Journal of managed care & specialty pharmacy最新文献

Background: Chronic kidney disease (CKD) is common in older adults and is often associated with type 2 diabetes (T2DM) and heart failure (HF). However, little is known about the burden of newly diagnosed CKD in Medicare Fee-for-Service (FFS) beneficiaries, including those with comorbid T2DM or HF.

Objective: To quantify the clinical and economic burden of CKD in Medicare FFS beneficiaries, including those with comorbid T2DM or HF.

Methods: In this retrospective cohort study using 100% Medicare FFS claims data (Parts A, B, D) from 2014 to 2022, beneficiaries with incident CKD (based on a diagnosis code on 2 distinct dates) from January 1, 2015, to December 31, 2021, were included; index date was the date of earliest CKD diagnosis. Beneficiaries with a diagnosis of CKD, acute kidney injury, dialysis, kidney transplantation, or a claim for any condition other than T2DM that could cause kidney disease during a 365-day baseline period prior to index date were excluded. Beneficiaries with CKD were categorized into 4 mutually exclusive cohorts: CKD-only; CKD+HF; CKD+T2DM; and CKD+HF+T2DM based on claims during the baseline period. Clinical burden was measured as prevalence at baseline and incidence of key clinical outcomes at 12 months of follow-up. Economic burden was measured as all-cause and CKD-related health care resource utilization (HCRU) and inflation-adjusted costs in the baseline period and at 12 months of follow-up.

Results: The overall cohort consisted of 2,260,075 patients, mean (SD) age 78.3 (7.7) years (56.5% CKD-only; 7.2% CKD+HF; 30.9% CKD+T2DM; and 5.4% CKD+HF+T2DM). The CKD+HF+T2DM cohort generally exhibited the highest incidence of clinical outcomes within 12 months. Cohorts with HF had higher HCRU across all claim types in both the baseline and 12-month follow-up periods compared with the other cohorts (P < 0.001 for comparison across cohorts). All-cause total costs at 12 months were numerically highest for the cohorts defined by the presence of HF (CKD+HF, mean [SD] $47,668 [$53,978]; CKD+HF+T2DM, mean [SD] $54,477 [$57,430] compared with the other cohorts (CKD-only, mean [SD] $24,180 [$37,623]; CKD+T2DM, $29,602 [$40,963]) (P < 0.001 for comparison across cohorts). Relative spending across cohorts was similar for CKD-related total costs at 12 months.

Conclusions: Older adults with a new diagnosis of CKD experienced considerable clinical and economic burden, and presence of T2DM and HF was associated with larger burden. All-cause mean total costs at 12 months after a new diagnosis of CKD ranged from $24,180 for the CKD-only cohort to $54,477 for the CKD+HF+T2DM cohort.

Background: Three covalent Bruton's tyrosine kinase inhibitors (BTKis) are approved first-line (1L) treatments for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). However, limited real-world data, especially in veterans, evaluate long-term outcomes associated with the different BTKis.

Objective: To describe and compare real-world overall survival (rwOS) among patients with CLL/SLL treated with BTKis in the Veterans Health Administration electronic medical record database.

Methods: This was a retrospective cohort study of patients with CLL/SLL who initiated 1L monotherapy of ibrutinib, acalabrutinib, or zanubrutinib between November 2019 and September 2023. Patients were grouped into 3 cohorts based on the BTKi initiated: (1) ibrutinib, (2) acalabrutinib, or (3) acalabrutinib or zanubrutinib (given small sample initiating zanubrutinib). Key inclusion criteria were 1L monotherapy treatment with a BTKi, at least 2 diagnoses of CLL/SLL, and continuous enrollment at least 12 months prior to and at least 28 days after the initiation of the BTKi. rwOS comparing the BTKi cohorts was analyzed using Kaplan-Meier methodology and adjusted Cox proportional hazards models. Sensitivity analyses adjusting for different sets of covariates were conducted.

