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Background: Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment.

Objectives: To explore the prevalence of AEs and treatment tolerability problems as reported by patients who received therapies for AL amyloidosis, and to examine the effects of AEs on treatment continuation and on health-related quality of life (HRQOL).

Methods: Patients with AL amyloidosis were recruited for this noninterventional, longitudinal, online survey. The patients responded to survey items regarding demographics, disease characteristics, most recent AL amyloidosis treatment, and HRQOL. The study analyses are based on data collected during the 6-month follow-up survey and are restricted to patients who completed the baseline and 6-month surveys and received treatment for AL amyloidosis within 6 months before the follow-up survey.

Results: A total of 100 patients met the inclusion criteria and were included in the study. The patients self-reported having a variety of AEs, which ranged in severity. Overall, 69.4% of patients had problems tolerating their treatment in the past 6 months, of whom 22% discontinued at least 1 therapy. In addition, approximately 33% of patients reduced their AL amyloidosis treatment because of AEs. Most often reported AEs included fatigue (83%), shortness of breath (53%), nausea (52%), and diarrhea (51%). Overall, 50% of the patients reported that their treatment was moderately well-tolerated and 41% said it was very well-tolerated. Those whose treatment was not well-tolerated had significantly worse HRQOL than patients whose treatment was well-tolerated.

Conclusions: Patient-reported experiences should be considered by clinicians when making treatment-related decisions. More research is needed to explore additional factors that may contribute to treatment discontinuation in patients with AL amyloidosis.

Background: As psoriatic arthritis (PsA) treatment choices continue to expand, it is important to consider patient preferences for treatment modalities for PsA. Involving patients in treatment decisions can influence adherence to treatment and outcomes of therapy.

Objective: To determine patient preferences for medication attributes prescribed for patients with PsA.

Methods: A choice-based conjoint survey was mailed to 2800 randomly selected patients with PsA who were enrolled in Humana Medicare and commercial plans. Patients had been diagnosed with PsA between January 1, 2012, and September 30, 2016. The medication attributes included in the survey were the medication route of administration, frequency of administration, ability to reduce daily joint pain and swelling, likelihood of serious infections, improvement in the patient's ability to perform daily activities, achieving clear or almost clear skin, and cost. Hierarchical Bayesian models were used to score patient preferences after adjusting for demographic and clinical characteristics. The mean attribute importance scores were used to rank patient preferences.

Results: A total of 468 patients (258 with a Medicare plan and 210 with a commercial plan) completed the survey. The top 3 medication attributes for patients in Medicare plans were route of administration, cost, and improvement in the ability to perform daily activities. For patients in commercial plans, the top 3 medication attributes were cost, route of administration, and frequency of administration. Within the top 2 attributes for patients in both plans, the oral route of administration and lower cost were most preferred.

Conclusion: Medication route of administration and cost were the 2 most important considerations for patients diagnosed with PsA who were enrolled in Medicare or commercial plans with Humana. As PsA treatment choices continue to expand, considering patient preferences may improve patient adherence and treatment outcomes and should be considered when making treatment decisions for this patient population.

Background: Partial-onset seizures are the most common type of seizures in patients with epilepsy. In addition to the significant impact on patients, the unpredictability of seizures often also affects family members or caregivers. Caregiver burden in relation to patient treatment may help to guide treatment choices for patients. Quantitative evidence about the relationship between workplace absences, costs, and treatment burden among caregivers of patients with partial-onset seizures is lacking.

Objective: To compare direct and indirect healthcare costs and absences among employed caregivers of patients with partial-onset seizures who are receiving monotherapy or adjunctive therapy with antiepileptic drugs (AEDs).

Methods: This retrospective study analyzed data of employed caregiver spouses of patients with partial-onset seizures and paired them with the patients into 2 groups based on the patient's therapy: the monotherapy cohort or the adjunctive therapy cohort (ie, >90 days of concomitant use of ≥2 AEDs). Patients and caregivers had to have ≥12 months of continuous data after the index date. Separate 2-part regression models were used to compare direct medical and prescription costs; indirect costs (ie, sick leave, short-term and long-term disability, and workers' compensation); and differences in work absences for caregivers.

Results: The baseline caregivers' characteristics were similar in the monotherapy cohort (N = 238) and the adjunctive therapy cohort (N = 129). Caregivers' total direct costs were $4231 in the monotherapy cohort and $7217 in the adjunctive therapy cohort. The caregivers of patients in the monotherapy cohort were less likely to use inpatient hospital services than caregivers of patients in the adjunctive therapy cohort (1.3% vs 9.9%, respectively; P = .0016). The caregivers' total indirect costs were $912 and $1192 in the monotherapy and adjunctive therapy cohorts, respectively. Sick days were significantly lower in the monotherapy cohort (2.4 days vs 4.4 days annually; P <.0001), with an associated cost difference of $541.

