Harvard Radiation Oncology Program, Boston, MA, USA.
Department of Radiation Oncology, Dana-Farber/Brigham and Women's Cancer Center, Boston, MA, USA.
Neurotherapeutics. 2022 Oct;19(6):1844-1854. doi: 10.1007/s13311-022-01284-x. Epub 2022 Aug 15.
Scientific and technologic advances have led to a boon of candidate therapeutics for patients with malignancies of the central nervous system. The path from drug development to clinical use has generally followed a regimented order of sequential clinical trial phases. The recent increase in novel therapies, however, has strained the regulatory process and unearthed limitations of the current system, including significant cost, prolonged development time, and difficulties in testing therapies for rarer tumors. Novel clinical trial designs have emerged to increase efficiencies in clinical trial conduct to better evaluate and bring impactful drugs to patients in a timely manner. In order to better capture meaningful benefits for brain tumor patients, new endpoints to complement or replace traditional endpoints are also an increasingly important consideration. This review will explore the current challenges in the current clinical trial landscape and discuss novel clinical trial concepts, including consideration of limitations and risks of novel trial designs, within the context of neuro-oncology.
科学技术的进步为中枢神经系统恶性肿瘤患者带来了大量候选治疗药物。药物研发到临床应用的过程通常遵循严格的顺序临床试验阶段。然而，最近新型疗法的增加，给监管过程带来了压力，也暴露出当前系统的局限性，包括成本高、开发时间长，以及为罕见肿瘤测试疗法的困难。新型临床试验设计的出现，提高了临床试验的效率，以便及时、更有效地评估和为患者带来有意义的药物。为了更好地为脑肿瘤患者带来有意义的获益，补充或替代传统终点的新终点也是一个越来越重要的考虑因素。这篇综述将探讨当前临床试验格局中的挑战，并讨论神经肿瘤学背景下的新型临床试验概念，包括对新型试验设计的局限性和风险的考虑。
