Biology Department, Master of Biotechnology Program, University of Toronto, Toronto, ON.
J Pharm Pharm Sci. 2011;14(2):227-35. doi: 10.18433/j39p45.
To quantify the clinical trial risk of new drug development in Non-Hodgkin's lymphoma (NHL). Risk estimates for this disease have not been reported before.
We undertook a retrospective review of clinical trials in (NHL) in four subtypes to compare the success rate with the industry average. Our inclusion criteria required that a drug must initiate its phase I trial in one of the four NHL subtypes between 1998 and June 2008 in the US. In addition, clinical trials of new drug candidates that pertain to four subtypes of NHL were retrieved from clinicaltrial.gov. Drug candidates that did not meet these criteria were excluded from the study.
The overall success rate (8-11%) was significantly lower than the industry standard (17%). Overall survival (OS) as a secondary outcome appeared more predictive than primary endpoints that were surrogate, of overall success. Further, targeted therapies appear more successful in these lymphoma sub-types than broad acting drugs.
Clinical trial risk in NHL, with an 89% failure rate reported here, may be reduced by basing decisions on OS secondary endpoints and biologic drugs.
定量评估非霍奇金淋巴瘤（NHL）新药开发的临床试验风险。此前尚未有针对该疾病的风险评估报告。
我们对四种 NHL 亚型的临床试验进行了回顾性研究，以比较成功率与行业平均水平。我们的纳入标准要求药物必须在 1998 年至 2008 年 6 月期间在美国的四种 NHL 亚型之一中启动其 I 期临床试验。此外，还从 clinicaltrial.gov 检索了与 NHL 四种亚型相关的新药候选物的临床试验。未满足这些标准的候选药物被排除在研究之外。
总体成功率（8-11%）明显低于行业标准（17%）。次要终点总生存期（OS）似乎比替代的主要终点更能预测总体成功率。此外，与广泛作用的药物相比，靶向疗法在这些淋巴瘤亚型中似乎更成功。
NHL 的临床试验风险较高，这里报告的失败率为 89%，通过基于 OS 次要终点和生物药物做出决策，可能会降低风险。
Zotero 插件