Department of Clinical Immunology, Medical University of Warsaw, Warsaw, Poland.
Centre for Studies on Research Integrity, Institute of Law Studies, Polish Academy of Sciences, Warsaw, Poland.
J Med Internet Res. 2021 Oct 28;23(10):e26890. doi: 10.2196/26890.
ClinicalTrials.gov (CT.gov) is the most comprehensive internet-based register of different types of clinical studies. Expanded access is the use of unapproved drugs, biologics, or medical devices outside of clinical trials. One of the key problems in expanded access is the availability to both health care providers and patients of information about unapproved treatments.
We aimed to evaluate CT.gov as a potential source of information about expanded access programs.
We assessed the completeness of information in the records of 228 expanded access programs registered with CT.gov from February 2017 through May 2020. Moreover, we examined what percentage of published expanded access studies has been registered with CT.gov. Logistic regression (univariate and multivariate) and mediation analyses were used to identify the predictors of the absence of some information and a study's nonregistration.
We found that some important data were missing from the records of many programs. Information that was missing most often included a detailed study description, facility information, central contact person, and eligibility criteria (55.3%, 54.0%, 41.7%, and 17.5% of the programs, respectively). Multivariate analysis showed that information about central contact person was more likely to be missing from records of studies registered in 2017 (adjusted OR 21.93; 95% CI 4.42-172.29; P<.001). This finding was confirmed by mediation analysis (P=.02). Furthermore, 14% of the programs were registered retrospectively. We also showed that only 33 of 77 (42.9%) expanded access studies performed in the United States and published from 2014 through 2019 were registered with CT.gov. However, multivariate logistic regression analysis showed no significant association between any of the variables related to the studies and the odds of study nonregistration (P>.01).
Currently, CT.gov is a quite fragmentary source of data on expanded access programs. This problem is important because CT.gov is the only publicly available primary source of information about specific programs. We suggest the actions that should be taken by different stakeholders to fully exploit this register as a source of information about expanded access.
ClinicalTrials.gov（CT.gov）是基于互联网的最全面的不同类型临床研究注册库。扩大准入是指在临床试验之外使用未经批准的药物、生物制剂或医疗器械。扩大准入的一个关键问题是向医疗保健提供者和患者提供有关未经批准治疗的信息。
我们旨在评估 CT.gov 是否可作为获取扩大准入计划信息的潜在来源。
我们评估了 2017 年 2 月至 2020 年 5 月期间在 CT.gov 注册的 228 个扩大准入计划记录中的信息完整性。此外，我们还研究了已在 CT.gov 注册的已发表的扩大准入研究的百分比。使用逻辑回归（单变量和多变量）和中介分析来确定某些信息缺失和研究未注册的预测因素。
我们发现许多计划的记录中缺少一些重要数据。最常缺失的信息包括详细的研究描述、机构信息、中央联络人和资格标准（分别为 55.3%、54.0%、41.7%和 17.5%的计划）。多变量分析表明，2017 年注册的研究记录中更有可能缺少关于中央联络人的信息（调整后的 OR 21.93；95%CI 4.42-172.29；P<.001）。这一发现通过中介分析得到证实（P=.02）。此外，14%的计划是事后注册的。我们还表明，在美国进行的、2014 年至 2019 年发表的 77 项扩大准入研究中，仅有 33 项（42.9%）在 CT.gov 上注册。然而，多变量逻辑回归分析显示，与研究相关的任何变量与研究未注册的几率之间均无显著关联（P>.01）。
目前，CT.gov 是扩大准入计划数据相当零碎的来源。这个问题很重要，因为 CT.gov 是唯一一个可公开获取的特定计划的信息的主要来源。我们建议不同利益相关者采取的行动，以充分利用该注册处作为扩大准入信息的来源。
