Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy currently approved for relapsed/refractory multiple myeloma (RRMM). This study aimed to assess the value of cilta-cel against a real-world standard of care (RW-SoC) basket using a novel cost per responder (CPR) model.

Research design and methods: The model was aligned with the patient population in the CARTITUDE-4 trial and incorporated progression-free survival (PFS), post-progression survival (PPS), and death states. The base-case analysis was conducted from a mixed US payer perspective with a 36-month time horizon, and modeled outcomes included the total cost per treated patient, total cost per complete responder, and cost per month during PFS.

Results: In the base-case analysis, total cost per treated patient over 36 months was estimated to be lower for cilta-cel ($792,243) compared with RW-SoC ($815,023), with the difference driven predominantly by the lower costs for cilta-cel over the PPS period. Total costs per complete responder and per month during PFS were $1,070,599 and $25,203 for cilta-cel compared with $5,101,186 and $38,018, respectively, for RW-SoC.

Conclusions: The CPR model suggests that cilta-cel offers substantial clinical and economic benefit for patients with RRMM compared with RW-SoC therapies.

Background: This study evaluates the economic impact of incorporating a fully liquid combined acellular hexavalent vaccine within Expanded Program on Immunizations (EPI) of the Dominican Republic (DR).

Research design and methods: A cost-consequence analysis model was conducted comparing two different vaccination schemes in 1 year. Inputs and costs were extracted from both official and literature sources. Two vaccination schedules were assessed: 3 + 0 and 3 + 1. Additionally, a 5-year budget impact analysis evaluated the implications of introducing the hexavalent vaccine into the EPI. The analysis was performed from both a third-party payer and considering productivity losses. (1 USD = 55.67, 2023).

Results: The hexavalent vaccine resulted in an increase in total costs of 43.1% and 57.9% for the 3 + 0 and 3 + 1 scenarios. Vaccine acquisition costs drove this increase. However, adverse events, logistics cost and productivity losses were reduced by 51.87%-74.87%, 38.20%-49.23%, and 28.5%-44.9% (3 + 0-3 + 1) respectively. The budget impact analysis showed an overall budget increase of 61.7% and 80.2% with a 37.5% and 50% reduction in total vaccine doses administered, for the 3 + 0 and 3 + 1 scenarios.

Conclusion: The incorporation of the hexavalent vaccine increases spending but reduces adverse events, logistics cost, and productivity losses, supporting modernization of the DR immunization program.

Introduction: The integration of digital endpoints into Health Technology Assessment (HTA) marks a significant advancement in modern healthcare evaluation, especially in the context of post-pandemic growth in telehealth and remote patient monitoring.

Areas covered: Digital endpoints, which are defined by their use of sensor-generated data collected in non-clinical settings, provide a comprehensive, real-time view of patient health. This enhances the precision of HTA by uncovering nuanced aspects of disease burden and improving the evaluation of health technologies. Examples of digital endpoints include smartphone-based diagnostics for cognitive impairment and wearable devices that measure the impact of diseases, such as nocturnal activity in patients with sickle cell disease. Earlier diagnoses, and cost reductions in drug discovery, their integration into HTA faces challenges- data privacy, standardization, and methodological validation.

Expert opinion: This paper explores the potential of digital endpoints to revolutionize HTA by enabling more dynamic and patient-centered evaluations, underscoring the need for established frameworks and standards to guide their effective incorporation. Initiatives such as the Digi-HTA process and the Digital Endpoints Ecosystem and Protocols (DEEP) highlight emerging frameworks that could shape the future of digital health assessments, ultimately enhancing healthcare decision-making and policy. Collaborative efforts across healthcare, technology, and regulatory bodies are essential to overcome these barriers.

Background: The psychological aftermath of the COVID-19 pandemic and ongoing regional stressors have influenced mental health needs in Jordan. Understanding antidepressant utilization and expenditure trends is essential for improving treatment access and resource planning.

Research design and methods: A retrospective market analysis was conducted using national IQVIA sales data (2019-2023), standardized by the World Health Organization's Defined Daily Dose methodology. Prescription-level data from Advanced Marketing Statistics (AMS) for 2021-2022 were analyzed to assess prescribing patterns by age, gender, and clinical indication. Descriptive and correlation analyses were performed.

