Sharing of clinical trial data and results reporting practices among large pharmaceutical companies: cross sectional descriptive study and pilot of a tool to improve company practices.
Sharing of clinical trial data and results reporting practices among large pharmaceutical companies: cross sectional descriptive study and pilot of a tool to improve company practices.
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DOI：
10.1136/bmj.l4217
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发表时间：
2019-07-10
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影响因子：
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通讯作者：
Mello, Michelle M
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作者：
Miller, Jennifer;Ross, Joseph S;Mello, Michelle M
目的：开发和试验一种工具来衡量和改进制药公司的临床试验数据共享政策和实践。设计：横断面描述性分析。集合：拥有2015年美国食品和药物管理局批准的新药的大型制药公司。数据来源：数据共享措施改编自专家机构的10个重要数据共享指南，并通过患者、行业、学者、监管机构和其他人参与的多方利益相关者审议过程进行细化。使用来自ClinicalTrials.gov、Pharmics@FDA、公司网站、数据共享平台和注册表(例如，耶鲁大学开放数据访问(Yoda)项目和临床研究数据请求(CSDR))以及与制药公司的个人交流的数据来评估数据共享做法和政策。主要结果指标：公司级别，参与者级别临床试验数据的可访问性的多成分度量(例如，分析就绪数据集和元数据)；药物和试验级别的注册、结果报告和发布的度量；公司级别的总体透明度排名；结果：只有25%的大型制药企业完全达到了数据共享措施。公司数据共享得分的中位数为63%(四分位数范围为58-85%)。在得到反馈并有机会改进政策以满足这一措施的情况下，三家公司进行了修改，将完全合规的公司比例提高到33%，公司数据共享得分中值提高到80%(73%-100%)。公司最初没有满足数据共享措施的最常见原因是未能在指定的最后期限前共享数据(75%)，以及未能报告其数据请求的数量和结果。在新药申请中，患者试验的中位数为100%(四分位数范围91-100%)，65%(36-96%)报告结果，45%(30-84%)发表，95%(69-100%)在FDA药物批准后6个月以某种形式公开可用。当检查药物水平的结果时，不到一半(42%)的受评药物在FDA批准后6个月内以某种形式公开了其在患者中的所有新药应用试验的结果。结论：开发一种工具来衡量大公司之间的数据共享政策和做法是可行的，并对改善公司做法产生影响。在大公司中，25%的公司根据当前研究的措施向外部调查人员提供参与者级别的试验数据，以获得新药批准；应用排名工具后，这一比例提高到33%。其他衡量审判透明度的指标也更高。然而，一些公司在临床试验的透明度和数据共享方面仍有很大的改进空间。
OBJECTIVES: To develop and pilot a tool to measure and improve pharmaceutical companies' clinical trial data sharing policies and practices.DESIGN: Cross sectional descriptive analysis.SETTING: Large pharmaceutical companies with novel drugs approved by the US Food and Drug Administration in 2015.DATA SOURCES: Data sharing measures were adapted from 10 prominent data sharing guidelines from expert bodies and refined through a multi-stakeholder deliberative process engaging patients, industry, academics, regulators, and others. Data sharing practices and policies were assessed using data from ClinicalTrials.gov, Drugs@FDA, corporate websites, data sharing platforms and registries (eg, the Yale Open Data Access (YODA) Project and Clinical Study Data Request (CSDR)), and personal communication with drug companies.MAIN OUTCOME MEASURES: Company level, multicomponent measure of accessibility of participant level clinical trial data (eg, analysis ready dataset and metadata); drug and trial level measures of registration, results reporting, and publication; company level overall transparency rankings; and feasibility of the measures and ranking tool to improve company data sharing policies and practices.RESULTS: Only 25% of large pharmaceutical companies fully met the data sharing measure. The median company data sharing score was 63% (interquartile range 58-85%). Given feedback and a chance to improve their policies to meet this measure, three companies made amendments, raising the percentage of companies in full compliance to 33% and the median company data sharing score to 80% (73-100%). The most common reasons companies did not initially satisfy the data sharing measure were failure to share data by the specified deadline (75%) and failure to report the number and outcome of their data requests. Across new drug applications, a median of 100% (interquartile range 91-100%) of trials in patients were registered, 65% (36-96%) reported results, 45% (30-84%) were published, and 95% (69-100%) were publicly available in some form by six months after FDA drug approval. When examining results on the drug level, less than half (42%) of reviewed drugs had results for all their new drug applications trials in patients publicly available in some form by six months after FDA approval.CONCLUSIONS: It was feasible to develop a tool to measure data sharing policies and practices among large companies and have an impact in improving company practices. Among large companies, 25% made participant level trial data accessible to external investigators for new drug approvals in accordance with the current study's measures; this proportion improved to 33% after applying the ranking tool. Other measures of trial transparency were higher. Some companies, however, have substantial room for improvement on transparency and data sharing of clinical trials.