Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: Respiratory syncytial virus (RSV) vaccination could improve health equity by protecting individuals who are disproportionally at increased risk of RSV infection and severe RSV-related outcomes. However, limited information is available about RSV-related disparities among United States (US) adults.

Areas covered: We reviewed US-specific literature regarding disparities across adult populations in having risk factors for severe RSV disease (cardiopulmonary disease, diabetes, liver disease, kidney disease). We summarize available evidence regarding disparities in having or being diagnosed with RSV, as well as experiencing severe RSV-related health outcomes. Disparities are analyzed by race, ethnicity, socioeconomic status, and other social determinants of health.

Expert opinion: RSV-related disparities are observed across all outcomes of interest, although RSV-specific data are limited in some cases. Racial and ethnic minority groups and socioeconomically disadvantaged populations are more likely to have risk factors for severe RSV disease, overall and at younger ages, yet individuals from these groups are more often underdiagnosed. Disparities in RSV-related hospitalizations, emergency department visits, and deaths are observed, especially among adults from racial and ethnic minority groups, of lower socioeconomic status, and in poorer or more crowded neighborhoods. Findings highlight the importance of RSV vaccination among these groups to improve health equity.

Background: Pharmaceutical expenditures (PE) are increasing worldwide, raising concerns about sustainability. However, the current price index provides an incomplete picture of this trend due to the rapid introduction of new drugs on the market.

Objective: The aim of this study is to decompose PE into their components and investigate the development in Sweden from 1990 to 2022.

Methods: The PE index was broken down into separate indices for price, quantity, and a residual. The residual reflects changes in expenditure driven by shifts in drug treatment patterns.

Results: PE increased by 227% during the study period. The decomposition showed that this increase was mainly driven by the residual (215%). Drug quantity increased by 105%, while the relative prices decreased by 50%. When dividing the whole study period into three 11-year-subperiods, the increase in real drug expenditure, drug quantity, and the residual was the highest from 1990 to 2000.

Conclusions: The finding that the residual is the main driver indicates that the increase in PE is due to the introduction of and shift to more expensive pharmaceutical treatments, while existing treatments tend to become cheaper. Further research is needed to determine whether newer, more expensive drugs are indeed worth the extra cost.

Background: India faces over 220 000 lakh new kidney failure cases annually, requiring approximately 34 million dialysis sessions, creating a significant economic burden on the healthcare system. This study estimates the costs of providing hemodialysis (HD) and Continuous Ambulatory Peritoneal Dialysis (CAPD) under a Public-Private Partnership (PPP) in a tertiary hospital.

Methods: Economic health system costs (October 2021-2022) were estimated using a bottom-up approach. Resources consumed were identified, measured, and valued. Capital costs were annualized and discounted at 3%. Sensitivity analysis assessed the impact of variations in input costs.

Results: The total annual economic cost for HD (n = 103) under PPP was INR 32 611 618 (USD 393 432), and for CAPD (n = 12) was INR 4 103 781 (USD 49 509). The annual cost per beneficiary for HD and CAPD was INR 316 618 (USD 3820) and INR 341 979 (USD 4126), respectively. Unit cost per HD session was INR 1856 (USD 22) and per CAPD exchange was INR 323 (USD 4).

Conclusion: This study provides detailed costs of HD and CAPD services under PPP, offering insights for expanding dialysis services under the Pradhan Mantri National Dialysis Programme and supporting cost-effectiveness analysis for resource allocation.

Introduction: This systematic review aims to explore the existing evidence on the cost-effectiveness of brexu-cel across different international jurisdictions.

Methods: A systematic search of articles on Embase, Medline, Econlit, Web of Science, Scopus, gray literature, and a manual search of HTA reports was done until 24 June 2024. Original English articles and reports from different countries assessing the cost-effectiveness of brexu-cel in relapsed/refractory acute lymphoblastic leukemia (R/R ALL) and mantle cell lymphoma (R/R MCL) were included. This review was registered in the Open Science Framework (OSF) registry.

