Department of General Practice, Royal College of Surgeons in Ireland, Dublin, Ireland
Royal College of Surgeons in Ireland, HRB Centre for Primary Care Research, Dublin, Ireland.
BMJ Open. 2023 May 15;13(5):e068981. doi: 10.1136/bmjopen-2022-068981.
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 Access to unpublished trial information should be open and timely to support decision-making for patients.
描述欧洲药品管理局（EMA）发布的临床研究报告（CSR）文件的特点，并对纳入的关键性试验进行定量分析，比较从 CSR 获得试验结果的及时性与传统已发表来源。
对 EMA 2016 年至 2018 年发布的 CSR 文件进行横断面分析。
从 EMA 下载 CSR 文件和药物总结信息。使用文件名识别每份提交材料中的单独试验。确定文件和试验的数量和长度。对于关键性试验，获取试验阶段、EMA 文件发布日期以及匹配的期刊和注册处出版物。
EMA 发布了 142 种药物的文件，这些药物均已提交药物监管审批。提交的初始营销授权占 64.1%。每份提交材料中位数有 15 份（IQR 5-46）文件、5 份（IQR 2-14）试验和 9629 页（IQR 2711-26673），每份试验中位数有 1 份（IQR 1-4）文件和 336 页（IQR 21-1192）。在所有确定的关键性试验中，60.9%为 3 期试验，18.5%为 1 期试验。在向 EMA 提交的 119 份独特材料中，46.2%由单一关键性试验支持，其中 13.4%基于单一关键性 1 期试验。26.1%的试验未在试验注册处找到结果，16.7%的试验未在期刊上发表，13.5%的试验两者均未发表。EMA 发布是 5.8%关键性试验的最早信息来源，最早发布日期前中位数 523 天（IQR 363-882 天）可获得。
EMA 临床数据网站包含冗长的临床试验文件。向 EMA 提交的材料中近一半基于单一关键性试验，其中许多为 1 期试验。CSR 是许多试验的唯一来源和更早的信息来源。应开放和及时获取未发表的试验信息，以支持患者的决策。
