Assistance Publique-Hôpitaux de Paris, Hôpital Hôtel-Dieu, Paris, France.
J Clin Oncol. 2013 Aug 20;31(24):2998-3003. doi: 10.1200/JCO.2012.46.9577. Epub 2013 Jul 22.
To evaluate to what extent results of completed trials of cancer drugs conducted in the United States are publicly available at ClinicalTrials.gov, as required by the Food and Drug Administration Amendments Act (FDAAA), or are published in journals.
We searched ClinicalTrials.gov for cancer trials governed by the FDAAA: phase II to IV trials assessing drugs in the United States with a primary completion date between December 26, 2007, and May 31, 2010. For each trial, we also searched PubMed to identify the publication of results. We assessed the cumulative percentages of posted or published results over time by using the Kaplan-Meier method.
We identified 646 trials, including 209 randomized controlled trials (RCTs). At 12 months after completion of the trials, the cumulative percentages of trials with results posted at ClinicalTrials.gov, published in journals, and available either at ClinicalTrials.gov or in journals were 9% (95% CI, 7% to 11%), 12% (95% CI, 10% to 15%), and 20% (95% CI, 17% to 23%), respectively, and for RCTs, the percentages were 12% (95% CI, 8% to 16%), 5% (95% CI, 2% to 8%), and 17% (95% CI, 12% to 22%), respectively. At 36 months, these percentages were 31% (95% CI, 28% to 35%), 35% (95% CI, 31% to 39%), and 55% (95% CI, 51% to 59%), respectively, and for RCTs, they were 38% (95% CI, 31% to 45%), 32% (95% CI, 25% to 39%), and 56% (95% CI, 48% to 62%), respectively. Public availability of phase III trials was 15% (95% CI, 7% to 23%) at 12 months, 39% (95% CI, 27% to 49%) at 24 months, and 64% (95% CI, 50% to 73%) at 36 months.
Despite the FDAAA, results for nearly half the trials of cancer drugs in the United States were not publicly available 3 years after completion of the trials.
评估根据《食品和药物管理局修正案》（FDAAA）的要求，在美国完成的癌症药物试验的结果在 ClinicalTrials.gov 上公开的程度，或者在期刊上发表的程度。
我们在 ClinicalTrials.gov 上搜索了受 FDAAA 管辖的癌症试验：在美国进行的 II 期至 IV 期试验，评估了主要完成日期在 2007 年 12 月 26 日至 2010 年 5 月 31 日之间的药物。对于每个试验，我们还在 PubMed 上搜索了结果的发表情况。我们使用 Kaplan-Meier 方法评估了随时间推移发布或发表结果的累积百分比。
我们确定了 646 项试验，包括 209 项随机对照试验（RCT）。在试验完成后 12 个月时，在 ClinicalTrials.gov 上发布结果、在期刊上发表结果以及在 ClinicalTrials.gov 或期刊上均可获得结果的试验的累积百分比分别为 9%（95%CI，7%至 11%）、12%（95%CI，10%至 15%）和 20%（95%CI，17%至 23%），对于 RCT，百分比分别为 12%（95%CI，8%至 16%）、5%（95%CI，2%至 8%）和 17%（95%CI，12%至 22%）。在 36 个月时，这些百分比分别为 31%（95%CI，28%至 35%）、35%（95%CI，31%至 39%）和 55%（95%CI，51%至 59%），对于 RCT，百分比分别为 38%（95%CI，31%至 45%）、32%（95%CI，25%至 39%）和 56%（95%CI，48%至 62%）。美国 III 期试验的公开程度为 12 个月时为 15%（95%CI，7%至 23%），24 个月时为 39%（95%CI，27%至 49%），36 个月时为 64%（95%CI，50%至 73%）。
尽管有 FDAAA，但在美国完成的近一半癌症药物试验的结果在试验完成 3 年后仍未公开。
