Journal of managed care & specialty pharmacy最新文献

Background: Non-small cell lung cancer (NSCLC) presents a formidable global health challenge owing to significant morbidity, high mortality rates, and substantial economic burden. Recent advances in targeted therapies and immunotherapies have transformed NSCLC treatment, but efficacy varies across patients. Tailoring treatment to patients can improve outcomes and potentially improve cost-effectiveness (ie, value for money) as well. For NSCLC, cost-effectiveness must often be estimated using economic modeling, and estimates are only as good as the models. Existing cost-effectiveness models are not necessarily suitable for evaluating personalized medicines.

Objective: To identify and assess cost-effectiveness models of NSCLC.

Methods: We searched for studies indexed in PubMed and Embase from 2012 to October 2023 that described cost-effectiveness models of NSCLC. Study details were extracted, summarized, and evaluated for adherence to the Consolidated Health Economic Evaluation Reporting Standards.

Results: We identified 237 unique models, 40% of which were published in 2022 or 2023. Despite cross-model heterogeneity, most models used the same 3 health states (progression-free survival, progressive disease, and death) combined with time-to-event equations that characterize risks. Thirty models included a diagnostic component, most of which considered guiding treatment selection using biomarkers. Adherence to the overall Consolidated Health Economic Evaluation Reporting Standards checklist was generally incomplete, and adherence to a subset of model-related questions even more so.

Conclusions: The large number of models that were found, almost half of which were published since 2022, underscores the importance of cost-effectiveness analysis in NSCLC. Variable adherence to best practices suggests opportunities for improvement, however, and making high-quality, open-source models available to researchers may be valuable.

Background: Heart failure is a prevalent disease state associated with limitations in function, hospitalization, and death. The 2022 American Heart Association/American College of Cardiology/Heart Failure Society of America guidelines recommend medications including sacubitril/valsartan to decrease morbidity and mortality in patients with heart failure. However, if patients are nonadherent to treatment or experience barriers to care, they will forgo these benefits.

Objective: To assess the pharmacy staff compliance rate to a workflow protocol for sacubitril/valsartan prescriptions received by Cleveland Clinic Home Delivery (HD) and Adherence Pharmacy (AP) and determine the nonclinical benefits experienced by the patients enrolled in the protocol.

Methods: At Cleveland Clinic, there are 2 mail-order pharmacies: HD and AP. Both pharmacies offer a variety of benefits and adherence services, with each pharmacy having their own unique services offered. With numerous adherence services provided by both pharmacies, it is likely that patients with heart failure would see clinical and nonclinical benefits, such as cost savings. This project created a triage protocol for patients deemed to experience the most benefit from services offered through AP. The primary endpoint of this project was determining the feasibility of a medication-specific workflow protocol for sacubitril/valsartan prescriptions at HD and AP.

Results: There were 114 qualifying prescriptions per the protocol, and 98 of those prescriptions were appropriately screened by the pharmacy staff, equating to an 86% compliance rate for the primary outcome. Of the 98 patients included in the workflow protocol, prior authorization was completed by pharmacy staff for 41 patients (41.8%), manufacturer copay card was applied for 13 patients (13.3%), 17 patients (17.3%) were enrolled in grant funding programs, and patient assistance program enrollment was initiated for 9 patients (9.2%).

Conclusions: Medication-specific workflows may be a feasible option to implement for pharmacies to ensure the offering of adherence services to patients with high-risk disease states using treatment with expensive, branded medications.

