The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
复制标题
通过欧洲药品管理局的适应性途径方法进行药物的迭代开发
DOI：
10.3389/fmed.2019.00148
复制
发表时间：
2019
影响因子：
3.9
通讯作者：
P. Pierimarchi
中科院分区：
文献类型：
--
作者：
G. Nicotera;Gianluca Sferrazza;A. Serafino;P. Pierimarchi
开发新的调控工具，如适应性临床试验设计和真实世界证据的利用，是人们高度感兴趣的话题。最近，欧洲药品管理局(EMA)推出了适应性路径(AP)，这是医疗保健系统中的一种创新工具，允许与多个利益相关者就高度未得到满足的医疗需求领域的有前途的创新医疗产品进行早期对话。AP的创新方面是几个利益相关者的早期参与，如制药业、学术界、卫生技术评估(HTA)机构和患者代表，带来了他们对这种疾病的真实经验和他们对治疗的期望。AP不是一个新的许可工具，而是一个机会，在开始第二阶段研究之前，所有利益相关者，包括监管机构、公司、HTA机构和患者代表，在高度未满足的医疗需求领域就一种新的潜在药物进行非常早期的讨论。本文件的目的是描述AP方法从试点项目开始到目前为止的演变，重点介绍在欧洲一级取得的主要进展和成就。
The development of novel regulatory tools such as adaptive clinical trial design and utilization of real-world evidence are topics of high interest. Recently, the European Medicines Agency (EMA) introduced the Adaptive Pathways (AP) that represents an innovative tool in healthcare systems allowing the early dialogue with multiple stakeholders on promising and innovative medicinal products in areas with an high unmet medical need. The innovative aspect in the AP is the early involvement of several stakeholders such as pharmaceutical industry, the Academia, Health Technology Assessment (HTA) bodies, and patient representatives bringing their real experience with the disease and their expectations about the treatment. AP is not a new licensing tool but an opportunity for a very early discussions, before starting the phase II studies, among all stakeholders, including regulators, companies, HTA bodies, and patient representatives on a new potential medicine in areas of high unmet medical need. The aim of this paper is to describe the evolution of the AP approach from the beginning of the pilot project to date, highlighting major advances, and achievement at European level.
影响因子：
158.5
作者：
Hutchins, LF;Unger, JM;Albain, KS
通讯作者：
Albain, KS