Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: Lung cancer (LC) is a leading cause of cancer mortality in Argentina. Low-dose computed tomography (LDCT) had demonstrated higher efficacy than chest radiography as a screening method for early detection and reducing LC mortality. This study estimates the Budget Impact of implementing annual LDCT screening for individuals aged 55-74 with at least 30 pack-years of smoking in Argentina.

Research design and methods: A budgetary impact model for a dynamic cohort was developed from the Argentine health system's perspective, accounting for LC incidents, prevalents and death probability, distinguishing by disease stage and survival years. Model parameters were sourced from literature and official databases, with costs expressed in 2023 USD.

Results: In Argentina, 726,420 individuals were eligible for annual LC screening. The average annual budget was USD 196,611,452 in the current scenario and USD 207,439,560 in the projected scenario, leading to a 5.20% incremental cost due to more false positives in LDCT and screening costs. The budget impact per member per month was USD 0.019, slightly above the estimated threshold of USD 0.015.

Conclusions: The introduction of LDCT for LC screening increased the health system budget in Argentina but improved early disease detection.

Introduction: Unprofessional behaviors (UB) such as bullying and incivility among healthcare staff are a significant and pervasive issue, impacting staff wellbeing, and patient safety and experiences of care. Despite its prevalence, misconceptions persist regarding the impact of UB and the mechanisms through which it affects patients.

Areas covered: We draw upon evidence including outcomes from an extensive realist review and empirical research, to demonstrate the impacts of UB on patient safety and experience, particularly focusing on how even mild forms of incivility can compromise care quality. Recent research demonstrates a correlation between UB among healthcare professionals and reduced performance, increased medical errors, compromised safety culture, and inequities in patient outcomes. Additionally, we highlight the disproportionate impact of UB on minoritised staff and its resulting effects on patient care, which has the potential to perpetuate healthcare inequities via reducing staff diversity.

Expert opinion: Emerging evidence indicates that subtle UB such as incivility and undermining may be more damaging to patient outcomes than previously acknowledged. Addressing UB requires effective, tailored interventions, especially considering the disproportionate impact on diverse healthcare teams. Urgently combating UB is required to not only enhance patient safety but also to improve equity in care in healthcare systems worldwide.

Objectives: To investigate the impact of COVID-19 on hospitalization and consequent diabetes-related complications in patients with type 2 diabetes mellitus (diabetes).

Methods: We conducted a retrospective cohort study of patients with diabetes. Interrupted time series analysis (ITS) was used to analyze the monthly trends in diabetes-related hospitalization rates, including short- and long-term complications, 1-year before and after onset of COVID-19.

Results: Persons with diabetes experienced a significant (p < 0.001) rapid drop in monthly hospital admission rates at onset of COVID-19, then rose significantly (p = 0.003) to higher than pre-COVID-19 levels. Older age, lower education, and income levels were associated with higher base-level monthly hospital admission rates and a greater rate reduction at COVID-19 onset. ITS analysis showed monthly hospital admission rates from short-term complications surged to higher level 6 months after COVID-19 onset. Hospital admissions due to long-term complications decreased immediately post-COVID-19, but rose significantly (p < 0.001) to higher than pre-COVID levels, with patients experiencing higher nephropathy, angiography, and dermatological complications post-COVID-19.

Conclusion: COVID-19 had a negative impact on diabetes-related hospitalization access, resulting in increased short- and long-term complications. Long-term effects of deferred care due to COVID-19 on diabetes-related complications may persist, emphasizing the need for continued education toward improved diabetes self-management.

Objective: To evaluate the cost-effectiveness of trastuzumab emtansine (T-DM1) compared to both lapatinib plus capecitabine (lapcap) and trastuzumab plus capecitabine (trascap) for treating human epidermal growth factor 2-positive advanced breast cancer patients, who have received prior treatment with trastuzumab and a taxane, in Singapore.

