,591; linaclotide, n = 11,105; plecanatide, n = 1,329). After adjusting for confounding factors, the HRs for discontinuation were significantly higher for all comparator medications compared with prucalopride after 2 months (HR [95% CI]: lubiprostone, 1.70 [1.48-1.95]; linaclotide, 1.25 [1.10-1.41]; plecanatide, 1.31 [1.13-1.51], all P < 0.001). The unadjusted mean (SD) PDC was 0.53 (0.32) with prucalopride compared with 0.41 (0.31); P less than 0.001 with lubiprostone, 0.48 (0.31), P less than 0.05 with linaclotide, and 0.48 (0.29), P = 0.98 with plecanatide. The comparator medications were all associated with lower odds of achieving PDC of at least 80% relative to prucalopride (odds ratio [95% CI]: lubiprostone, 0.52 [0.40-0.69], P < 0.001; linaclotide, 0.73 [0.58-0.93], P = 0.009; plecanatide, 0.70 [0.53-0.93], P = 0.015).

Conclusions: The findings of this study indicate that prucalopride has higher treatment persistence and adherence compared with other CIC prescription medications. This research represents the first instance of a real-world claims study showcasing such outcomes.

Background: In 2022-2023, the US Food and Drug Administration approved 2 novel gene therapies, valoctocogene roxaparvovec and etranacogene dezaparavovec, for hemophilia A and B, respectively. These one-time-administered gene therapies have been marketed at prices that create financial challenges for payers and patients. Understanding the magnitude and uncertainties around the long-term value of these therapies and how they can potentially relate to managed care practices is of high interest to the payer and patient community.

Objective: To conduct a systematic review of cost-effectiveness analysis (CEA) studies to assess (1) the long-term value of valoctocogene roxaparvovec and etranacogene dezaparavovec and (2) the relevance and validity of the underlying data and assumptions used in the CEA models and discuss how they relate to the challenges identified for CEAs of gene therapies.

Methods: A systematic review of cost-effectiveness studies of novel hemophilia A and B gene therapy was conducted. PubMed and Embase were searched for published studies from inception to January 12, 2024. Original research articles published in English that conducted a CEA on gene therapy treatments for hemophilia A and B, with a comparison of incremental costs and health effects, were considered. Critical appraisal of the quality of reporting and the underlying modeling assumptions were conducted to assess the relevance and validity of the results.

Results: Two hundred thirty-eight studies were identified, of which 4 met the inclusion criteria. Three studies were conducted from a US health care perspective and 1 from a Dutch societal perspective. Despite the high upfront costs of the gene therapies, all included studies' (3 hemophilia A and 1 hemophilia B) modeled results showed that gene therapies had lower overall costs and better health outcomes compared with factor concentrate replacement therapies and emicizumab. The results were driven by the assumption that gene therapies will have a durable effect of at least 10 years and offset the high cost of the current standard of care. The modeled health improvements varied substantially across studies, showing that the long-term value is sensitive to varying clinical and economic assumptions.

Conclusions: The novel hemophilia gene therapy treatments can potentially be a cost-effective use of treatment resources if the treatment effects are durable over time. To reduce the risk for payers while still facilitating patient access, outcomes-based agreements similar to what has recently been proposed by the Centers for Medicare & Medicaid Services for sickle-cell therapies are well supported.

Background: The increasing prevalence of diabetes in the United States continues to drive a steady rise in health care resource utilization, especially emergency department visits and all-cause hospitalizations, and the associated costs.

Objective: To investigate the impact of continuous glucose monitoring (CGM) on emergency department visits and all-cause hospitalizations among Medicaid beneficiaries with type 2 diabetes (T2D) treated with multiple daily insulin injections (MDIs) or basal insulin therapy (BIT) in a real-world setting.