Pulmonary hypertension associated with interstitial lung disease (PH-ILD) is a complex condition in which 2 consequential diseases interact and increase negative outcomes. Although the pathophysiologic mechanisms of PH-ILD are not yet well understood, the pronounced effect on functional status, supplemental oxygen requirements, health care resource utilization, and mortality that frequently accompany this diagnosis are well documented. A critical feature that complicates pathophysiologic understanding of PH-ILD is that progression of the pulmonary vascular disease does not always appear to be driven by the underlying lung disease. As PH-ILD is a progressive disease, early recognition and treatment initiation have the potential to delay the increased burden it creates. Historically, therapeutic development within pulmonary hypertension has concentrated on pulmonary arterial hypertension (PAH). However, PH-ILD and PAH are categorically distinct-belonging to distinct PH groups owing to differing pathophysiological mechanisms and therapeutic implications. PAH and PH-ILD may have numerous similarities; however, when PAH therapies have been studied in patients with PH-ILD, inconclusive efficacy (bosentan, sildenafil, tadalafil, iloprost) and at times deleterious safety findings (riociguat, ambrisentan) have been observed. Despite the paucity of evidence to support PAH therapy use in this patient population, widespread off-label use of PAH therapies arose as a result of a historical lack of PH-ILD-approved treatment. Recently, inhaled treprostinil-a prostacyclin analog-has become the first therapy approved for treatment of PH-ILD. In the phase 3 INCREASE trial, inhaled treprostinil was effective in improving 6-minute walk distance (the primary endpoint; P < 0.001) as well as N-terminal pro-B-type natriuretic peptide levels (P < 0.001). The approval of inhaled treprostinil in 2022 facilitates evidence-based therapeutic management. In addition, the 7th World Symposium on Pulmonary Hypertension has recently published an extensive summary of clinical research to date in PH-ILD. The proceedings from the 7th World Symposium on Pulmonary Hypertension provide timely recommendations for investigation of PH-ILD and a framework for assessing treatment needs. The therapeutic landscape advances are poised to transform PH-ILD care and improve outcomes for patients with PH-ILD.

Background: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.

Objective: To assess the cost-effectiveness of caplacizumab in iTTP from the US payer perspective.

Methods: The National Institute for Health and Care Excellence's model was adapted to the US setting using US costs and discount rates. In contrast to previous cost-effectiveness analyses that accounted only for acute outcomes, our model consisted of a 3-month decision tree for an acute iTTP episode, followed by a Markov model to project long-term costs and outcomes (time horizon: up to 55 years; 3-monthly cycles).

Results: Patients taking caplacizumab with PEX+IS experienced an incremental gain of 2.96 life years (LYs) and 1.75 quality-adjusted LYs relative to PEX+IS alone, at an increased lifetime cost of $256,000. The incremental cost-effectiveness ratio was $86,400 per LY and $146,300 per quality-adjusted LY gained.

Conclusions: Considering willingness-to-pay thresholds of $150,000 to $200,000, the addition of caplacizumab to PEX+IS may be cost-effective compared with PEX+IS alone for the treatment of iTTP in a US setting.

Pharmacoequity is a health system and policy goal of ensuring equitable access to high-quality medications for all individuals, regardless of factors such as race, ethnicity, socioeconomic status, or resource availability to reduce health disparities. Although measurement frameworks have been widely used in health equity contexts, a focused framework for pharmacoequity remains a critical gap. In this article, we introduce a novel pharmacoequity measurement framework anchored in the patient medication-use journey. The framework includes the following domains: (1) access to health care services, (2) prescription generation, (3) primary medication nonadherence, (4) secondary medication nonadherence, and (5) medication monitoring. For each domain, we provide examples of outcome measures and potential data sources that can be used for evaluation. We also outline an implementation workflow of the pharmacoequity measurement framework that population health stakeholders can use across various settings (eg, health systems, health plans). The framework provides a structured approach to identify existing gaps in the path toward achieving pharmacoequity and lay the foundation for targeted interventions. Additionally, it enables ongoing monitoring of progress toward achieving pharmacoequity while identifying interventions that are effective, scalable, and sustainable.

Background: The incidence of melanoma has increased significantly in the past few decades, posing a significant public health challenge. However, there is an evidence gap regarding the marginal costs of treating melanoma.

Objective: To examine the marginal health care expenditures for melanoma compared with other nonskin cancers among US adults.

Methods: This study examined individuals aged 18 years or older with melanoma, nonmelanoma skin cancer (NMSC), and other cancers from the 2011-2020 Medical Expenditure Panel Survey datasets. Direct health care expenditures involving hospital inpatient, outpatient, prescription medications, dental, vision, home health care, and other medical services for melanoma were analyzed using generalized linear models, and comparisons were made with expenditures for other types of cancers while adjusting for other patient characteristics.

Results: There were 0.70 million individuals (95% CI = 0.61-0.78) diagnosed with melanoma annually. Total health care expenditures among individuals with melanoma, NMSC, and other cancers were $19,427, $13,744, and $23,741, respectively. A generally increasing trend of expenditure was observed over the years. Notably, office-based care (30.46%), inpatient services (28.78%), and prescription (18.27%) costs primarily accounted for the health care burden of patients with melanoma. Adjusted marginal total health care expenditures for melanoma were found to be lower ($-3,369.01 [95% CI = -$5,934.15 to -$803.85]) than other cancers but higher ($2,844.75 [95% CI = $2,204.77-$3,484.72]) compared with NMSC. Prescription expenditures were similar across the 3 cancer study groups.

