Eve Hanna,Cécile Rémuzat,Pascal Auquier et al.
Eve Hanna et al.
Background: In 1989, the concept of human gene therapies has emerged with the first approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as promising therapies applicable to a broad range of diseases. Objectiv...
Value added medicines: what value repurposed medicines might bring to society? [0.03%]
老药新用：药品重新定向的社会价值何在？
Mondher Toumi,Cécile Rémuzat
Mondher Toumi
Background & objectives: Despite the wide interest surrounding drug repurposing, no common terminology has been yet agreed for these products and their full potential value is not always recognised and rewarded, creating a disincentive for ...
What makes a compliant Phase III and pre-launch patient advocacy strategy? [0.03%]
合规的III期和上市前患者倡导策略的特点是什么？
Nick Hicks,Keith Allan,Christoph Thalheim et al.
Nick Hicks et al.
Background: A key task for the pharmaceutical industry is to understand the compliance implications of engaging with a patient advocacy group (PAG). This presents challenges for the industry to negotiate the ethical and r...
A training paradigm to enhance performance and safe use of an innovative neuroendovascular device [0.03%]
一种增强创新神经血管设备性能和安全使用的培训方案
Donald R Ricci,Thomas R Marotta,Howard A Riina et al.
Donald R Ricci et al.
Training has been important to facilitate the safe use of new devices designed to repair vascular structures. This paper outlines the generic elements of a training program for vascular devices and uses as an example the actual training req...
Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement [0.03%]
确定治疗极罕见病的医学技术价值：共识声明
Michael Schlander,Silvio Garattini,Peter Kolominsky-Rabas et al.
Michael Schlander et al.
Background: In most jurisdictions, policies have been adopted to encourage the development of treatments for rare or orphan diseases. While successful as assessed against their primary objective, these policies have promp...
Perceptions and factors affecting pharmaceutical market access: results from a literature review and survey of stakeholders in different settings [0.03%]
药政市场准入的相关因素及各方认知：文献回顾和多国多方调查结果摘要
Semukaya Sendyona,Isaac Odeyemi,Khaled Maman
Semukaya Sendyona
Background: A change in the pharmaceutical environment has occurred from previously only needing to convince regulators of a product's safety and efficacy to obtain marketing authorisation to now needing to satisfy the va...
The quality of life of Spanish patients with Huntington's disease measured with H-QoL-I and EQ-5D [0.03%]
西班牙亨廷顿舞蹈病患者的生活质量评估：H-QoL-I与EQ-5D量表的使用
Julie Dorey,Emilie Clay,Amine Khemiri et al.
Julie Dorey et al.
Background and objective: Huntington's disease (HD) is an inherited neurodegenerative disorder that heavily affects the patient's motor, cognitive, and psychological functions. Yet, very few studies have measured the impa...
A price comparison of recently launched proprietary pharmaceuticals in the UK and the US [0.03%]
英国与美国近期上市的专利药价格比较分析报告
Jesper Jørgensen,Panos Kefalas
Jesper Jørgensen
Objective: To explore the relationship between prices charged by manufacturers of proprietary pharmaceuticals in the US and in the UK in recent years (2013-2016), expressed as a multiplier, and to detail to what extent th...
Risk of discontinuation of Advanced Therapy Medicinal Products clinical trials [0.03%]
先进治疗药物临床试验中断的风险
Eve Hanna,Cecile Rémuzat,Pascal Auquier et al.
Eve Hanna et al.
Objective: Advanced therapy medicinal products (ATMPs) constitute a class of innovative products that encompasses gene therapy, somatic cell therapy, and tissue-engineered products (TEP). There is an increased investment ...
Challenges in striving to simultaneously achieve multiple resource allocation goals: the pan-Canadian Oncology Drug Review (pCODR) example [0.03%]
同时实现多种资源配置目标的挑战：以泛加拿大肿瘤药物审查（pCODR）为例
Heather McDonald,Cathy Charles,Laurie Elit et al.
Heather McDonald et al.
The pan-Canadian Oncology Drug Review (pCODR) makes recommendations to Canada's provinces and territories (except Quebec) to guide their cancer drug funding decisions. The objective of this paper is to explore, using an economic perspective...