Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13.
Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13.
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DOI：
10.1136/bmj.j4530
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发表时间：
2017-10-04
期刊：
影响因子：
--
通讯作者：
Aggarwal A
中科院分区：
文献类型：
--
作者：
Davis C;Naci H;Gurpinar E;Poplavska E;Pinto A;Aggarwal A
目的确定欧洲批准的癌症药物的总生存期和生活质量获益数据的可用性。 设计回顾性队列研究。 2009年至2013年欧洲药品管理局（EMA）关于癌症批准的公开可访问的监管和科学报告。 主要结果测量抗癌药物的上市前和上市后试验，根据其设计特征（随机化，交叉，盲法），对照药物和终点。批准时和进入市场后确定的总体生存期或生活质量获益的可用性和程度。经验证的欧洲医学肿瘤学会临床获益量表（ESMO-MCBS），用于评估已发表的癌症药物研究中报告的获益的临床价值。 从2009年到2013年，EMA批准了48种癌症药物用于68种适应症。其中，8种适应症（12%）是基于单组研究获得批准的。在上市批准时，68例患者中有24例（35%）的生存期显著延长。总生存期的获益幅度为1.0至5.8个月（中位数2.7个月）。在批准上市时，68种适应症中有7种（10%）的生活质量有所改善。在44种适应症中，在上市许可时没有证据表明生存率增加，在随后的上市后期间，有3种（7%）有证据表明寿命延长，5种（11%）报告了生活质量获益。在EMA批准的68种癌症适应症中，中位随访时间为5.4年（最短3.3年，最长8.1年），只有35种（51%）显示出生存率或生活质量的显著改善，而33种（49%）仍不确定。在与生存获益相关的23种适应症中，可以使用ESMO-MCBS工具进行评分，不到一半（11/23，48%）的受益被判定为具有临床意义。 EMA对2009- 2013年批准的肿瘤药物的系统评价显示，大多数药物进入市场时没有证据表明对生存或生活质量有益。在进入市场至少3.3年后，仍然没有确凿的证据表明这些药物延长或改善了大多数癌症适应症的生命。当与现有的治疗方案或安慰剂相比有生存增益时，它们通常是边际的。
Objective To determine the availability of data on overall survival and quality of life benefits of cancer drugs approved in Europe. Design Retrospective cohort study. Setting Publicly accessible regulatory and scientific reports on cancer approvals by the European Medicines Agency (EMA) from 2009 to 2013. Main outcome measures Pivotal and postmarketing trials of cancer drugs according to their design features (randomisation, crossover, blinding), comparators, and endpoints. Availability and magnitude of benefit on overall survival or quality of life determined at time of approval and after market entry. Validated European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) used to assess the clinical value of the reported gains in published studies of cancer drugs. Results From 2009 to 2013, the EMA approved the use of 48 cancer drugs for 68 indications. Of these, eight indications (12%) were approved on the basis of a single arm study. At the time of market approval, there was significant prolongation of survival in 24 of the 68 (35%). The magnitude of the benefit on overall survival ranged from 1.0 to 5.8 months (median 2.7 months). At the time of market approval, there was an improvement in quality of life in seven of 68 indications (10%). Out of 44 indications for which there was no evidence of a survival gain at the time of market authorisation, in the subsequent postmarketing period there was evidence for extension of life in three (7%) and reported benefit on quality of life in five (11%). Of the 68 cancer indications with EMA approval, and with a median of 5.4 years’ follow-up (minimum 3.3 years, maximum 8.1 years), only 35 (51%) had shown a significant improvement in survival or quality of life, while 33 (49%) remained uncertain. Of 23 indications associated with a survival benefit that could be scored with the ESMO-MCBS tool, the benefit was judged to be clinically meaningful in less than half (11/23, 48%). Conclusions This systematic evaluation of oncology approvals by the EMA in 2009-13 shows that most drugs entered the market without evidence of benefit on survival or quality of life. At a minimum of 3.3 years after market entry, there was still no conclusive evidence that these drugs either extended or improved life for most cancer indications. When there were survival gains over existing treatment options or placebo, they were often marginal.
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影响因子：
50.5
作者：
Douillard, J. Y.;Siena, S.;Sidhu, R.
通讯作者：
Sidhu, R.
DOI：
10.1017/s0266462314000300
发表时间：
2014-07-01
影响因子：
3.2
作者：
Ciani, Oriana;Cantrell, Anna;Taylor, Rod S.
通讯作者：
Taylor, Rod S.
影响因子：
8.4
作者：
Amir, Eitan;Seruga, Bostjan;Ocana, Alberto
通讯作者：
Ocana, Alberto
影响因子：
45.3
作者：
Booth, Christopher M.;Cescon, David W.;Krzyzanowska, Monika K.
通讯作者：
Krzyzanowska, Monika K.
影响因子：
45.3
作者：
Blumenthal, Gideon M.;Karuri, Stella W.;Pazdur, Richard
通讯作者：
Pazdur, Richard