Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: Tumor-agnostic treatments represent paradigm shift in oncology, targeting specific genetic mutations irrespective of the tumor's anatomical origin. This personalized approach relies on companion diagnostic testing to identify patients likely to respond to treatment. However, incorporating molecular diagnostics poses challenges to healthcare systems, particularly regarding costs and accessibility. This must be addressed in economic evaluations to guide health technology assessment and reimbursement decisions.

Areas covered: This study reviewed economic evaluations of tumor-agnostic therapies, focusing on the role of diagnostic testing. A literature search identified 13 compounds targeting nine molecular alterations, with eight relevant publications analyzed. The review revealed heterogeneity in how diagnostic testing is incorporated into cost-effectiveness models. While some studies excluded the costs and consequences of diagnostic tests entirely, others included them partially or fully. Key findings underscore the substantial impact of testing, particularly for low-prevalence biomarkers.

Expert opinion: Integrating diagnostic testing into economic evaluations is essential for accurately assessing the health economic value of tumor-agnostic therapies. Economic models must account for test-treatment combinations and consider shifts in treatment pathways compared to routine practice. As the field evolves, the development of robust frameworks and clear guidance on the requirements are crucial to support sustainable and equitable healthcare decision-making.

Introduction: U.S. FDA designations: Breakthrough Therapy (BTD), Fast Track (FTD), Orphan Drug (ODD), and Regenerative Medicine Advanced Therapy (RMAT) aim to expedite drug development, yet their combined economic and developmental effects have not been extensively studied. This systematic review evaluates their impacts on Day 1 cumulative average abnormal returns (CAAR) and timelines from Investigational New Drug submission to approval.

Methods: A systematic search of PubMed, Scopus, ScienceDirect, GreyNet, OpenGrey, ProQuest, and Cochrane Library was conducted for articles published between January 1997 and September 2024. A random-effects model generated pooled estimates with 95% confidence intervals (CI), and heterogeneity was assessed using the Cochrane-Q and I² statistic. Study quality was evaluated using Drummond's checklist and an adapted Barker checklist.

Results: Twenty-five studies were included. The pooled Day 1 CAAR across designations was 6.12% (95% CI: 3.64-8.61). Subgroup analysis revealed FTD with the strongest immediate market impact (8.20%%, 95% CI: 4.38-12.03) and BTD with the shortest mean approval timeline (69.96 months, 95% CI: 60.25-79.67).

Conclusions: FDA designations provide economic advantages, especially for smaller companies, and can expedite approvals for high-priority therapies. Notable heterogeneity, particularly with RMAT, warrants further research to clarify how disease area and company size shape real-world outcomes.

Registration: ResearchRegistry ID11080.

Introduction: Enhanced recovery after surgery (ERAS) pathways are widely adopted in both major and minimally invasive surgeries. However, ERAS pathway implementation in ventral hernia repair (VHR) surgery remains an area of ongoing research given the variability in hernia complexity and surgical approach. To address this, our institution proposed and developed a stratified ERAS pathway to deliver effective, tailored perioperative care.

Areas covered: This narrative describes the development of the ERAS pathway stratified to address the varied perioperative needs of VHR procedures, outline the evidence-based interventions comprising the bundle, describe the implementation process, and discuss the potential economic impact of implementing this pathway. We conducted a systematic literature search, last updated on 15 February 2024.

Expert commentary: By leveraging the common elements of ventral hernia repair ERAS pathways, while addressing the perioperative needs of patients undergoing more complex procedures, a stratified pathway approach provides a practical and adaptable framework that balances intervention specificity with ease of implementation. While the introduction of conditional element modifications increases pathway complexity, it also facilitates patient-centered delivery of care. Operational expertise in organizing such pathways, as well as the implementation science behind it, is an opportunity to advance the frontiers of ERAS program developments.

Introduction: This systematic scoping review aimed to identify and analyze current methodological approaches used in model-based economic evaluations (EEs) of prognostic and predictive companion diagnostics (pCDx), highlighting methodological gaps and challenges.

