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.

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 socioeconomic 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: 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.

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: 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.

Objectives: This study quantified the public value (PV) of the criteria and sub-criteria in the current drug reimbursement systems in South Korea and examined sociodemographic factors that associated with PV.

Methods: The Analytic Hierarchy Process (AHP) was used to quantify the PVs of criteria and sub-criteria. We developed a questionnaire to generate pairwise comparison matrices among criteria and sub-criteria. From 27 March to 1 April 2023, we recruited 1,000 study participants using a quota sampling method stratified by age, sex, and region based on Korean census data.

Results: The PVs for the criteria were highest for clinical usefulness (28.5%), followed by cost-effectiveness (27.1%), budget impact (24.3%), and reimbursement in other countries (20.1%). The sociodemographic characteristics of the participants had a significant impact on the PVs of the criteria. Willingness to pay additional premiums for national health insurance was negatively associated with PV for clinical usefulness and cost-effectiveness and positively associated with PV for reimbursement in other countries.

Conclusions: The public prioritized clinical usefulness and cost-effectiveness as the main criteria. However, the PVs of the criteria were divergent and associated with sociodemographic factors. Divergent public interests require an evidence-informed deliberative process for reimbursement decisions.

Introduction: In breast cancer clinical trials utilizing digital endpoints, wearable sensors record participants' health information during activities of daily living.  These sensors are worn on the wrist or finger, placed as a skin patch or headband, or embedded on clothing. Data collected from wearable sensors form the basis of a digital endpoint, useful for determining effects of novel treatments on health outcomes, particularly quality-of-life outcomes.

Areas covered: References for this article were selected from a PubMed search spanning from 1 January 2017,to 1 July 2024, using the terms 'wearable sensors,' 'digital endpoints,' 'virtualtrials,' 'breast cancer.'  Additional articles from the authors' personal collection of papers and reviewers suggestions were also used.

Expert opinion: Digital endpoints must be validated as proper surrogate measures for healthcare outcomes, prior to their use in breast cancer trials.  Wearable sensors may introduce biases, such as 'missing not-at-random bias,' and perhaps even exacerbate disparities in healthcare outcomes if patients not comfortable with their use are excluded from clinical trials, or if the accuracy of sensors varies between racial and ethnic groups. Therefore, before embarking on trials with digital endpoints, validation studies are required, and limitations and risks of such trials need to be addressed.

Introduction: Pharmacovigilance (PV) plays a central role as a quality benchmark for healthcare systems in any country. Adverse drug reactions (ADRs) contribute significantly to patient hospitalization and are major contributors to morbidity and mortality worldwide. Achieving improvements in health infrastructure and employing precise monitoring tools are essential components of drug safety. As reliance on drug therapy increases, patient exposure to potential risks rises, emphasizing the importance of minimizing ADRs.

Area covered: A search for studies published from January 2010 to November 2023 was retrieved from PubMed, Medline, and Google Scholar databases. We developed the search strategies using the Mesh terms and keywords. Only English-language literature was included.

Expert opinion: Twenty-nine studies met the inclusion criteria and utilized to evaluate the pharmacovigilance and its outcomes. The Saudi 2030 vision outlines an initiative to enhance patient care through a robust, safety- and quality-centered culture, fostering collaboration between drug manufacturers and regulatory authorities. This collaborative approach is expected to result in higher-quality care for the public. Moreover, a unified, simple, and advanced ADR reporting portal, in collaboration with stakeholders, is recommended to enhance the quality of ADR reporting. Also, commitment to training, updating courses, and incorporating PV practices into curricula demonstrates progress in Saudi PV System.