Results: The study included samples of 1,059, 504, and 612 patients treated with ibrutinib, acalabrutinib, and acalabrutinib or zanubrutinib (108 received zanubrutinib), respectively. Median rwOS was not reached in any cohort. In the main analysis comparing the ibrutinib and acalabrutinib cohorts, after adjustment for baseline characteristics, treatment with acalabrutinib was associated with an increased risk of death compared with ibrutinib (hazard ratio [HR], 1.33; 95% CI, 1.01-1.76; P = 0.042). In the main analysis comparing the ibrutinib and acalabrutinib or zanubrutinib cohorts, the adjusted risk of death was numerically higher for acalabrutinib- or zanubrutinib-treated patients compared with ibrutinib (HR, 1.32; 95% CI, 1.00-1.74; P = 0.050). For both comparisons, sensitivity analyses indicated similar trends in rwOS.

Conclusions: As new therapies emerge, this study highlights the comparative effectiveness of BTKis in the real world, potentially informing current clinical practice.

Background: Substantial rates of nonadherence are observed among people with hemophilia A (PwHA) receiving standard- or extended-half-life factor (F)VIII replacement products, although better adherence is associated with fewer bleeding events and better quality of life. Emicizumab is the first bispecific FIXa-/FX-directed antibody approved for routine prophylaxis in PwHA. More real-world adherence and persistence data on emicizumab and other new hemophilia treatments are needed to better inform decisions on population health care and individualized treatments for PwHA.

Objective: To describe real-world emicizumab adherence and persistence among PwHA in the United States.

Methods: This retrospective, observational cohort study used adjudicated health plan claims data from IQVIA PharMetrics Plus. This study included PwHA with at least 1 claim noting hemophilia A diagnosis between May 1, 2017, and September 30, 2023; at least 2 claims for emicizumab fills between November 1, 2017, and September 30, 2023; and continuous medical and pharmacy insurance benefit coverage for at least 6 months before and at least 12 months after the first emicizumab fill (index date). Adherence to emicizumab was measured during the 12-month post-index period using the proportion of days covered (PDC). Persistence was defined as the proportion of PwHA who did not discontinue emicizumab during the post-index period, where discontinuation was defined as a treatment gap of at least 60 days. Among adherent PwHA (PDC ≥ 80%), the number of post-index claims for FVIII were evaluated.

Results: A total of 280 PwHA were included in the study, of whom 98% were male and the mean age was 26 (SD = 16) years. Overall, 76% of PwHA were adherent to and 85% of PwHA were persistent with emicizumab. Rates of adherence and persistence were high across age- and geography-based subgroups, ranging from 61% to 97%. Among PwHA adherent to emicizumab (n = 213), 57% had no post-index claims for FVIII.

Conclusions: In the first year following emicizumab initiation, high adherence and low discontinuation rates were observed among PwHA in a real-world setting.

The mucopolysaccharidoses (MPS) are a group of rare genetic disorders that cause chronic, progressive cellular damage, which can affect multiple organ systems and lead to reduced life expectancy. Diagnosis and treatment of MPS are often delayed, during which irreversible organ damage can occur. Treatments are not currently available for all MPS subtypes, and those that are available are associated with significant limitations. The therapeutic pipeline for MPS is active; however, bringing new treatments for rare conditions to market is difficult. To discuss the role of managed care in improving patient care and outcomes in MPS, AMCP Market Insights virtually convened an expert panel of managed care stakeholders in September 2025. Key insights from the discussion were that the earliest possible intervention is crucial to improving outcomes in MPS; expertise is limited because of the rarity of these disorders; patients and caregivers face significant challenges accessing treatments and other interventions for MPS; and the therapeutic pipeline for MPS holds promise for addressing some critical treatment gaps but leaves others unmet. Suggested payer practices for MPS also emerged from the discussion.

Clinical programs are essential to managed care pharmacy, supporting safe, effective, and affordable medication use across populations and improving outcomes. This primer provides an overview of program types, as well as design, implementation, and evaluation strategies across health plans, pharmacy benefit managers, accountable care organizations, and integrated delivery systems. It highlights key goals, emerging trends, and the importance of value-based alignment.

