PDF(Pubmed)
Abstract：
To describe the characteristics of clinical study report (CSR) documents published by the European Medicines Agency (EMA), and for included pivotal trials, to quantify the timeliness of access to trial results from CSRs compared with conventional published sources.
Cross-sectional analysis of CSR documents published by the EMA from 2016 to 2018.
CSR files and medication summary information were downloaded from the EMA. Individual trials in each submission were identified using document filenames. Number and length of documents and trials were determined. For pivotal trials, trial phase, dates of EMA document publication and matched journal and registry publications were obtained.
The EMA published documents on 142 medications that were submitted for regulatory drug approval. Submissions were for initial marketing authorisations in 64.1%. There was a median of 15 (IQR 5-46) documents, 5 (IQR 2-14) trials and 9629 (IQR 2711-26,673) pages per submission, and a median of 1 (IQR 1-4) document and 336 (IQR 21-1192) pages per trial. Of all identified pivotal trials, 60.9% were phase 3 and 18.5% were phase 1. Of 119 unique submissions to the EMA, 46.2% were supported by a single pivotal trial, with 13.4% based on a single pivotal phase 1 trial. No trial registry results were identified for 26.1% trials, no journal publications for 16.7% and 13.5% of trials had neither. EMA publication was the earliest information source for 5.8% of pivotal trials, available a median 523 days (IQR 363-882 days) before the earliest publication.
The EMA Clinical Data website contains lengthy clinical trial documents. Almost half of submissions to the EMA were based on single pivotal trials, many of which were phase 1 trials. CSRs were the only source and a timelier source of information for many trials. Access to unpublished trial information should be open and timely to support decision-making for patients.
Cross-sectional analysis of CSR documents published by the EMA from 2016 to 2018.
CSR files and medication summary information were downloaded from the EMA. Individual trials in each submission were identified using document filenames. Number and length of documents and trials were determined. For pivotal trials, trial phase, dates of EMA document publication and matched journal and registry publications were obtained.
The EMA published documents on 142 medications that were submitted for regulatory drug approval. Submissions were for initial marketing authorisations in 64.1%. There was a median of 15 (IQR 5-46) documents, 5 (IQR 2-14) trials and 9629 (IQR 2711-26,673) pages per submission, and a median of 1 (IQR 1-4) document and 336 (IQR 21-1192) pages per trial. Of all identified pivotal trials, 60.9% were phase 3 and 18.5% were phase 1. Of 119 unique submissions to the EMA, 46.2% were supported by a single pivotal trial, with 13.4% based on a single pivotal phase 1 trial. No trial registry results were identified for 26.1% trials, no journal publications for 16.7% and 13.5% of trials had neither. EMA publication was the earliest information source for 5.8% of pivotal trials, available a median 523 days (IQR 363-882 days) before the earliest publication.
The EMA Clinical Data website contains lengthy clinical trial documents. Almost half of submissions to the EMA were based on single pivotal trials, many of which were phase 1 trials. CSRs were the only source and a timelier source of information for many trials. Access to unpublished trial information should be open and timely to support decision-making for patients.
摘要：
目的：描述欧洲药品管理局（EMA）发布的临床研究报告（CSR）文件的特征，对于包括关键试验，量化从CSRs获取试验结果的及时性，与传统公布的来源相比。
方法：EMA发布的2016年至2018年CSR文件的横截面分析。
方法：从EMA下载CSR文件和用药摘要信息。使用文件文件名识别每个提交的个体试验。确定文件和试验的数量和长度。对于关键的审判，试验阶段,获得了EMA文件出版日期以及匹配的期刊和注册表出版物日期。
结果：EMA公布了142种药物的文件，这些药物被提交给监管药物批准。提交的是64.1%的初始营销授权。中位数为15(IQR5-46)份文件，每次提交5(IQR2-14)次试验和9629(IQR2711-26,673)页，每个试验的中位数为1（IQR1-4）文档和336（IQR21-1192）页。在所有确定的关键试验中，60.9%为第3阶段，18.5%为第1阶段。在向EMA提交的119份独特作品中，46.2%得到了一项关键试验的支持，基于单个关键阶段1试验的13.4%。26.1%的试验没有确定试验登记结果，16.7%和13.5%的试验中,没有期刊出版物两者都没有.EMA出版物是5.8％关键试验的最早信息来源，最早出版前的中位数为523天（IQR363-882天）。
结论：EMA临床数据网站包含冗长的临床试验文件。提交给EMA的几乎一半是基于单一关键试验，其中许多是1期试验.CSR是许多试验的唯一信息来源和更及时的信息来源。对未公布的试验信息的访问应该是开放和及时的，以支持患者的决策。
方法：EMA发布的2016年至2018年CSR文件的横截面分析。
方法：从EMA下载CSR文件和用药摘要信息。使用文件文件名识别每个提交的个体试验。确定文件和试验的数量和长度。对于关键的审判，试验阶段,获得了EMA文件出版日期以及匹配的期刊和注册表出版物日期。
结果：EMA公布了142种药物的文件，这些药物被提交给监管药物批准。提交的是64.1%的初始营销授权。中位数为15(IQR5-46)份文件，每次提交5(IQR2-14)次试验和9629(IQR2711-26,673)页，每个试验的中位数为1（IQR1-4）文档和336（IQR21-1192）页。在所有确定的关键试验中，60.9%为第3阶段，18.5%为第1阶段。在向EMA提交的119份独特作品中，46.2%得到了一项关键试验的支持，基于单个关键阶段1试验的13.4%。26.1%的试验没有确定试验登记结果，16.7%和13.5%的试验中,没有期刊出版物两者都没有.EMA出版物是5.8％关键试验的最早信息来源，最早出版前的中位数为523天（IQR363-882天）。
结论：EMA临床数据网站包含冗长的临床试验文件。提交给EMA的几乎一半是基于单一关键试验，其中许多是1期试验.CSR是许多试验的唯一信息来源和更及时的信息来源。对未公布的试验信息的访问应该是开放和及时的，以支持患者的决策。
