Differences in drug approval processes of 3 regulatory agencies: a case study of gemtuzumab ozogamicin
Differences in drug approval processes of 3 regulatory agencies: a case study of gemtuzumab ozogamicin
复制标题
3个监管机构药品审批流程差异：以吉妥珠单抗奥佐米星为例
作者：
T. Tanimoto;M. Tsubokura;Jinichi Mori;Monika Pietrek;S. Ono;M. Kami
在监管机构之间，风险效益评估和监管行动的重大差异是经常发生的。我们通过仔细检查急性髓性白血病（AML）患者使用吉妥珠单抗ozogamicin （GO）的病例来探讨差异。我们从美国食品和药物管理局（FDA）、欧洲药品管理局（EMA）和日本监管机构的网站上检索了氧化石墨烯的评估报告，并查阅了已发表的临床试验。虽然GO于2000年通过美国FDA的加速审批程序获得批准，但基于上市后承诺的失败，它于2010年退出市场。2008年，EMA以没有随机对照试验（RCTs）为由拒绝批准氧化石墨烯的上市许可。GO于2005年在日本被批准为孤儿药，日本监管机构决定在加强上市后监管的条件下，于2010年继续批准。在这种情况下，2011年出现了有希望的rct新结果，氧化石墨烯在AML治疗中的作用在全球范围内得到了重新关注。对于靶向治疗的孤儿药，严格的监管可能并不合适，应该留有更多的空间，以促进新的抗肿瘤药物的有效开发。
SummaryMajor discrepancies concerning risk-benefit assessments and regulatory actions are frequent among regulatory agencies. We explored the differences by scrutinizing a case of gemtuzumab ozogamicin (GO) in patients with acute myeloid leukaemia (AML). Assessment reports of GO were retrieved form the websites of the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Japanese regulatory agency, and we also reviewed published clinical trials. While GO was approved by the US FDA under the accelerated approval program in 2000, it was withdrawn from the market in 2010, based on the required post-marketing commitment failure. The EMA refused granting marketing authorization for GO in 2008 on the grounds that there were no randomised controlled trials (RCTs). GO was approved as an orphan drug in Japan in 2005, and the Japanese regulatory authority decided to continue with the approval in 2010 on the condition that post-marketing surveillance is strengthened. Under these situations, promising new results of RCTs appeared in 2011, and the role of GO in AML treatment was refocused worldwide. The stringent regulation may not be suitable in case of an orphan drug of targeted therapy, and more room should be kept to facilitate effective developments of new anti-neoplastic agents.
影响因子：
158.5
作者：
Lowenberg, Bob;Pabst, Thomas;Ossenkoppele, Gert J.
通讯作者：
Ossenkoppele, Gert J.
影响因子：
20.3
作者：
Walter, Roland B.;Appelbaum, Frederick R.;Bernstein, Irwin D.
通讯作者：
Bernstein, Irwin D.
吉妥珠单抗奥佐米星和常规化疗序贯给药作为老年急性髓系白血病患者的一线治疗：EORTC 和 GIMEMA 白血病组的 II 期研究 (AML-15)。
影响因子：
10.1
作者：
Amadori,Sergio;Suciu,Stefan;Willemze,Roel;Mandelli,Franco;Selleslag,Dominique;Stauder,Reinhard;Ho,Anthony;Denzlinger,Claudio;Leone,Giuseppe;Fabris,Piero;Muus,Petra;Vignetti,Marco;Hagemeijer,Anne;Beeldens,Filip;Anak,Ozlem;DeW
通讯作者：
DeW