زیرشاخه پژوهشیاقتصاد دارو
مقالهها، منابع و پژوهشهای تازه حوزه اقتصاد دارو
جستوجوی چندمنبعی
PubMed2026
American journal of therapeuticsOana-Monica Leah, Florin G Leaşu, Mihaela Badea, Mihaela Constantinescu, Mihai Vârciu, Liliana M Rogozea
BACKGROUND: Cardiovascular disease remains the leading cause of global mortality (19.8 million deaths in 2022; 32% of all deaths worldwide). Drug repurposing-extending approved agents beyond their original indications-has emerged as a high-impact strategy in cardiovascular prevention, offering reduced development timelines, established safety profiles, and faster implementation than de novo molecular development. STUDY QUESTION: Which repurposed cardiovascular agents demonstrate the most favorable pharmacoeconomic profiles, and how does the convergence of clinical benefit, patient risk stratification, and economic sustainability define the optimal hierarchy for cardiovascular prevention? STUDY DESIGN: Narrative review synthesizing evidence from 19 pivotal cardiovascular outcomes trials and European and American guidelines. No formal meta-analysis was applied. MEASURES AND OUTCOMES: For 13 agents across 8 therapeutic classes, efficacy was quantified as relative and absolute risk reductions, and as the number needed to treat or to harm. Pharmacoeconomic value was assessed via incremental cost-effectiveness ratio in US dollars per quality-adjusted life year, integrating mortality in years of life lost and morbidity in years lived with disability. Primary outcomes included all-cause mortality, cardiovascular mortality, major adverse cardiovascular events, and heart failure hospitalizations. RESULTS: Three Incremental Cost-Effectiveness Ratio (ICER) tiers were identified: Low-cost (< $20,000/ Quality-Adjusted Life Year (QALY)): ramipril (Number Needed to Treat (NNT) 28), carvedilol (NNT 29), metformin ( NNT 9-15, highest Relative Risk Reduction (RRR) 38-42%), and generic statins - robust mortality benefits at negligible cost; Moderate-cost ($3,000-$50,000/QALY): empagliflozin (↓38% cardiovascular mortality, NNT 61), dapagliflozin (NNT 20 in Heart Failure with Reduced Ejection Fraction), liraglutide (NNT 51), semaglutide (NNT 43), colchicine (NNT 36, morbidity benefit only), and branded statins; High-cost ($80,000-$300,000/QALY): Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) inhibitors (evolocumab NNT 63; alirocumab NNT 64) - proven benefit compromised by unaffordable biologic pricing. CONCLUSIONS: Pharmacoeconomic stratification of repurposed cardiovascular agents identifies 3 distinct tiers. Generic agents-ramipril, carvedilol, metformin, and statins-demonstrate the most favorable cost-effectiveness profiles and should form the basis of any prevention protocol. SGLT2 inhibitors and GLP-1 receptor agonists offer clinically meaningful benefits in secondary prevention when applied to populations meeting pivotal trial eligibility criteria. PCSK9 inhibitors remain cost-effective only in patients with very high cardiovascular risk and inadequate LDL control on maximally tolerated statin therapy.
PubMed2026
Expert review of pharmacoeconomics & outcomes researchMarta Trapero-Bertran
INTRODUCTION: Economic evaluation (EE) is a key input for pharmaceutical pricing and reimbursement (P&R), yet countries differ in how EE is specified, interpreted and integrated into decisions. Spain is a timely case: despite long-standing legal recognition of efficiency, EE has historically been weakly operationalized and difficult to trace in P&R. AREAS COVERED: This narrative review analyses Spain's reform trajectory through the lenses of specification, interpretation and integration, focusing on the work of the Advisory Committee for Pharmaceutical Financing (CAPF) (2019-2024). Evidence was identified through searches in PubMed, Scopus, Web of Science and Google Scholar (2000-2025), complemented by Spanish and EU legislative and policy documents. The review synthesizes CAPF recommendations to reorganize the evaluation pathway, including structured model linking clinical assessment, EE and budget impact analysis, and publication of national guideline and reference case. It also examines how these initiatives interact with draft national reforms and the EU HTA Regulation, highlighting a hybrid transitional context (late 2025) in which Joint Clinical Assessments are progressively incorporated while national procedural reform remains incomplete. EXPERT OPINION: Spain illustrates how EE standardization can strengthen deliberative, multi-criteria P&R by improving transparency and coherence, although impact depends on regulatory enactment, capacity and implementation.
