A discrete choice experiment on oral and injection treatment preferences among moderate-to-severe psoriasis patients in Japan.

Long-term psoriasis (PsO) management remains challenging. With growing variation in treatment efficacy, cost, and modes of administration, patient preferences for different treatment characteristics are not well understood. A discrete choice experiment (DCE), informed by qualitative patient interviews, was conducted to assess patient preferences for different attributes of PsO treatments; 222 adult patients with moderate-to-severe PsO receiving systemic therapy participated in the DCE web survey. Better long-term efficacy and lower cost were preferred (preference weights p < 0.05). Long-term efficacy had the highest relative importance (RI) and mode of administration was as important as the outcome attributes (efficacy and safety). Patients also preferred oral to injectable administration. In subgroup analyses by disease severity, residence, psoriatic arthritis as a comorbidity, and gender, the trends for each subgroup were the same as the overall population although the extent of RI for administration mode varied. Mode of administration was more important for patients with moderate versus severe disease, or rural versus urban residence. This DCE utilized attributes related to both oral and injectable treatment as well as a broad study population of systemic treatment users. Preferences were further stratified by patient characteristics to explore trends in different subgroups. Understanding the RI of treatment attributes and the attribute trade-offs acceptable to patients helps inform moderate-to-severe PsO systemic treatments decisions.

Treatment patterns, healthcare resource utilization, and costs in patients with moderate-to-severe psoriasis treated with systemic therapy in Japan: A retrospective claims database study.

The real-world treatment landscape for patients with moderate-to-severe psoriasis receiving systemic therapies in Japan is not well understood. This study describes the demographic and clinical characteristics, treatment patterns, healthcare resource utilization, and psoriasis-associated costs in these patients. This retrospective observational study used data from the Japan Medical Data Center database between January 2016 and December 2020. Eligible patients had a confirmed diagnosis of psoriasis, ≥1 claim for a systemic treatment of interest, medical history for ≥6 months, and follow-up data for ≥12 months. Systemic therapies comprised biologics (tumor necrosis factor and interleukin inhibitors) and oral treatments (a phosphodiesterase-4 inhibitor, immunosuppressants, and vitamin A). Patient demographics and clinical characteristics, treatment patterns, healthcare resource utilization, and costs were evaluated. The study identified 1770 patients satisfying all inclusion criteria. The mean age was 49.0 years, with 68% of patients aged 20-54 years. Overall, 90.6% and 9.4% of patients received oral medications and biologics as index treatment, respectively. Treatment patterns, healthcare resource utilization, and costs were assessed for treatments received by ≥20 patients (n = 1730). During the 12-month follow-up period, 1102/1730 patients (63.7%) discontinued index treatment, of whom 9.9% switched to alternative systemic treatments. The persistence rate was ≥70% for most biologics and <50% for oral systemic treatments. All 1730 patients had ≥1 all-cause outpatient visit (2.0 visits per person per month) and hospitalization frequency was ≤0.01 per person per month. Persistent patients incurred inflation-adjusted costs of Japanese Yen (JPY) 88 667 per person per month. Treatment switching was associated with an increase in total cost: JPY 128 039 per person per month after switching versus JPY 117 504 before switching. This study of Japanese patients with moderate-to-severe psoriasis demonstrated low persistence, high discontinuation, and low rates of treatment switching with systemic therapies. Switching was associated with increased total cost. These results indicate unmet needs for new treatments.

Economic evaluation of nivolumab combined with ipilimumab in the first-line treatment of advanced melanoma in Japan.

AIMS: The objective of this study was to evaluate the cost-effectiveness of nivolumab in combination with ipilimumab (nivo + ipi) compared to current therapeutic alternatives in first-line treatment of patients with advanced melanoma from the Japanese national healthcare payer perspective using 48-month survival data from the CheckMate 067 Phase III trial. MATERIALS AND METHODS: A three-state partitioned survival model was developed from projections of overall survival (OS) and progression-free survival (PFS) to estimate accrued quality-adjusted survival and costs over a 30-year time horizon. The analysis included nivo + ipi, nivolumab, and ipilimumab monotherapies (the three treatments included in CheckMate 067). Drug acquisition, administration, disease management, subsequent therapy, and adverse event (AE) costs were obtained via published sources and expert input (solicited via Delphi panel). AE frequencies were collected from the Checkmate 067 trial. Utility weights were estimated from the Checkmate 067 trial, based on Japanese tariffs. Results were presented as incremental cost-utility ratios (ICURs, cost per quality-adjusted life-year (QALY)). RESULTS: Nivo + ipi had the greatest estimated survival among the three competing treatments, followed by nivolumab monotherapy accruing the second greatest survival. The incremental cost-effectiveness of nivo + ipi was ¥778,000 per QALY vs. nivolumab and ¥1,584,000 per QALY vs. ipilimumab. The results indicate that nivo + ipi is cost-effective in Japan when compared to a threshold of ¥7,500,000 per QALY. This finding was found to be generally robust to sensitivity and scenario analyses. LIMITATIONS: Limitations include uncertainty in long-term survival extrapolations and lack of Japan-specific clinical data. CONCLUSIONS: This analysis indicates that adding ipilimumab to nivolumab therapy represents a cost-effective new treatment option for patients with unresectable malignant melanoma in Japan.

