End points to establish the efficacy of new agents in the treatment of acute leukemia
End points to establish the efficacy of new agents in the treatment of acute leukemia
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DOI：
10.1182/blood-2006-08-041152
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发表时间：
2007-03-01
期刊：
影响因子：
20.3
通讯作者：
Tallman, Martin S.
中科院分区：
文献类型：
--
作者：
Appelbaum, Frederick R.;Rosenblum, Daniel;Tallman, Martin S.
联邦法规提供了2种批准用于治疗急性白血病的新药的途径，即定期和加速批准。定期批准需要临床获益的证据，临床获益通常定义为延长生命或改善生活质量，或对作为临床获益替代指标的终点的影响。可以根据对替代指标的影响的证明来获得加速批准，该替代指标“合理可能”预测临床获益，但也需要在批准后证明临床获益。急性白血病是一组异质性和相对罕见的疾病。设计和执行前瞻性随机临床试验，证明这些疾病患者的生命延长或生活质量改善可能是困难和昂贵的，需要长期随访。因此，需要开发新的试验设计并纳入经验证的替代标记物以获得临床益处。为了探讨与急性白血病药物审批终点选择相关的一些问题，美国食品药品监督管理局邀请美国血液学会参与组织和举办联合研讨会。在本报告中，我们介绍了这一努力的成果。
Federal regulations provide 2 pathways for approval of new agents for the treatment of acute leukemia, regular and accelerated approval. Regular approval requires evidence of clinical benefit, which is generally defined as either prolongation of life or improved quality of life, or an effect on an end point established as a surrogate for clinical benefit. Accelerated approval can be obtained based on demonstration of an effect on a surrogate measure "reasonably likely" to predict clinical benefit, but requires demonstration of clinical benefit after approval as well. The acute leukemias are a heterogeneous and relatively uncommon group of diseases. The design and execution of prospective randomized clinical trials demonstrating prolongation of life or improved quality of life for patients with these disorders can be difficult and costly and require lengthy follow-up. Thus, the development of novel trial design and inclusion of validated surrogate markers for clinical benefit are needed. To explore some of the issues pertinent to the choice of end points for drug approval in acute leukemia, the Food and Drug Administration invited the American Society of Hematology to participate in the organization and conduct of a joint workshop. In this report, we present the results of that effort.