Results: Antidepressant utilization in Jordan's private sector increased steadily between 2019 and 2023, dominated by selective serotonin reuptake inhibitors (SSRIs), which comprised 79% of total use. Escitalopram was most prescribed (40%), followed by sertraline (18.9%) and citalopram (3.3%). Prescriptions were more frequent among males (55.6%) and adults aged 30-54 years. Major depressive disorder accounted for 46.7% of indications. Fluoxetine use was higher among females, while tricyclic antidepressants were more common in males.

Conclusions: SSRIs, particularly escitalopram, dominate antidepressant prescribing in Jordan's private sector. Patterns vary by age and gender, reflecting clinical diversity. Findings highlight the growing economic burden of antidepressants and the need for cost-containment and targeted mental health strategies.

Background: Multiple myeloma (MM) is a hematologic cancer with rising incidence worldwide. Autologous stem cell transplantation (ASCT) is a key treatment for eligible patients, but mobilization failure remains a major obstacle. Plerixafor enhances stem cell mobilization, but its high costs and lack of standardized protocol prevent its widespread use.

Objective: To evaluate the cost-utility of preemptive versus rescue use of plerixafor for hematopoietic stem cell mobilization inMM patients.

Methods: A Markov model was developed using real-world data from 196 MM patients undergoing stem cell mobilization at two SãoPaulo transplant centers in Brazil. The model compared two strategies: (1) preemptive plerixafor and (2) rescue plerixafor use following mobilization failure. Transition probabilities, utilities, and costs were informed by clinical data and literature. Deterministic and probabilistic sensitivity analyses were performed.

Results: The preemptive strategy led to higher rates of successful mobilization and ASCT, resulting in greater quality-adjusted life years (QALYs), but also higher costs. Still, it demonstrated favorable results compared to the rescue approach and passed the Brazilian willingness to pay thresholds of acceptability.

Conclusion: Preemptive plerixafor passes the cost-utility guidelines to be used in MM patients in Brazil, potentially guiding policy decisions on resource allocation within the national health system.

Background: Lebrikizumab and tralokinumab significantly treat moderate-to-severe atopic dermatitis. Nevertheless, pharmacoeconomic analyses and head-to-head clinical studies for both are lacking. This study aimed to compare the cost per responder of lebrikizumab and tralokinumab in these patients.

Research design and methods: To indirectly compare the efficacy of lebrikizumab and tralokinumab for in the treatment of moderate-to-severe atopic dermatitis based on published clinical trial data. The number needed to treat and the cost per responder in comparison to a placebo were used to compare the cost-effectiveness of the two therapies. The cost per responder was calculated based on United States drug acquisition costs by multiplying the cost of treatment by the number needed to treat for each therapy.

Results: At Week 16, the cost per responder for lebrikizumab and tralokinumab was $67,932 versus $130,655 for EASI-75 and $109,412 versus $176,622 for IGA 0/1.For key secondary endpoints, the cost per responder was consistently lower for lebrikizumab compared to tralokinumab, including 16-week ∆NRS ≥4 ($95,282vs$156,262), and 4-week IGA 0/1 ($68,955vs$151,874).

Conclusions: In comparison to tralokinumab, lebrikizumab had a much lower number needed to treat and cost per responder. These results, based on US pricing, indicate that lebrikizumab appears to be more cost-effective treatment option based on indirect comparisons.

Background: This study evaluates the cost-effectiveness of larotrectinib compared to the standard of care for treating metastatic NTRK fusion colorectal cancer (CRC) from the perspective of healthcare payers in China.

Research design and methods: An economic evaluation utilizing a 3-state partitioned survival model assessed the cost-effectiveness of larotrectinib therapy vs regorafenib therapy or larotrectinib therapy vs trifluridine/tipiracil therapy.