Patient Preferences for Anti-Vascular Endothelial Growth Factor Treatment for Wet Age-Related Macular Degeneration in Japan: A Discrete Choice Experiment.

BACKGROUND: In Japan, intravitreal anti-vascular endothelial growth factor (anti-VEGF) dosing regimens for wet age-related macular degeneration (wAMD) include pro re nata, every 2 months, and treat-and-extend, resulting in different outcomes and patient burden. Although reflecting patient preferences in treatment decision-making is desirable, few studies have examined this in Japan. This study assessed the patients willingness to trade-off between different dosing regimens. PATIENTS AND METHODS: Patients with wAMD were recruited from four Japanese university hospitals to complete a face-to-face cross-sectional survey. In a discrete choice experiment, patients were asked to choose their preferred option from two anti-VEGF treatment profiles shown side-by-side across a series of choice tasks. The profiles varied on four attributes: number of injections in 12 months, number of physician consultations in 12 months, chance of 1-year visual acuity (VA) improvement, and chance of 2-year VA maintenance. Preference weights were estimated using hierarchical Bayes' models. RESULTS: Overall, 120 patients (30 treatment naïve and 90 anti-VEGF experienced) completed the survey. Patients were willing to accept an increase from three to approximately eight injections in 12 months to increase the chance of 1-year VA improvement from 25% to 40%. They would be willing to accept 11 injections in 12 months if the chance of 2-year VA maintenance increased from 80% to 96%. The most valued attributes were increasing the chance of 2-year VA maintenance and reducing the number of injections in 12 months, which were each about twice as important as decreasing physician consultations in 12 months and increasing the chance of 1-year VA improvement (p<0.001). Among the dosing regimens, patients most preferred treat-and-extend because of its higher chance of 2-year VA maintenance. CONCLUSION: Informing patients with wAMD about the likelihood of long-term VA maintenance when selecting treatment may increase the acceptance of an optimal treatment regimen and number of injections.

Vision-related quality of life in Japanese patients with wet age-related macular degeneration treated with intravitreal aflibercept in a real-world setting.

PURPOSE: To evaluate vision-related quality of life (QoL) in wet age-related macular degeneration (wAMD) patients receiving intravitreal aflibercept (IVT-AFL). STUDY DESIGN: Prospective, observational Japanese postmarketing surveillance study. METHODS: All decisions were made by the treating physician. QoL was assessed using the 25-item National Eye Institute-Visual Functioning Questionnaire (NEI-VFQ-25) composite score administered at baseline, 6 months, and 12 months (primary assessment). Secondary assessments included NEI-VFQ-25 subscale scores, resource use, and best-corrected visual acuity (BCVA; logarithm of the minimum angle of resolution [logMAR]). RESULTS: In total, 576 patients (baseline), 555 patients (6 months), and 446 patients (12 months) were included. The mean (SD) number of IVT-AFL injections was 3.5 (1.2) at 6 months and 4.6 (2.2) at 12 months. The mean (SD) improvement from baseline in the NEI-VFQ-25 composite score was 3.1 (11.1) at 6 months and 2.7 (12.3) at 12 months (P < .0001). For the NEI-VFQ-25 subscale scores, the mean change was ≥ 4 (minimally important difference) for general vision, near vision, and mental health at 6 months, and for general vision and mental health at 12 months (all P < .0001). A significant improvement from baseline was found in mean BCVA (logMAR) at 6 months (-0.1) and 12 months (-0.1) (P < .0001). The mean change from baseline in the NEI-VFQ-25 scores was greatest in patients with improved BCVA (gain of ≤ -0.3 logMAR units or ≥ 15 letters) after treatment. CONCLUSION: IVT-AFL was associated with significant improvements in QoL and visual acuity in Japanese patients with wAMD in a real-world setting.