Methods: Systematic searches were conducted in PubMed and Scopus (January 2009-March 2023). Included studies were model-based EEs, methodological papers, or reviews specifically addressing prognostic or predictive CDx. Data extraction followed the modified CHEERS checklist. Results were synthesized narratively across six methodological domains. No formal risk of bias assessment was done per scoping review conventions.

Results: Eighty-eight studies were included, of which 60 were model-based EEs. Most studies utilized Markov cohort models (37%) or decision tree-Markov hybrids (30%). Quality-adjusted life-years (QALYs) were the main outcome (88%). Only 15% of studies derived clinical utility from randomized controlled trials, and fewer than half explicitly modeled diagnostic accuracy. Methodological limitations included inconsistent modeling of real-world test-treatment pathways, insufficient consideration of pretest probabilities, diagnostic thresholds, and inadequate uncertainty analyses.

Conclusions: This review identified variability and methodological gaps in economic evaluations of pCDx. Standardizing evaluation methods, integrating real-world evidence, and systematically considering the diagnostic accuracy and uncertainty could improve the robustness of pCDx evaluations. Limitations of this study included overrepresentation of breast cancer studies.

Registration: OSF Registries (22 February 2023) DOI 10.17605/OSF.IO/GVFMQ.

Background: Prostate cancer is a leading cause of cancer death among men. Metastatic castration-resistant prostate cancer (mCRPC) treatment has changed dramatically since the introduction of novel hormonal agents (NHAs); however, real-world data including healthcare resource utilization (HCRU) and costs are lacking in Greece.

Methods: A subset of patients with HCRU data from the PROSPECT retrospective chart review study of patients with mCRPC who initiated first line (1 L) systemic therapy with chemotherapy or NHAs were included; HCRU and costs incurred during mCRPC treatment were estimated.

Results: HCRU remained mostly stable across lines for the 119 patients included. Overall, median HCRU cost was €2,363.8 per patient per month (PPPM); 1 L was €2,475.0, second line (2 L) was €1,698.9, third line (3 L) was €2,499.0. Cost was mostly made up of systemic treatment (~90%). Overall HCRU 2 L costs were €688,795.1, contributing the least to the total HCRU cost of the three lines studied (€3,820,561.1). 2 L cost was lower as a greater proportion of patients received chemotherapy than in 1 L or 3 L, and chemotherapy was cheaper than NHAs.

Conclusions: Cost was impacted by type of systemic treatment; 2 L treatment costs were lower as a higher proportion of patients were treated with chemotherapy rather than NHAs.

Introduction: In low-income and middle-income countries (LIMCs), defined based on the World Bank classification, non-adherence to respiratory therapies contributes to increasing mortality and morbidity due to chronic respiratory diseases. To address this issue, it is essential to identify and tackle underlying factors such as cultural beliefs, socioeconomic disparities, and limited access to healthcare resources and infrastructures. The absence of strategies that integrate community involvement, healthcare professional's training, economic policies, and educational programs exacerbates the disproportionate burden of chronic respiratory diseases in LIMCs.

Areas covered: This review is based on a structured literature search across PubMed, Scopus, and Google Scholar (2000-2023) using terms relevant to asthma, COPD, adherence, and LMICs. The review examines key factors that hinder patients' adherence to Asthma and COPD medications in LIMCs, providing some insights into the issue and proposing concrete solutions.

Expert opinion: Addressing non-adherence requires a multifaceted approach involving community engagement, educational initiatives, and improved healthcare infrastructure. Future research should focus on tailored interventions to enhance adherence and ultimately improve health outcomes for patients with chronic respiratory diseases in LMICs.

Objectives: We evaluated the cost-effectiveness of implementing different pneumococcal conjugate vaccines (PCV) - 20-valent (PCV20; 3 + 1), 13-valent (PCV13; 2 + 1), and 15-valent (PCV15; 2 + 1) - into the Spanish pediatric national immunization program (NIP) for pneumococcal disease prevention.

Methods: A Markov model adopting a Spanish National Healthcare System perspective and annual cycles estimated the health and cost impact of PCV20, PCV13, and PCV15 over 10 years among children. Epidemiological, cost, and utility inputs were derived from published literature and official databases; vaccine efficacy inputs were based on PCV13 clinical effectiveness and 7-valent PCV efficacy and impact studies. Sensitivity analyses evaluated model robustness.

