Development of an academic disease registry for spinal muscular atrophy
Development of an academic disease registry for spinal muscular atrophy
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DOI：
10.1016/j.nmd.2019.08.014
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发表时间：
2019-10-01
影响因子：
2.8
通讯作者：
D'Amico, Adele
中科院分区：
文献类型：
--
作者：
Mercuri, Eugenio;Finkel, Richard;D'Amico, Adele
我们报告了一个新的SMA疾病登记处的发展，这是美国、意大利和英国三个国家网络与一家生物技术公司合作并在倡导团体的支持下合作的结果。高度策划的数据，将对所有SMA患者进行深入的表型分析，并对其进行数年的纵向随访。本文描述了该登记研究的开发过程，包括识别相关数据元素，设计带有共享数据字典的电子CRF，第一个版本的试点和最终版本的定义。登记中心将提供一个中央结构，用于进行基于比单个网络中可用的患者大得多的患者队列的学术研究。由于对收集的数据进行了质量控制，该登记也可用于上市后目的，允许以透明和受控的方式与制药合作伙伴，药品监管机构和倡导团体共享真实世界数据，以更好地了解新治疗的安全性和有效性。(C)2019由Elsevier B.V.出版
We report the development of a new disease registry on SMA as the result of a collaboration among three national networks in United States, Italy, and United Kingdom in partnership with a biotechnology company and with the support of advocacy groups.The aim of establishing a large collaborative registry within academic centers was to establish a structured but flexible system for collection of prospective, highly curated data that will deeply phenotype all patients with SMA and follow them longitudinally over several years.This paper describes the process leading to the development of the registry including the identification of the relevant data elements, the design of an electronic CRF with a shared data dictionary, the piloting of the first version and the definition of the final version.The registry will provide a central structure for conducting academic studies based on a much larger cohort of patients than those available in the individual networks. Due to the quality control of the data collected the registry can also be used for postmarketing purposes, allowing to share, in a transparent and controlled way, real-world data with pharmaceutical partners, drug regulatory agencies, and advocacy groups for better understanding of safety and effectiveness of new treatments. (C) 2019 Published by Elsevier B.V.