Wu Can, Ono Shunsuke
Laboratory of Pharmaceutical Regulatory Science, Graduate School of Pharmaceutical Sciences, The University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo, 113-0033, Japan.
Ther Innov Regul Sci. 2025 Jan;59(1):9-19. doi: 10.1007/s43441-024-00718-2. Epub 2024 Oct 31.
We analyzed factors shaping the choice of the lead indication (i.e., cancer type) in the first clinical development projects of new oncological drugs in the United States (US), and how the type of pharmaceutical company is related to this choice. We selected 576 new clinical development projects in the US since 2000 for analysis. These projects were characterized according to three potential perspectives detected by multiple correspondence analysis: the morbidity of the cancer type which corresponds to market size of each cancer type, the company's previous experience with the cancer type, and the company's attitude to development risks. Mega firms tend to choose cancer types with higher morbidity (and large-market), previously experienced cancer types, while diverse small firms choose both major and rare cancers and both high- and low-risk projects, indicating that different sizes of firms utilize different development entry patterns. Common tendencies concerning the choice of lead indication were found across all companies. Cancer types the company had developed and launched in the past were more likely to be chosen; cancer types with high five-year survival rates and those with high competition were less likely to be chosen. The study showed that pharmaceutical companies seem to enter clinical development from cancer types where they can demonstrate their strengths and advantages through experience, depending on each cancer type's different market sizes and development difficulties. The results could provide clues for considering what support measures and incentives are appropriate to balance the efficiency of industrial development and the fulfillment of society's unmet medical needs.
我们分析了在美国新肿瘤药物的首个临床开发项目中影响主要适应症（即癌症类型）选择的因素，以及制药公司类型与这一选择之间的关系。我们选取了自2000年以来美国的576个新临床开发项目进行分析。这些项目根据多重对应分析检测出的三个潜在视角进行了特征描述：与每种癌症类型市场规模相对应的癌症类型发病率、公司此前在该癌症类型方面的经验，以及公司对开发风险的态度。大型公司倾向于选择发病率较高（且市场规模大）、此前有经验的癌症类型，而多样化的小公司则同时选择主要癌症和罕见癌症，以及高风险和低风险项目，这表明不同规模的公司采用不同的开发进入模式。在所有公司中都发现了关于主要适应症选择的共同趋势。公司过去开发并推出过的癌症类型更有可能被选中；五年生存率高的癌症类型和竞争激烈的癌症类型被选中的可能性较小。该研究表明，制药公司似乎会从那些它们能够凭借经验展示自身优势的癌症类型进入临床开发，这取决于每种癌症类型不同的市场规模和开发难度。研究结果可为思考何种支持措施和激励措施适合平衡产业发展效率与满足社会未满足的医疗需求提供线索。