Results: PCV20 implementation was predicted to reduce the pneumococcal disease burden, preventing > 1,000,000 pneumococcal disease cases and > 150 deaths, versus both comparators. The adoption of PCV20 was estimated to result in cost-savings of approximately €1 billion versus PCV13 and PCV15. PCV20 demonstrated dominance over both alternatives, with 100% of 1,000 probabilistic sensitivity analysis iterations indicating PCV20 dominance.

Conclusion: Incorporating PCV20 3 + 1 into the Spanish pediatric NIP was predicted to be more effective at a lower cost than PCV13 2 + 1 and PCV15 2 + 1 due to its broader serotype coverage and enhanced protection against pneumococcal disease.

Objective: Variant transthyretin (ATTRv) amyloidosis is a rare genetic multisystem disease inducing progressive morbidity. Evidence about its economic burden is scarce. This study estimated annual use and costs of healthcare-resource-utilization (HRU) and indirect costs for ATTRv mutation carriers and patients with polyneuropathy from a societal perspective in Spain.

Methods: A cross-sectional, retrospective non-interventional multicenter study was conducted in 2018-2020 to characterize asymptomatic carriers of mutations in the TTR gen (AC) and Coutinho stage 1 transthyretin amyloidosis polyneuropathy patients (PA). HRU, associated costs, and indirect wages were assessed during 12 months prior to enrollment.

Results: One-hundred-five participants were analyzed: 86 AC and 19 PA. Average healthcare cost-per-person-per-year was significantly higher in PA: €2,323.6 vs. €953.3 (p < 0.001), due to higher utilization of medical specialty visits, hospitalizations, and nerve conduction studies. Indirect costs were statistically not different (€380.1 for AC and €781.3 for PA, p = 0.581). Days worked with fatigue/pain were significantly higher in PA: 53.9 vs 5.5 (p = 0.034). Total cost was significantly higher in PA than AC (€3,105.0 vs €1,333.4; p < 0.001).

Conclusions: Both AC and early-stage ATTRv-PN patients showed a meaningful economic burden to the National Health System and society. We observed an annual incremental economic burden of €1,771.5 in PA versus AC.

Objectives: Lacking national policy for adult pneumococcal vaccination, local Indian guidelines recommend 13-valent pneumococcal polysaccharide conjugate vaccine (PCV13) followed by 23-valent pneumococcal polysaccharide vaccine (PPV23). However, no vaccination ('NV') or PPV23 alone are most common in clinical practice. We evaluated cost-effectiveness of PCV13 versus NV and, alternatively, PPV23 in India.

Methods: Cohort model with Markov-type process projected lifetime cases, deaths, and costs associated with invasive pneumococcal disease and all-cause non-bacteremic pneumonia (discounting = 5%/year). Cost per quality-adjusted life year (QALY) gained was assessed among at-risk/high-risk adults aged 50-59 years and all aged 60-99 years (N = 209.5 M) from private/patient and government/payer perspectives, which differed on medical costs, vaccination costs, and uptake rates.

Results: From private/patient perspective, PCV13 versus NV yielded ₹480,908/QALY (ΔCosts=₹27.2B;ΔQALYs = 56,560), whereas PCV13 was dominant versus PPV23 (ΔCosts=-₹359.1 M; ΔQALYs = 53,861).From government/payer perspective, corresponding ratios were ₹610,178/QALY (ΔCosts=₹84.4B;ΔQALYs = 138,382), and ₹456,048/QALY (ΔCosts=₹60.2B; ΔQALYs = 131,933). In probabilistic sensitivity analyses, cost-effectiveness was <₹600,000/QALY in 72.9%-99.7% of replications (N = 1000/analysis), depending on comparison/perspective.

Conclusions: PCV13 versus NV among at-risk/high-risk adults aged 50-59 years and all aged ≥ 60 years would be cost-effective from both perspectives considering willingness-to-pay equaling approximately 3× gross domestic product/capita. Furthermore, PCV13 versus PPV23 would be cost-effective and cost saving from government/payer and private/patient perspectives, respectively.