Research design and methods: A three-state partitioned survival model was used to evaluate the cost-effectiveness from the local healthcare system perspective. Key effectiveness data, including progression-free survival and overall survival from the pivotal EMILIA trial, were used alongside local clinician inputs. Health state utilities (in quality-adjusted life years [QALYs]) were obtained from the literature and direct medical costs obtained from the public health institutions in Singapore. Scenario, one-way, and probabilistic sensitivity analyses were conducted to explore the impact of uncertainties on cost-effectiveness.

Results: The base-case incremental cost-effectiveness ratios (ICERs) for T-DM1, compared to lapcap and trascap, were S$472,600 and S$495,200 per QALY gained, respectively. Sensitivity and scenario analyses showed high ICERs, with a 50% price reduction for T-DM1 resulting in an ICER above S$200,000.

Conclusion: At current prices, T-DM1 is unlikely to be a cost-effective use of Singapore's limited healthcare budget. These findings can help inform policymakers, alongside other considerations, on funding decisions.

Introduction: Rheumatoid arthritis (RA) is a systemic inflammatory disease associated with an increased risk of cardiovascular disease (CVD) and premature mortality. The risk of CVD is closely associated with RA disease activity, and achieving RA remission using disease-modifying anti-rheumatic drugs (DMARDs) can significantly mitigate this risk. However, despite the availability of highly effective DMARDs, many veterans fail to achieve sustained RA remission.

Areas covered: We will discuss DMARD adherence in U.S. veterans with RA as it relates to RA disease activity and CVD risk, describe factors associated with DMARD non-adherence in individuals with RA, and discuss intervention strategies to improve DMARD adherence. For this review, the authors performed an extensive literature search using Embase, PubMed, Google Scholar, MEDLINE, Cochrane Library, Web of Science, and Duke University library resources.

Expert opinion: Barriers to DMARD adherence in veterans with RA are multifactorial and include patient-related factors, systemic barriers, and suboptimal adherence screening practices. Additional research is needed to create validated screening tools for DMARD adherence, train rheumatology providers on how to assess DMARD adherence, develop effective interventions to promote veteran self-efficacy in DMARD management, and to learn how to sustainably utilize multidisciplinary resources to support DMARD adherence in veterans with RA.

Objectives: Advanced therapy medicinal products (ATMPs) are transformative healthcare interventions, however, there has been limited research exploring their value. The objective of this study was to conduct a thematic analysis as part of a documentary analysis to identify value attributes of ATMPs.

Methods: As part of the NICE assessment processes in England, stakeholders are invited to provide comments on the technology. Nineteen NICE technology assessments (TAs) related to ATMPs were identified and subsequently included in a thematic analysis. All codes (and themes) were generated inductively from the text to reduce bias.

Results: This study found that the attributes of value of ATMPs can be categorized into three themes: treatment factors, innovation factors, and wider factors. That is, attributes of value of these interventions include not only direct treatment factors, although this was the largest category, but also additional factors such as those related to innovation and wider impacts the intervention may have, beyond the patient receiving therapy.

Conclusions: This thematic analysis of stakeholder comments submitted to NICE as part of TAs for ATMPs enhances the understanding of attributes of value of ATMPs. The findings are applicable to a wider geography.

Introduction: Patient-reported experience measures (PREMs) are patient reports about their healthcare, whereas patient-reported outcome measures (PROMs) are reports about their functioning and well-being regarding physical, mental, and social health. We provide a perspective on using PREMs and PROMs in ambulatory healthcare.

Areas covered: We conducted a narrative review of the literature about using PREMs and PROMs in research and clinical practice, identified challenges and possibilities for addressing them, and provided suggestions for future research and clinical practice.

Expert opinion: Substantial progress in using PREMs and PROMs has occurred during the last half-century. Collecting and reporting PREMs to clinicians in ambulatory care settings has improved communication with patients, diagnosis, and treatment, which may improve patients' health. Optimal use requires appropriate data analysis, minimizing implementation barriers, and facilitating interpretation of PREMs and PROMs in clinical practice. Also, formal structures and processes that include patient and family input into care improvement are needed (e.g. patient and family advisory councils as partners in co-design and coproduction of quality improvement). PREMs and PROMs have been used primarily in more affluent countries (e.g. the United States, Australia, United Kingdom, Netherlands, Japan, and Portugal), but this is expected to increase in many countries.