Results: When the time horizon was 10 years, the anticipated expenditure for larotrectinib therapy exceeded the cost for regorafenib therapy or trifluridine/tipiracil therapy (21,588.50 USD vs 1579.09 USD; 21,588.50 USD vs 2411.16 USD). The estimated utility of larotrectinib therapy was also greater compared to that of regorafenib therapy or trifluridine/tipiracil therapy (1.14 QALYs vs 0.28 QALYs; 1.14 QALYs vs 0.29 QALYs). The ICER of larotrectinib therapy vs regorafenib therapy or larotrectinib therapy vs trifluridine/tipiracil therapy was calculated at 23,321.46 USD/QALY or 22,585.39 USD/QALY.

Conclusions: From the perspective of healthcare payers in China, larotrectinib was cost-effective compared to standard of care as a second-line treatment or subsequent treatment for advanced or metastatic CRC patients with NTRK gene fusion-positive.

Background: The review of healthcare costs in multiple sclerosis (MS) has been of increased interest. In this study, we aimed to estimate the economic burden of MS patients, treated with dimethyl fumarate (DMF) in Greece.

Research design and methods: Four hundred and fifty-six participants were followed every 6 months after DMF initiation. Healthcare resource utilization, out-of-pocket expenses, and productivity loss data were recorded. Treatment cost and effectiveness of DMF were compared with those of previous treatments.

Results: From a societal perspective, the total mean biennial burden was 13,113 € per patient, mainly attributed to direct healthcare costs (10,818 €, with medication accounting for almost 96%). From the payer perspective, the cost per patient was estimated at 10,462 €. The main driver of indirect cost was early retirement (87.8%). The treatment cost in the 2 years before the study was ~25% higher than the biennial DMF cost. A total of 43.9% of the patients experienced adverse events, mostly mild/moderate.

Conclusions: DMF displayed a societal cost mainly driven by medication, disease severity, disease duration and patients' age. There was evidence of beneficial effects on disease activity and quality of life with no new safety signals emerging.

Trial registration: The trial is registered at ClinicalTrials.gov (ID: NCT03101735).

Background: Functional genomics approaches, such as transcriptomics and proteomics, can provide valuable insights into rare diseases when genomic sequencing fails to yield informative findings. This study estimated the monetary value that parents, carers and individuals with undiagnosed rare diseases place on functional genomics testing.

Research design and methods: A triple-bounded dichotomous choice contingent valuation survey was completed by carers and individuals with suspected rare monogenic disorders recruited as part of the Australian Undiagnosed Disease Network. A multilevel interval regression model was used to analyze response data and estimate the monetary value of functional genomics, in terms of willingness to pay (WTP).

Results: There was a total of 57 respondents (48%), primarily carers (95%). The mean WTP for functional genomics testing was estimated to be $2,522 (95% CI: $817-$4,228) [US $1,568 (95% CI: $508-$2,629)].

Conclusions: Our findings indicate that individuals with undiagnosed rare diseases and their parents or caregivers place high value on functional genomics testing. The estimated WTP is comparable to findings from contingent valuation studies of other genomic interventions and exceeds the expected economic cost of proteomics testing. These insights can inform a preference-based evaluation of the diagnostic outcomes and net benefits achieved through functional genomics, thereby guiding decision-making and clinical implementation.

Background: Patient-reported outcome (PRO) data capture patients' perspectives on their disease and its treatments; however, use of such data in health technology assessments (HTA) and pricing/reimbursement processes is limited. We provide considerations to support the incorporation of PROs in payer decision-making.

Research design and methods: A review of guidance documents released by payers until 2024 regarding requirements for using PRO evidence and scientific literature published during 2015-2024 on the trends of PRO use by payers informed the development of a discussion guide. Using this, 15 experts from seven countries were interviewed (one-on-one) to seek their opinions on PRO use in HTAs and identify potential barriers to its adoption.

Results: PRO-related guidance focused on the validity and reliability of PRO instruments, risk for bias, missing data, and economic modeling. Guidance varied between countries with essential details often missing. Expert interviews revealed that PRO use in payer evaluations may depend on established endpoints for indications and varied decision approaches and frameworks. Barriers to PRO use include the lack of capacity or technical expertise, instrument validity concerns, and data accuracy.

Conclusion: Barriers to the use of PRO data for evaluations require further efforts from all relevant stakeholders to promote PRO incorporation in payer decision-making.