Conclusions: This study found that adjusted marginal expenditures for melanoma were higher than those with NMSC but lower than other nonskin cancers, with office-based care and inpatient expenditures contributing to most of the expenditures. The findings suggest that concerted efforts are needed to control the primary cost drivers to reduce the associated burden of potentially preventable skin cancer like melanoma.

Background: Per capita spending on drugs in the United States is double that of Canada. One commonly debated point when comparing the 2 countries is whether this additional spending allows residents of the United States access to valuable therapies not available in Canada.

Objective: To characterize the therapeutic value of prescription drugs used in the United States that are not marketed in Canada.

Methods: This cross-sectional study used IQVIA Multinational Integrated Data Analysis System data to identify drugs purchased in the United States but not in Canada from 2017 to 2021. Drug listing and regulatory review statuses were obtained. We categorized the drugs into 8 mutually exclusive groups: listing status in Canada ("cancelled post-market" or "dormant; approved but not marketed; cancelled pre-market"), other alternatives available ("formulation unavailable," "existing drug class," or "therapeutically similar"), "pre-approval," "atypical access available," or "unavailable without alternatives marketed" in Canada. Therapeutic value assessments of drugs in the last category were obtained from 3 international organizations.

Results: 2,084 products were purchased in the United States but not in Canada from 2017 to 2021; 1,685 were excluded because they were not prescription drugs, were combinations in which each active pharmaceutical ingredient was already available in the United States as a separate drug, had been discontinued in the United States by August 30, 2023, or were marketed in Canada by August 30, 2023. After exclusions, there were 399 drugs; 120 (30%) were "cancelled post-market," 38 (10%) were "dormant; approved but not marketed; cancelled pre-market," 49 (12%) were "formulation unavailable," 130 (33%) were "existing drug class," 35 (9%) were "therapeutically similar," 3 (1%) were "preapproval," 15 (4%) were "atypical access available," and 9 (2%) were "unavailable" in Canada. 6 of the 9 drugs had been evaluated by 1 or more independent organizations, and all 6 were rated as offering minor to no additional therapeutic value compared with existing drugs.

Conclusions: There was similar access to important prescription drug therapies in the United States and Canada. Overall, the additional spending in the United States may not have necessarily translated into access to important therapeutic innovations.

Background: For patients with metastatic non-small cell lung cancer (mNSCLC), next-generation sequencing (NGS) biomarker testing has been associated with a faster time to appropriate targeted therapy and more comprehensive testing relative to polymerase chain reaction (PCR) testing. However, the impact on payer costs and clinical outcomes during patients' treatment journeys has not been fully characterized.

Objective: To assess the costs and clinical outcomes of NGS vs PCR biomarker testing among patients with newly diagnosed de novo mNSCLC from a US payers' perspective.

Methods: A Markov model assessed costs and clinical outcomes of NGS vs PCR testing from the start of testing up to 3 years after. Patients entered the model after receiving biomarker test results and then initiated first-line (1L) targeted or nontargeted therapy (immunotherapy and/or chemotherapy) depending on actionable mutation detection. A few patients with an actionable mutation were not detected by PCR and inappropriately initiated 1L nontargeted therapy. At each 1-month cycle, patients could remain on treatment with 1L, progress to second line or later (2L+), or die. Literature-based inputs included the rates of progression-free survival (PFS) and overall survival (OS), targeted and nontargeted therapy costs, total costs of testing, and medical costs of 1L, 2L+, and death. Per patient average PFS and OS as well as cumulative costs were reported for NGS and PCR testing.

Results: In a modeled population of 100 patients (75% commercial and 25% Medicare), 45.9% of NGS and 40.0% of PCR patients tested positive for an actionable mutation. Relative to PCR, NGS was associated with $7,386 in savings per patient (NGS = $326,154; PCR = $333,540) at 1 year, driven by lower costs of testing, including estimated costs of delayed care and nontargeted therapy initiation before receiving test results (NGS = $8,866; PCR = $16,373). Treatment costs were similar (NGS = $305,644; PCR = $305,283). In the PCR cohort, the per patient costs of inappropriate 1L nontargeted therapy owing to undetected mutations were $6,455, $6,566, and $6,569 over the first 1, 2, and 3 years, respectively. Relative to PCR testing, NGS was associated with $4,060 in savings at 2 years and $1,092 at 3 years. Patients who initiated 1L targeted therapy had an additional 5.4, 8.8, and 10.4 months of PFS and an additional 1.4, 3.6, and 5.3 months of OS over the first 1, 2, and 3 years, respectively, relative to those who inappropriately initiated 1L nontargeted therapy.