Results: Of the 149 records, 22 articles underwent full-text review after the title and abstract screening, five met the inclusion criteria along with seven HTA reports from Australia, Canada, Scotland, and England. The CEA studies were from the US, England, Canada, and Italy, with varying perspectives, mainly adopting a partitioned survival model and lifetime horizons. The model input data from the ZUMA-2 and ZUMA-3 trials were used for brexu-cel, with comparisons from their respective trials or literature.

Conclusion: Brexu-cel was found cost-effective in all the CEA studies and an HTA report from Scotland, but the other HTA agencies reported uncertainties around the cost-effectiveness of brexu-cel for R/R ALL and R/R MCL.

Registration: Open Science Framework. (Reg doi: https://doi.org/10.17605/OSF.IO/JZU6Y).

Introduction: Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts.

Areas covered: This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research.

Expert opinion: We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials.

Introduction: The new European Medical Device Regulation has raised the bar for the clinical evaluation of medical devices to gain marketing authorization by Notified Bodies (NBs) regarding certificates of conformity in Europe. Restrictions applied for High-risk medical devices (HRMD) may require further evidence generation. Some other jurisdictions apply similar schemes that may be useful to the European Union. This systematic review focused on extracting lessons from similar schemes worldwide to the European context.

Methods: A systematic review of peer-reviewed and gray literature was performed based on 'Device approval' and 'conditional approval' keywords. Databases such as Medline, Embase, and WoS retrieved documents assessed with the AMSTAR-2 checklist. A descriptive and narrative analysis was conducted detailed in CRD42023431233 - PROSPERO.

Results: We obtained eight documents where conditional approvals for High-risk medical devices in the United States of America, China, and Canada were subject to generate further evidence. In Europe, NBs impose restrictions or limitations to certificates of conformity instead.

Conclusion: Further development of policies, supporting access to HRMD subject to further evidence generation, would help Europe in further defining the appropriate situations for the application of determined regulatory routes, to enhance access to HRMD with promising evidence and further evidence development.

Registration: PROSPERO (CRD42023431233).

Background: The DAPA-CKD study showed that dapagliflozin added to standard treatment reduced the risk of chronic kidney disease progression, and death from renal or cardiovascular causes compared to placebo.

Objective: Assess the cost-effectiveness of dapagliflozin and standard treatment versus standard treatment alone for chronic kidney disease within the Colombian health system.

Methods: We employed a Markov model based on the DAPA-CKD study, tailored to the Colombian scenario. The model forecasted hospitalizations for heart failure, overall and cardiovascular mortality, and chronic kidney disease progression over a 10-year horizon with a 5% discount rate.

Results: Dapagliflozin combined with standard treatment is a cost-effective intervention in treating stage 2-4 CKD. In the base case, the ICER was US $5,366, below 1 GDP (US $6.558) per capita. This was consistent in the sensitivity analyses.

Conclusion: Our study showed that dapagliflozin, when combined with standard treatment, is cost-effective against standard treatment alone, aligning with Colombia's willingness-to-pay threshold.

Introduction: Next-generation sequencing (NGS) identifies genetic variants to inform personalized treatment plans. Insufficient evidence of cost-effectiveness impedes the integration of NGS into routine cancer care. The complexity of personalized treatment challenges conventional economic evaluation. Clearly delineating challenges informs future cost-effectiveness analyses to better value and contextualize health, preference-, and equity-based outcomes.

Areas covered: We conducted a scoping review to characterize the applied methods and outcomes of economic evaluations of NGS in oncology and identify existing challenges. We included 27 articles published since 2016 from a search of PubMed, Embase, and Web of Science. Identified challenges included defining the evaluative scope, managing evidentiary limitations including lack of causal evidence, incorporating preference-based utility, and assessing distributional and equity-based impacts. These challenges reflect the difficulty of generating high-quality clinical effectiveness and real-world evidence (RWE) for NGS-guided interventions.

Expert opinion: Adapting methodological approaches and developing life-cycle health technology assessment (HTA) guidance using RWE is crucial for implementing NGS in oncology. Healthcare systems, decision-makers, and HTA organizations are facing a pivotal opportunity to adapt to an evolving clinical paradigm and create innovative regulatory and reimbursement processes that will enable more sustainable, equitable, and patient-oriented healthcare.