Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate [0.03%]
英国SACT数据集和EBMT登记处的升级以根据肿瘤学成果实施基于结果的报销：缺口分析及初步成本估算
Jesper Jørgensen,Panos Kefalas
Jesper Jørgensen
Background: Outcomes-based reimbursement (OBR) can reduce decision uncertainty and accelerate patient access to cell and gene therapies, however, OBR is rarely applied in practice in England. Oncology is the therapy area with the most cell ...
Cost-effectiveness of pirfenidone compared to all available strategies for the treatment of idiopathic pulmonary fibrosis in France [0.03%]
吡非尼酮与法国特发性肺纤维化所有可用疗法相比的性价比分析
Emilie Clay,Olivier Cristeau,Romain Chafaie et al.
Emilie Clay et al.
Objective: To update the health economic evaluation of pirfenidone in the treatment of idiopathic pulmonary fibrosis (IPF) compared to all available alternatives strategies (Best supportive care - BSC and nintedanib), based on a cost-utilit...
Measuring the health-related quality of life in young children: how far have we come? [0.03%]
测量儿童健康相关生活质量的方法：我们取得了哪些进展？
Nicola Germain,Samuel Aballéa,Mondher Toumi
Nicola Germain
The importance of understanding the impact of disease and treatment on children's Health-Related Quality of Life (HRQoL) has given rise to an increasing use of child self-report and observer or proxy instruments. In this article, we review ...
Management and clinical practice of multiple face and scalp actinic keratosis in France [0.03%]
法国多发性面部和头皮光化性角化病的管理及临床实践
Jacques Savary,Marie Christelle Tine,Anne Caroline Weber et al.
Jacques Savary et al.
Background: Actinic keratosis (AK) is characterized by the occurrence of thick and scaly skin areas caused by damage from ultraviolet radiation. The management of AK aims to reduce lesions and prevent their recurrence by regular monitoring....
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients [0.03%]
治疗脊髓性肌萎缩症1型患者的onasemnogene abeparvovec (AVXS-101)使用成本效益分析
Daniel C Malone,Rebecca Dean,Ramesh Arjunji et al.
Daniel C Malone et al.
Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of ...
Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption [0.03%]
技术预测：晚期临床研究、EMA审批或通过医院豁免进行临床应用的先进疗法
Claudia Eder,Claudia Wild
Claudia Eder
Background: The umbrella term ATMPs (Advanced Therapy Medicinal Products) comprises cell therapies, gene therapeutics and tissue engineered products. After implementation of the Regulation 1394/2007, only a couple of products have obtained ...
From investigational product to active reference: evolution of oral sumatriptan efficacy versus placebo for the treatment of acute migraine episodes and potential impact in comparative analyses [0.03%]
从试验药物到活性对照药：口服舒马曲普坦治疗急性偏头痛发作的疗效相对于安慰剂的变化及其在比较分析中的潜在影响
Katia Thokagevistk,Clément François,Mélanie Brignone et al.
Katia Thokagevistk et al.
Background: The relative efficacy and safety can vary among drugs over time. Sumatriptan, a first choice drug for acute migraine, can illustrate this phenomenon. Objective: To assess the evolution of the relative efficacy and tolerability o...
Government pharmaceutical pricing strategies in the Asia-Pacific region: an overview [0.03%]
亚太地区政府医药产品定价策略概览
Naina R Verghese,Jon Barrenetxea,Yukti Bhargava et al.
Naina R Verghese et al.
Background and objectives: Governments in Asia Pacific (APAC) are increasingly using pharmaceutical pricing strategies to contain rising healthcare costs. The objective of this narrative review is to discuss formal pricing strategies for re...
Małgorzata Biernikiewicz,Vanessa Taieb,Mondher Toumi
Małgorzata Biernikiewicz
Objective: Doctor-shopping has significant consequences for patients and payers and can indicate misuse of drugs, polypharmacy, less continuity of care, and increased medical expenses. This study reviewed the literature describing doctor-sh...