Choudhury Mohua Chakraborty, Chakraborty Indraneel, Saberwal Gayatri
Institute of Bioinformatics and Applied Biotechnology, Bengaluru, India.
PLOS Glob Public Health. 2022 Apr 22;2(4):e0000261. doi: 10.1371/journal.pgph.0000261. eCollection 2022.
Clinical trial registries such as ClinicalTrials.gov (CTG) hold large amounts of data regarding trials. Drugs for rare diseases are known as orphan drugs (ODs), and it is particularly important that trials for ODs are registered, and the data in the trial record are accurate. However, there may be discrepancies between trial-related data that were the basis for the approval of a drug, as available from Food and Drug Administration (FDA) documents such as the Medical Review, and the data in CTG. We performed an audit of FDA-approved ODs, comparing trial-related data on phase, enrollment, and enrollment attribute (anticipated or actual) in such FDA documents and in CTG. The Medical Reviews of 63 ODs listed 422 trials. We used study identifiers in the Medical Reviews to find matches with the trial ID number, 'Other ID' or 'Acronyms' in CTG, and identified 202 trials that were registered with CTG. In comparing the phase data from the 'Table of Clinical Studies' of the Medical Review, with the data in CTG, there were exact matches in only 75% of the cases. The enrollment matched only in 70% of the cases, and the enrollment attribute in 91% of the cases. A similar trend was found for the sub-set of pivotal trials. Going forward, for all trials listed in a registry, it is important to provide the trial ID in the Medical Review. This will ensure that all trials that are the basis of a drug approval can be swiftly and unambiguously identified in CTG. Also, there continue to be discrepancies in trial data between FDA documents and CTG. Data in the trial records in CTG need to be updated when relevant.
诸如ClinicalTrials.gov（CTG）之类的临床试验注册机构拥有大量与试验相关的数据。用于罕见病的药物被称为孤儿药（OD），对孤儿药试验进行注册且试验记录中的数据准确尤为重要。然而，作为药物批准依据的试验相关数据（可从美国食品药品监督管理局（FDA）的文件如医学审评中获取）与CTG中的数据之间可能存在差异。我们对FDA批准的孤儿药进行了审核，比较了此类FDA文件和CTG中关于试验阶段、入组人数及入组属性（预期或实际）的试验相关数据。63种孤儿药的医学审评列出了422项试验。我们利用医学审评中的研究标识符在CTG中查找与试验ID号、“其他ID”或“首字母缩写”的匹配项，确定了202项在CTG中注册的试验。在比较医学审评“临床研究表”中的阶段数据与CTG中的数据时，仅有75%的情况完全匹配。入组人数仅有70%的情况匹配，入组属性有91%的情况匹配。关键试验子集也发现了类似趋势。展望未来，对于注册机构列出的所有试验，在医学审评中提供试验ID很重要。这将确保在CTG中能迅速且明确地识别出作为药物批准依据的所有试验。此外，FDA文件和CTG之间的试验数据仍存在差异。CTG中试验记录的数据在相关时需要更新。
