Journal of managed care & specialty pharmacy最新文献

Background: Although bevacizumab and its biosimilars are commonly used, there are limited real-world data on bevacizumab use in the United States, especially biosimilar bevacizumab used in ophthalmologic conditions.

Objective: To evaluate use patterns and patient characteristics for the originator bevacizumab relative to its biosimilars for labeled and off-label oncology and ophthalmology conditions and characterize adverse events in patients using bevacizumab for oncologic indications.

Methods: We conducted a retrospective cohort study with the Biologics and Biosimilars Collective Intelligence Consortium-distributed database to identify patients aged 21 years and older who received bevacizumab between January 1, 2010, and June 30, 2021. Oncology indications included colon, lung, and gynecologic (cervical, uterine, and ovarian) cancers. Ophthalmologic indications included neovascular age-related macular degeneration (AMD), retinal vein occlusion (RVO), choroidal neovascularization (CNV), and proliferative diabetic retinopathy (PDR). We also captured patients' demographic and clinical characteristics.

Results: Total bevacizumab product (originator and biosimilars) use increased over time for RVO, CNV, and PDR starting in 2015 but decreased for AMD after 2016. For ophthalmology, bevacizumab product users were primarily male (56.8%), had a mean age of 62.9 years (SD = 0.08), and had a mean Charlson/Elixhauser combined comorbidity score ranging from 0.7 (CNV) to 2.7 (PDR). Bevacizumab users for oncology indications were mostly female (61.8%), had a mean age of 62.9 years (SD = 12.2), and had a mean Charlson/Elixhauser combined comorbidity score of 7.4 (SD = 3.0). Oncologic biosimilar product use increased over time between 2019 and 2020 as follows: colon cancer, 6.2% to 49.4%; lung cancer, 1.9% to 36.2%; and gynecologic cancer, 2.4% to 38.1%.

Conclusions: Bevacizumab product use increased across most indications during the study period. Use for biosimilars increased in later years relative to the originator once available on the market. Limited data are available on real-world biosimilar use in the United States; future research should include monitoring for use and adverse events of these products.

Background: Duchenne muscular dystrophy (DMD) is a rare neuromuscular disorder, and data on the impact of patients' race on treatment outcomes and health care resource utilization are lacking.

Objective: To describe the real-world treatment and health care utilization among patients with DMD, by race, in a Medicaid population.

Methods: This was a retrospective cohort study of patients with DMD in the Merative Multi-State Medicaid Database between January 2017 and June 2021. Patients with DMD were identified using a validated algorithm and included male patients with at least 2 DMD diagnoses (earliest DMD diagnosis date = index date), aged 40 years or younger, with at least 12 months of continuous enrollment prior to index date, and with at least 12 months (or evidence of death) following the index date were selected. Demographics, clinical characteristics, treatment utilization, and health care utilization and costs were reported by race and ethnicity in the 12-month baseline and 12-month follow-up periods.

Results: A total of 561 patients were included in the study, of which 360 (64.2%) were White, 50 (8.9%) were Black, 33 (5.9%) were Hispanic, and 118 (21.0%) were of other/unknown race and ethnicity. The median age on the index date was 16, 13, 14, and 15 years among the race and ethnicity categories, respectively. In the follow-up, period clinical characteristics were similar across cohorts. Corticosteroids were the most commonly received treatment, with the highest use among Hispanic patients (73%) and lowest use among Black patients (52%). A third of patients treated with corticosteroids received deflazacort, with similar utilization across groups. Exon-skipping therapy use was rare, with 3% utilization overall, and highest use among White patients (4.2%). In both the baseline and follow-up periods, differences in health care costs were not statistically significant. White patients had the highest total costs in the follow-up period (mean [SD] = $108,895 [$346,934]) compared with $59,501 [$85,758] in the Black cohort, $61,199 [$67,021] in the Hispanic cohort, and $65,247 [$119,733] in the unknown/other cohort. Differences in total health care costs were driven by outpatient pharmacy costs, likely because of the larger proportion of White patients having a prescription for an exon-skipping therapy.

Conclusions: Differences were seen across race and ethnicities in select clinical characteristics, DMD treatments, and health care utilization and costs in a Medicaid population.

