Reichert Janice M, Rochon Stephanie L, Zhang Bodi D
Tufts Center for the Study of Drug Development, Boston, Massachusetts 02111, USA.
Drug News Perspect. 2009 Jan-Feb;22(1):53-8. doi: 10.1358/dnp.2009.22.1.1303819.
The U.S. Food and Drug Administration's (FDA) Fast Track program, created in 1997, was designed to facilitate the development and expedite the review of drugs and biologics intended to treat serious or life-threatening conditions, and that demonstrate the potential to address unmet medical needs. Although the intent is laudable, the significance of designations and effectiveness of the program have recently come into question. Tufts Center for the Study of Drug Development has collected data on fast track candidates since 1998. We analyzed the current dataset of 344 fast track candidates granted nearly 400 designations, representing approximately 70% of the fast track designations granted by FDA, to address questions regarding common metrics. We found that fast track candidates were widely diverse in characteristics and development histories. The complexity and limitations of the data introduced biases in metrics such as clinical phase lengths and phase transition probabilities, although these could be determined for subsets of the candidates. Our results suggest that evaluation of the Fast Track program requires a nuanced approach, and estimates of the program's value should include assessment of the resulting marketed products.
美国食品药品监督管理局（FDA）的快速通道计划创建于1997年，旨在促进旨在治疗严重或危及生命疾病的药物和生物制品的开发，并加快对其审查，这些药物和生物制品显示出满足未满足医疗需求的潜力。尽管其意图值得称赞，但该计划指定的意义和有效性最近受到了质疑。塔夫茨药物开发研究中心自1998年以来一直在收集快速通道候选药物的数据。我们分析了当前包含344个快速通道候选药物的数据集，这些候选药物获得了近400个指定，约占FDA授予的快速通道指定的70%，以解决有关常见指标的问题。我们发现，快速通道候选药物在特征和开发历史方面差异很大。数据的复杂性和局限性在临床阶段时长和阶段转换概率等指标中引入了偏差，尽管可以针对候选药物的子集确定这些指标。我们的结果表明，对快速通道计划的评估需要一种细致入微的方法，并且对该计划价值的估计应包括对最终上市产品的评估。