Methods: In this retrospective, 12-month analysis, we used the Inovalon Insights claims dataset to evaluate the effects of CGM acquisition on emergency department visits and all-cause hospitalizations in the Managed Medicaid population. The analysis included 44,941 beneficiaries with T2D who were treated with MDIs (n = 35,367) or BIT (n = 9,574). Primary outcomes were changes in the number of emergency department visits and all-cause hospitalizations following 6 months after acquisition of CGM (post-index period) compared with 6 month prior to CGM acquisition (pre-index period). The first claim for CGM was the index date. Inclusion criteria were as follows: aged younger than 65 years, diagnosis of T2D, claims for short- or rapid-acting insulin (MDI group) or basal insulin (not rapid-acting) (BIT group), acquisition of a CGM device between January 1, 2017, and September 30, 2022, and continuous enrollment in their health plan throughout the pre-index and post-index periods.

Results: In the MDI group, all-cause inpatient hospitalization rates decreased from 3.25 to 2.29 events/patient-year (hazard ratio = 0.12; 95% CI = 0.11-0.13; P < 0.001) and emergency department visit rates decreased from 2.15 to 1.86 events/patient-year (hazard ratio = 0.52; 95% CI = 0.50-0.53; P < 0.001). In the BIT group, all-cause inpatient hospitalization rates decreased from 1.63 to 1.39 events/patient-year (hazard ratio = 0.11; 95% CI = 0.09-0.12; P < 0.001) and emergency department visit rates decreased from 1.60 to 1.43 events/patient-year (hazard ratio = 0.47; 95% CI = 0.44-0.50; P < 0.001).

Conclusions: Acquisition of CGM is associated with significant reductions in emergency department visits and all-cause hospitalizations among people with T2D treated with MDIs or BIT.

Background: Because of concerns of cost-effectiveness and low utilization, in 2018, manufacturers initiated a 60% price reduction for PCSK9 inhibitors, reducing the list price from more than $14,000 to $5,850. The goal of the reduction was to increase access and lower patient cost sharing for PCSK9 inhibitors.

Objective: To determine whether list price reductions resulted in a statistically significant decrease in patient cost sharing for PCSK9 inhibitors. The secondary objective is to quantify the change in monthly out-of-pocket (OOP) cost in the years following the price reduction policies.

Methods: This analysis uses a cross-sectional quasi-experimental design, with 2 time periods, to estimate the change in monthly OOP cost. A 2-stage cost model was used to quantify the difference in mean monthly OOP cost between the preprice and postprice reduction periods. This analysis was completed using IQVIA PharMetrics Plus for Academics health plan claims for PSCK9 inhibitors between January 2016 and December 2021 for commercially insured individuals in the United States. The primary exposure of interest is a manufacturer-initiated list price reduction in October 2018. The primary outcome of interest is the difference in the predicted monthly OOP cost between the prereduction and postreduction periods.

Results: There was a 50% decrease in the predicted monthly OOP cost, from $235.22 (SD = $241) in the prereduction period to $116.75 (SD = $152) in the postreduction period.

Conclusions: This claims level analysis used robust statistical modeling techniques to quantify the effect of manufacturer-initiated price reductions on monthly OOP cost. This unique manufacturer decision resulted in a statistically significant decrease in the monthly OOP cost for beneficiaries using PCSK9 inhibitors. Manufacturer-initiated price reductions could be a strategy to reduce the cost for other therapies with access and cost concerns. Further research is needed on the downstream patient-level effects of cost reductions, particularly among individuals who experience multiple barriers to care.

Background: Migraine, characterized by recurrent, severe headaches, presents a considerable challenge for patients, health care systems, and employers in the United States. However, there is a lack of recent estimates of the economic and humanistic burden in this population.

Objective: To assess the incremental burden of migraine on the total all-cause health care costs and health-related quality of life (HRQoL) in the United States, using data from the Medical Expenditure Panel Survey (MEPS).

