Blumenthal G M, Goldberg K B, Pazdur R
Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clin Pharmacol Ther. 2017 May;101(5):572-574. doi: 10.1002/cpt.623. Epub 2017 Apr 4.
As a result of enhanced understanding of genetic and immunologic underpinnings of cancer, there has been progress in development of targeted and immunotherapies in oncology. The traditional linear sequential model of drug development has evolved. Early clinical trials of breakthrough therapies often include expansion cohorts, termed "seamless drug development." The US Food and Drug Administration (FDA) uses expedited programs, such as breakthrough designation and accelerated approval ensuring that transformative therapies are available to patients earlier in the cycle of evidence generation.
由于对癌症的遗传和免疫基础有了更深入的了解，肿瘤学中的靶向治疗和免疫治疗取得了进展。药物开发的传统线性顺序模式已经演变。突破性疗法的早期临床试验通常包括扩展队列，称为“无缝药物开发”。美国食品药品监督管理局（FDA）采用加速计划，如突破性认定和加速批准，以确保变革性疗法能在证据生成周期中更早地提供给患者。