Conclusion: Caregivers of patients with partial-onset seizures in the adjunctive therapy cohort had significantly greater medical and sick day costs than caregivers in the monotherapy cohort. These findings suggest that higher treatment burden among patients with epilepsy is associated with greater direct and indirect healthcare costs for their caregivers.

Background: Current national estimates for acute myelogenous leukemia (AML) indicate this disease accounts for 1.1% of new cancer diagnoses and 1.8% of cancer deaths in the United States. The 5-year overall survival rate for patients with AML was 27.4% between 2008 and 2014. The standard induction for patients with AML includes cytarabine, infused for 7 days, with 3 once-daily injections of an anthracycline, such as daunorubicin, known as the 7+3 regimen. Daunorubicin plus cytarabine liposomal encapsulation for injection was approved in the United States in 2017 for adults with newly diagnosed therapy-related AML (tAML) or AML with myelodysplasia-related changes (AML-MRC).

Objective: To estimate the annual budget impact of introducing daunorubicin-cytarabine liposome as induction treatment for patients with tAML or AML-MRC in the United States over a 3-year period.

Methods: The model consisted of a simple decision analytic framework for a 1- to 3-year period. We used an incidence-based approach to estimate the annual number of patients newly diagnosed with tAML or AML-MRC in a hypothetical 1-million-member plan. Patients were allocated to 2 groups based on when daunorubicin-cytarabine liposome became available, with the base-case group allocated to the 7+3 regimen, and another group allocated to daunorubicin-cytarabine liposome treatment. The incidence of AML was estimated as 4.3 per 100,000 people. Efficacy measures included the proportion of complete responders, proportion of patients who had undergone transplantation, and survival at 180 and 365 days. Inpatient drug and hospitalization costs were based on diagnosis-related group rates, and outpatient drug costs on wholesale acquisition costs.

Results: Based on this hypothetical 1-million-member health plan, 15.1 members would receive intensive induction for newly diagnosed tAML or AML-MRC annually. Increasing the use of daunorubicin-cytarabine liposome (assumption of year 1, 20%; year 2, 50%; year 3, 80%) resulted in a 3-year incremental cumulative budget impact of $72,041 (1.7% increase for patients with tAML or AML-MRC), with a per-member per-month cost of $0.0032 at year 3. Over a 3-year period, the use of daunorubicin-cytarabine liposome would result in an estimated increase in the number of patients with a complete response to therapy by 2.72 (23.1%), which would lead to an incremental cost decrease of $179,956 per responding patient compared with the use of the 7+3 regimen in the base-case group.

Conclusions: Based on these results, induction treatment with daunorubicin-cytarabine liposome for patients with tAML or AML-MRC instead of the 7+3 regimen may have a limited economic impact on the budget of commercial health plans and may result in cost offsets, particularly in patients who respond to therapy.

Background: Computed tomography (CT) colonography's effectiveness, its associated patient advantages, and its potential role to increase colorectal cancer (CRC) screening rates have been demonstrated in previous research, but whether CT colonography has a cost advantage relative to optical colonoscopy for the commercially insured US population has not been assessed.

Objective: To compare the costs of CRC screening using CT colonography or optical colonoscopy for commercially insured people in the United States.

Methods: Using retrospective commercial healthcare claims data and peer-reviewed studies, we performed a simulated multiyear, matched-case comparison of the costs of CT and optical colonoscopies for CRC screening. We estimated commercial optical colonoscopy costs per screening based on the 2016 Truven Health MarketScan Commercial Database and ancillary services, such as bowel preparation, anesthesia, pathology, and complication costs. We developed 4 scenarios for CT colonography cost per screening using the ratio of commercial to Medicare fees, and calculated ancillary service and follow-up costs from payers' costs for these services when associated with optical colonoscopies. For comparison, we converted the costs per screening to the costs per screening year per person using real-world screening intervals that were obtained from peer-reviewed studies.

Results: In 2016, the average optical colonoscopy screening cost for commercial payers was $2033 (N = 406,068), or $340 per screening year per person. With our highest-cost CT colonography scenario, CT colonography costs 22% less, or $265 per screening year, than optical colonoscopy, mostly because of the advantages for patients of no anesthesia and the greatly reduced use of pathology services.

Conclusions: The use of CT colonography for CRC testing offers effective screening, patient-centered advantages, and lower costs compared with optical colonoscopy, and may be particularly appealing to the currently unscreened population with commercial health insurance. If the availability of CT colonography expands to meet the increased demand for it, CT colonography could cost up to 50% less than optical colonoscopy per screening year.