Iberoamerican Cochrane Centre, Sant Pau Biomedical Research Institute (IIB Sant Pau), Barcelona, Spain ; CIBER Epidemiología y Salud Pública (CIBERESP), Barcelona, Spain ; Universitat Autònoma de Barcelona, Barcelona, Spain.
PLoS One. 2013 Nov 13;8(11):e79684. doi: 10.1371/journal.pone.0079684. eCollection 2013.
To describe the characteristics of randomized controlled clinical trials (RCT) on cancer drugs conducted in Spain between 1999 and 2003 based on their protocols.
We conducted an observational retrospective cohort study to identify the protocols of RCTs on cancer drugs authorized by the Agencia Española del Medicamento y Productos Sanitarios (AEMPS) (Spanish Agency for Medicines and Medical Devices) during 1999-2003. A descriptive analysis was completed and the association between variables based on the study setting and sponsorship were assessed.
We identified a total of 303 protocols, which included 176,835 potentially eligible patients. Three-quarter of the studies were internationally-based, 61.7% were phase III, and 76.2% were sponsored by pharmaceutical companies. The most frequently assessed outcomes were response rate (24.7%), overall survival (20.7%), and progression-free survival (14.5%). Of all protocols, 10.6% intended to include more than 1000 patients (mean: 2442, SD: 2724). Compared with their national counterparts, internationally-based studies were significantly larger (p<0.001) and were more likely to implement centralized randomization (p<0.001), blinding of the intervention (p<0.001), and survival as primary outcome (p<0.001). Additionally, most internationally-based studies were sponsored by pharmaceutical companies (p<0.01). In a high percentage of protocols, the available information was not explicit enough to assess the validity of each trial. Compared to other European countries, the proportion of Spanish cancer drugs protocols registered at www.clinicaltrials.gov (7%) was lower.
RCTs on cancer drugs conducted in Spain between 1999 and 2003 were more likely to be promoted by pharmaceutical companies rather than by non-profit national groups. The former were more often part of international studies, which generally had better methodological quality than national ones. There are some worldwide on-going initiatives that aim to increase the transparency and quality of future research.
根据方案描述 1999-2003 年期间在西班牙开展的癌症药物随机对照临床试验（RCT）的特征。
我们开展了一项观察性回顾性队列研究，以确定西班牙药品和医疗器械管理局（AEMPS）在 1999-2003 年期间批准的癌症药物 RCT 方案。完成了描述性分析，并根据研究背景和资助情况评估了变量之间的关系。
我们共确定了 303 项方案，其中包括 176835 名潜在合格患者。76.2%的研究为国际研究，61.7%为 III 期研究，76.2%由制药公司赞助。最常评估的结局是缓解率（24.7%）、总生存率（20.7%）和无进展生存率（14.5%）。所有方案中，有 10.6%的方案计划纳入超过 1000 名患者（平均：2442，标准差：2724）。与国内研究相比，国际研究的规模明显更大（p<0.001），更有可能实施中心化随机分组（p<0.001）、干预措施设盲（p<0.001）和将生存作为主要结局（p<0.001）。此外，大多数国际研究由制药公司赞助（p<0.01）。在大多数方案中，可用信息不够明确，无法评估每个试验的有效性。与其他欧洲国家相比，在 www.clinicaltrials.gov 注册的西班牙癌症药物方案比例（7%）较低。
1999-2003 年期间在西班牙开展的癌症药物 RCT 更有可能由制药公司而不是非营利性的国家团体推动。前者更常参与国际研究，这些研究的方法学质量通常优于国内研究。目前有一些全球性的正在进行的倡议旨在提高未来研究的透明度和质量。
