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首页> 美国卫生研究院文献>British Journal of Cancer >Ten years of marketing approvals of anticancer drugs in Europe: regulatory policy and guidance documents need to find a balance between different pressures
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Ten years of marketing approvals of anticancer drugs in Europe: regulatory policy and guidance documents need to find a balance between different pressures

机译：欧洲抗癌药物获得十年市场批准：监管政策和指导文件需要在不同压力之间找到平衡

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Despite important progress in understanding the molecular factors underlying the development of cancer and the improvement in response rates with new drugs, long-term survival is still disappointing for most common solid tumours. This might be because very little of the modest gain for patients is the result of the new compounds discovered and marketed recently. An assessment of the regulatory agencies' performance may suggest improvements. The present analysis summarizes and evaluates the type of studies and end points used by the EMEA to approve new anticancer drugs, and discusses the application of current regulations. This report is based on the information available on the EMEA web site. We identified current regulatory requirements for anticancer drugs promulgated by the agency and retrieved them in the relevant directory; information about empirical evidence supporting the approval of drugs for solid cancers through the centralised procedure were retrieved from the European Public Assessment Report (EPAR). We surveyed documents for drug applications and later extensions from January 1995, when EMEA was set up, to December 2004. We identified 14 anticancer drugs for 27 different indications (14 new applications and 13 extensions). Overall, 48 clinical studies were used as the basis for approval; randomised comparative (clinical) trial (RCT) and Response Rate were the study design and end points most frequently adopted (respectively, 25 out of 48 and 30 out of 48). In 13 cases, the EPAR explicitly reported differences between arms in terms of survival: the range was 0–3.7 months, and the mean and median differences were 1.5 and 1.2 months. The majority of studies (13 out of 27, 48%) involved the evaluation of complete and/or partial tumour responses, with regard to the end points supporting the 27 indications. Despite the recommendations of the current EMEA guidance documents, new anticancer agents are still often approved on the basis of small single arm trials that do not allow any assessment of an ‘acceptable and extensively documented toxicity profile' and of end points such as response rate, time to progression or progression-free survival which at best can be considered indicators of anticancer activity and are not ‘justified surrogate markers for clinical benefit'. Anticipating an earlier than ideal point along the drug approval path and the use of not fully validated surrogate end points in nonrandomised trials looks like a dangerous shortcut that might jeopardise consumers' health, leading to unsafe and ineffective drugs being marketed and prescribed. The present Note for Guidance for new anticancer agents needs revising. Drugs must be rapidly released for patients who need them but not be at the expense of adequate knowledge about the real benefit of the drugs.
机译：尽管在理解癌症发展的分子因素和新药反应率的改善方面取得了重要进展，但是对于大多数常见的实体瘤，长期生存仍然令人失望。这可能是因为对患者的微不足道的收益很少是由于最近发现并投放市场的新化合物的结果。对监管机构绩效的评估可能会提出改进建议。本分析总结并评估了欧洲，中东和非洲用于批准新抗癌药物的研究类型和终点，并讨论了当前法规的应用。该报告基于EMEA网站上的可用信息。我们确定了该机构发布的有关抗癌药物的现行法规要求，并在相关目录中进行了检索；从欧洲公共评估报告（EPAR）中检索了有关通过集中程序支持实体药物批准的经验证据的信息。我们调查了从1995年1月成立EMEA到2004年12月的药物申请和以后的延期的文件。我们确定了针对27种不同适应症的14种抗癌药物（14种新用途和13种延期）。总体而言，有48项临床研究被用作批准的依据。随机比较（临床）试验（RCT）和缓解率是研究设计和最常采用的终点（分别是48个中的25个和48个中的30个）。 EPAR在13例病例中明确报告了两组间生存率的差异：范围为0-3.7个月，平均和中位数差异为1.5和1.2个月。大多数研究（27个研究中的13个，占48％）涉及评估支持27个适应症的终点的完整和/或部分肿瘤反应。尽管有现行的EMEA指导文件中的建议，但新的抗癌药仍经常在小型单臂试验的基础上获得批准，这些试验不允许对“可接受的且广泛记录的毒性概况”和终点（例如缓解率，进展时间或无进展生存时间最多可被视为抗癌活性的指标，而不是“临床获益的合理替代指标”。在非随机试验中预期沿药物批准途径的早于理想的点以及使用未经充分验证的替代终点似乎是危险的捷径，可能危及消费者的健康，从而导致市场上销售和开处方的不安全和无效的药物。目前有关新抗癌药物的指导说明需要修改。必须为有需要的患者迅速释放药物，但不能以对药物真正益处的充分了解为代价。

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