Method: This retrospective cross-sectional study included adults (≥18 years) with and without migraine on the 2019-2021 full-year consolidated MEPS Household Component and Medical Provider Component data files. Descriptive analyses were conducted to compare health care expenditures and HRQoL among patients with and without migraine. To estimate the marginal effect of migraine on total health care spending, a two-part model generalized linear models was employed. HRQoL was evaluated using physical component summary (PCS) and mental component summary (MCS) scores based on the items in the Veterans Rand 12 Health Survey. A multivariate linear regression with log-link was conducted to understanding the factors associated with PCS and MCS scores. All analyses accounted for complex survey design of MEPS.

Results: The study included approximately 1.14 million patients with migraine and approximately 184 million patients without migraine. The patients with migraine were majorly female (82.81%), aged 18-45 years (50.24%), and residing in the southern region of the United States (41.45%). A two-part model revealed that marginal total health care expenditures among patients with migraine were $6,078.56 (95% CI = $4,618.45-$8,141.34) higher compared with those without migraine. In terms of HRQoL, average PCS scores in migraine and nonmigraine groups were 39.79 and 42.15, respectively. The average MCS scores were 46.63 and 49.95 for migraine and nonmigraine groups, respectively. After adjusting for sociodemographic characteristics, multivariable linear regression models revealed that the PCS score was 2.14 (95% CI = 1.17-4.55) units lower, and the MCS score was 3.19 (95% CI = 2.51-6.07) units lower among patients with migraine compared with those without.

Conclusions: Migraine imposes a substantial economic burden on both health care payers and patients in the United States. Notably, prescription drugs make up nearly half of the overall cost. Additionally, patients with migraine experience lower levels of physical and mental HRQoL compared with those without migraine.

Background: Trastuzumab is an antihuman epidermal growth factor receptor 2 monoclonal antibody used to treat breast and other cancers. Trastuzumab biosimilars were approved in the United States beginning in 2017. Utilization information on these biosimilars is limited.

Objective: To examine utilization patterns and characteristics of patients treated with trastuzumab (biosimilars and reference) and other human epidermal growth factor receptor 2 products.

Methods: We evaluated health care claims data from the Biologics and Biosimilars Collective Intelligence Consortium distributed research network, representing a large, geographically diverse US population of commercially insured individuals. We queried 4 distributed research network health plan partners to capture product usage data and patient information from October 1, 2016, to October 31, 2022. Patients were required to be continuously enrolled in their health plan for at least 365 days before their first observed trastuzumab utilization date in this study period. Data were aggregated across data partners.

Results: More than 16 million eligible health plan members representing more than 31 million person-years of data were evaluated. We identified 5,984 incident treatment episodes; 3,878 (64.8%) episodes were with the reference trastuzumab. The mean ages were consistent across trastuzumab products (60.2 to 65.1 years) and at least 80% of the episodes were among female patients. The mean comorbidity index score was 1.2 (SD = 1.9) among users of the reference vs the biosimilars (range 1.2-2.5). Other clinical characteristics (eg, diabetes, hypertension) were comparable across products. The proportion of total incident episodes of the reference trastuzumab decreased substantially over time (96% in 2016 vs 28% in 2021) as utilization of the biosimilars increased (eg, use of trastuzumab-anns increased from 2% [2019] to 36% [2021]). Similar utilization trends were seen among patients with and without metastatic breast cancer.

Conclusions: Trastuzumab biosimilars utilization has grown since their introduction to the US market. Exploration of these biosimilars' comparative effectiveness and safety to their reference product is warranted.

Value-based diabetes care is a proactive approach to providing quality care to individuals with diabetes. This approach focuses on improving clinical outcomes rather than the volume of services provided. Implementation of value-based diabetes care requires an established set of standardized quality measures against which all stakeholders can assess and benchmark their performance. The National Committee for Quality Assurance recently added the Glucose Management Indicator to its Healthcare Effectiveness Data and Information Set. The Glucose Management Indicator can be used as a measure of glucose control. This article discusses the benefits of value-based care, the importance of diabetes quality measures, and how the rapidly increasing adoption of continuous glucose monitoring is impacting these measures while improving the lives of individuals with diabetes.