Conclusions: In this Markov model, as early as year 1, and over 3 years following biomarker testing, patients with newly diagnosed de novo mNSCLC undergoing NGS testing are projected to have cost savings and longer PFS and OS relative to those tested with PCR.

Background: Individuals with depression who do not respond to initial antidepressant may switch to a different antidepressant, add a second antidepressant, or add an atypical antipsychotic. Previous studies comparing these strategies' efficacy and safety reported conflicting results, and the impact of these strategies on subsequent health care utilization is unknown.

Objective: To compare health care utilization between individuals with depression who switched antidepressants, added a second antidepressant (ie, combination), or added an atypical antipsychotic (ie, augmentation) following their initial antidepressant.

Methods: This retrospective cohort study used a 25% random sample of the IQVIA PharMetrics Plus for Academics health plan claims database. The study cohort included individuals aged 10-64 years who newly initiated an antidepressant at any point from January 2016 to December 2020. New use was defined as no evidence of an antidepressant in the 180 days preceding the antidepressant dispensing. Individuals had to have a depression diagnosis and a treatment change in the 180 days following the initial antidepressant. The index date was the date of the first observed antidepressant change, which was defined as a switch, combination, or augmentation. Health care utilization, measured as the number of outpatient visits, any all-cause hospitalization, and any emergency department (ED) visit, was assessed in the 180 days after the index date. Negative binomial regression models evaluated the rate ratio of the number of outpatient visits. Logistic regression models estimated the odds ratio of a hospitalization, and modified Poisson regression estimated the relative risk of an ED visit. Models were adjusted for demographics, clinical characteristics, and previous health care utilization.

Results: Among 3,847 individuals with depression who had the first treatment change following the initial antidepressant, we identified 2,418 (62.9%) who switched, 1,268 (33.0%) who combined, and 161 (4.2%) who augmented their antidepressant. The augmentation group had a significantly higher rate of outpatient visits than the combination group (adjusted rate ratio = 1.14, 95% CI = 1.04-1.25). There was no statistically significant difference in hospitalizations or ED visits between the switch and augmentation vs combination groups.

Conclusions: Augmentation comprised 4% of our antidepressant cohort but had higher outpatient health care utilization than those who combined treatment.

Background: In 2021, Medicare Part D gross prescription drug spending amounted to $216 billion, a number that has more than doubled over the last 10 years. Spending in Medicare Part D is concentrated on a small number of drugs, and spending on specialty drugs has increased in recent years. However, the extent to which concentration in Part D spending has changed over time and the drivers of this change have not been described.

Objective: To quantify the time trends in Medicare Part D spending and utilization, the concentration of spending, and the share of spending accounted for by specialty drugs from 2012 to 2021.

Methods: In this repeated cross-sectional study, we used data from the Centers for Medicare & Medicaid Services Part D Drug Spending Dashboard to investigate the time trends in total gross spending, prescriptions claims, and the average cost of a prescription claim for Part D drugs. We assessed the concentration based on the share of total gross spending and prescriptions by the drugs with the top 1%, 5%, and 10% of the highest spending and Lorenz curves and Gini coefficients. In addition, we stratified our analyses by specialty and nonspecialty drugs.

Results: Over the last 10 years, total gross drug spending in Medicare Part D increased by 103.5%, with a compounded annual growth rate of 8.2%. This change was driven by both increases in prescription claims and price increases of existing drugs to a similar degree. The concentration of spending intensified, with the top 1% of drugs accounting for an escalating share of total spending (from 31.4% to 41.1%). Over the 10-year study period, these top-spending drugs accounted for 5.6% of prescriptions but 34.6% of spending. Lorenz curves and increased Gini coefficients similarly showed that a smaller number of drugs accounted for increased spending over the study period. Specialty drug spending increased by 566.5%, with a compounded annual growth rate of 23.5%. The share of total spending on specialty drugs increased from 21.7% in 2012 to 71.1% in 2021. In 2021, specialty drugs accounted for 6.2% of prescriptions but 71.1% of total spending.

Conclusions: Medicare Part D gross drug spending became increasingly more concentrated from 2012 to 2021, which was especially pronounced for specialty drugs. Increases in prices for specialty and other brand-name drugs will likely continue to drive gross spending upward. Although the Inflation Reduction Act provisions will likely reduce net spending on selected drugs, other policy changes may be warranted.