Cost-effectiveness analysis of mosunetuzumab for treatment of relapsed or refractory follicular lymphoma after two or more lines of systemic therapy in the United States.

AIMS: Mosunetuzumab has received accelerated approval by the US Food and Drug Administration for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. We evaluated the cost-effectiveness of mosunetuzumab for the treatment of R/R FL from a US private payer perspective. MATERIALS AND METHODS: A partitioned survival model simulated lifetime costs and outcomes of mosunetuzumab against seven comparators: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), tazemetostat (taz, EZH2 wild-type only), rituximab plus lenalidomide (R-Len) or bendamustine (R-Benda), obinutuzumab plus bendamustine (O-Benda), and a retrospective real-world cohort (RW) based on current patterns of care derived from US electronic health records (Flatiron Health). Efficacy data for mosunetuzumab were from the pivotal Phase II GO29781 trial (NCT02500407). Relative treatment efficacy was estimated from indirect treatment comparisons (ITCs). Costs included were related to treatment, adverse events, routine care, and terminal care. Except for drug costs (March 2023), all costs were inflated to 2022 US dollars. Costs and quality-adjusted life-years (QALYs) were used to calculate incremental cost-effectiveness ratios (ICERs). Net monetary benefit (NMB) was calculated using a willingness-to-pay (WTP) threshold of $150,000/QALY. RESULTS: Mosunetuzumab dominated taz, tisa-cel, and axi-cel with greater QALYs and lower costs. Mosunetuzumab was projected to be cost-effective against R-Benda, O-Benda, and RW with ICERs of $78,607, $42,731, and $21,434, respectively. Mosunetuzumab incurred lower costs but lower QALYs vs. R-Len. NMBs showed that mosunetuzumab was cost-effective against comparators except R-Len. LIMITATIONS: Without head-to-head comparative data, the model had to rely on ITCs, some of which were affected by residual bias. Model inputs were obtained from multiple sources. Extensive sensitivity analyses assessed the importance of these uncertainties. CONCLUSION: Mosunetuzumab is estimated to be cost-effective compared with approved regimens except R-Len for the treatment of adults with R/R FL.

Budget Impact of Introducing Fixed-Duration Mosunetuzumab for the Treatment of Relapsed or Refractory Follicular Lymphoma After Two or More Lines of Systemic Therapy in the USA.

OBJECTIVE: This study aimed to assess the budget impact of introducing fixed-duration mosunetuzumab as a treatment option for adult patients with relapsed or refractory follicular lymphoma after at least two prior systemic therapies and to estimate the total cumulative costs per patient in the USA. METHODS: A 3-year budget impact model was developed for a hypothetical 1-million-member cohort enrolled in a mixed commercial/Medicare health plan. Comparators were: axicabtagene ciloleucel, tisagenlecleucel, tazemetostat, rituximab plus lenalidomide, copanlisib, and older therapies (rituximab or obinutuzumab ± chemotherapy). Costs per patient comprised treatment-associated costs including the drug, its administration, adverse events, and routine care. Dosing and safety data were ascertained from respective package inserts and clinical trial data. Drug costs (March 2023) were estimated based on the average wholesale acquisition cost reported in AnalySource®, and all other costs were based on published sources and inflated to 2022 US dollars. Market shares were obtained from Genentech internal projections and expert opinion. Budget impact outcomes were presented on a per-member per-month basis. RESULTS: Compared with a scenario without mosunetuzumab, its introduction over 3 years resulted in a budget increase of $69,812 (1% increase) and an average per-member per-month budget impact of $0.0019. Among the newer therapies, mosunetuzumab had the second-lowest cumulative per patient cost (mosunetuzumab = $202,039; axicabtagene ciloleucel = $505,845; tisagenlecleucel = $476,293; rituximab plus lenalidomide = $263,520; tazemetostat = $250,665; copanlisib = $127,293) and drug costs, and its introduction only increased total drug costs by 0.1%. By year 3, the cumulative difference in the per patient cost with mosunetuzumab was -$303,805 versus axicabtagene ciloleucel, -$274,254 versus tisagenlecleucel, -$61,481 versus rituximab plus lenalidomide, -$48,625 versus tazemetostat, and $74,747 versus copanlisib. Older therapies were less costly with 3-year cumulative costs that ranged from $36,512 to $147,885. CONCLUSIONS: Over 3 years, the estimated cumulative per patient cost of mosunetuzumab is lower than most available newer therapies, resulting in a small increase in the budget after its formulary adoption for the treatment of relapsed or refractory follicular lymphoma.

Health disparities among Burmese diaspora: an integrative review.

Tens of thousands of displaced Burmese ethnic minorities have endured various adversities for over six decades but are largely underserved. This study aimed to illuminate the health impacts of their misfortunes and unmet areas of concern. Using a holistic lens, we conducted an integrative review of 47 papers spanning the years 2004 to 2022 from diverse data sources. The results revealed widespread multimorbidity, triggered mainly by displacement. The diaspora's problematic health conditions were worse than their host country's general population. There was a strong indication that the diaspora's unfortunate health trajectory is determined early in life. Ongoing human rights violations and grossly inadequate health care interventions deepened pre-existing health conditions. Noteworthy emerging treatment initiatives, including integrative health care, were underutilized. The persisting health and intervention needs among the diaspora warrant advanced studies to facilitate much-needed resource mobilization and collaboration among stakeholders to promote health equity. Funding: There was no financial support for this manuscript.

