Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: Economic evaluation guidelines (EEGs) serve as a valuable tool to assist appraisers in making consistent and transparent recommendations, standardize EE studies, enhance their quality, and minimize methodological uncertainties. As other LMICs, Lebanon aims for UHC where EEG is a necessity. This paper aims to report on the Lebanese health EEG (LEEG) and its reference case, including the intermediate results leading to the final decisions.‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬.

Research design and methods: The LEEG followed a structured, systematic, and transparent process: (1) identifying the rationale and the guideline scope; (2) establishing the Guideline Development Group; (3) searching the evidence; (4) planning the development process; (5) selecting the panel for the deliberative process; (6) surveying Lebanese stakeholders; (7) deliberating on the results; (8) drafting the guideline; and (9) consulting with international experts.

Results: The LEEG includes three general characteristics, 19 key features, a reference case, and an action plan.

Conclusions: The LEEG is the first national EEG for health interventions. It will help decision-makers, researchers, and healthcare providers improve the quality and assessment of EE in Lebanon to identify the most cost-effective health interventions. Implementing LEEG is crucial to promoting an equitable, efficient, and high-quality health system with a more consistent decision-making process.

Background: This retrospective observational study described the epidemiology and the burden on the Italian healthcare service (SSN) of patients with polyneuropathy (PN) associated with hereditary transthyretin amyloidosis (ATTRv).

Research design and methods: From the Fondazione ReS (Ricerca e Salute) administrative healthcare database (~5.5 million inhabitants in 2021), patients were identified as having ATTRv-PN in 2021 if they had received treatments for ATTRv-PN under SSN reimbursement (i.e. tafamidis, patisiran, or inotersen) from 1 January 2014 to 31 December 2021 (index date in 2021). Demographics and comorbidities at the baseline, healthcare resource consumption, and related direct costs reimbursed by the SSN throughout the one-year follow-up were described.

Results: In 2021, 36 patients with ATTRv-PN (prevalence: 7.4/1,000,000) were identified (males were 83.3%; patients with ≥2 comorbidities were 61.1%; the mean age was 73 ± 8 years). During follow-up, of patients, 91.7% received drugs for ATTRv-PN; >50% received antiepileptics and acid suppressants; 22.2% were admitted to overnight hospitalizations; 30.6% accessed the emergency department; 97.2% received local outpatient specialist care. The per patient mean annual cost was € 122,017; drugs for ATTRv-PN accounted for 94.7% of the total expenditure.

Conclusions: This study of real-world patients with ATTRv-PN showed a high rate of comorbidities, and substantial direct healthcare and economic burdens on the SSN.

Introduction: The 2022 Inflation Reduction Act (IRA) 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 (CER) 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.

Objectives: Obstructive sleep apnea (OSA) is a sleep-related breathing disorder characterized by recurrent episodes of nocturnal breathing cessation resulting from upper airway collapse. Given the absence of a comprehensive review of the cost-effectiveness of OSA treatments, we undertook an extensive systematic review and meta-analysis to calculate the pooled incremental net benefit (INBp).

Methods: A systematic search of PubMed, Embase, Scopus, and Tufts cost-effectiveness analysis registry was conducted. INBp with 95% confidence intervals (CI) was estimated using a random-effects model, and heterogeneity was assessed through the Cochrane-Q test and I² statistic. Study quality was evaluated using the modified ECOBIAS Checklist, and GRADE framework was applied to assess the certainty of outcomes.

Results: Thirty-four studies were included in the systematic review, fifteen qualifying for meta-analysis. CPAP was cost-effective compared to other treatments, with a INBp of $13,024 (95%CI $6,813 to $19,236), and substantial heterogeneity (I² = 97.48%). Compared to no treatment and oral appliances (OAs), CPAP showed cost-effective INB values of $30,834 ($21,325 to $40,343) and $2,708 ($645 to $4,771) respectively.

Conclusion: CPAP is cost effective compared to all treatments collectively, as well as specifically to OAs and no treatment though with low certainty.

Background: This study aims to examine the short-term, population-level effects of the 2023 Australian Pharmaceutical Benefits Scheme (PBS) copayment reduction on prescription volume, patients' out-of-pocket (OOP) expenditure, and government contributions.

Research design and methods: We conducted a quasi-experimental study using national data from January 2021 to April 2024. For system-level analysis, we examined all drugs used by general patients, focusing on 252 drugs that were 'above copayment' during 2022-2023. We also performed drug category-specific analyses on six broad groups of drugs. Paired-sample t-tests and segmented regression analyses were used to compare prescription volumes, OOP expenditure, and government contributions before and after the copayment reduction.

Results: The copayment reduction was not associated with significant changes in prescription volumes or government contributions for general patients. However, the copayment reduction led to an immediate, but not gradual, decrease in inflation-adjusted OOP expenditure. Specifically, there was a relative reduction of 26.1% at 15 months post-policy for drugs above the general copayment (95% confidence interval (CI): -34.10, -18.10; p-value < 0.001). Similar immediate declines were observed across the six selected drug categories.

Conclusions: Further research is needed to assess the longer-term effects of copayment reductions, particularly their impact on medication adherence and overall healthcare costs.

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.

Introduction: Cervical cancer is almost entirely preventable by vaccination and screening. Population based vaccination and screening programs are effective and cost effective, but millions of people do not have access to these programs, causing immense suffering. The WHO Global Strategy for the elimination of cervical cancer as a public health problem calls for countries to meet ambitious vaccination, screening and treatment targets.

Areas covered: Epidemiological evidence indicates marked socioeconomic gradients in the burden of cervical cancer and vaccination, screening, and treatment coverage. The unacceptable socio-economic burden of cervical cancer is largely a function of inequitable access to these programs. We discuss these inequities, and highlight strategies enabled by new evidence and technology. Single dose HPV vaccination, HPV based screening, and the rapidly moving technology landscape have enabled task-shifting, innovation in service delivery and the possibility of scale. Equitable access to optimal care for the treatment of invasive cancers remains a challenge.

Expert opinion: Cervical cancer can be eliminated equitably. It will require global political will, sustained public and private investment, and community leadership to safely and sustainably embed proven tools, technology and infrastructure in local health and knowledge systems.

Background: Biologics are recommended for use in patients with psoriatic arthritis (PsA) after the failure of conventional systemic disease-modifying anti-rheumatic drugs (csDMARDs). However, compared to csDMARDs, biologics are significantly more expensive. The aim of this study was to evaluate the cost-effectiveness of biologic treatments for active PsA patients who have failed treatment with csDMARDs, from the perspective of the Chinese healthcare system.

Research design and methods: A discrete event simulation model was constructed to estimate health and economic outcomes of patients. The seven biologics recommended by the Chinese psoriasis treatment guidelines were included in the evaluation. One-way and probabilistic sensitivity analysis were performed to ensure that our results were robust.

Results: Our results found that compared to the standard of care (SoC) and all other biologics strategies, secukinumab (SEC) had the highest quality-adjusted life years, and at a willingness-to-pay (WTP) threshold of US $38,161 per QALY, SEC was the most cost-effective option, with an incremental cost-effectiveness ratio of $14,968 per QALY. One-way sensitivity analysis and probabilistic sensitivity analysis confirmed the robust of this result.

Conclusions: From the perspective of the Chinese healthcare system, biologics are estimated to be cost-effective compared to SoC. Among these, SEC is the most cost-effective option.

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 3.052 QALYs and a total cost of $60,813, with an ICER of $19,066 per QALY, 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.