Journal of managed care & specialty pharmacy最新文献

Background: Hemophilia B is characterized by a deficiency of clotting factor IX (FIX), leading to excessive bleeding. Hemophilia B is commonly treated using replacement FIX therapy, which may be administered prophylactically or on-demand following a bleeding episode. Previous research has found high health care resource use (HCRU) and costs among Medicare and commercially insured people with hemophilia B (PwHB), with FIX therapy being a primary driver of health care costs.

Objective: To assess HCRU, outcomes, and costs among US Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B.

Methods: This study employed a retrospective comparative cohort design to assess HCRU, outcomes, and costs among adult male Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B, relative to a matched comparator population of beneficiaries without bleeding disorders. Nationwide Medicaid claims and enrollment data from 2015 to 2020 were used for this analysis. Adult male PwHB who received FIX prophylaxis, defined as not having identified gaps in FIX therapy exceeding 60-days during a 1-year measurement period, and were continuously enrolled in Medicaid for at least 2 years, were matched 1:4 to comparator beneficiaries without bleeding disorders based on baseline demographic and clinical characteristics. Key measures of HCRU and outcomes included inpatient hospital admissions, outpatient hematologist visits, and bleeding events. Measures of health care costs were assessed among a subset of beneficiaries enrolled in fee-for-service Medicaid.

Results: PwHB receiving FIX prophylaxis were significantly more likely to have multiple inpatient hospital admissions and had a longer cumulative length of stay per person relative to comparator beneficiaries (30.2 vs 14.8 days, respectively; P = 0.0473). PwHB receiving FIX prophylaxis also had significantly higher rates of bleeding events relative to comparator beneficiaries (0.54 vs 0.02 per person, respectively; P < 0.0001) and outpatient hematologist visits (1.58 vs 0.20 per person, respectively; P < 0.0001). Annual costs among PwHB receiving FIX prophylaxis were significantly higher than costs among comparator beneficiaries ($928,370 vs $34,553 per person, respectively; P < 0.0001) and were overwhelmingly driven by costs associated with FIX therapy.

Conclusions: This analysis found higher rates of HCRU and costs among Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B relative to a matched comparator population of beneficiaries without bleeding disorders. Future research should examine hemophilia B costs and outcomes within the context of new treatments with innovative mechanisms of action, such as gene therapies, RNA interference therapies, and antitissue factor pathway inhibitor therapies.

Demodex infestation is the cause of more than two-thirds of all cases of blepharitis in the United States. Although symptoms may include crustiness, redness, or itching of the eyelids, diagnosis can be accomplished through a simple examination of the eyelashes. The presence of a waste product of the Demodex mite, known as collarettes, on the base of the eyelashes is a pathognomonic sign of Demodex blepharitis. Demodex infestation that results in blepharitis may cause blockage and ultimately atrophy of the meibomian glands, worsening dry eye disease. Until recently, management of Demodex blepharitis has been limited by a lack of approved therapy options. Lotilaner ophthalmic solution 0.25%, the first approved therapy for treatment of Demodex blepharitis, has not only been shown to eradicate Demodex mites in one-half to two-thirds of patients following short-term treatment but also demonstrated continued benefits through 1 year of follow-up. In addition to managing Demodex blepharitis, treatment with lotilaner ophthalmic solution 0.25% may aid in the management of dry eye disease and other forms of ocular surface disease caused by complications of Demodex infestation. As a result, it is possible that successful management of Demodex blepharitis may reduce chronic use of health care resources dedicated to managing other chronic ocular conditions. As eye care professionals recognize Demodex infestation as a key mediator of ocular surface disease, increasing diagnostic awareness and addressing this underlying cause of Demodex blepharitis may reduce the need for specialist follow-up care, decrease the need for chronic therapy, and improve patient outcomes. Through routine screening for Demodex infestation and Demodex blepharitis, eye care professionals can now address an underlying factor in ocular surface disease to improve use of health care resources in the community.

Background: Disease-modifying Alzheimer treatments are becoming available. The value of the treatments will be attenuated by their complexity of delivery and monitoring, creating additional medical cost and caregiver burden.

Objective: To estimate net treatment value using different assumptions for treatment duration and intensity.

Methods: We estimated the lifetime value of hypothetical treatments that reduce disease progression by 30% from a payer perspective, which considers cost offsets, i.e., reduced medical and formal social care costs, and quality-adjusted life-year gains, and a societal perspective, which adds reduction in caregiver burden. Estimates for gross value of the treatment were based on a prior publication, medical cost on Medicare payment rates, and caregiver time use on a survey of 21 clinics. We analyzed 5 hypothetical treatment scenarios: treatment until progression to moderate dementia with (1) biweekly and (2) 4-weekly infusions, and time-limited infusions every 4 weeks for (3) 72, (4) 52, and (5) 24 weeks.

Results: Treatment until progression to moderate dementia would take 5.7 years and generate gross value of $20,734 in direct cost offsets, $83,761 from a payer and $87,749 from a societal perspective, respectively. Added medical cost and caregiver burden for the 5 scenarios would be $44,179, $24,875, $21,632, $20,416, and $14,350, respectively. The maximum value-based price per year would be $7,687, $11,088, $47,708, $67,273, and $158,954.

Conclusions: Assuming identical efficacy and safety, the net value generation of time-limited treatment is projected to be larger than that of chronic treatment. Such determination of net lifetime value can be useful to determine value-based prices for different treatment types.

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.

Ongoing innovations in glucose monitoring, insulin delivery, and telehealth technologies have created a digital diabetes ecosystem populated by connected tools and technologies that have been shown to improve clinical outcomes, lower costs, and reduce the burden of diabetes. Advances in connected continuous glucose monitoring devices, insulin pumps, and insulin pens have led to the development of automated insulin delivery systems that modulate insulin infusion based on sensor glucose data. Similar integrations of continuous glucose monitoring and connected blood glucose meter data into "smart" pens have lessened the guesswork of intensive insulin management for individuals who prefer traditional injection therapy. A growing number of health apps that can be accessed through smartphones and wearable devices provide information and advice that support individuals in adopting healthier lifestyles. The differences in features and functionality give users the ability to select the devices that best meet their unique requirements and preferences. This article reviews the most current digital diabetes technologies and discusses how the connectivity of these tools can create an overarching architecture of feedback mechanisms that monitor an individual's health status, motivate and enhance adherence to self-management, and provide advice and decision-support tools to clinicians as well as other members of the health care team to make living with diabetes more manageable.

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: 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.