Introduction: The 2022 Inflation Reduction Act is expected to result in lower drug prices for Medicare beneficiaries in the United States (US). The Centers for Medicare & Medicaid Services (CMS) released the most recent draft guidance for the medicare drug price negotiation (DPN) program in May 2024.

Areas covered: In August 2023, the list of 10 drugs selected for the DPN were published and the first round of negotiations are now complete. While the latest CMS guidance highlights the importance of considering study limitations, bias, uncertainty, and generalizability, there were scarce methodological guidelines provided for the comparative effectiveness research studies. We conducted searches on PubMed and reviewed resources from websites of regulatory authorities.

Expert opinion: Broader domains of data quality, transparency, and methodology including study design, outcomes assessment, bias, confounding and uncertainty should be considered when developing methodological guidelines for evidence submitted for DPN. There is opportunity to align with and leverage existing guidelines published by federal and non-federal organizations in the US and globally while customizing the new guidance based on the specific requirements of DPN, keeping in mind CMS's interest in specific populations as well as health equity.

Background: Empirical evidence regarding temporal trends in cost per quality-adjusted life year (QALY) gained remains limited. This study investigates the evolution of cost-effectiveness for diabetes mellitus treatments over time.

Research design and methods: We analyzed cost-effectiveness analyses of anti-diabetic pharmaceuticals extracted from the Tufts Medical Center Cost-Effectiveness Analysis Registry (CEAR). Incremental cost-effectiveness ratios (ICERs) were normalized by GDP per capita and categorized into four threshold-based groups. In addition, we examined temporal trends in incremental QALYs. Analyses stratified by Organisation for Economic Co-operation and Development (OECD) membership were also conducted.

Results: Among 239 eligible studies, the proportion of highly cost-effective interventions in OECD countries decreased from 62.50% (1999-2005) to 35.48% (2021-2023), while interventions exceeding 3хGDP per capita/QALY increased from 12.50% to 54.84%. This trend was less pronounced in non-OECD countries. Mean incremental QALYs remained stable (range: 0.19-0.47) across periods and between OECD and non-OECD countries, suggesting that rising costs rather than diminishing health benefits drive the declining cost-effectiveness.

Conclusions: The cost-effectiveness of anti-diabetic medications has deteriorated substantially over time, particularly in OECD countries, despite stable therapeutic benefits. These findings highlight the need for dynamic approaches to cost-effectiveness thresholds and robust price negotiations to ensure sustainable access to innovative therapies.

Background: To investigate the cost-effectiveness of toripalimab plus chemotherapy versus chemotherapy alone for advanced non-small cell lung cancer (NSCLC) patients from a societal perspective.

Methods: A partitioned-survival model estimated the costs and cost-effectiveness of toripalimab plus chemotherapy versus standard chemotherapy for advanced NSCLC over 20 years. Clinical data were derived from the CHOICE-01 trial, and cost and utility inputs were gathered from Yaozh.com, expert interviews, and a nationwide hospital-based survey. Costs were reported in 2022 US dollars, and outcomes included quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs), with a 5% discount rate was applied. Sensitivity, subgroup, and scenario analyses verified the robustness of results.

Results: Toripalimab plus chemotherapy resulted in 3.048 QALYs and a total cost of $60,813, with an ICER of $19,066 per QALY gained, below China's 3 times GDP per capita threshold ($38,223). Robustness is confirmed through sensitivity, subgroup and scenario analyses.

Conclusions: Toripalimab plus chemotherapy is a cost-effective option for treatment-naive advanced NSCLC compared to chemotherapy alone, providing valuable evidence for clinical and reimbursement decision-making.