PubMedدسترسی آزاد2026
The journal of headache and painSimona Guerzoni, Lanfranco Pellesi, Lisa Giannessi, Flavia Lo Castro, Maria Chiara Olivieri, Luca Degli Esposti, Francesca Gandolfi
BACKGROUND: Migraine is a leading cause of disability worldwide, impairing quality of life and productivity. Preventive therapies aim to reduce attack frequency, severity and the need for acute medications. Oral atogepant (60 mg), a calcitonin gene-related peptide (CGRP) receptor antagonist, has recently expanded migraine prevention options. This study evaluated changes in triptan use after atogepant initiation using real-world data. METHODS: This retrospective observational study used administrative healthcare databases from the Local Health Unit of Modena (Italy). Adults with chronic or high-frequency episodic migraine initiating atogepant between November 2023 and December 2024 were identified. Triptan use and related costs were assessed in the 6 months before and after treatment initiation (index date), measured as dispensed dosage units. RESULTS: Among 95 patients (86% female; mean age 53.5 years), 55 triptan users were included in the analysis. Triptan use decreased in 45 patients (82%), with ≥ 60% reduction in 27 (49%) and complete discontinuation in 10. Median consumption decreased from 76 to 24 dosage units, and mean consumption from 88.1 to 45.2. This reduction was statistically significant (Wilcoxon signed-rank test, p < 0.001). This reduction corresponded to a decrease of €2,591 in triptan-related pharmaceutical expenditure within the study cohort. CONCLUSION: Atogepant preventive treatment may substantially reduce acute migraine medication use in real-world practice, with potential clinical and economic benefits.
PubMedدسترسی آزاد2026
Frontiers in public healthYang Li, Yang Zou, Canhua Liang, Ziwei Feng, Shaohuan Lu, GuangZhao Wang, Guangyi Meng
OBJECTIVE: To evaluate the cost-effectiveness of first-line tislelizumab plus chemotherapy for extensive-stage small cell lung cancer (ES-SCLC) within the Chinese healthcare context. Furthermore, this study aims to comparatively validate the impact of traditional survival models vs. advanced machine learning models on the robustness of research findings, thereby providing refined empirical evidence for healthcare decision-making. METHODS: Based on data from the Phase III RATIONALE-312 clinical trial, a partitioned survival model (PSM) was constructed with a 10-year time horizon. From the perspective of the Chinese healthcare system, only direct medical costs were included. A willingness-to-pay (WTP) threshold was set at three times the 2025 per capita GDP of China (298,995 CNY/QALY). A dual-validation strategy was employed for survival extrapolation: a base-case analysis using the optimal parametric model (Log-logistic distribution) selected via Akaike Information Criterion/Bayesian Information Criterion (AIC/BIC) criteria, followed by a comparative validation using DeepSurv deep learning and random survival forest (RSF) models utilizing a simplified feature set. Uncertainty was assessed through one-way sensitivity analysis and probabilistic sensitivity analysis (PSA) using 1,000 Monte Carlo simulations. RESULTS: The base-case analysis (Log-logistic model) revealed that, compared with chemotherapy alone, the tislelizumab group yielded an incremental cost of 101,506.9 CNY and incremental quality-adjusted life years (QALYs) of 0.4044, resulting in an incremental cost-effectiveness ratio (ICER) of 251,030.5 CNY/QALY, which remains below the WTP threshold. Machine learning validation demonstrated exceptional consistency, with ICERs of 261,718.45 CNY/QALY for the DeepSurv model and 248,299.41 CNY/QALY for the RSF model. Sensitivity analysis identified the utility of progression-free survival (PFS) and the unit price of tislelizumab as the primary drivers of the model. PSA confirmed that the probability of tislelizumab being cost-effective exceeded 85% across all three survival-fitting logics, reaching a maximum of 94.90%. CONCLUSION: First-line tislelizumab plus chemotherapy demonstrates a significant cost-effectiveness advantage for ES-SCLC in China. The integration of machine learning survival models effectively reduced the inherent uncertainties of non-linear extrapolation in immunotherapy data, confirming the robustness of the conclusions. These findings provide a solid empirical basis for national health insurance negotiations and precision clinical applications.