The impact of anti-vascular endothelial growth factor agents on visual impairment/blindness prevention in patients with diabetic macular edema and on associated patient and caregiver burden in Japan.

AIMS: To estimate the impact of anti-vascular endothelial growth factor (VEGF) agents on visual impairment and blindness avoided in patients with diabetic macular edema (DME) and on associated patient and caregiver productivity loss in Japan. METHODS: This study compared the impact of current care (estimated at 53.8% utilization of anti-VEGF agents using current data) with that of hypothetical care (characterized by a higher utilization of anti-VEGF agents [80.0%], as estimated by an expert panel) of DME patients. A population-based Markov model (two-eye approach) simulated visual acuity (Early Treatment Diabetic Retinopathy Study [ETDRS] letters) transitions over 5 years with DME treatments (intravitreal aflibercept, ranibizumab, and triamcinolone acetonide, and grid/focal laser) in patients with DME. Patient and caregiver productivity loss was determined using the human capital method. RESULTS: In total, 570,000 DME patients were included in the model over 5 years. Increased utilization of anti-VEGF agents resulted in 6,659 fewer cases of severe visual impairment (SVI; 26-35 ETDRS letters) or blindness (0-25 ETDRS letters) compared with the current care approach (26,023 vs 32,682 cases; 20.38% reduction) over this period. Increased utilization of anti-VEGF agents also contributed to productivity loss savings of ¥12.58 billion (US $115.64 million) (i.e., 17.01%) at the end of year 5. The total overall saving over 5 years was ¥45.83 billion (US $421.27 million) (13.45%). LIMITATIONS: Few Japanese data were available, and assumptions were made for some inputs. Vision changes dependent on the function of both eyes were not studied. Only intravitreal (not sub-Tenon's) injections of triamcinolone were considered in this model. Direct costs were not considered. CONCLUSIONS: Increased utilization of anti-VEGF agents can reduce SVI and legal blindness in patients with DME in Japan. This would also be associated with substantial savings in patient and caregiver productivity loss.

The impact of different anti-vascular endothelial growth factor treatment regimens on reducing burden for caregivers and patients with wet age-related macular degeneration in a single-center real-world Japanese setting.

OBJECTIVE: To describe the burden associated with different anti-vascular endothelial growth factor (VEGF) treatment strategies for wet age-related macular degeneration (wAMD) in a real-word setting in Japan. METHODS: Single-center, cross-sectional survey of caregivers of patients with wAMD performed in a hospital in Mito-City, a rural area in Japan. Caregiver burden was evaluated using the Burden Index of Caregivers (BIC-11), and depressive symptoms were assessed by the Center for Epidemiologic Studies Depression scale. Retrospective medical chart review was conducted to monitor resource use and visual acuity outcomes in patients. The productivity loss of caregivers accompanying patients on hospital visits was estimated using the human capital method. RESULTS: Seventy-one patient-caregiver pairs were included. Most caregivers were female (74.6%), spouse/partner (54.9%), employed (46.5%), and the primary caregiver (85.9%). Patients received anti-VEGF treatment as follows: treat-and-extend (T&E; n = 42), switch (from as-needed [PRN] to T&E; n = 18), PRN (n = 10), and other (n = 1). Caregiver-related burden (total BIC-11 scores) were 4.29 (T&E) 4.60 (PRN), and 5.33 (switch) (p = NS). The mean number of hospital visits was lower with T&E than PRN (7.88 vs. 14.0 [p = 0.00674] in year 1 and 5.68 vs. 9.0 in year 2). For patients who switched from PRN to T&E, the mean number of hospital visits decreased from 13.21 to 7.43 (p<0.0001) in the first year after switch. The productivity loss associated with accompanying patients to the hospital was lower for caregivers of patients receiving T&E than PRN (mean differences: 74,456.04 JPY [p = 0.00284] in year 1 and 40843.14 JPY in year 2), and was also reduced for caregivers of patients who switched from PRN to T&E. CONCLUSION: wAMD treatment with anti-VEGF agents via T&E reduced hospital visits compared with PRN, where associated monitoring visits are necessary to provide good patient outcomes. T&E was associated with a reduction trend in caregiver burden, including time and costs.