The Global Initiative for Asthma, the most widely cited international guidelines for asthma management, currently recommends the use of maintenance and reliever therapy with an inhaled corticosteroid-formoterol inhaler based on evidence showing reductions in asthma exacerbations and hospitalizations vs short-acting relievers alone. A review of Medicare Part D plans based on a Kaiser Family Foundation report reveals that the formulary design of many plans is not aligned with this recommendation, resulting in restricted prescriber adherence to the guidelines and limited patient access to standard-of-care treatments. To improve clinical outcomes, it is key that Medicare Part D plans minimize barriers to access through prioritization of inhaled corticosteroid-formoterol regimens as preferred formulary agents.

Background: In 2022, the US Food and Drug Administration approved cemiplimab in combination with chemotherapy (CCT) as a first-line treatment for advanced non-small cell lung cancer (aNSCLC). However, whether CCT presents a cost-effective alternative to the previously preferred first-line treatment, pembrolizumab plus chemotherapy (PCT), remains uncertain.

Objective: To evaluate the cost-effectiveness of CCT vs PCT as the first-line treatment for aNSCLC from a US health care payer perspective.

Methods: A 3-state partitioned survival model with a 10-year horizon was constructed. Clinical data were derived from the EMPOWER-Lung 3, KEYNOTE-407, and KEYNOTE-189 trials. Costs and quality of life were obtained from published 2024 US list prices and literature. The cost, quality-adjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER) were calculated. All outcomes were discounted at a rate of 3% per year. Scenario analyses, deterministic and probabilistic sensitivity analyses, and subgroup analyses were performed for patients with different programmed death ligand 1 (PD-L1) levels.

Results: In the base-case analysis, the total cost of PCT was $207,926 with 1.609 QALYs, whereas CCT had a total cost of $175,247 with 1.657 QALYs. Results from the scenario analyses were consistent with the base-case analysis, indicating that CCT was a dominant treatment strategy over PCT (ICER =-$675,304 per QALY). The cost of pembrolizumab highly impacted the ICER. At a willingness-to-pay threshold of $150,000 per QALY, CCT would be accepted as a cost-effective option 96.9% of the time. In subgroup analyses, CCT remained a dominant alternative to PCT for patients with PD-L1 levels of at least 50% and 1%-49%.

Conclusions: This cost-effectiveness analysis suggests that CCT is a dominant first-line treatment option for aNSCLC with PD-L1 levels of at least 1% compared with PCT.

Background: Many payers and pharmacy benefit managers (PBMs) use step therapy requirements and formulary alternatives to reduce prescription spending. The clinical utility and ultimate therapy outcomes for patients participating in these programs is an area of needed research.

Objective: To evaluate medication outcomes and time requirements to access therapy in patients required to use a step therapy or formulary alternative after being prescribed a specialty medication for psoriasis (PsO) or atopic dermatitis (AD).

Methods: A single-center, retrospective review of data collected from electronic health records and the specialty pharmacy patient management system was conducted. Patients were included if they had a referral for a specialty medication by a dermatology provider for PsO or AD that was discontinued because of the payer/PBM requiring step therapy or formulary alternative use from January 2021 to June 2022. The primary outcome was the number of days from the time of patient referral for specialty medication until the second specialty medication referral. Secondary outcomes included the number of patients that failed step therapy or formulary alternative and were referred back to the specialty pharmacy as well as the number of patients not started on a specialty medication and the reason.

Results: The included patients (N = 83) were predominantly White (75%) and female (61%) with a median age of 50 years (interquartile range [IQR] = 37-61 years). 51% of patients had an indication of AD. 62 patients were required to use step therapy, and 21 were required to use a formulary alternative. Of the patients required to use step therapy, 37 (60%) had a second referral for a specialty medication because of failing step therapy requirements, with a median time to a second referral of 75 days (IQR = 27-135). The remaining 25 patients were not started on a specialty medication: 5 patients (8%) benefited from and remained on step therapy, 15 (24%) were lost to follow-up, 3 (5%) decided not to start step therapy, and 2 (3%) were referred for psoriatic arthritis evaluation. All patients required to use a formulary alternative initiated specialty medication with a median time from the initial referral to second referral of 3 days (IQR = 1-9 days).

Conclusions: Most patients required to complete nonspecialty step therapy ultimately initiated specialty medications after a lengthy delay, demonstrating the potential impact of step therapy requirements on initiating clinically appropriate treatment for AD and PsO. Further research assessing the financial burden and clinical impact of requiring step therapy before specialty medications is warranted.