Background: Medication adherence varies across socioeconomic and demographic groups. Prior work has examined these relationships in selected populations and with zip code-level socioeconomic measures, but less is known about adherence patterns across all medications when using more granular community measures and location proxies.

Objective: To quantify associations between socioeconomic, demographic, and medication class factors and outpatient medication adherence.

Methods: We conducted a retrospective cohort study of 1,252,986 outpatient medication orders for 189,832 adults placed at a large health system in the Mountain West (University of Utah Health) from January 1, 2022, to January 1, 2024. Pharmacy, electronic health record, and Surescripts data were used. Medication adherence (proportion of days covered) was modeled with multivariate β regression with covariates including insurance, race, sex, marital and employment status, medication class, and Social Vulnerability Index socioeconomic quartile.

Results: Medicaid (adjusted odds ratio [aOR] = 0.87, P < 0.001) or no insurance (aOR = 0.80, P < 0.001) were associated with lower adherence than commercial coverage, whereas Medicare was associated with improved adherence (aOR = 1.06, P < 0.001, when compared with commercial insurance). Black, Native Hawaiian/Pacific Islander, and American Indian/Alaska Native patients had lower adherence compared with White patients (aOR = 0.93, P < 0.001 and aOR = 0.96, P < 0.001, respectively). The most vulnerable Social Vulnerability Index quartile (Q4) modestly reduced adherence (aOR = 0.98, P < 0.001) compared with Q1. Antihypertensive, cholesterol-lowering, antidepressant, and heart failure drugs had markedly higher adherence (aORs 1.35-1.60, P < .01, when compared with other medication classes).

Conclusions: Several individual-level factors had strong associations with adherence. Community-level socioeconomic vulnerability had a modest association.

Background: Clinical outcome assessments (COAs) are tools widely used in clinical trials to evaluate treatment efficacy by capturing patient experience. However, little is known about how COAs are used in specialty drug coverage decisions.

Objective: To describe the frequency, type, and role of COAs in US commercial health plans' specialty drug coverage policies.

Methods: We analyzed coverage data from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database, which tracks specialty drug coverage policies from 18 large US commercial health plans. Researchers reviewed each policy to identify disease-specific COAs, excluding tools assessing objective physiological data or clinical algorithms (eg, biomarkers, risk prediction scores). We categorized COAs by (1) type (ie, patient-reported outcomes [PROs], clinician-reported outcomes [ClinROs], etc), (2) time point (initial approval vs reauthorization), and (3) application (ie, supporting diagnosis, documenting baseline, limiting access for initial criteria; or defining meaningful treatment response for reauthorization criteria).

Results: As of April 2024, SPEC included 13,933 coverage policies for 440 specialty drugs for 386 indications. These policies incorporated COAs for 169 (43.8%) indications. In total, 2,188 (15.7%) policies referenced at least 1 COA, and of these 763 (34.9%) referenced multiple COAs. Because policies can incorporate COAs into both initial and reauthorization criteria, we identified 4,305 total COA references. Inclusion of COAs varied across plans, ranging from 5% to 27.2% of coverage decisions. COAs appeared 2,077 times in initial criteria, most commonly as ClinROs (69.0%) and infrequently as PROs (5.7%). In initial criteria, plans primarily used COAs to limit access (50.5%), whereas in reauthorization criteria, plans used COAs to determine treatment response.

Conclusions: COA inclusion varied across plans, with most using them to limit access. ClinROs were the most frequently used COA type, underscoring the pivotal role of providers on behalf of patients and health plans in shaping treatment access decisions. Plans used PROs relatively infrequently, with some including PROs in their policies more than others, suggesting a missed opportunity to consistently incorporate patients' voices to complement the assessment of treatment outcomes when determining coverage. Future research should explore the rationale behind COA use and its implications for treatment access.