Health care utilization and expenditures of parents of children with and without hemophilia A.

BACKGROUND: Caring for children with hemophilia A (HA) impacts many aspects of parents' lives. How this translates to caregivers' utilization of health services is unknown, and its elicitation can inform future evaluations of interventions that address caregiver burden for HA. OBJECTIVE: To understand the impact of caring for children with HA on parents' utilization of nonmental and mental health services by comparing 1-year costs and number of claims with parents of children without HA. METHODS: Retrospective matched cohort study using MarketScan commercial medical and pharmacy claims from 2011 to 2019. Children with HA were male sex, aged younger than 18 years, dependent policyholders, and had at least 1 HA-related medical claim from 2011 to 2018 and either an HA-related procedure or drug claim. Parents of children with HA (PCH) were primary or secondary policyholders, shared the same family ID as children with HA, and were continuously enrolled for 1-year post-index. PCH were matched (1:2) with parents of children without HA on age, sex, beneficiary type, child's age, number of children, index month and year, health plan type, employment status, and region. Primary outcomes were nonmental and mental health care costs (2020 US dollars). Secondary outcomes were number of nonmental health outpatient claims and utilization of mental health outpatient or drug claim. Subgroup analyses excluding parents with HA were also conducted. Productivity loss was also explored. Outcomes were compared using 2-sided, paired t-tests, and McNemar test. RESULTS: 1,068 PCH met inclusion criteria and were matched to 2,122 control parents. Mean 1-year cost for PCH was higher for nonmental health (mean difference $1,826 [95% CI = -1,000 to 4,652; P = 0.20]) and similar for mental health services (mean difference $14 [95% CI = -77 to 105; P = 0.76]). When parents with HA were excluded in the subgroup analyses, mental health cost was significantly higher for PCH (mean difference $676 [95% CI = 399 to 953; P < 0.001]). PCH had more nonmental health outpatient claims compared with parents of children without HA (mean difference 1.9 [95% CI = -1.1 to 4.9; P = 0.21]) and were 1.2 times (95% CI = 0.99 to 1.45; P = 0.07) more likely to have a mental health outpatient or drug claim. CONCLUSIONS: PCH had moderately higher health care costs and utilization compared with parents of children without HA; however, these results were not statistically significant. Future studies to better characterize HA disease severity and assess its impact on caregiver burden or to expand caregivers to spouses of adult patients with HA may be warranted. Limitations include inability to ascertain severity of HA in children and the use of claims data to capture complex effects on health care utilization. DISCLOSURES: Dr. Kim's postdoctoral fellowship is supported by Genentech, Inc. Dr. Raimundo is an employee of Genentech, Inc.

Alternative Approaches to Quality-Adjusted Life-Year Estimation Within Standard Cost-Effectiveness Models: Literature Review, Feasibility Assessment, and Impact Evaluation.

OBJECTIVES: The quality-adjusted life-year (QALY) has been long debated, but alternative estimation approaches have not been comprehensively evaluated. Our objective was to identify alternatives, characterize them by implementation feasibility, and evaluate the impact of implementing feasible options in cost-effectiveness models developed for the Institute for Clinical and Economic Review reports. METHODS: We conducted a literature review combining keywords relating to QALYs, methodology alternatives, and cost-effectiveness in PubMed, EconLit, Web of Science, and MEDLINE. Articles that discussed alternatives to the conventional QALY were included. Alternatives were characterized by type, data availability, calculation burden, and overall implementation feasibility. The subset of feasible alternatives, that is, sufficient data and methodology compatible with incorporation into common modeling approaches, were evaluated according to impact on incremental QALYs, incremental net monetary benefit (iNMB), intervention rankings, and proportion of interventions with a positive iNMB. RESULTS: We identified 28 articles discussing 9 alternatives. Feasible alternatives were using patient preference (PP) data; equity weighting according to baseline utility, fair innings, or proportional QALY shortfall; and the equal value of life-years-gained approach. All alternatives affected the incremental QALY and iNMB outcomes, rankings, and proportion of interventions with a positive iNMB. The PP alternative had the largest and most consistent impact. The PP impact on the proportion of interventions with a positive iNMB, was in the negative direction. CONCLUSIONS: Our work is the first comprehensive evaluation of proposed alternatives to the conventional QALY. We found robust literature but few options that were feasible to be implemented in current healthcare decision-making processes.

The Supercar Stays in the Garage: Factors Preventing Indirect Comparisons of Novel Medicines Targeting the Same Condition.

DISCLOSURES: No outside funding supported the writing of this letter. Ollendorf reports advisory board and consulting fees from DBV Technologies, EMD Serano, Gerson Lehman Group, and Sarepta Therapeutics, unrelated to the content of this research letter. Naci reports a grant from the Commonwealth Fund, unrelated to the content of this letter. The other authors have nothing to disclose.

Continents divided: Understanding differences between Europe and North America in acceptance of GM crops.

The differences between GM policies in the US and Europe have several causes. GM technology holds a home court advantage in the US and European chemical companies did not support its introduction. The technology did not seem to provide benefits to consumers, and the crops it applied to were not so significant in Europe. The technology was introduced during a time when the political influence of green parties in Europe was especially significant, and European trust of government capacity to enter food security issues was at its lowest.