生物制药公司临床试验透明度和数据共享，以及公司规模、地点和产品类型的作用：一项横断面描述性分析。

Clinical trial transparency and data sharing among biopharmaceutical companies and the role of company size, location and product type: a cross-sectional descriptive analysis.

机构信息

Internal Medicine, Yale University School of Medicine, New Haven, CT, USA.

Center for Health Policy/Primary Care and Outcomes Research, Department of Medicine, Stanford University School of Medicine; Stanford University Law School, Stanford, CA, USA.

出版信息

BMJ Open. 2021 Jul 19;11(7):e053248. doi: 10.1136/bmjopen-2021-053248.

DOI:10.1136/bmjopen-2021-053248

PMID:34281933

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8291313/

Abstract

OBJECTIVES

To examine company characteristics associated with better transparency and to apply a tool used to measure and improve clinical trial transparency among large companies and drugs, to smaller companies and biologics.

DESIGN

Cross-sectional descriptive analysis.

SETTING AND PARTICIPANTS

Novel drugs and biologics Food and Drug Administration (FDA) approved in 2016 and 2017 and their company sponsors.

MAIN OUTCOME MEASURES

Using established Good Pharma Scorecard (GPS) measures, companies and products were evaluated on their clinical trial registration, results dissemination and FDA Amendments Act (FDAAA) implementation; companies were ranked using these measures and a multicomponent data sharing measure. Associations between company transparency scores with company size (large vs non-large), location (US vs non-US) and sponsored product type (drug vs biologic) were also examined.

RESULTS

26% of products (16/62) had publicly available results for all clinical trials supporting their FDA approval and 67% (39/58) had public results for trials in patients by 6 months after their FDA approval; 58% (32/55) were FDAAA compliant. Large companies were significantly more transparent than non-large companies (overall median transparency score of 95% (IQR 91-100) vs 59% (IQR 41-70), p<0.001), attributable to higher FDAAA compliance (median of 100% (IQR 88-100) vs 57% (0-100), p=0.01) and better data sharing (median of 100% (IQR 80-100) vs 20% (IQR 20-40), p<0.01). No significant differences were observed by company location or product type.

CONCLUSIONS

It was feasible to apply the GPS transparency measures and ranking tool to non-large companies and biologics. Large companies are significantly more transparent than non-large companies, driven by better data sharing procedures and implementation of FDAAA trial reporting requirements. Greater research transparency is needed, particularly among non-large companies, to maximise the benefits of research for patient care and scientific innovation.

摘要

目的

研究与透明度提高相关的公司特征，并应用一种工具来衡量和提高大公司和药物临床试验的透明度，使其适用于小公司和生物制剂。

设计

横截面描述性分析。

设置和参与者

2016 年和 2017 年新批准的食品和药物管理局 (FDA) 新药和生物制剂及其公司赞助商。

主要观察指标

使用已建立的良好制药记分卡 (GPS) 措施，对临床试验注册、结果传播和 FDA 修正案法案 (FDAAA) 实施情况对公司和产品进行评估；使用这些措施和多部分数据共享措施对公司进行排名。还研究了公司透明度评分与公司规模（大型与非大型）、地点（美国与非美国）和赞助产品类型（药物与生物制剂）之间的关联。

结果

26%的产品（16/62）有支持其 FDA 批准的所有临床试验的公开结果，67%（39/58）在 FDA 批准后 6 个月内有患者试验的公开结果；58%（32/55）符合 FDAAA 规定。大公司比非大公司更加透明（整体透明度中位数为 95%（IQR 91-100）与 59%（IQR 41-70），p<0.001），这归因于更高的 FDAAA 合规性（中位数为 100%（IQR 88-100）与 57%（0-100），p=0.01）和更好的数据共享（中位数为 100%（IQR 80-100）与 20%（IQR 20-40），p<0.01）。公司所在地或产品类型没有观察到显著差异。